Scientific program | Pediatric Care | Child health

New Canadian guidelines offer comprehensive approach to managing pediatric obesity

2025-04-17 - 2025-04

Recommendations to manage pediatric obesity
In the current study, the researchers categorized their recommendations into five behavioral and psychological interventions, three pharmacologic interventions, and two surgical interventions. Multicomponent interventions that incorporate at least two interventions based on nutrition, physical activity, psychology, and technology were strongly recommended.

Nutritional interventions involving specific dietary patterns, such as low-fat, low glycemic index, and Mediterranean-style diets, have successfully been used to manage child obesity. Research has also demonstrated the beneficial effects of physical activity interventions including aerobic or resistance training, with the combination of both aerobic and resistance training most effective.

The certainty of evidence on the efficacy of psychological interventions was moderate. Nevertheless, the researchers recommend psychological interventions for managing obesity in children 18 years of age and younger, including counseling and standardized cognitive behavioral therapies.

The recommendations were neither for nor against technological interventions.

Among children 12 years of age and older, behavioral and psychological interventions should be combined with glucagon-like peptide-1 receptor agonists (GLP-1RAs), such as exenatide, liraglutide, and semaglutide. Biguanides can also be combined with behavioral and psychological interventions to manage obesity in this pediatric patient population. Lipase inhibitors were not recommended to manage obesity in children.

Gut Instincts: The Microbiome’s Role in Pediatric Vasculitis

2025-04-09 - 2025-04

When a child presents with a stubborn high fever, an angry rash and red eyes, the pediatrician’s impulse might be to prescribe antibiotics, since such symptoms can indicate a serious infection that requires swift treatment. But for patients with Kawasaki disease, a rare pediatric vasculitis that can masquerade as a bacterial infection, prescribing antibiotics before obtaining a definitive diagnosis is a risky gamble.

The onset of Kawasaki disease is sudden and confounding. And recent research reveals a surprising twist: Antibiotics aren’t just ineffective at treating Kawasaki disease—they may make things worse.

"Antibiotic treatment may help children with bacterial infections but not patients with Kawasaki disease. Antibiotics compromises their gut microbiome, which plays an important role in protecting children from long-term complications such as cardiac dysfunction, arrythmias and even death," said Magali Noval Rivas, PhD, a cellular and molecular immunologist at Cedars-Sinai.

In studying the gastrointestinal system for more than 10 years, Noval Rivas has discovered that imbalances in gut microbiota, called dysbiosis, also fuel more common pediatric immune conditions, including allergies and asthma.

Noval Rivas recently co-authored a preclinical study in Circulation Research, showing that altering the gut microbiome by introducing beneficial bacteria could help stall disease progression and improve cardiac abnormalities in patients with Kawasaki disease.

"If we're able to grow specific anti-inflammatory bacteria in the laboratory and deliver them back to the patient, it may help reduce intestinal dysbiosis and promote intestinal barrier functions—and that can be relevant for several inflammatory diseases, not just Kawasaki disease," said Moshe Arditi, MD, executive vice chair of the Department of Pediatrics for Research, part of Cedars-Sinai Guerin Children’s, who works closely with Noval Rivas.

Compared to healthy children, patients with Kawasaki disease have an abundance of harmful bacteria in the gut and lower levels of beneficial short chain fatty acid-producing bacteria, which are protective against the disease."

— Dr. Moshe Arditi

An Underappreciated Player in Pathology
The gut microbiome is home to trillions of diverse microorganisms that regulate mucosal barriers and support immune function. This complex ecosystem is established at birth and remains for a lifetime, but it’s constantly in flux. Specific alterations in the intestinal microbiota composition can contribute to a host of inflammatory and metabolic diseases, including Kawasaki disease and cardiovascular disease.

Kawasaki disease, first identified in 1967 by Japanese pediatrician Tomisaku Kawasaki, primarily affects children under 5 years old. The disease is rare in the United States, affecting 18 to 25 per 100,000 children under age 5 annually. It is the leading cause of acquired heart disease in children. The incidence is 10 to 30 times higher in Japan, South Korea, China and Taiwan, suggesting that genetic variation and environmental factors are responsible for this increased risk.

The exact cause of the condition remains unclear, and there is no diagnostic test for the disorder. Clinicians rely on symptoms such as high fever, rash, red eyes, and swollen hands and feet.

How pre-URS tamsulosin use in pediatric patients may reduce repeat procedures

2025-04-08 - 2025-04

In a recent interview with Urology Times®, Kate H. Kraft, MD, MHPE, FACS, FAAP, discussed a recent study regarding the use of tamsulosin (Flomax) in pediatric patients undergoing ureteroscopy.1 During the interview, she was asked about the implications of reducing repeat anesthetic exposures in children.

“With regard to anesthesia exposure in the pediatric population, there's been some data in the anesthesiology literature that suggests that there may be an association with neurotoxicity or cognitive development over time, whether it's anesthesia exposure at an early age or repeated anesthesia exposure. And so in pediatric urology and all pediatric surgical fields, we're very cognizant of that, and want to try to reduce exposure as much as possible. Now, [with] the literature essentially, the jury is out. The literature is not very definitive [and] still somewhat controversial, but I think if we can minimize anesthesia exposure for our patients, that is certainly the best for them. That’s where I think the findings of our study are really exciting, because if we can do a simple prescribing practice to be able to minimize return trips to the operating room for ureteroscopic access, I think that would really benefit our patients,” said Kraft, chief of Pediatric Urology, Mark C. McQuiggan, MD, and Carolyn A. McQuiggan Research Professor, and professor of urology at the University of Michigan, Ann Arbor.
Asked for the take-home message of the study, Kraft commented, “I would say the take-home message for this study is that prescribing tamsulosin for a week prior to an anticipated surgery that involves flexible ureteroscopy is a very simple practice that certainly is within standard of care within pediatric urology. It’s easy for patients to take. If they don't swallow pills, they can sprinkle the capsule contents on some food and easily take it on a daily basis. It's a once-daily medication, so it's not too cumbersome. It has minimal [adverse events], and has been shown to be very safe and effective for children, even as young as in the toddler range. So simply, just adding this to one's daily practice might save the patient return trips to the operating room, afford more successful outcomes and make our lives easier too, so that we can have successful treatment in 1 fell swoop, in a single surgery.”

Systematic Review Highlights Post-Pandemic Disparities in Pediatric Atopic Dermatitis

2025-04-07 - 2025-04

A recent update on published data related to pediatric atopic dermatitis disparities highlights the results of several studies around the time of the COVID-19 pandemic, highlighting that children of color and those with specific socioeconomic factors showed increased prevalence and severity in terms of atopic dermatitis.1

This updare was authored by a team of investigators led by Simone Gottlieb—an MD candidate from the Department of Dermatology at Northwestern University’s Feinberg School of Medicine—who noted that a significant body of literature on health disparities had increased since the beginning of the COVID-19 pandemic.2

“...[An] update on recently published research on pediatric [atopic dermatitis], which affects up to 20% of children in the United States, is needed,” Gottlieb and colleagues wrote.1 “In this scoping review, we provide an update on recently published pediatric [atopic dermatitis] disparities literature and identify knowledge gaps to guide future research.

Review Process
The investigative team carried out a systematic scoping review using a comprehensive search strategy developed in collaboration with a medical librarian. Searches were done via the Embase (Elsevier), MEDLINE (PubMed) databases as well as the Cochrane Database of Systematic Reviews and Central Register of Controlled Trials (Wiley).

Keywords and database-specific controlled vocabulary that were focused on atopic dermatitis, pediatric populations, and health disparities were used. The team restricted their analysis to studies published from January 2021 onward, as it was a prior scoping review. Searches were done on May 9, 2024, with studies included being required to be published in English and involve either those under 18 or the parents or guardians of pediatric patients within the US.

Additionally, studies that the investigators included also were required to address 1 or more disparities associared with pediatric atopic dermatitis. Two reviewers collaboratively developed and piloted a data extraction spreadsheet for this review, and 53 studies met their inclusion criteria. Among these, 49 studies were shown to have examined disparities based on race or ethnicity, 18 based on socioeconomic status, 11 focused on sex or gender differences, 6 on regional or environmental factors, and 1 study had assessed disparities related to preferred language.

Notable Findings on Disparities
The researchers noted that 17 of 49 studies on racial and ethnic disparities looked into disease prevalence, 12 into comorbidities linked to atopic dermatitis, 11 into access to care, 6 into medical outcomes, and the remainder assessed several other disparity-related issues.

The review highlighted several notable conclusions from various studies, including 1 indicating that compared to non-Hispanic White children, the odds of having atopic dermatitis were elevated among American Indian/Alaskan Native (1.2 times greater), Hispanic (1.4 times greater), and Asian children (2.45 times greater). The investigative team highlighted many studies which had identified additional racial and ethnic disparities, particularly affecting Black and Hispanic children.

These children were shown in several studies to be less likely to be given allergy tests, to receive standard treatments or be assessed by healthcare professionals, and were shown to have higher rates of missed prescriptions. Other findings suggested that such patients experienced longer delays prior to diagnosis and initial appointments, and faced greater rates of delayed care as a result of transportation barriers.

Overall, children of color experienced an absolute prevalence of atopic dermatitis that was 2%–10% were noted by the investigators to be higher than the prevalence observed in White children, along with a 1%–4% greater absolute increase in prevalence over time. Additionally, the team reported that hospitalizations and urgent or ambulatory care visits among such populations were 8%–24% more frequent than those of of White children, with odds ratios ranging from 1.5 - 3.8.

Black children were specifically noted in some of these findings to have 1.24 times the odds (95% CI: 0.99–1.55) of experiencing poorer levels of disease control. They were also 2.5 to 4.12 times more likely in certain of the team's highlighted studies to have early and persistent atopic dermatitis forms compared to their White counterparts.

Disproportionate financial and social burden impacts were also demonstrated among such patients, with studies included in the review pointing to the 1.32 times greater odds (95% CI: 1.09–1.60) among these children of food insecurity, 3.86 times higher odds (95% CI: 1.66–8.98) of harmful financial effects, and increased sleep disturbance (B = 3.89, P < 0.01). These findings were some highlights of the dramatic disparities identified in this review, suggesting the need to address disparities in the field of dermatology.

“The persistence of health disparities among an increasingly diverse pediatric population necessitates further health equity research,” Gottlieb and colleagues concluded. “Analyzing health disparities is the first step to paving the way for future research and interventions that promote health equity for all marginalized patient populations.”1

Prognostic factors in pediatrics TAPVC: a 10-year retrospective study

2025-04-06 - 2025-04

Total anomalous pulmonary venous connection (TAPVC) is a rare congenital heart defect where the pulmonary veins abnormally connect to the systemic venous circulation, rather than the left atrium (LA). This study retrospectively analyzed 48 children diagnosed with TAPVC from January 2014 to January 2024 to investigate how specific echocardiographic and morphological characteristics impact prognosis. Perioperative records, including echocardiography, computed tomography angiography (CTA) images, surgical details, and patient outcomes, were reviewed. Data were statistically analyzed using tools such as t-tests, Mann-Whitney U tests, χ² tests, and logistic regression. Key factors examined included age at surgery, anatomical subtype of TAPVC, presence of preoperative obstruction, surgical techniques used, and postoperative complications. By identifying the impact of these variables on survival rates and hospital recovery, the study aims to improve risk stratification and management strategies for better outcomes in children with TAPVC.
Total anomalous pulmonary venous connection (TAPVC) is a relatively rare and severe cyanotic congenital heart disease, occurring in approximately 0.5-2% of congenital heart diseases1. In this condition, all pulmonary veins fail to connect to the left atrium (LA); instead, they connect to the right atrium (RA) through systemic veins or drain directly into the RA. Based on the drainage pathways, TAPVC can be divided into four types: supracardiac, cardiac, infracardiac, and mixed. The supracardiac type is the most common, representing about 43% of all cases. Additionally, about 25-50% of patients with TAPVC have associated pulmonary venous obstruction (PVO)2,3,4.

The clinical symptoms of children with TAPVC are closely related to the presence of PVO, these symptoms can range from mild pulmonary hypertension to severe pulmonary congestion and acidosis5. In case where PVO is present, emergency surgery is required. Without timely intervention, most affected neonates do not survive the neonatal period.

Advancements in surgical techniques, diagnostic procedures, and perioperative care have significantly reduced mortality rates in TAPVC in recent years. Various studies have focused on prognostic factors such as preoperative general conditions (age, weight, oxygen saturation, pH value), surgical techniques, and cardiopulmonary bypass time6. While much of the research has concerned on the type and presence of PVO, less attention has been given to specific echocardiographic parameters.

This study aims to bridge that gap by retrospectively analyzing the correlation between detailed echocardiographic measurements and the prognosis of children with TAPVC. By understanding these relationships, better predictive models and tailored management strategies can be developed, potentially improving outcomes for patients with TAPVC.

Results
Clinical characteristics
In this cohort of 48 children diagnosed with TAPVC, the median age was 35 days (range 1-2615days), and the median weight was 3.8 kg (range 1.5–13.5 kg). The median surgical duration for these cases was 215 min, with a range from 135 to 485 min. The gender distribution included 34 males (approximately 70.8%) and 14 females (approximately 29.2%). Among of TAPVC connections, 15 cases (31.3%) were supracardiac, 20 cases (41.7%) were cardiac, 10 cases (20.8%) were infracardiac, and 3 cases (6.3%) had mixed connections.

Top 10 Most-Feared Diagnostic Errors for Trainees in Pediatric Neuroradiology Emergencies

2025-04-03 - 2025-04

Survey of senior radiology residents revealed on-call potential errors most challenging to trainees
Shortened image acquisition times and greater MR availability are allowing even the youngest patients to undergo imaging, which is especially helpful in the emergency department, where infants and young children present with life-threatening conditions. However, for radiologists in training, interpreting the imaging studies of pediatric patients presents unique challenges.

“Pediatric neuroradiology is a subspecialty within a subspecialty,” said Agustín Cárdenas, MD, clinical assistant professor of pediatric neuroradiology at the Hospital Universitario Doce de Octubre, in Madrid, and the Children’s Hospital of Alabama/University of Alabama at Birmingham. “So many trainees and attending radiologists are fearful whenever they see a CT of a two-year-old or an MRI of a five-year-old because the pediatric brain, pathology and myelination are different, and the differential diagnosis changes.”

Throughout his career, Dr. Cárdenas said he has noticed a pattern of common fears among trainees when interpreting pediatric neuroradiology CT and MRI studies.

“There are differences in the appearance of pathophysiologic conditions and disease in pediatric patients compared to adults,” he said. “Limited exposure during training contributes to the complexity of interpreting these imaging studies, especially in the emergency setting.”

To better understand these challenges, Dr. Cárdenas and a team of researchers surveyed 35 senior radiology residents in 22 different residency programs in the U.S. and Europe to identify their most feared diagnostic errors when on call. Once the top 10 potential errors were identified, the researchers generated practical tips and tricks to help prevent them.

“Trainees are regularly exposed to adult brains, making them more familiar with what is considered ‘normal’,” he said. “However, acute emergency findings can be easy for trainees to overlook, especially when they are accustomed to the appearance of a typical 50-year-old brain.”
Breaking Down the Top Five
Ranking No. 1 in the survey as the most dreaded potential error was the detection of early cerebral edema. Nearly all survey respondents feared missing this diagnosis.

“Not only are the initial findings subtle, but many trainees are unfamiliar with cerebrospinal fluid (CSF) spaces in children,” he said. “Early edema can be very difficult to see, and there are grave implications for missing it.”

To prevent the condition from being missed, the researchers suggested becoming familiar with the normal appearance of basal cisterns. “Basal cisterns and CSF spaces along convexities are your friend,” Dr. Cárdenas said.

Coming in at No. 2 was detecting dural sinus thrombosis with non-contrast CT. Missing this relatively uncommon condition was feared by 73% of survey respondents.

According to Dr. Cárdenas, vague symptoms, dural sinuses that appear normal, and a relatively high hematocrit level (typical in neonates) make this diagnosis challenging. “Clinically, dural sinus thrombosis can be very silent and subtle,” he said.

Prevention tips include conducting a quantitative analysis and adjusting for hematocrit levels.

Correctly distinguishing between retropharyngeal edema and early abscess was No. 3 on the list, with 63% of senior residents fearing a potential error in such a case. Dr. Cárdenas said looking for the enhancement pattern and mass effect is helpful for trainees faced with answering this question.

More than half of the survey respondents considered detecting early shunt failure challenging, giving it a No. 4 ranking. In addition to always comparing with prior images when available, Dr. Cárdenas said trainees should look for sulcal effacement near the vertex. “Early shunt dysfunction can be easily missed, particularly in patients with developmental delays,” he said.

At No. 5—feared by 56% of respondents—was incorrectly diagnosing a craniocervical junction (CCJ) fracture. “Radiology trainees often overcall fractures because they lack familiarity with the imaging appearance and timing of the normal ossification centers of C1 and C2 on CT,” he said.

Learning more about the imaging appearance of normally developing ossification centers will help trainees accurately identify fractures.

“The craniocervical junction in pediatric patients is more flexible and has more laxity than adults,” he said. “Fractures are rare in infants and young children, but MRI may help assess ligamentous injuries.”

What it's really like in pediatrics—from 3 physicians who know

2025-04-02 - 2025-04

As a medical student, do you ever wonder what it’s like to specialize in pediatrics? Over the years, three pediatricians from around the country who are working in academic settings have taken the time to be featured doctors in the AMA’s "Shadow Me” Specialty Series, which offers advice directly from physicians about life in their specialties. Check out their insights to help determine whether a career in pediatrics might be a good fit for you.
Shadowing Peter Hotez, MD, PhD
Dr. Hotez, an AMA member, is an academic pediatrician-scientist and vaccinologist. He is also a world-renowned advocate in the fields of global health, vaccinology and neglected tropical disease control. He said every pediatrician in training should have health and public communication skills. “We need a new generation of physicians who know how to engage the public and communicate effectively. There’s a vacuum on this front,” he said, adding that pediatricians should ideally have management skills to head an organization.
He also said too many young physicians and scientists are excessively focused on their immediate next steps, instead of asking what problem they want to solve in 10 or 15 years’ time. “In this way, you can construct a roadmap to get from here to there. It also makes getting into a prestigious residency, fellowship or academic appointment less important by focusing on the area in which you want to achieve. To be provocative I often say: Where you go to medical school or residency or fellowship ranks among the least important decisions you will make. What you choose to study ranks among the most important.”
Shadowing Nusheen Ameenuddin, MD, MPH, MPA
Dr. Ameenuddin has more than 15 years in practice, largely in an academic medical center. She said one of the hardest parts of her work has been dealing with “this growing group of loud anti-vaccine and anti-science voices controlling the media and popular narrative on immunizations and spreading mass misinformation and disinformation.” Since she was in residency, she has been “dealing with some type of vaccine hesitance or outright refusal, which are actually two very different issues and require very different approaches.”
This “erosion of trust and expertise, coupled with the rise of conspiracy theories, has been especially difficult,” said Dr. Ameenuddin, an AMA member. “On the other hand, kids are the absolute best! Even though we see some really sad things, one of the great things about children is that we see resilience and joy even under extremely difficult circumstances.” In fact, “Kids can always say something or do something that makes me smile and remind me of why I chose to become a pediatrician.”
Shadowing Kanani Titchen, MD
A pediatrician and an adolescent medicine physician, Dr. Titchen is employed by an academic health system. She said one of the skills every physician in training should have for adolescent medicine is the ability to connect, the “ability to listen—really listen with one’s whole self and with love in mind—skills in motivational interviewing and ability to be authentic and in the moment with our patients are essential. Understanding and embodying trauma-sensitive care is also key and currently there’s not much education for this, let alone testing for it.”
She also said her lifestyle is different from what she had envisioned in training. “I’m much busier than I thought I’d be. Partly, that’s because I’ve chosen to work at an academic practice where I really have three jobs: patient care, teaching and research. In terms of work-life balance, I think it’s hard to beat pediatrics and academic medicine. Still, whatever field one enters, there are daily choices to be made about prioritizing ourselves, our families, our patients, our colleagues and our communities. I don’t really know if any of us ever ‘arrive’ at striking that balance. To me, it feels like an ongoing process and a daily decision.”

Anaphylm sublingual film demonstrates positive results in pediatric patients with severe allergic reactions

2025-04-01 - 2025-04

On April 1, 2025, Aquestive Therapeutics announced positive topline results from its pediatric study evaluating Anaphylm (epinephrine) sublingual film in patients aged 7 to 17 years.1
The study included children weighing more than 30 kilograms with a history of allergic reactions. These results mark the completion of the Anaphylm clinical program and support the company’s New Drug Application (NDA) submission to the FDA.1,2
“We are extremely pleased with the positive results from our pediatric study, which further validate Anaphylm's potential as the first-ever sublingual film and convenient treatment option for all patients with severe allergic reactions, including anaphylaxis,” said Daniel Barber, president and CEO of Aquestive.

Anaphylm is a polymer matrix-based epinephrine prodrug candidate designed for the treatment of severe allergic reactions, including anaphylaxis.
It is similar in size to a postage stamp, weighs less than an ounce, and begins to dissolve upon contact without the need for water or swallowing. The packaging is compact, smaller than a credit card, and designed to withstand environmental factors like rain and sunlight, according to the company.
The multi-site, single-treatment study evaluated the pharmacokinetics (PK), pharmacodynamics (PD), safety, and tolerability of Anaphylm.
A total of 32 patients completed the study. The PK results were consistent with previous adult studies. Additionally, Anaphylm was well-tolerated, with no serious adverse events reported.

“These results demonstrate that Anaphylm maintains its consistent PK profile in pediatric patients between the ages of seven and seventeen and weighing greater than thirty kilograms,” Barber stated.

He added, “These data are an important component of the FDA review process and could enable Anaphylm to have a label that includes this pediatric patient population. We continue to prepare for commercial readiness and plan to launch Anaphylm in the first quarter of 2026, if approved by the FDA.”
Aquestive has submitted the Anaphylm NDA to the FDA and anticipates potential acceptance during the second quarter of 2025.
References:
1. Aquestive Therapeutics. Aquestive Therapeutics Announces Positive Topline PK Results from its Pediatric Study and Completes the NDA Submission for Anaphylm™ (epinephrine) Sublingual Film. Global Newswire. April 1, 2025. Accessed April 1, 2025. https://www.globenewswire.com/news-release/2025/04/01/3053173/0/en/Aquestive-Therapeutics-Announces-Positive-Topline-PK-Results-from-its-Pediatric-Study-and-Completes-the-NDA-Submission-for-Anaphylm-epinephrine-Sublingual-Film.html
2. Aquestive Therapeutics. Aquestive Therapeutics Reports Fourth Quarter and Full Year 2024 Financial Results and Provides Business Update. Aquestive.com. April 1, 2025. Accessed April 1, 2025. https://investors.aquestive.com/news-releases/news-release-details/aquestive-therapeutics-reports-fourth-quarter-and-full-year-2024

Navigating challenges in pediatric trial conduct: integrating bayesian sequential design with semiparametric elicitation for handling primary and secondary endpoints

2025-03-31 - 2025-03

This study presents a Bayesian Adaptive Semiparametric approach designed to address the challenges of pediatric randomized controlled trials (RCTs). The study focuses on efficiently handling primary and secondary endpoints, a critical aspect often overlooked in pediatric trials. This methodology is particularly pertinent in scenarios where sparse or conflicting prior data are present, a common occurrence in pediatric research, particularly for rare diseases or conditions.

Method
Our approach considers Bayesian adaptive design, enhanced with B-Spline Semiparametric priors, allowing for the dynamic updating of priors with ongoing data. This improves the efficiency and accuracy of the treatment effect estimation. The Semiparametric prior inherent flexibility makes it suitable for pediatric populations, where responses to treatment can be highly variable. The design operative characteristics were assessed through a simulation study, motivated by the real-world case of the REnal SCarring Urinary infEction Trial (RESCUE).

Result
We demonstrate that Semiparametric prior parametrization exhibits an improved tendency to correctly declare the treatment effect at the study conclusion, even if recruitment challenges, uncertainty, and prior-data conflict arise. Moreover, the Semiparametric prior design demonstrates an improved ability in truly stopping for futility, with this tendency varying with the sample size and discontinuation rates. Approaches based on Parametric priors are more effective in detecting treatment efficacy during interim assessments, particularly with larger sample sizes.

Conclusion
Our findings indicate that these methods are especially effective in managing the complexities of pediatric trials, where prior data may be limited or contradictory. The flexibility of Semiparametric prior design in incorporating new evidence proves advantageous in addressing recruitment challenges and making informed decisions with restricted data.

Strategic Pathways for Yale Pediatrics

2025-03-28 - 2025-03

"This is an exciting time for Yale Pediatrics. Healthcare is undergoing a major transformation focused on innovative ways to care for populations of patients. Advances in treatments, such as gene editing, are becoming a reality because of cutting edge research efforts. Yet many gaps remain in our knowledge, implementation, and dissemination of approaches and potential treatments of many health problems children face, not the least of which is pervasive inequities in how populations of patients are treated and in their health outcomes. With vexing problems comes the opportunity for innovative and transformational solutions. That dynamic is the reason for our development of this strategic plan. Our department has grown significantly in many ways - in the number of faculty, clinical programs, grant funding, diversity of perspectives and experiences, and new leaders. We now are taking the time to reflect on our shared values and aspirations to develop a roadmap towards addressing the many challenges we and our patients face. We are dreaming big! I am extremely impressed with the dedication and accomplishments of our faculty and trainees. By focusing our efforts on some big and audacious goals, there is no limit to what we can achieve and the impact we can have. Achieving these goals will accelerate our efforts of realizing our vision of Yale Pediatrics being a world leader in improving the health of children and adolescents by transforming and integrating pediatric education, research, advocacy, and clinical care. I cannot wait for us to get started!"

Music intervention for neurodevelopment in the pediatric population: a systematic review and meta-analysis

2025-03-26 - 2025-03

This systematic review and meta-analysis aim to summarize and analyze current research on the effects of MI on neurodevelopment in children of all ages and health statuses to provide a reference for music therapy (MT) and its clinical research in pediatrics. We conducted a comprehensive search of PubMed, Embase, Web of Science, and Cochrane Library databases up to December 2023, focusing on randomized controlled trials that evaluated neurodevelopmental outcomes following MI. Our analysis, which included seven studies involving 337 participants, employed standardized mean difference (SMD) calculations to assess outcomes across multiple neurodevelopmental scales. While no significant cognitive improvements were observed on the Bayley-III scale, positive effects were noted in language, motor skills, and IQ scores when assessed via Gesell, CSBQ, and IQ scales. Our research underscores the potential of music intervention in the process of children’s neurodevelopment, including cognitive function, language, motor and IQ. Based on the limitations, researchers should carefully design their MI protocols, ensuring standardization and avoiding probable confounding factors such as regional specificity, age ranges and special populations, it will contribute to more robust results and improve the comparability of findings across studies.

How dangerous is a measles outbreak?

2025-03-24 - 2025-03

Measles is a very contagious and dangerous disease — but it’s also preventable.

Thanks to a coordinated and highly effective effort to deliver the measles, mumps and rubella (MMR) vaccine, it was declared eliminated from the U.S. in 2000.

But increasing numbers of measles infections — driven by misinformation about vaccines and reduced vaccination rates in some communities — have been reported over the last five years.

As of mid-March 2025, there were 301 new cases of measles and 2 measles-related deaths in the U.S., surpassing the 285 cases reported for all of 2024, according to the Centers for Disease Control and Prevention.

As a pediatric infectious diseases specialist at UChicago Medicine, I want to share important facts about measles prevention that can help keep you and your loved ones safe.

What is measles — and what are the symptoms and complications?
Measles is an infection caused by a virus. Symptoms of measles include fever, cough, runny nose, rash and red eyes ("pink eye," or conjunctivitis), and they typically show up eight to 12 days after initial exposure to the virus.

The characteristic measles rash appears three to five days after the first symptoms appear. Additional complications, such as ear infections, pneumonia and diarrhea, can occur.

A serious complication of measles is acute encephalitis, or inflammation of the brain, which can result in permanent brain damage in one of every 1,000 cases. In the U.S., death from neurologic or respiratory complications of measles occurs in one to three of every 1,000 cases.

In addition, there is a late-onset complication called subacute sclerosing panencephalitis (SSPE). This rare, degenerative and fatal central nervous system disease can occur seven to 11 years after a primary measles infection, with the highest rates seen in children infected before 2 years of age.

How contagious is measles and how long is a person contagious?
Measles is one of the most highly communicable infections. It’s spread through direct contact with infectious droplets or through the air. Patients with a measles infection are contagious from four days before the rash through four days after the rash appears.

For people who are not vaccinated and have never been exposed to the virus before, the likelihood of contracting measles is 90% in close-contact settings.

How effective is the measles vaccine? Can I get the measles if I’ve been vaccinated?
More than 97% of people who receive two doses of the MMR vaccine develop immunity to measles. The second dose of the vaccine protects those who fail to respond to their primary dose.

While it is possible to contract measles after two doses of the vaccine, it is very uncommon. Importantly, immunization is not harmful to those with established measles immunity and immunized persons do not transmit measles vaccine virus.

When should children get vaccinated for measles?
Children should be vaccinated after their first birthday and receive an additional dose of MMR vaccine before entering kindergarten (when they’re 4 to 6 years old). The second dose provides additional protection to the approximately 5% of people who do not respond to their first dose.

Given the risk of measles associated with international travel, a first dose can be given as early as 6 months of age for infants who will be traveling abroad. This does not, however, replace the need for additional MMR doses administered at the traditional timepoints of over 1 year of age and 4 to 6 years of age.

Should adults get vaccinated for measles?
Adults are considered immune to measles if they have documentation of at least one dose of MMR vaccine on or after their first birthday, documentation of two doses of MMR vaccine for individuals at high risk, laboratory evidence of measles immunity, laboratory confirmation of measles infection, or birth before 1957.

Adults who do not meet these criteria should be vaccinated with at least one dose of the MMR vaccine — unless their physician recommends against it due to special circumstances, such as severely compromised immunity.

During measles outbreaks, health departments may provide additional recommendations to protect their communities, including a second dose of MMR for adults who have received only one dose previously. Currently, a third dose of MMR vaccine during measles outbreaks is not recommended.

How dangerous is measles?
Fortunately, for most people, measles is not life-threatening — but the problem is that you can't predict who will become seriously ill. Three in 1,000 people who contract measles will die. For those who are vulnerable due to preexisting conditions that impact their immune system, measles can be even more dangerous.

However, if everyone is vaccinated, no one will die. Before the vaccine, there were up to 500 U.S. deaths each year from measles, and now it's completely preventable.

With vaccine-preventable illnesses, most people don't get sick, but that doesn’t mean it’s smart to forego the vaccine. Why take the risk when the protection is safe and effective?

Should vitamin A and cod liver oil be used to prevent measles infection?
The only way to prevent measles is with the MMR vaccine. Vitamin A does not prevent measles, and in high doses, it can lead to toxic side effects such as nausea, vomiting, headache, blurred vision, liver damage and coma.

Treatment with once-daily vitamin A for two days is recommended for those with an established diagnosis of a measles infection to help the illness from becoming more severe.

Cod liver oil contains high amounts of vitamin A, vitamin D and fatty acids, which can be harmful if intake exceeds the recommended daily amount; it should not be administered to prevent measles infection.

The Mount Sinai Health System on Monday announced its new Center for Artificial Intelligence in Children’s Health, which will explore new ways to develop, test and embed AI directly into pediatric healthcare to enable earlier diagnoses, preventive approac

2025-03-24 - 2025-03

WHY IT MATTERS

To accelerate AI research and personalized treatment in children’s health, Mount Sinai said it will create an integrated data infrastructure and advance multimodal AI research and leverage computer-augmented imaging, multi-omics research, rare disease identification and pharmacogenomics to enhance health economics and care delivery.

"While AI has advanced at a remarkable pace in many areas of medicine, pediatric medicine has unfortunately lagged due to stricter privacy considerations, more complicated regulatory pathways and limited data infrastructure," said Benjamin Glicksberg, a digital health and clinical informatics expert, in a statement.

Glicksberg, who will also serve as associate professor of artificial intelligence and human health at the Icahn School of Medicine at Mount Sinai, will lead the center. He'll focus on enabling the health system to offer more precise diagnostics and personalized treatments for young patients, according to Dr. Brendan Carr, Mount Sinai's chief executive officer and Dr. Kenneth L. Davis Distinguished Chair.

The Center, established under The Mindich Child Health and Development Institute, will also spearhead clinical trials at Mount Sinai Kravis Children’s Hospital to enhance AI-driven diagnostics, predictive modeling and real-time monitoring.

"The Center for AI in Children’s Health underscores Mount Sinai’s commitment to pioneering AI-driven technologies that will enable Mount Sinai to deliver world-class care to our children," Carr said in a statement.

"Our kids are our future, and under Dr. Glicksberg’s leadership, the Center for AI in Children’s Health will advance children’s health outcomes for generations to come," added Dr. Girish Nadkarni, Mount Sinai's Windreich Department of Artificial Intelligence and Human Health chair, which is co-sponsoring the new Center.

THE LARGER TREND

Health systems have been using AI imaging tools to improve patient access and treatment and to predict diseases.

Pediatrics researchers at the Children's Hospital of Philadelphia developed a deep-learning model to enhance their understanding of disease progression and made the AI available to others for tumor analysis.

The model can learn patterns and make predictions or classifications faster than previous approaches, CHOP researchers said.

The "approach could redefine how we understand complex tissues at the cellular level, paving the way for transformative breakthroughs in healthcare," Kai Tan, the study's lead author and a professor in the Department of Pediatrics at CHOP, said in December.

ON THE RECORD

"This new Center is dedicated to addressing these challenges by safely developing, testing, and embedding AI directly into child healthcare – enabling earlier diagnoses, preventive measures, computer-augmented imaging for complex conditions, expedited drug discovery and highly personalized treatment plans," said Glicksberg in a statement.

"By harnessing the power of advanced data science and clinical expertise, we aim to usher in a new era for child healthcare – delivering faster diagnoses, personalized treatments and transformative outcomes," added Nadkarni.

Consortium for Technology & Innovation in Pediatrics Adds Avulux Into Its Portfolio

2025-03-24 - 2025-03

MIAMI—Avulux, a migraine and light sensitivity lens manufacturer, has announced that the Consortium for Technology & Innovation in Pediatrics (CTIP), which connects clinicians, engineers, regulators, patients and businesses across its network of 25 organizations spanning 8 states, has added Avulux into its product portfolio. Avulux lens technology filters up to 97 percent of migraine-triggering light in the blue, red and amber spectrums, the company notes, while letting in over 70 percent of green light, providing support for people living with migraine and light sensitivity. In the pediatric population, eyecare and migraine present unique challenges, the announcement advises, as children struggle to describe symptoms, complicating diagnoses between visual disturbances and migraine.
“Migraine in children is often underdiagnosed and undertreated, despite the profound impact on development, education and overall well-being," said Juan Espinoza, MD, director and principal investigator of CTIP. "At CTIP, we are committed to supporting innovations like Avulux that offer non-invasive and effective solutions to improve the lives of pediatric patients.”
"Visual symptoms such as light sensitivity are often the most disabling component of migraine for children, thus optometrists are well positioned to make a real difference in their lives," said Charles Posternack, MD, CEO of Avulux. "Migraine prevalence in children also increases with age, with up to 24 percent of teenagers experiencing at least one migraine attack. Avulux looks forward to joining CTIP and contributing to advancing pediatric health."
According to the National Institutes of Health (NIH), about 1 in 10 children experience migraine, and up to 18 percent of patients in the pediatric emergency room are found to be migraine related. Further, migraine can also significantly impair school performance and quality of life in children.

Free Drug Concepts: A Lingering Problem in Drug Discovery

2025-03-21 - 2025-03

Free drug concepts underpin all pharmacokinetic–pharmacodynamic relationships and therefore should be a foundational element of drug discovery projects. The concepts are not new and have been widely accepted, (1−6) yet there remains misunderstanding of the principles, and confusion as to how best to apply them in drug design.
The lack of impact of plasma protein binding modulation on overall drug quality was discussed by Liu et al. in 2014 (6) in this journal. However, the current authors’ experience working within the industry, and reviewing drug discovery projects in academic and charitable research institutions, is that free drug concepts are not fully embedded in many organizations . We also see that many published strategies aimed at “improved DMPK properties” are not founded on free drug principles and thus are flawed and misleading. This leads to the propagation of incorrect data interpretation and inefficient optimization strategies.
Why Does This Continue to Be Such a Problem?Click to copy section link
Potential reasons include:
The concepts, while not complex, can be counterintuitive.
The processes governing free drug concentrations in vivo are different from those in in vitro assays but are often assumed to be the same.
The most common calculation performed (conversion of total drug concentration to free drug concentration) requires an equation in a form that, while mathematically correct, has led to a mechanistic misunderstanding. This misunderstanding results in many published studies being incorrect in their use of pharmacokinetic data as drivers of compound optimization.
Pharmacokinetic training focuses on total drug concentrations and parameters, and these also dominate clinical pharmacokinetics where the focus is on a single compound, and for which plasma binding is usually constant. Compound optimization in drug discovery is anchored on the understanding of unbound drug exposure and its relationship to target engagement; thus, comparisons between molecules requires the use of unbound exposure and unbound PK parameters.
Even today, authors use the term “Free drug hypothesis”, as though there is some doubt as to its validity. It is not a hypothesis; it is a fundamental principle.
What Are the Key Free Drug Concepts?
Unbound drug concentrations at the target site determine the degree of target engagement
Unbound concentrations in vivo are not determined by the extent of plasma or tissue binding; instead, plasma and tissue binding determine the concentrations of bound drug
Unbound drug concentrations in vivo are determined by the rate of drug input (absorption) and the rate of elimination (intrinsic clearance)
At equilibrium, free drug concentrations in cytosol approximate those in plasma (with a few specific exceptions, described below

Multibagger Pharma Company Expands into New Territory with Innovative Pediatric Solution

2025-03-22 - 2025-03

Piramal Pharma Limited, a prominent player in the pharmaceutical industry, has achieved a significant milestone with its Piramal Critical Care division and BrePco Biopharma Limited. The two companies have received approval from the UK Medicines and Healthcare Products Regulatory Agency (MHRA) for Neoatricon®, a pioneering pediatric formulation of Dopamine Hydrochloride.
This marks the first authorized treatment specifically designed for hypotension in neonates, infants, and children. Neoatricon® addresses a critical gap in pediatric care by providing precise dosing, reducing the risk of under- or overdosing, and minimizing preparation time in emergency settings. The commercialization rights for Neoatricon® have been secured for the EU, UK, and Norway, with Piramal Critical Care responsible for distribution. The approval is a testament to Piramal's strategic focus on expanding into new therapeutic areas and improving health outcomes for pediatric patients. The collaboration with BrePco Biopharma has enabled the introduction of this innovative solution, setting a new standard in pediatric critical care.
Piramal Pharma Limited is a key player in the pharmaceutical sector, operating through three major segments: Contract Development and Manufacturing Organisations (CDMO), Complex Hospital Generics (CHG), and India Consumer Healthcare (ICH). The company has a significant presence in North America, Europe, and India, with 15 CDMO sites and a strong foothold in regulated markets. Piramal Pharma is among the top CDMO players globally and the fourth-largest player in inhalation anaesthesia worldwide.
The company has a diverse portfolio, with a focus on high-quality products and strategic partnerships. It has achieved substantial growth through mergers and acquisitions and aims to become a global leader in pharma, health, and wellness by FY30. With a strong emphasis on quality control and regulatory compliance, Piramal Pharma continues to expand its capabilities and market reach, making it a compelling choice for investors. Piramal Pharma Limited has a market capitalization of over Rs 30,000. The stock has delivered 85 per cent in 1 year and gave multibagger returns of 240 per cent in 2 years.

The diagnostic accuracy of presepsin for late-onset neonatal sepsis: a multicenter prospective cohort study

2025-03-21 - 2025-03

Background
Antibiotic overtreatment in infants is a significant problem, due to lack of accurate diagnostic tools for late-onset neonatal sepsis (LONS). We aimed to investigate the diagnostic accuracy of presepsin for LONS at initial suspicion.
Methods
In this multicenter prospective observational cohort study, we consecutively included all term and preterm infants who started on antibiotics empirically for a nosocomial LONS suspicion. Presepsin concentrations were determined at initial LONS suspicion before antibiotic initiation (t = 0), and 12 and 24 h afterwards. Diagnostic accuracy measures for LONS were calculated.
Results
A total of 63 episodes of suspected LONS (32 classified as LONS, including 23 culture-positive and 9 culture-negative episodes) in 50 infants were included. Presepsin concentrations were significantly higher in LONS cases compared with non-LONS at all time-points. The AUC for all LONS cases at t = 0 was 0.77 (95% CI 0.66–0.89) and 0.80 (95% CI 0.67–0.92) for culture-positive LONS cases only.
Conclusion
Presepsin seems to have insufficient accuracy as single biomarker to serve as a biomarker for ruling out LONS in infants suspected of LONS. Future larger studies are warranted to validate our findings and to investigate the clinical impact of presepsin, in combination with other biomarkers, as diagnostic tool to facilitate decision-making regarding the initiation of antibiotics, thereby supporting antibiotic stewardship.
Impact
Presepsin seems to have insufficient accuracy as single biomarker for the decision to treat or not at initial suspicion of late-onset neonatal sepsis.
This is the first prospective observational cohort study on the diagnostic accuracy of presepsin for late-onset neonatal sepsis consecutively recruiting all infants suspected of late-onset neonatal sepsis, minimizing bias.
Future larger studies are warranted to investigate the clinical impact of presepsin in facilitating decision-making regarding the initiation of antibiotics in infants, thereby supporting antibiotic stewardship.

Pitt researchers develop a new way to profile pediatric brain tumors

2025-03-20 - 2025-03

Researchers and pediatric neurosurgeons at the University of Pittsburgh School of Medicine and UPMC Children's Hospital of Pittsburgh developed a new way to profile brain cancers in children, paving the way for improved diagnostics and treatments.
Today in Science Translational Medicine, researchers describe a diagnostic platform that could classify brain tumors based on the body's cancer-fighting immune response. This approach, which is complementary to traditional microscopic and genetic cancer cell analyses, establishes an opportunity to tailor cancer therapies to each patient's unique immune response and harness the success of immunotherapies that revolutionized the treatment of childhood leukemias.
"The success of T cell-based therapies for non-brain tumors, including childhood leukemias, suggests tremendous potential for brain cancers," said senior author Gary Kohanbash, Ph.D., assistant professor of neurological surgery at Pitt. "Having access to an unprecedented dataset of pediatric tumors and new bioinformatic tools allowed us to investigate how T cell immune response and clonal expansion could be used as markers for treatment classification and prognosis independent of other diagnostic tools."
As part of the study, the researchers profiled nearly 1,000 pediatric brain tumor samples, which were collected through the Children's Brain Tumor Network (CBTN), a medical research consortium of 35 medical centers across the nation and globally. The study was first to look at the T cell clonal repertoire and expansion in this sample group.
The group observed that very aggressive tumor types are associated with less T cell expansion than less aggressive ones, suggesting that clonality can inform patient outcomes across tumor types. On the other hand, studying T cell response can shed light on which antigens on the surface of cancer cells can be exploited therapeutically, offering avenues for development of cancer-antigen peptide immunotherapy.
"Ultimately, we hope for a future where clonal T cell expansion is incorporated into pediatric cancer diagnosis," said co-author Ian Pollack, M.D., distinguished professor of neurological surgery at Pitt, and a founding institutional principal investigator of CBTN. "UPMC Children's Hospital is committed to supporting brain tumor research and developing new life-saving treatments, and we think that this landmark study represents a foundational shift to how the field will be considering pediatric tumors in the future."

Pediatric investigation review explores the link between anemia and retinopathy of prematurity

2025-03-18 - 2025-03

Retinopathy of prematurity (ROP) is a major cause of vision impairment in preterm infants, particularly those born weighing ≤1,250 g. Anemia is commonly observed in these infants, with nearly 90% of extremely low birth weight (ELBW) infants requiring at least one blood transfusion. The primary cause of anemia in this population is frequent blood draws needed for medical monitoring during critical illness. Additionally, preterm infants miss the crucial third trimester of pregnancy, during which 70% of maternal iron is transferred to the fetus, placing them at an increased risk for iron deficiency. As anemia and ROP frequently co-occur in infants, understanding the potential link between these two conditions is essential for improving clinical outcomes and treatment strategies.
A recent review published online in Pediatric Investigation on 03 February 2025 explores the complex association between anemia and ROP, focusing on the role of iron deficiency and its potential molecular mechanisms in ROP development. The review highlights several factors that may underlie this association, including tissue hypoxia, oxidative stress, inflammation, and the timing of anemia onset relative to the different phases of ROP—all of which contribute to retinal damage. “Given the high prevalence of anemia and iron deficiency in preterm infants, understanding these mechanisms is vital for improving prevention and treatment strategies for ROP,” explains corresponding author Dr. Ellen C. Ingolfsland from the Department of Pediatrics, Division of Neonatology, University of Minnesota, USA.
The review begins with the discussion on the potential role of iron deficiency in the development of ROP. Anemia reduces oxygen delivery to the retina, and iron deficiency further exacerbates hypoxia by inhibiting the prolyl hydroxylase domain, which normally activates hypoxia-inducible factor 1-alpha (HIF-1α), a protein essential for the body’s response to low oxygen levels or hypoxia. Under conditions of hypoxia and iron deficiency, HIF-1α is translocated to the nucleus, where it activates the transcription of angiogenic factors such as vascular endothelial growth factor (VEGF). While VEGF promotes normal angiogenesis under physiological conditions, its overexpression in ROP leads to pathological neovascularization, contributing to the abnormal retinal vessel growth characteristic of the condition.
In addition to hypoxia, oxidative stress and inflammation play significant roles in the development of ROP. Preterm infants, with lower antioxidant levels, are more vulnerable to reactive oxygen species (ROS) produced during oxidative metabolism. The accumulation of ROS in the retina, particularly during the first phase of ROP, damages endothelial cells and delays retinal vasculature development. Anemia and iron deficiency further increase ROS production, accelerating retinal damage. Additionally, anemia triggers a proinflammatory response, leading to higher levels of cytokines such as interferon-gamma and tumor necrosis factor-alpha, which further exacerbate retinal injury.
Furthermore, the review highlights a paradox in the relationship between anemia and ROP severity. It suggests that early anemia, particularly in the first few weeks of life, is associated with a significantly increased risk for ROP development. Whereas chronic, non-transfused anemia may decrease this risk with iron deficiency having a protective effect. This paradox is likely due to the timing, severity, and treatment of anemia, which vary across clinical settings. The review points out that transfusion protocols and iron supplementation strategies differ significantly between neonatal intensive care units, complicating efforts to determine the precise role of anemia in ROP.
“In light of these findings, our review emphasizes the need for well-designed randomized controlled trials to clarify the impact of anemia on ROP and to explore the molecular mechanisms involved. Such studies should consider factors such as the timing, severity, and management of anemia, along with other confounding factors like comorbidities,” explains Dr. Ingolfsland.
Overall, while anemia remains a common and significant issue for preterm infants, its exact role in the development and progression of ROP is still not fully understood. “Our review calls for further focused research to better understand the molecular mechanisms linking iron deficiency anemia to ROP. Addressing this knowledge gap is essential for developing more effective prevention and treatment strategies that could improve clinical outcomes for vulnerable premature infants,” concludes Dr. Ingolfsland.

Upcoming Opportunities in Pediatric Telehealth Solutions Market: Future Trend and Analysis of Key Segments and Forecast 2025 to 2032 | Teladoc Health, Amwell (formerly American Well)

2025-03-17 - 2025-03

A newly released report on the "Pediatric Telehealth Solutions Market 2025" provides a comprehensive view of the industry with market insights on the competitive scenarios and market segments with complete representation through graphs, tables, and charts to study the market easy to use and compare the numbers and user-friendly. The Pediatric Telehealth Solutions Market research report is the hub of market information, which precisely expounds on critical challenges and future market growth prospects. Also, The research study provides a complete qualitative and quantitative analysis to help shareholders obtain a thorough grasp of the Pediatric Telehealth Solutions Market and its crucial dynamics.
Moreover, The report provides a professional in-depth examination of the Pediatric Telehealth Solutions Market's current scenario, CAGR, gross margin, revenue, price, production growth rate, volume, value, market share, and growth are among the market data assessed and re-validation in the research. The report will also cover key agreements, collaborations, and global partnerships soon to change the dynamics of the market on a global scale. Detailed company profiling enables users to evaluate company shares analysis, emerging product lines, the scope of New product development in new markets, pricing strategies, innovation possibilities, and much more.
The purpose of this market analysis is to estimate the size and growth potential of the market based on the kind of product, the application, the industry analysis, and the area. Also included is a comprehensive competitive analysis of the major competitors in the market, including their company profiles, critical insights about their product and business offerings, recent developments, and important market strategies.

Biofeedback Therapy: An Effective, Noninvasive Approach to Voiding Dysfunction in Kids

2025-03-14 - 2025-03

Voiding dysfunction is a very common problem in children, accounting for an estimated 40% of visits to pediatric urology. Biofeedback therapy is one of the many tools available to providers for the management of pediatric voiding dysfunction.
Lynn Woo, MD, Section Director of Pediatric Urology at Cleveland Clinic, explains, “Biofeedback therapy is a noninvasive, child-friendly approach to help target the pelvic floor muscle groups and teach children how to isolate and control these muscles,” she says. “It can aid in proper relaxation and contraction, which can help improve a number of urinary symptoms including urgency, frequency, involuntary leakage and incomplete bladder emptying.”
The video game-inspired platform allows providers, patients and their families to visualize the child’s muscle activity in real-time through adhesive sensors affixed to the child’s skin. Exercises and games are then “played” by active contraction and relaxation of the pelvic muscles. The colorful graphics and achievement of various tasks motivate kids to reach muscle activity goals. In doing this, they are retraining these muscle groups and learning how to store and empty urine normally.
The technology can be individualized based on the type of voiding dysfunction, the age of the patient, and their attention span.
Biofeedback in children is effective, with data showing that more than 50% of patients have improved symptoms with treatment. While not every patient may experience the same amount of success with the program, it can provide some helpful tools for children and their families struggling with voiding problems and give them a way to be proactive in improving their symptoms.
Voiding dysfunction: A complicated pathway
The term “voiding dysfunction” encompasses a number of symptoms and common urinary complaints, including recurrent urinary tract infections, incontinence, and incomplete or difficult bladder emptying.
“We often take for granted how our bodies control urination, since it is considered an automatic process that we learn with toilet training,” Dr. Woo explains. “But storing and emptying urine properly is a complex pathway involving an elastic bladder, normal sensation of bladder fullness, and appropriate pelvic muscle control.”
Voiding dysfunction is learned abnormal behavior in which the normal coordination is disrupted, she says. “Sometimes, this is due to an initial infection that causes pain or relates to a history of constipation and discomfort in passing hard stools. Children then adopt withholding behaviors, which lead to involuntary contraction of their pelvic floor muscles at the wrong times,” explains Dr. Woo. Once this behavior is in place, it can create a vicious cycle that leads to poor emptying, urine accidents, and more infections.
Considerations for care
All children with suspected voiding dysfunction need a comprehensive evaluation, including a complete patient history, urinalysis, and potentially imaging or functional studies to determine an underlying voiding dysfunction. Cleveland Clinic’s pediatric urology team performs a thorough assessment for each patient, provides education for families and will devise an individualized treatment plan that might include behavioral modifications (termed “urotherapy”), medications, and pelvic floor therapy in the form of biofeedback.
“While some patients may require medicines or more invasive interventions, the vast majority can improve their symptoms with urotherapy and/or biofeedback exercises. I think parents and providers alike would generally prefer to begin with conservative measures and, if possible, avoid drugs or surgery,” notes Dr. Woo.
Early intervention is key
Recurrent urinary tract infections or lack of consistent urinary control after the age of 7 may be signs of voiding dysfunction, and these children should have additional evaluation. She stresses, “It’s important to be proactive about these issues to prevent long-term urinary tract complications.”

Updates on the Use of Biosimilars for Pediatric Patients With Rheumatic Disease

2025-03-12 - 2025-03

Although traditional disease-modifying antirheumatic drugs (DMARDs) are effective, their high cost has been a barrier to widespread accessibility for some patients. Now, as patents for these biologics begin to expire, the emergence of biosimilars has the potential to improve access to care.1
Biosimilars are highly similar to the reference product in clinical potency and toxicity but may have slight differences in components that do not appear to affect their effectiveness or toxicity.2 Observational studies consistently demonstrate that biosimilars are comparable to the reference product with regard to safety and effectiveness.1,2 Regardless, several barriers, including limited pediatric studies and patient misconceptions, have limited their clinical adoption in pediatric populations.
A team of researchers in Turkey conducted a comprehensive literature review evaluating studies that address the use of biosimilar products in pediatric rheumatic diseases. They included a total of 10 studies in their review, which aimed to provide insight and guidance to clinicians regarding biosimilars’ safety, efficacy, and economic impact in pediatric patients with rheumatic diseases.1
They first looked at the biosimilar development process, which can be nearly 10% more cost-effective than the process used for the original products, largely because the FDA approves biosimilars based on extrapolation. Biosimilars can potentially come to market with only a single randomized clinical trial designed to show equivalent efficacy and immunogenicity between the biosimilar and the reference product. To obtain regulatory approval, the FDA requires a pharmacokinetic/pharmacodynamic study in humans, either healthy volunteers or patients, and at least 1 randomized controlled trial. The design of the randomized controlled trials should be based on an equivalence margin related to the primary end point difference between active treatment and placebo. The study authors recommend that clinicians review the FDA Purple Book to obtain more information on the comparability programs associated with biosimilar products.1
The literature review also discussed the safety and efficacy of biosimilar products. Although they found a lack of clinical trials involving children and young adults, the few studies reviewed showed similar efficacy and safety between the reference product and the biosimilar. One of the main challenges with the uptake of biosimilars is that randomized controlled trial data are generally limited, despite robust data for the original product. Additionally, when biosimilars are FDA-approved, approval for a single indication based on similarity and efficacy of the original molecule, along with a single randomized clinical trial, is typically sufficient. These factors can contribute to the nocebo effect, which the authors describe as expectations that lead to negative outcomes, thereby playing a potential role in the discontinuation of biosimilars.1
The authors conclude that more research is needed on the use of biosimilars in pediatric patients with rheumatic diseases. However, they acknowledge that these therapies hold significant potential in this population, particularly with regard to cost savings. They reiterate that observational studies have continued to consistently demonstrate that biosimilars are comparable to original biologics with no significant difference in treatment discontinuation or disease activity. Providing ongoing education to both patients and providers, maintaining transparent communication, and conducting further research will enhance the success of biosimilars in pediatric populations in the future.

Juan Carlos Salazar named chair of Pediatrics and physician-in-chief for Monroe Carell Jr. Children’s Hospital at Vanderbilt

2025-03-11 - 2025-03

Juan Carlos Salazar, MD, MPH, professor and chair of the Department of Pediatrics at the University of Connecticut School of Medicine and Executive Vice President and physician-in-chief at Connecticut Children’s, has been named the chair of the Department of Pediatrics at Vanderbilt University Medical Center and physician-in-chief for Monroe Carell Jr. Children’s Hospital at Vanderbilt. Effective Aug. 15, his appointment will include professor of Pediatrics and the James C. Overall endowed chair.
A nationally and internationally recognized pediatric infectious diseases specialist and investigator, Salazar joins VUMC with more than three decades of experience in academic leadership, medical education and NIH-funded research. During his tenure with Connecticut Children’s and the University of Connecticut School of Medicine, his leadership team has driven significant growth in clinical care, established a foundation for a new pediatric research institute and spearheaded significant increases in health services and research funding.
“Monroe Carell is a destination of hope for children and families throughout our region and across the nation seeking the highest level of care and is anchored by the broad and cutting-edge programs in our nation-leading Department of Pediatrics. Dr. Salazar, having served for more than a decade in leadership roles with Connecticut Children’s, is ideally suited to help us further advance our ambitious goals for child health,” said Jeff Balser, MD, PhD, President and CEO of VUMC and Dean of Vanderbilt University School of Medicine.
“As we welcome Dr. Salazar, I want to express my gratitude to Dr. William Cooper for his excellent service as interim chair and to the members of the search committee for their outstanding work.”
Vanderbilt’s Department of Pediatrics currently comprises some 460 primary faculty, 270 community-based volunteer faculty, 82 clinical fellows, 127 residents and over 300 administrative and research staff. The department is currently ranked No. 1 in the nation for NIH funding among departments of pediatrics.
The department provides care at 31 off-campus locations in a statewide network that includes clinics, after-hours care, emergency room coverage, specialty care, and support for several
nurseries and neonatal intensive care units located within other hospitals across Tennessee. The department’s training programs continue to attract the best and brightest trainees from across the country and internationally.
“Monroe Carell Jr. Children’s Hospital at Vanderbilt and our pediatric specialists fulfill a crucial role within our health system. Continued growth of the Department of Pediatrics, along with expanding the hospital’s unique offerings is essential to our mission,” said C. Wright Pinson, MBA, MD, Deputy CEO and Chief Health System Officer for VUMC. “I want to welcome Dr. Salazar to this important role and look forward to working with him as we seek to enhance these services.”
Serving more than 1,800 children and families daily, Monroe Carell is completing construction on two additional floors, and by 2027 will have over 400 beds for children. Patients from 48 states seek care from an extraordinary range of pediatric specialties and subspecialty services, including many only available at select national centers.
“I am excited to welcome Dr. Salazar to our leadership team at Monroe Carell and VUMC. He comes to us with deep experience in both building and leading mission-based clinical, research and training programs. Throughout the interview process, I appreciated his spirit of collaboration and passion about these missions and his true commitment to the people who carry out these endeavors. I look forward to partnering with him to continue to advance our care for children and families, support discovery and ensure the best possible training for our future pediatric providers,” said Meg Rush, MD, MMHC, professor of Pediatrics and President of Monroe Carell Jr. Children’s Hospital at Vanderbilt. “I am grateful to Dr. Cooper for his close partnership as we await Dr. Salazar’s arrival later this year.”
Salazar, who also led the Division of Pediatric Infectious Diseases and Immunology at Connecticut Children’s and is the current director of Connecticut Children’s Pediatric, Youth and Family HIV Program, has received NIH grants to study the human innate immune response to the causative agents of Lyme disease and syphilis. He is currently funded by the NIH to develop a first-of-its-kind syphilis vaccine. Most recently his team received funding from the National Institute of Child Health and Human Development to study the pathogenesis of COVID-19 and the multisystem inflammatory syndrome in children (MIS-C), a rare but serious condition associated with COVID-19.
Salazar is a current recipient of Ryan White Care Act federal funding to coordinate and provide statewide HIV treatment and prevention services in Connecticut for women and children. Having led several NIH and pharmaceutical industry-sponsored pediatric and adolescent HIV clinical trials and long-term follow-up studies, he is frequently interviewed by local and national news media regarding pediatric health and infectious diseases topics.
“It has been the greatest honor of my career to serve the faculty, the health care team members and most importantly the children of Connecticut. My leadership has been informed by over three decades of experience in pediatric health care, medical education, research and community advocacy,” said Salazar.
“My vision as the new chair of the Department of Pediatrics at VUMC and physician-in-chief at Monroe Carell Jr. Children’s Hospital at Vanderbilt is to transform the landscape of pediatric health care through innovative, compassionate and comprehensive care for every child and family served by the health system. I will do so by inspiring confidence, involving our community, training the best generation of pediatricians, and advancing pediatric research and innovation. The goal is to provide a brighter, healthier future for the children and communities we serve and be recognized as one of the best children’s health systems in the nation.”

Enhancing pediatric health evaluations through improved PROMs

2025-03-10 - 2025-03

Children's health assessments differ significantly from adults due to varying cognitive abilities, language skills, and developmental stages. Patient-reported outcome measures (PROMs), which are self-reported questionnaires, offer valuable insights into a child's health status, but their design and implementation in pediatrics are complex. Challenges include determining the appropriate age for self-reporting and ensuring the reliability of proxy reports from parents or caregivers. Previous studies have shown that children as young as 8 can reliably report their health, but younger children often require proxy reports, which may not accurately reflect their experiences. Based on these challenges, there is a pressing need for more in-depth research to optimize the use of PROMs in pediatric healthcare.
A team of researchers from Children's Hospital of Chongqing Medical University and other institutions published a review (DOI: 10.1002/pdi3.77) on June 1, 2024, in Pediatric Discovery, providing an extensive overview of systematic reviews on pediatric PROMs. The study analyzed 44 SRs, identifying 123 recommended PROMs and evaluating their methodological quality. The research highlights the most frequently used PROMs, such as the Pediatric Quality of Life Inventory, and calls for improvements in the reporting of psychometric properties and the development of tailored tools for assessing pediatric PROMs.
The study reviewed 44 SRs, encompassing 1,888 studies and 635 different PROMs, with 123 PROMs being recommended. The most frequently recommended PROM was the Pediatric Quality of Life Inventory (PedsQL), followed by the EuroQol five-dimension questionnaire. These PROMs were primarily used to assess quality of life, body functions, and emotional functions. The study found that 36 different conditions were addressed, with mental, behavioral, or neurodevelopmental disorders being the most common. However, the methodological quality of the SRs varied, with only 22.7% scoring high on the AMSTAR tool. Key issues included inconsistent reporting of measurement properties, such as content validity and internal consistency, and a lack of clarity on the appropriate age for self-reporting. The study also highlighted the need for more diverse PROMs to cover a broader range of pediatric conditions and improve the evidence base for their use in clinical practice.

HME Home Health puts focus on pediatrics

2025-03-03 - 2025-03

RICHMOND, B.C. – HME Home Health on Feb. 26 held its Annual Pediatrics Day at its Richmond corporate office. The event brought together pediatric therapists from across British Columbia – in-person and via live stream – with pediatric equipment manufacturers and representatives from key pediatric funding sources. “The pediatric equipment space is an ever-evolving landscape with new technology and best practices constantly being updated,” said Robert Boscacci, CEO and co-founder. “From our first Pediatrics Day in 2023 to our event this year, we’ve seen how valuable these training events are for clinicians.” Led by HME’s team of 15 pediatric specialists, the day focused on funding, product development, and first-hand product training and demonstrations. “HME is B.C.’s largest pediatric equipment provider, and we are honored to have so many clinicians from all areas of the province join us for an excellent day of learning, networking and fun,” said Cameron Fleming, CIO and co-founder. “HME looks forward to continuing this vital event annually to keep moving the Pediatric equipment space forward.

Guest column: Los Robles Hospital shouldn't close pediatric ward

2025-03-02 - 2025-03

I am sure all of you have heard that Los Robles Hospital plans to close our pediatric ward on July 1. Though I have been told that this is a done deal, I would like to go on record in stating that I respectfully disagree with this decision. Closing this unit would be a great mistake for our community, not just medically, but bad for the Conejo Valley, as we have always been known as a family friendly area and a wonderful place to raise our children.
I have been a pediatrician in town for over 40 years. I have served as Chief of Pediatrics at Los Robles for the last four years, have been on the Medical Executive Board for over 10 years, and have served two terms on the Los Robles Board of Trustees. I also hold a board position on the local chapter of the American Academy of Pediatrics.
I speak from experience and an understanding of how our local medical community operates. Here is the issue. Los Robles Hospital, (operated by Hospital Corporation of America) is closing the Pediatric Unit; stating that the ward is not busy enough. Historically, pediatric wards do not make as much money as adult wards and, admittedly, at times there is low volume on the floor, because of the good care our patients do receive, and children are generally healthy.
However, many people in our community do not agree that a low census figure should warrant closing the entire ward. There are many times when the patient load is up to six and there will be critical times when we need those beds. I have also been told that low patient volumes may not generate enough experience to maintain quality care. I do not agree with this either because of our superior staff. One of the reasons why HCA wants to close the ward is that the hospital has an issue in the ER with a logjam of adult patients needing admission consequently needing more hospital beds. But why risk the welfare of our children?
In a future scenario, this same boy could end up with a ruptured appendix on the 101 leading to a very long hospitalization. Appendicitis and sepsis happen frequently and progress quickly. We need our own unit as other hospitals are often full and reluctant to admit. Another issue to consider are the jaundice cases that would need to be sent out of town at 2-3 days of age. And what about next flu season or other epidemics?
In closing, I would like to add that I am familiar with most medical communities across Southern California and I truly believe the Los Robles Pediatric unit is the best for the Conejo Valley for mild to moderate acuity care. The pediatric nurses are the best I have ever worked with, and the hospitalists are the best I have ever partnered with. The nurses are caring and skilled. The hospitalists are excellent, efficient and collaborate with me and other specialists as needed.
The bottom line is that Conejo Valley needs a pediatric ward, maybe with some reimagining and reduction, but it needs one. I have several ideas on how to make it work while also providing the hospital a good portion of the extra beds it also needs. Los Robles is an excellent hospital with great leadership; therefore, I hope they will listen to some of these ideas and come to a healthy compromise.
I need the help of the community, and I am urging our citizens to call our city council, our local representatives, or anyone you think could help. Our children need your support.

Dime Releases the Playbook: Pediatric Digital Medicine Edition

2025-02-25 - 2025-02

BOSTON, Feb. 25, 2025 /PRNewswire/ today, the Digital Medicine Society (Dime), a global nonprofit and the professional home for digital medicine, released The Playbook: Pediatric Digital Medicine, an industry roadmap to address the unique challenges of pediatric digital health innovation and create solutions that optimize care for children.
Today, children make up 23% of the U.S. population, but they receive less than 10% of total healthcare funding and 5% of venture capital funding. Where investment does exist, best practices for developing digital health solutions that are optimized for the unique needs of pediatric care and research are largely absent.
In response to the lack of investment and barriers to innovation in the pediatric digital health space, Dime partnered with Boston Children's Hospital and other key industry partners to create The Playbook: Pediatric Digital Medicine.
"The future of pediatric healthcare depends on the choices we make today. In my practice, I see the urgent need for pediatric specific digital health tool. But need alone isn't enough," Amy Molten, Chair, Section on Advances in Therapeutics & Technology, American Academy of Pediatrics, "True progress requires turning good intentions into meaningful action. By applying the same rigor, care, and creativity to pediatric digital health as we do to lifesaving interventions, we can create technology that truly serves children and their caregivers. These resources pave the way for thoughtful innovation technology that grows alongside them, remaining responsive, ethical, and built for their future."

Opus Genetics advances gene therapy in pediatric trial

2025-02-18 - 2025-02

DURHAM, N.C. - Opus Genetics, Inc. (NASDAQ:IRD), a biotechnology firm focusing on gene therapies for inherited retinal diseases, has dosed the first pediatric patient in a Phase 1/2 clinical trial of OPGx-LCA5, an investigational treatment for Leber congenital amaurosis (LCA). According to InvestingPro data, the company maintains a strong financial position with a current ratio of 6.63, indicating robust liquidity to support its clinical programs. The company expects to release initial data from the pediatric cohort by the third quarter of 2025.

The trial, which includes adult participants, has shown early signs of success, with the first three adult patients experiencing meaningful visual improvement after treatment. These results initially observed at six months and to be further detailed with 12-month follow-up data, will be presented at a major medical conference in the second quarter of 2025. InvestingPro analysis shows that while the company is currently pre-profit, analysts have set price targets ranging from $8 to $15, suggesting significant upside potential if the trials succeed.

Jordan Noble’s path from patient to pediatric heart doctor

2025-02-13 - 2025-02

Three-year-old Jordan Noble told her parents she was going to be a baby heart doctor.
She was diagnosed with congenital heart disease as a toddler and saw a pediatric cardiologist every 6 months to a year from then on.
As I got older, I was really interested in understanding my heart disease, understanding what was normal and what was abnormal, she said. I actually had the privilege of shadowing my own pediatric cardiologist when I was in high school.
It was shortly after high school when an MRI showed her aorta was much larger than it appeared on previous echocardiograms. At 20, she underwent open heart surgery, followed by a 5-day hospital stay. A complication had her in and out of the hospital multiple times between her sophomore and junior years of college. That’s when her childhood declaration became more than a dream.
It really became a plan after I had heart surgery and got to experience all the wonderful people that went into that and see what that looked like. I decided that I was for sure going to do medicine, Noble said.

Abstracts sought for CHRI Pediatric Research Forum

2025-02-18 - 2025-02

The Child Health Research Institute, a partnership between UNMC and Children’s Nebraska, seeks abstract submissions for the 2025 CHRI Pediatric Research Forum.
The annual research forum is an opportunity for researchers to showcase their current projects to the Child Health Research Institute community. The forum accepts all types of research with pediatric applications. Case reports/case studies and quality improvement projects will not be considered for an award but still may be featured in the forum.
Associate member: Interprofessional health care workers and research personnel from the University of Nebraska, Children’s Nebraska and Nebraska Medicine are invited to submit abstracts in the newly formed Associate Member category. The new category is reserved for researchers that do not align with preexisting categories. CHRI membership is not required but is encouraged for award consideration.

500 Bedded Pediatric Hospital Bemina: A Premier Healthcare Institution

2025-02-15 - 2025-02

SRINAGAR, FEBRUARY 15: Government Children Hospital Bemina, a 500-bedded hospital, is a premier healthcare institution catering to the population of Kashmir Division, Chenab Valley, Poonch, Rajouri and UT of Ladakh. The hospital provides comprehensive healthcare services to approximately 1200 patients daily in its Outpatient Department (OPD), with around 80-90 admissions in its Inpatient Department (IPD). Additionally, the hospital witnesses a significant footfall of around 6000 people, including patients & attendants
The hospital has highest number of critical care beds in whole UT, and critical care provided in NICU and PICU is of national and international standard.
The hospital has got fellowship programme for doctors and nurses approved in neonatology and pediatric neurology.
Further to ensure high end critical care free of cost , the government enhanced the fund allocation to the hospital and all very costly drugs like IVIG, ALBUMIN and Surfactant are available free of cost for patients.

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