What's new in pediatrics

The following represent additions to UpToDate from the past six months that were considered by the editors and authors to be of particular interest. The most recent What's New entries are at the top of each subsection.

ORTHOPEDICS AND SPORTS MEDICINE

Ice for treating musculoskeletal injury (November 2024)

For many years, ice has been a standard treatment for acute muscle and tendon injuries. However, evidence supporting the effectiveness of cryotherapy is largely unstudied. The authors of a systematic review identified only 27 controlled studies of cryotherapy for acute soft tissue injury, of which 26 were animal studies, most involving muscle injury [1]. No randomized trials in humans were identified. While ice can provide analgesia and may offer benefits for acute treatment of minor injuries, its longer-term effects on more severe muscle or tendon injury are largely unknown. Pending further research, we believe it is reasonable to apply ice as part of the acute treatment of soft tissue musculoskeletal injury. (See "Initial management of soft tissue musculoskeletal injuries", section on 'Evidence'.)

GENERAL PEDIATRICS AND ADOLESCENT MEDICINE

Coated versus standard peripherally inserted central catheters (April 2025)

Whether specialized materials or coatings reduce complications from peripherally inserted central catheters (PICC) has been uncertain. In a randomized trial including over 1000 patients, those assigned to receive a chlorhexidine PICC had a higher complication rate than those assigned to a hydrophobic or standard polyurethane PICC (approximately 39, 22, and 22 percent respectively) [2]. The device failure rate was not significantly different among the three groups and was defined as the cessation of PICC function or the need to remove the PICC before completion of the intended therapy. The results of this trial support our practice of using standard PICCs. (See "Central venous access: Device and site selection in adults", section on 'Antimicrobial-impregnated catheters'.).

Consensus guideline on melatonin for sleep in typically developing children (March 2025)

An international group of pediatric sleep experts has published a new consensus guideline on the use of melatonin for sleep in typically developing children [3]. The panel recommends performing a thorough clinical evaluation to rule out other causes of chronic insomnia before considering melatonin, using behavioral approaches before and along with melatonin, limiting duration to as short of a time period as possible and no longer than three to six months in most cases, and storing melatonin safely in locked containers out of the reach of children. The guideline provides typical dose ranges of melatonin by age, up to a maximum of 5 mg nightly in adolescents. Our approach is consistent with these guidelines. (See "Pharmacotherapy for insomnia in children and adolescents: A rational approach", section on 'Melatonin'.)

Early sugar intake and long-term health outcomes (January 2025)

In a new study that analyzed the effects of sugar rationing in the United Kingdom after World War II, reduced sugar consumption to levels within current dietary guidelines, from in utero exposure up to two years of age, was associated with a decreased risk of type 2 diabetes, hypertension, and obesity in adulthood by 20 to 35 percent [4]. Approximately two-thirds of the risk reduction was attributable to sugar restriction between six months and two years of age. These findings expand the evidence of "metabolic programming" and demonstrate that limiting sugar exposure during the first two years of life has important long-term health benefits. (See "Definition, epidemiology, and etiology of obesity in children and adolescents", section on 'Infancy and early childhood'.)

Health behavior intervention to prevent obesity during the first two years of life (January 2025)

Interventions to prevent the development of obesity during early childhood often show little or no effect and are difficult to implement on a large scale. In a recent, randomized trial involving 900 American children enrolled shortly after birth, enhanced counseling (clinic-based health behavior counseling plus individually tailored responsive text messages) reduced the number of patients at age 24 months who were either overweight or had obesity by World Health Organization Z-score criteria compared with standard care (clinic-based health behavior counseling alone) [5]. These findings suggest that early intervention with a digital counseling intervention can help prevent obesity during the first two years of life. (See "Prevention and management of childhood obesity in the primary care setting", section on 'Prevention interventions'.)

Atherosclerotic cardiovascular disease (ASCVD) risk factors in adolescence and ASCVD events in adulthood (January 2025)

A growing body of evidence suggests that vascular changes predisposing to adult atherosclerotic cardiovascular disease (ASCVD) begin in childhood. In a recent study using data from seven different longitudinal studies including over 11,000 participants who were followed from adolescence to a mean age of 50 years, the presence of ASCVD risk factors in adolescence (eg, hypertension, obesity, smoking, or hypercholesterolemia) was independently associated with experiencing fatal and nonfatal ASCVD events in adulthood [6]. The risk increased with increasing number of risk factors. These findings highlight the importance of screening for ASCVD risk factors during childhood and adolescence and intervening at a young age. (See "Overview of pediatric risk factors for premature atherosclerotic cardiovascular disease (ASCVD)", section on 'Evidence linking childhood risk factors to ASCVD events in adulthood'.)

Long-term safety of oral propranolol for infantile hemangiomas (December 2024)

Oral propranolol is the standard-of-care treatment for infantile hemangiomas (IH) at risk of impairing function or causing permanent disfigurement. Several studies have addressed the concerns about potential long-term adverse effects of propranolol in children. The largest study using data from a database of electronic medical records from multiple health institutions did not find any statistically significant differences in the risk of growth impairment, sleep disorders, learning disabilities, and diabetes mellitus between a cohort of over 1000 children aged 10 to 17 years who had received propranolol for IH in the first year of life and an age-, sex-, and ethnicity-matched cohort of children who had never received propranolol [7]. These findings support propranolol as a safe treatment for IH in infants who do not have contraindications to its use. (See "Infantile hemangiomas: Management", section on 'Long-term safety'.)

2025 immunization schedule for infants, children, and adolescents published by the CDC (December 2024)

The United States Centers for Disease Control and Prevention has provided the 2025 updates for the routine immunization schedules for infants, children, and adolescents ≤18 years of age [8]. Notable changes in the 2025 schedules include an additional dose of COVID-19 vaccine for moderately or severely immunocompromised individuals and the addition of Vaxelis to PedvaxHIB as a preferred Hib vaccine for American Indian and Alaska Native infants. (See "Standard immunizations for children and adolescents: Overview", section on 'Routine schedule'.)

Long-term outcomes after weight loss surgery in adolescents (November 2024)

In a recent report of 10-year outcomes after weight loss surgery in 218 adolescents (mean age 17 years), the weight outcomes did not differ by procedure (Roux-en-Y gastric bypass versus sleeve gastrectomy) and were excellent in approximately 20 percent (body mass index [BMI] reduced 44 percent), very good in 30 percent (BMI reduced 27 percent), and good in 40 percent (BMI reduced 13 percent) [9]. The remaining 10 percent of patients experienced initial weight loss followed by weight regain. Long-term remission of type 2 diabetes, hypertension, and dyslipidemia occurred in 55, 57, and 54 percent of patients, respectively. These findings support weight loss surgery as an important management option for adolescents with severe obesity that can provide durable improvement in BMI and related comorbidities. (See "Surgical management of severe obesity in adolescents", section on 'Weight loss'.)

ALLERGY, IMMUNOLOGY, AND RHEUMATOLOGY

Neuropsychiatric events with initiation of montelukast versus long-acting beta-agonist for asthma (April 2025)

Whether leukotriene antagonist administration results in increased neuropsychiatric events (NPEs) in children and adolescents is controversial. Large observational studies often demonstrate an association with increased risk; however, this may be related to worsening asthma control in those who required leukotriene antagonist initiation. In a new national database study from Sweden, investigators compared new montelukast users with new long-acting beta-agonist (LABA) users to help avoid this confounding. Incident neuropsychiatric adverse events were uncommon and nearly identical in those with new montelukast use compared with those with LABA use (2.39 versus 2.41 per 100 patient-years; hazard ratio 0.99) [10]. These data should offer reassurance to clinicians and caregivers of those who require these medications. (See "Antileukotriene agents in the management of asthma", section on 'Leukotriene receptor antagonists'.)

Egg allergy no longer a concern for any vaccines (February 2025)

Some vaccines contain trace amounts of egg protein (table 1), but none contain enough to cause reactions in egg-allergic patients. For the last several years, it has been recommended that patients not be asked about egg allergy prior to receiving influenza vaccine. More recently, data have accumulated to show that egg allergy is similarly not a concern for administration of the yellow fever vaccine. In the largest study to date, 171 children with egg allergy, including 24 percent with a history of anaphylaxis, underwent skin testing with the yellow fever vaccine and then received it regardless of skin test results, with no allergic reactions [11]. Thus, we no longer inquire about egg allergy prior to the administration of any vaccine. Vaccine providers should remain prepared to treat rare allergic reactions that may occur after any vaccine, but no special precautions are necessary for recipients with egg allergy. (See "Allergic reactions to vaccines", section on 'Hen's egg'.)

Transcriptional profiling of asthma in children and adolescents (January 2025)

Childhood-onset asthma traditionally is thought to be primarily mediated by allergic processes (typically characterized by hypereosinophilia and elevated levels of interleukins 4, 5, and 13). However, a new cross-sectional analysis of nasal epithelial transcription profiles from three studies of predominantly African American and Puerto Rican youths with asthma (aged 6 to 20 years) found that nearly 75 percent of participants did not have an allergic (type 2) gene signature [12]. Instead, these patients had transcriptomic profiles consistent with either Th17-mediated inflammation or a third endotype that included neither type 2 nor Th17-responsive genes. This finding may explain why pediatric asthma may not respond to therapies that target type 2 immune responses and implies that many patients would benefit from treatments that focus on other inflammatory pathways. (See "Characterizing severe asthma phenotypes", section on 'Neutrophilic asthma'.)

CARDIOLOGY

Updated guidance on newborn pulse oximetry screening (January 2025)

The American Academy of Pediatrics (AAP) has released updated guidance on newborn pulse oximetry screening (POS) for critical congenital heart disease [13]. Key changes from the 2011 guidelines include endorsement of a simplified screening algorithm that requires only one repeated screen if the initial screen is equivocal (algorithm 1), and clarification that screening must be performed in room air. For newborns receiving oxygen therapy, pulse oximetry monitoring should be performed as clinically indicated for their condition, and the screening procedure should be deferred until the infant has been weaned off oxygen. We agree with the approach recommended by the AAP. (See "Newborn screening for critical congenital heart disease using pulse oximetry", section on 'Screening algorithm'.)

Atherosclerotic cardiovascular disease (ASCVD) risk factors in adolescence and ASCVD events in adulthood (January 2025)

A growing body of evidence suggests that vascular changes predisposing to adult atherosclerotic cardiovascular disease (ASCVD) begin in childhood. In a recent study using data from seven different longitudinal studies including over 11,000 participants who were followed from adolescence to a mean age of 50 years, the presence of ASCVD risk factors in adolescence (eg, hypertension, obesity, smoking, or hypercholesterolemia) was independently associated with experiencing fatal and nonfatal ASCVD events in adulthood [6]. The risk increased with increasing number of risk factors. These findings highlight the importance of screening for ASCVD risk factors during childhood and adolescence and intervening at a young age. (See "Overview of pediatric risk factors for premature atherosclerotic cardiovascular disease (ASCVD)", section on 'Evidence linking childhood risk factors to ASCVD events in adulthood'.)

Association of myocardial fibrosis with sudden cardiac death risk in children with hypertrophic cardiomyopathy (January 2025)

In children with hypertrophic cardiomyopathy (HCM), growing evidence suggests that the burden of late gadolinium enhancement (LGE; a surrogate for myocardial fibrosis) on cardiac magnetic resonance (CMR) imaging provides valuable information for assessing the risk of sudden cardiac death (SCD). In a recent study of 700 patients <21 years of age with HCM, the risk of SCD events increased with increasing LGE burden, even after adjustment for multiple variables associated with SCD [14]. In patients whose risk of SCD is unclear, incorporating LGE burden into the overall risk assessment may influence the decision to place an internal cardioverter-defibrillator (ICD). (See "Hypertrophic cardiomyopathy in children: Management and prognosis", section on 'Other risk factors'.)

EMERGENCY MEDICINE

Reduction of radial head subluxation ("pulled elbow") in children (March 2025)

Radial head subluxation (RHS) commonly occurs in young children (<5 years old) when a parent or caregiver grabs the arm to prevent falling or pulling away (figure 1); the best method for reduction of an RHS is debated. In a meta-analysis of 11 randomized trials (almost 1200 children with RHS undergoing reduction), the pooled first-attempt success rate was higher following the use of the hyperpronation method (figure 2) compared with the supination/flexion method (figure 3) (91 versus 75 percent, respectively) [15]. Limitations of this analysis include the high risk of bias in most of the included studies and interstudy differences in the definition of a successful reduction. These findings support our suggestion for the hyperpronation method rather than the supination/flexion method to reduce an RHS; however, the supination/flexion method is a reasonable alternative if the clinician is more familiar and skilled with this technique. (See "Radial head subluxation (pulled elbow): Evaluation and management", section on 'Radial head subluxation reduction'.)

Fractures and child abuse in young children (March 2025)

The American Academy of Pediatrics has published a clinical report that provides guidance for the evaluation of fractures in young children [16]. This report reaffirms the importance of a careful history and physical examination with special attention to the reported mechanism of injury, the developmental capability of the child, associated injuries on examination, and the specific type of fracture. A skeletal survey (table 2), under the guidance of a pediatric radiologist or a multidisciplinary child abuse team led by a child abuse specialist, is indicated for children <24 months old who have a fracture that is concerning for abuse (table 3). (See "Orthopedic aspects of child abuse", section on 'Overview' and "Physical child abuse: Recognition", section on 'Fractures'.)

Nonoperative management of appendicitis in children (January 2025)

For children with early appendicitis, the role of nonoperative management with antibiotics (NOM) has been debated. In a multicenter trial of 846 children with appendicitis, 34 percent of children assigned to NOM required an appendectomy within one year. Approximately one-half of these patients required appendectomy at the index admission, of whom 35 percent had perforated appendicitis [17]. For children assigned to prompt appendectomy, 7 percent had normal pathology and 6 percent had perforation. Based on this first randomized trial of NOM in children with early appendicitis, we continue to perform appendectomy for most of these patients. However, NOM may be an option for selected children after shared decision-making (algorithm 2). (See "Acute appendicitis in children: Management", section on 'Nonoperative management'.)

ENDOCRINOLOGY

Crinecerfont receives regulatory approval for adjunctive management of hyperandrogenism in children ≥4 years with 21OHD CAH (April 2025)

Crinecerfont, a selective oral antagonist of the corticotropin-releasing factor (CRF) receptor type 1, was approved by the US Food and Drug Administration as an adjunctive therapy to glucocorticoids for patients age ≥4 years with classic congenital adrenal hyperplasia due to 21-hydroxylase deficiency (classic 21-OHD CAH) who require supraphysiologic doses of glucocorticoids to suppress adrenal androgen production or who have signs of glucocorticoid excess despite appropriate glucocorticoid dosing [18]. Approval was based on effective suppression of adrenal androgens with lower glucocorticoid doses in a randomized trial of more than 100 children aged 2 to 17 years with classic 21-OHD CAH [19]. Additional data are needed to determine optimal dosing and approach to adjustment of glucocorticoid doses for patients treated with crinecerfont. (See "Classic congenital adrenal hyperplasia due to 21-hydroxylase deficiency in infants and children: Treatment", section on 'Corticotropin-releasing factor (CRF) type 1 receptor antagonist (crinecerfont)'.)

Use of serum hormone testing to determine the cause of delayed puberty (April 2025)

In children and adolescents with delayed puberty, it can be challenging to distinguish between self-limited constitutional delay of growth and puberty (CDGP) and idiopathic hypogonadotropic hypogonadism (IHH). Some widely available hormone tests may help to differentiate between these diagnoses. In a case-control study of 65 male adolescents aged 13 to 18 years with delayed puberty, median basal serum luteinizing hormone (LH), follicle-stimulating hormone (FSH), anti-Mullerian hormone (AMH), and inhibin B were lower in patients with IHH than with CDGP [20]. Furthermore, combining these measurements showed high accuracy for distinguishing between CDGP and IHH. We typically measure LH, FSH, and gonadal steroids to evaluate delayed puberty. More data are needed to determine the utility of AMH and inhibin B testing in clinical practice. (See "Delayed puberty: Approach to evaluation and management", section on 'Low FSH and LH for pubertal stage'.)

Expanded approval for intranasal glucagon for hypoglycemia in young children with diabetes (April 2025)

The US Food and Drug Administration (FDA) has expanded regulatory approval for intranasal glucagon for the treatment of severe hypoglycemia in patients with type 1 and type 2 diabetes; while previously it was approved for those aged ≥4 years old, approval now includes those aged ≥1 year old. This expanded approval was based on a phase 1 single-arm study of seven patients age 1.8 to 4 years with type 1 diabetes [21]. All patients experienced an increase in blood glucose ≥20 mg/dL from baseline within 30 minutes of administration of 3 mg of intranasal glucagon. No serious adverse events were reported, although several minor events occurred (eg, vomiting, epistaxis, nasal discomfort and eye irritation). Based on these data, we consider intranasal glucagon to be a safe and effective alternative to intramuscular glucagon for the treatment of severe hypoglycemia in patients with diabetes aged 1 year and older. (See "Type 1 diabetes in children and adolescents: Prevention and management of hypoglycemia", section on 'Glucagon'.)

Global rates of overweight and obesity (March 2025)

Despite numerous associated health risks, rates of overweight and obesity continue to increase worldwide. In a new global analysis, the number of adults with overweight or obesity is expected to increase from 2.1 billion in 2021 to 3.8 billion in 2050, which represents nearly 60 percent of the predicted adult population [22]. Among children and young adults, the projected global prevalence of obesity by 2050 is 15.6 percent for those ages 5 to 14 years, and 14.2 percent for those 15 to 24 years [23]. We continue to promote screening and prevention for all patients, routine growth monitoring and counseling on healthy behaviors and lifestyle for all children, comprehensive lifestyle intervention for children and adults with overweight and obesity, and intensive lifestyle, pharmacologic, or surgical management for those at the highest risk of obesity-related morbidity and mortality. (See "Obesity in adults: Prevalence, screening, and evaluation", section on 'Global'.)

GASTROENTEROLOGY, HEPATOLOGY, AND NUTRITION

Updated guidelines on eosinophilic esophagitis (January 2025)

The American College of Gastroenterology has published updated guidelines on diagnosis and management of eosinophilic esophagitis (EoE) [24]. The guidelines establish the diagnosis of EoE based esophageal symptoms, ≥15 eosinophils per high-power field on biopsy, and excluding other causes of eosinophilia. Endoscopic evaluation includes obtaining multiple biopsies from two esophageal levels (ie, proximal/mid and distal esophagus). For initial treatment, the guidelines endorse a limited empiric food elimination diet or pharmacologic (proton pump inhibitor or topical glucocorticoid) therapy. Our approach is generally consistent with these guidelines. (See "Treatment of eosinophilic esophagitis (EoE)", section on 'Introduction'.)

HEMATOLOGY AND ONCOLOGY

Neutropenia/agranulocytosis from deferiprone (November 2024)

Deferiprone is an oral iron-chelating agent used to treat iron overload when phlebotomy cannot be tolerated. The most concerning adverse effect is neutropenia/agranulocytosis. A recent review of data from 15 clinical studies (977 patients) found 22 cases of agranulocytosis and 3 serious infections, 1 of which was fatal [25]. Review of post-marketing reports identified 176 cases of agranulocytosis, 19 serious infections, and 11 deaths, mostly from sepsis. Lower neutrophil counts (<200/microL) conferred the greatest risk, and the mean time to onset of agranulocytosis was approximately two years. These data emphasize the need to closely monitor neutrophil counts throughout deferiprone therapy. (See "Iron chelation: Choice of agent, dosing, and adverse effects", section on 'Deferiprone dosing + AEs (Ferriprox)'.)

INFECTIOUS DISEASES AND IMMUNIZATIONS

Universal screening for congenital cytomegalovirus infection (March 2025)

Newborn screening for congenital cytomegalovirus (cCMV) has been proposed by public health experts, but the most reliable and cost-effective method is uncertain. Two recent studies reported on the experience of performing universal cCMV screening with dried blood spots [26,27]. Combined, these studies screened >600,000 newborns, and 863 (0.14 percent) screened positive. The false-positive rate was 4 percent. Among confirmed cases, >80 percent were asymptomatic whereas 12 to 16 percent had either isolated hearing loss or findings consistent with symptomatic cCMV disease. These reports suggest that universal screening is feasible; however, important challenges and uncertainties remain (eg, ensuring timely follow-up and linkage to care after a positive screen, and lack of standardized criteria for initiating antiviral treatment). (See "Congenital cytomegalovirus (cCMV) infection: Clinical features and diagnosis", section on 'Universal newborn screening'.)

Pediatric deaths due to influenza-associated encephalopathy or encephalitis (March 2025)

Influenza-associated encephalopathy or encephalitis (IAE) is a rare complication of influenza that can be fatal. New surveillance data from the Centers for Disease Control and Prevention indicate that IAE accounts for a significant proportion of pediatric influenza-associated deaths [28]. Of nearly 1800 influenza-associated pediatric deaths occurring between the 2010-2011 season and the 2023-2024 season, 157 (9 percent) were caused by IAE. Additionally, of the 68 influenza-associated pediatric deaths reported from October 1, 2024 through February 8, 2025, 9 (13 percent) were due to IAE. It is not yet clear whether this represents a higher incidence of IAE this season; however, IAE should be considered in children with influenza and abnormal neurologic findings. (See "Seasonal influenza in children: Clinical features and diagnosis", section on 'Central nervous system'.)

Revised dosing schedule for MenB-4C (Bexsero) (February 2025)

In December 2024, the US Centers for Disease Control published an updated dosing schedule for MenB-4C (Bexsero), one of two available meningococcal serogroup B vaccines [29]. Routine vaccination of healthy individuals now consists of two doses, separated by ≥6 months (table 4). Three doses (zero, one to two, and six months) should be administered to those at increased risk for serogroup B meningococcal disease and during an outbreak (table 5 and table 6). Previously MenB-4C was administered as two doses, separated by ≥1 month. This change was based on improved immunogenicity with the revised dosing schedule. MenB-4C is also available as part of a pentavalent vaccine (Penmenvy), which was approved for use in February 2025 [30]; updated recommendations on the use of this vaccine are pending. When administering serogroup B meningococcal vaccines, a single manufacturer's MenB products must be used for each dose of the primary series and all booster doses. Vaccine formulations are not interchangeable. (See "Meningococcal vaccination in children and adults", section on 'Vaccine schedules'.)

Updated United States guidelines on perinatal HIV transmission (January 2025)

The United States Department of Health and Human Services has updated the perinatal HIV clinical guidelines [31]. They now categorize infants born to mothers with HIV into three risk stratification groups based on maternal HIV viral load during pregnancy, the most significant risk factor for transmission. The lowest-risk infants are born to mothers with sustained viral suppression (HIV RNA <50 copies/mL) since 20 weeks gestation. The highest-risk infants are born to mothers with a detectable HIV viral load within four weeks of delivery. All other infants fall into the middle-risk category. This risk stratification informs the antiretroviral regimen given to the infant to prevent HIV transmission, outlined in the table (table 7). Our approach to the management of infants exposed to HIV at birth is consistent with these updated guidelines. (See "Pediatric HIV infection: Management of infants born to mothers with HIV in resource-abundant settings", section on 'Approach to ARV drug management'.)

2025 immunization schedule for infants, children, and adolescents published by the CDC (December 2024)

The United States Centers for Disease Control and Prevention has provided the 2025 updates for the routine immunization schedules for infants, children, and adolescents ≤18 years of age [8]. Notable changes in the 2025 schedules include an additional dose of COVID-19 vaccine for moderately or severely immunocompromised individuals and the addition of Vaxelis to PedvaxHIB as a preferred Hib vaccine for American Indian and Alaska Native infants. (See "Standard immunizations for children and adolescents: Overview", section on 'Routine schedule'.)

Perinatal HIV transmission continues to occur in the United States (November 2024)

In the United States, when pregnant individuals with HIV and their infants receive all applicable perinatal transmission prevention interventions, perinatal HIV transmission rates can be reduced to less than 1 percent. However, cases of perinatal HIV transmission still occur, as outlined by a recently published case series of six children diagnosed with HIV due to perinatal transmission in 2022 [32]. Causes included delayed entry to prenatal care, discovery of HIV diagnosis during pregnancy rather than preconception, and delays in antiretroviral therapy (ART) initiation during pregnancy. Potential opportunities to reduce transmission include increasing pre-exposure prophylaxis (PrEP) utilization, routine HIV testing among individuals of childbearing age who are at high risk of HIV acquisition, and rapid start of ART for pregnant individuals with HIV. Additionally, once a mother is diagnosed with HIV, all biological children should be tested for HIV to avoid delayed identification. (See "Pediatric HIV infection: Epidemiology, clinical manifestations, and outcome", section on 'Resource-rich settings'.)

NEONATOLOGY

Universal screening for congenital cytomegalovirus infection (March 2025)

Newborn screening for congenital cytomegalovirus (cCMV) has been proposed by public health experts, but the most reliable and cost-effective method is uncertain. Two recent studies reported on the experience of performing universal cCMV screening with dried blood spots [26,27]. Combined, these studies screened >600,000 newborns, and 863 (0.14 percent) screened positive. The false-positive rate was 4 percent. Among confirmed cases, >80 percent were asymptomatic whereas 12 to 16 percent had either isolated hearing loss or findings consistent with symptomatic cCMV disease. These reports suggest that universal screening is feasible; however, important challenges and uncertainties remain (eg, ensuring timely follow-up and linkage to care after a positive screen, and lack of standardized criteria for initiating antiviral treatment). (See "Congenital cytomegalovirus (cCMV) infection: Clinical features and diagnosis", section on 'Universal newborn screening'.)

No clear benefit with intratracheal glucocorticoids plus surfactant in preventing bronchopulmonary dysplasia (December 2024)

Postnatal intratracheal glucocorticoids mixed with surfactant have been proposed to prevent bronchopulmonary dysplasia (BPD) in preterm infants while avoiding the adverse effects of systemic glucocorticoids; however, large randomized trials have not demonstrated efficacy. A randomized, multicenter trial of over 1000 extremely preterm infants did not show a clear difference in the rates of BPD or survival at 36 weeks postmenstrual age with budesonide plus surfactant compared with surfactant alone [33]. In addition, treatment with budesonide did not clearly reduce the need for postnatal systemic glucocorticoids or the duration of mechanical ventilation. Given the lack of supporting evidence, we do not use intratracheal glucocorticoids mixed with surfactant to prevent BPD. (See "Postnatal use of glucocorticoids for prevention of bronchopulmonary dysplasia (BPD) in preterm infants", section on 'No role for intratracheal glucocorticoids mixed with surfactant'.)

NEUROLOGY

Pediatric deaths due to influenza-associated encephalopathy or encephalitis (March 2025)

Influenza-associated encephalopathy or encephalitis (IAE) is a rare complication of influenza that can be fatal. New surveillance data from the Centers for Disease Control and Prevention indicate that IAE accounts for a significant proportion of pediatric influenza-associated deaths [28]. Of nearly 1800 influenza-associated pediatric deaths occurring between the 2010-2011 season and the 2023-2024 season, 157 (9 percent) were caused by IAE. Additionally, of the 68 influenza-associated pediatric deaths reported from October 1, 2024 through February 8, 2025, 9 (13 percent) were due to IAE. It is not yet clear whether this represents a higher incidence of IAE this season; however, IAE should be considered in children with influenza and abnormal neurologic findings. (See "Seasonal influenza in children: Clinical features and diagnosis", section on 'Central nervous system'.)

Impact of genetic testing on cerebral palsy management (March 2025)

Genetic testing is increasingly performed for individuals diagnosed with cerebral palsy (CP) to identify a specific underlying cause of symptoms, but the clinical implications of testing are unclear. In a meta-analysis of studies that assessed genetic variants in children with CP and included 1841 individuals who underwent exome sequencing, likely pathologic variants were identified in 27 percent, and results led to a change in management in 8 percent [34]. Specific interventions included the initiation of condition-specific medications, stem cell transplantation, dietary changes, and implementation of preventive measures and condition-specific monitoring. These results support the use of genetic testing both to identify underlying causes and to guide treatment of CP. (See "Cerebral palsy: Evaluation and diagnosis", section on 'Metabolic and genetic testing'.)

Tonsillotomy versus tonsillectomy for obstructive sleep apnea in children (January 2025)

Intracapsular tonsillectomy (also known as subtotal or partial tonsillectomy or "tonsillotomy") is increasingly used for the treatment of obstructive sleep apnea (OSA) in children, but its optimal role is unsettled. In a meta-analysis of 32 studies including 17 randomized trials of either tonsillotomy or traditional tonsillectomy in children with OSA, tonsillotomy was associated with improved recovery time and lower risk of postoperative complications but increased risk of tonsillar regrowth, recurrent OSA, and reoperation compared with traditional tonsillectomy [35]. Despite the high number of trials, the quality of the evidence remains limited by heterogeneity, high risk of bias, and lack of long-term follow-up. Further studies are needed to help identify individual patient characteristics predictive of long-term benefit from tonsillotomy. (See "Adenotonsillectomy for obstructive sleep apnea in children", section on 'Intracapsular tonsillectomy (tonsillotomy)'.)

New treatments for Niemann-Pick disease type C (November 2024)

Niemann-Pick disease type C (NPC) is a rare genetic disorder that typically presents in childhood with vertical supranuclear gaze palsy, cerebellar symptoms, cognitive deterioration, progressive dystonia, dysarthria, and dysphagia. Based on limited data, the US Food and Drug Administration recently approved two new therapies (arimoclomol and levacetylleucine) for patients with neurologic symptoms due to NPC [36,37]. A previously published 12-week trial of patients with NPC found that arimoclomol treatment (with concomitant miglustat in most patients) resulted in less disease progression from baseline compared with placebo. In a separate small 12-week trial, levacetylleucine treatment (also with concomitant miglustat in most patients) of NPC resulted in a better neurologic outcome compared with placebo [38]. While these trials are limited by issues including small size and absence of longer-term data, we suggest treatment with one of these agents for children and adults with neurologic symptoms due to NPC. (See "Overview of acid sphingomyelinase deficiency and Niemann-Pick disease type C", section on 'Disease-modifying therapies for NPC'.)

Cerebral cortical encephalitis as part of myelin oligodendrocyte glycoprotein antibody-associated disease (November 2024)

Myelin oligodendrocyte glycoprotein antibody-associated disease (MOGAD) is characterized by attacks of demyelination predominantly targeting the optic nerves, brain, and spinal cord, but may infrequently present as cerebral cortical encephalitis (CCE). A recent case series of five children and a literature review add to the knowledge of this MOGAD phenotype [39]. Most cases of CCE present with seizures, headache, and encephalopathy, and brain MRI typically shows cortical T2-hyperintense lesions and leptomeningeal enhancement; some patients have fever and focal neurologic deficits. The diagnosis of MOGAD requires one of the defining core clinical events (ie, optic neuritis, transverse myelitis, acute disseminated encephalomyelitis, cerebral monofocal or polyfocal deficits, brainstem or cerebellar deficits, or CCE), a positive MOG-IgG antibody test, and the exclusion of a better diagnosis. (See "Myelin oligodendrocyte glycoprotein antibody-associated disease (MOGAD): Clinical features and diagnosis", section on 'Cerebral cortical encephalitis'.)

Screening for anxiety and depression in children and adolescents with epilepsy (November 2024)

New consensus-based recommendations on the diagnosis and treatment of anxiety and depression in children and adolescents with epilepsy are available from the International League Against Epilepsy (ILAE) [40]. These recommendations include screening for anxiety and depression in all children and adolescents with epilepsy at age 7 years and annually thereafter, use of a formal screening questionnaire to assess symptoms of anxiety and depression, and closer surveillance for groups at higher risk (eg, those with suicidal behavior). Screening interviews should include the child with epilepsy and their parents or other caregivers, and they should be informed about the potential adverse behavioral effects of antiseizure medications. We agree with the ILAE recommendations, including referral of patients with moderate to severe depression or anxiety to a mental health care clinician. (See "Epilepsy in children and adolescents: Comorbidities, complications, and outcomes", section on 'Anxiety and depression'.)

PULMONOLOGY

Vanzacaftor-tezacaftor-deutivacaftor for treatment of cystic fibrosis (February 2025)

Vanzacaftor-tezacaftor-deutivacaftor (VTD) is a new triple combination cystic fibrosis transmembrane conductance regulator (CFTR) modulator drug that is administered once daily by month. In two randomized clinical trials with a total of over 900 adolescents and adults with cystic fibrosis (CF), clinically important outcomes including pulmonary function changes, number of pulmonary exacerbations, respiratory symptom-related quality of life, and adverse effects were similar for patients assigned to VTD compared with elexacaftor-tezacaftor-ivacaftor (ETI), the standard CFTR modulator drug for the treatment of CF [41]. Compared with ETI, VTD has the advantage of superior effects on sweat chloride and less frequent dosing, but it may be more costly and has less data on medium-term outcomes. Based on these trials, the US Food and Drug Administration has approved VTD for the treatment of CF in patients ≥6 years; we recommend that patients with CF and responsive genotypes receive treatment with either ETI or VTD. (See "Cystic fibrosis: Treatment with CFTR modulators", section on 'Efficacy'.)

Effects of CFTR modulator therapy on mental health (November 2024)

Patients with cystic fibrosis (CF) generally have improved emotional function and quality of life when receiving cystic fibrosis transmembrane regulator (CFTR) modulator therapy, but post-marketing surveillance has raised questions about adverse mental health effects soon after initiation in selected patients. In a new prospective, observational study of 93 adolescents and adults with CF, standardized measures found improved cognitive function and disease-specific quality of life and no increased development of depression or anxiety during the first six months of treatment [42]. Consistent with prior randomized trials, these findings suggest that CFTR therapy is not associated with adverse effects on mental health. (See "Cystic fibrosis: Treatment with CFTR modulators", section on 'Mental health effects'.)

Use of anti-inflammatory reliever therapies to reduce asthma exacerbations (November 2024)

Use of inhalers containing both a fast-acting bronchodilator and anti-inflammatory inhaled corticosteroids (ICS) for relief of symptoms has reduced the rate of asthma exacerbations compared with short-acting beta-agonists (SABA) alone in several randomized trials of adolescents and adults. In a new network meta-analysis, compared with SABA alone, severe asthma exacerbations were significantly reduced for both ICS-formoterol (13 trials, 19,184 patients; risk difference 10.3 percent, risk ratio [RR] 0.65) and ICS-SABA (4 trials, 4852 patients; risk difference 4.7 percent, RR 0.84) [43]. Our authors recommend these anti-inflammatory reliever therapies for those with variable asthma symptoms or frequent exacerbations and prefer them for all patients with asthma. (See "Ongoing monitoring and titration of asthma therapies in adolescents and adults", section on 'Anti-inflammatory reliever therapy (AIR) to reduce exacerbations'.)