Current Opinion in Pediatrics

PURPOSE OF REVIEW: Hematopoietic stem cell-based therapies, including allogeneic hematopoietic cell transplantation (HCT) and autologous gene therapy (GT), have been used as curative therapy for many inborn errors of immunity (IEI). As the number of genetically defined IEI and the use of HCT and GT increase, valuable data on outcomes and approaches for specific disorders are available. We review recent progress in HCT and GT for IEI in this article. RECENT FINDINGS: Novel approaches to prevention of allogeneic complications and experience in adolescents and young adults have expanded the use of HCT...

PURPOSE OF REVIEW: Severe combined immune deficiency (SCID) is the most devastating genetic disease of the immune system with an unfavorable outcome unless diagnosed early in life. Newborn screening (NBS) programs play a crucial role in facilitating early diagnoses and timely interventions for affected infants. RECENT FINDINGS: SCID marked the pioneering inborn error of immunity (IEI) to undergo NBS, a milestone achieved 15 years ago through the enumeration of T-cell receptor excision circles (TRECs) extracted from Guthrie cards...

PURPOSE OF REVIEW: This review examines the potential of rapid palatal expansion (RPE) as a treatment for pediatric obstructive sleep apnea (OSA). The focus is on recent findings related to its efficacy, safety, patient selection, timing, appliance options, cost considerations, and long-term outcomes. RECENT FINDINGS: Recent studies indicate that RPE can lead to significant improvements in pediatric OSA, with a 70% reduction in the Apnea Hypopnea Index (AHI) and increased oxygen saturation levels...

PURPOSE OF REVIEW: As the incidence of allergic conditions has increased in recent decades, the effects of climate change have been implicated. There is also increased knowledge on the effects of other physical influences, such as scratching and Staphylococcus aureus. The skin barrier is the first line of defense to the external environment, so understanding the ways that these factors influence skin barrier dysfunction is important. RECENT FINDINGS: Although the impact on environmental exposures has been well studied in asthma and other allergic disorders, there is now more literature on the effects of temperature, air pollution, and detergents on the skin barrier...

PURPOSE OF REVIEW: Pediatric acute pancreatitis is an infrequent but potentially serious condition in children. Most have mild cases with spontaneous resolution, but up to one-third of patients can have moderate or severe disease or progress to recurrent or chronic pancreatitis. RECENT FINDINGS: Significant advances have been made in the field of pediatric pancreatology with a recognition that pediatric acute pancreatitis can vary significantly from adult disease with different risk factors and outcomes...

PURPOSE OF REVIEW: This review focuses on sharing the current and changing cystic fibrosis (CF) care model. This includes changes in CF care as a chronic disease with availability of new revolutionary, highly effective therapies as well as incorporation of shared decision-making, coproduction of care, quality improvement, telemedicine, and remote patient monitoring. RECENT FINDINGS: Changes in the CF management, the CF patient population, and CF care team are described as well as how CF care has adapted to these changes...

PURPOSE OF REVIEW: Sudden cardiac death (SCD) is the leading cause of death in young athletes during sports participation. Preparticipation cardiovascular screening aims to identify those at an increased risk of SCD. This review aims to provide a background of SCD in young athletes, to discuss the various screening recommendations of major medical societies, and to review recent evidence and current practice. RECENT FINDINGS: Numerous studies have evaluated various preparticipation screening practices, particularly regarding the inclusion of ECG as part of an initial evaluation to identify conditions with an increased risk of SCD...

PURPOSE OF REVIEW: To review the incidence of congenital heart disease in the trisomies, highlight the history of cardiac surgery in trisomy 21 comparing it to the increase in cardiac surgery in trisomies 13 and 18, discuss ethical issues specific to trisomies 13 and 18, and suggest a pathway of shared decision-making in the management of congenital heart disease in trisomy 13 and 18, specifically congenital heart surgery. RECENT FINDINGS: Congenital heart disease is prevalent in the trisomies and the management of these defects, especially surgical intervention, has changed...

PURPOSE OF REVIEW: This review aims to summarize the most up-to-date criteria for diagnosis of pediatric irritable bowel syndrome (IBS) and treatment options. It also explores recent research that has been performed evaluating risk factors, pathophysiology, and treatment designed to improve quality of life for those who suffer from IBS. IBS is a common disorder in pediatrics and one of the most common causes of abdominal pain for children; thus, it can be quite debilitating for individuals to achieve a satisfactory quality of life on a routine basis...

PURPOSE OF REVIEW: Conventional treatments of atopic dermatitis have been inadequate, especially in patients with moderate-to-severe disease. RECENT FINDINGS: In the past 5 years, four immunomodulators have been approved for the treatment of atopic dermatitis in children. These include dupilumab, ruxolitinib, upadacitinib, and abrocitinib. The review summarizes the pivotal phase 3 trials of these medications. SUMMARY: The newer immunomodulators have transformed the treatment of atopic dermatitis, particularly in patients with moderate-to-severe disease...

PURPOSE OF REVIEW: To synthesize and critically assess recent clinical and research advancements in pediatric bicuspid aortic valve (BAV) and its associated aortopathy. RECENT FINDINGS: In pediatric patients with BAV, progressive aortic dilation (i.e. bicuspid aortopathy) is commonly present and associated with increased risk for aortic aneurysm, dissection, and surgery in adulthood. Ongoing research explores the cause, incidence, and progression of bicuspid aortopathy to promote earlier diagnosis and improve preventive management...

PURPOSE OF REVIEW: To discuss the role of ventilator induced lung injury (VILI) and patient self-inflicted lung injury in ventilated children supported on extracorporeal membrane oxygenation (ECMO). RECENT FINDINGS: While extracorporeal life support is used routinely used every day around the globe to support neonatal, pediatric, and adult patients with refractory cardiac and/or respiratory failure, the optimal approach to mechanical ventilation, especially for those with acute respiratory distress syndrome (ARDS), remains unknown and controversial...

PURPOSE OF REVIEW: Aerodigestive disorders encompass conditions that affect both the airway and gastrointestinal tract. These include conditions such as acquired and congenital defects of the airway and esophagus as well as neuromuscular disorders. Patients often suffer from dysphagia, aspiration, and respiratory disorders. This article will provide a review of current practices in the management of feeding disorders, oropharyngeal dysphagia, and nutritional support in the aerodigestive population...

PURPOSE OF REVIEW: The aim of this study was to provide pediatric providers with a review of the diagnosis and management of fetal cardiac disease in the current era. RECENT FINDINGS: Prenatal detection of congenital heart disease (CHD) has improved but is still imperfect. In experienced hands, fetal echocardiography can detect severe CHD as early as the first trimester and a majority of more subtle conditions in the second and third trimesters. Beyond detection, a prenatal diagnosis allows for lesion-specific counseling for families as well as for development of a multidisciplinary perinatal management plan, which may involve in-utero treatment...

PURPOSE OF REVIEW: Avoidant/restrictive food intake disorder (ARFID) and pediatric feeding disorder (PFD) are the newest evolutions of frameworks for dysfunctional feeding and share overlapping features but maintain notable differences. This review will compare the two frameworks, highlighting some of the latest advances in diagnosis and management. RECENT FINDINGS: Dysfunctional feeding, particularly withing the PFD definition, benefits from multidisciplinary care with equal attention to medical, nutritional, skill-based, and behavioral domains...

PURPOSE OF REVIEW: Food allergies are on the rise. Though allergen avoidance and management of acute reactions have been the backbone of therapy, complete avoidance and timely acute care is often not feasible. Food allergen immunotherapy (FAIT) is a novel and evolving treatment option intended to induce desensitization and potential sustained unresponsiveness (SU) to food allergens. This review addresses the methods, mechanisms, efficacy, and adverse effects of oral immunotherapy (OIT), sublingual immunotherapy (SLIT), and epicutaneous immunotherapy (EPIT) for food allergens in the published literature...

PURPOSE OF REVIEW: Hearing loss is the most common sensory deficit and in young children sensorineural hearing loss is most frequently genetic in etiology. Hearing aids and cochlear implant do not restore normal hearing. There is significant research and commercial interest in directly addressing the root cause of hearing loss through gene therapies. This article provides an overview of major barriers to cochlear gene therapy and recent advances in preclinical development of precision treatments of genetic deafness...

PURPOSE OF REVIEW: On top of existing rites of passage, adolescents with congenital heart disease (CHD) face additional challenges as related to their underlying medical condition. These include compliance with medications and appointments, establishing heart-healthy behaviors, navigating potential at-risk situations, and transition to adult-oriented care. RECENT FINDINGS: Teenagers with CHD should focus on the importance of exercise as this seems to have important long-term benefits and may help improve weight management...

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