Clinical efficacy and safety of intravenous ferric carboxymaltose treatment for restless legs symptoms and low serum ferritin in children with autism spectrum disorder.

Background: Restless legs syndrome (RLS) may be underdiagnosed in children with autism spectrum disorder (ASD) due to difficulty expressing the symptoms in their own words. In addition, administration of oral iron may be particularly difficult in children with ASD.

Methods: This was a retrospective, open-label case series of children with ASD, restless legs (RL) symptoms, and serum ferritin <30 μg/L, who either had failed or did not tolerate oral iron, and were subsequently treated with intravenous (IV) ferric carboxymaltose (FCM).

Kidney support for babies: building a comprehensive and integrated neonatal kidney support therapy program.

Kidney support therapy (KST), previously referred to as Renal Replacement Therapy, is utilized to treat children and adults with severe acute kidney injury (AKI), fluid overload, inborn errors of metabolism, and kidney failure. Several forms of KST are available including peritoneal dialysis (PD), intermittent hemodialysis (iHD), and continuous kidney support therapy (CKST). Traditionally, extracorporeal KST (CKST and iHD) in neonates has had unique challenges related to small patient size, lack of neonatal-specific devices, and risk of hemodynamic instability due to large extracorporeal circuit volume relative to patient total blood volume.

Risk factors for severe acute kidney injury after pediatric hematopoietic cell transplantation.

Background: Acute kidney injury (AKI) is common after hematopoietic cell transplantation (HCT) and is associated with poorer outcomes. Risk factors for AKI after pediatric HCT are not fully understood. The study objective was to assess unique risk factors for AKI in the HCT population and evaluate post-HCT AKI patterns.

Clonazepam for the management of sleep disorders.

Background: The objectives of this review and meta-analysis of polysomnographic data are those to focus on the clinical use of clonazepam for the management of sleep disorders by re-analyzing clinical trials and randomized clinical trials which have been published in peer-reviewed journals.

Methods: A review of the literature including clinical trials and randomized controlled trials was performed in PubMed®, in accordance with the Preferred Reporting Items for Systematic Reviews and Meta-Analyses (PRISMA) statement protocol. A random effects model meta-analysis was then carried out for the four more frequently reported polysomnographic measures: total sleep time, sleep latency, sleep efficiency, and periodic leg movement during sleep (PLMS) index.

Sirolimus for management of GI bleeding in blue rubber bleb nevus syndrome: A case series.

Blue rubber bleb nevus syndrome (BRBNS) commonly presents with anemia from bleeding gastrointestinal (GI) vascular malformations. Management is highly variable, as no consensus guidelines for medical treatment currently exist. Sirolimus has been used in BRBNS to decrease GI bleeding and seems well tolerated, though questions remain regarding dosing, duration of therapy, and adverse effects.

Effects of bupropion and SSRI antidepressants on leg movement activity and chin muscle tone during sleep in adolescents.

Study Objectives: To evaluate the effects of bupropion on periodic limb movements during sleep (PLMS) and chin electromyography tone in children taking it for their mood disorder, compared to the effects of selective serotonin reuptake inhibitors (SSRIs) and of bupropion combined with SSRIs.

Methods: Six adolescents (aged 16.0 ± 0.

Best Practice Guidelines for Surgical Site Infection in High-risk Pediatric Spine Surgery: Definition, Prevention, Diagnosis, and Treatment.

Background: Prior "best practice guidelines" (BPG) have identified strategies to reduce the risk of acute deep surgical site infection (SSI), but there still exists large variability in practice. Further, there is still no consensus on which patients are "high risk" for SSI and how SSI should be diagnosed or treated in pediatric spine surgery. We sought to develop an updated, consensus-based BPG informed by available literature and expert opinion on defining high-SSI risk in pediatric spine surgery and on prevention, diagnosis, and treatment of SSI in this high-risk population.

Gastroschisis and low incidence of early-onset infection: a case for antimicrobial stewardship.

Objective: Early onset infection (EOI) in gastroschisis is rare. Excess antibiotic exposure in neonates increases necrotizing enterocolitis and mortality. We evaluated antibiotic exposure and EOI in gastroschisis.

Trazodone affects periodic leg movements and chin muscle tone during sleep less than selective serotonin reuptake inhibitor antidepressants in children.

Study Objectives: To test the hypothesis that children taking trazodone have less leg movements during sleep (LMS) and higher rapid eye movement (REM) sleep atonia than children taking selective serotonin reuptake inhibitors (SSRIs) but more than normal controls.

Methods: Fifteen children (9 girls and 6 boys, mean age 11.7 years, standard deviation [SD] 3.

Prevalence and Risk Factors for Kidney Disease and Elevated BP in 2-Year-Old Children Born Extremely Premature.

Background And Objectives: Extremely low gestational age neonates born <28 weeks gestation are at risk for chronic disease. We sought to describe the prevalence of kidney outcomes by gestational age and determine risk factors for their development.

Design, Setting, Participants, & Measurements: The Recombinant Erythropoietin for Protection of Infant Renal Disease (REPAIReD) study examined kidney outcomes of extremely low gestational age neonates enrolled in the Preterm Epo NeuroProtection Trial (PENUT) study.

Patent foramen ovale in children: Unique pediatric challenges and lessons learned from adult literature.

A patent foramen ovale (PFO) is a frequent incidental finding during echocardiography in otherwise healthy children. In most healthy children with a diagnosis of isolated incidental PFO, no further follow-up or intervention is necessary. In some children, PFO is associated with certain clinical syndromes such as cryptogenic stroke, decompression sickness, migraine, and platypnea-orthodeoxia syndrome.

Epilepsy and proxy-reported health-related quality of life in children and young people with non-ambulatory cerebral palsy.

Aim: To assess the association between epilepsy characteristics and proxy-reported health-related quality of life (HRQoL) in children and young people with non-ambulatory cerebral palsy (CP) and seizures.

Method: This was a cross-sectional study of 164 children and young people (74 females, 90 males; mean age 10 years 6 months, range 2-21 years, SD 5 years 5 months). Caregivers completed the Child Health Index of Life with Disabilities (CPCHILD) in an outpatient setting.

Fronto-Orbital Advancement for Metopic and Unilateral Coronal Craniosynostoses.

Fronto-orbital advancement remains a powerful technique for the correction of anterior cranial vault differences related to metopic (trigonocephaly) or unilateral coronal (anterior plagiocephaly) craniosynostoses. Traditional fronto-orbital advancement requires access to the forehead and superior 2/3 of the orbit via a coronal incision. The frontal bone and orbital segment (bandeau) are then separated from the skull and reshaped.

Management of Pediatric Pulmonary Vein Stenosis.

Pediatric intraluminal pulmonary vein stenosis has evolved into a chronic illness, with improving survival. Although significant knowledge gaps remain, medical providers have found success in the management of patients with pulmonary vein stenosis using a comprehensive multimodality treatment strategy. This review discusses the core principles employed by 4 centers dedicated to improving pulmonary vein stenosis outcomes, including how to make the diagnosis, educating the family, treatment strategy, the importance of surveillance, and the management of symptoms and comorbidities.

Recurrent Gingival Lesions in a Pediatric Patient.

We present the case of a 13-year-old girl who developed numerous gingival masses that recurred after two prior resections. Following the initial resection as a child, she reported that there was a period of resolution for several years before recurrence as a teenager. After the second resection, the masses recurred after 4 months.

A unique cardiovascular presentation of Marfan syndrome.

We report a neonate with severe Marfan syndrome (MS), prenatally identified to have persistent atrial tachycardia, biventricular dysfunction, and an unusual structure within the atria. Detailed postnatal echocardiographic evaluation and cross-sectional imaging confirmed congenital pseudoaneurysm of the mitral-aortic intervalvular fibrosa. Emergent testing by next-generation sequencing identified a FBN1 pathological variant, key to establishing goals of care.

Rationale and design of ON-TRK: a novel prospective non-interventional study in patients with TRK fusion cancer treated with larotrectinib.

Background: Tropomyosin receptor kinase (TRK) fusion proteins resulting from neurotrophic tyrosine receptor kinase (NTRK) gene fusions are rare primary oncogenic drivers in a wide array of tumors. Larotrectinib is a first-in-class, highly selective, central nervous system-active TRK inhibitor approved by the US Food and Drug Administration (FDA), European Medicines Agency (EMA), and over 40 countries for the treatment of TRK fusion solid tumors in adult and pediatric patients. Due to the rarity of TRK fusion cancer, larotrectinib was granted accelerated approval based on a relatively small number of patients enrolled in three early phase trials.

Efficacy of post-induction therapy for high-risk neuroblastoma patients with end-induction residual disease.

Background: High-risk neuroblastoma patients with end-induction residual disease commonly receive post-induction therapy in an effort to increase survival by improving the response before autologous stem cell transplantation (ASCT). The authors conducted a multicenter, retrospective study to investigate the efficacy of this approach.

Methods: Patients diagnosed between 2008 and 2018 without progressive disease with a partial response or worse at end-induction were stratified according to the post-induction treatment: 1) no additional therapy before ASCT (cohort 1), 2) post-induction "bridge" therapy before ASCT (cohort 2), and 3) post-induction therapy without ASCT (cohort 3).

Gestational Age at Delivery and Neonatal Outcomes among Infants with Gastroschisis in the Children's Hospitals Neonatal Consortium (CHNC).

Objective: The effect of gestational age (GA) on gastroschisis outcomes is unclear and delivery timing varies in practice. We aimed to correlate clinical outcomes of infants with gastroschisis and GA at delivery in the Children's Hospitals Neonatal Consortium (CHNC).

Study Design: This was a retrospective multicenter cohort study of infants with gastroschisis admitted to CHNC neonatal intensive care units (NICUs) from 2010 to 2016.

Current State of Fetal Heart Disease Counseling and Training: Room for Improvement? : Endorsed by the Fetal Heart Society.

We sought to describe the fellowship experiences and current practice habits of pediatric cardiologists who counsel patients with fetal heart disease (FHD) and to identify fellowship experiences related to FHD counseling perceived as valuable by respondents as well as opportunities for improvement. A cross-sectional survey of attending pediatric cardiologists who care for patients with FHD was performed. The respondents' demographics, fellowship experiences related to FHD counseling, reflections on fellowship training, and current practice habits were collected.

Phase II Study of Selumetinib in Children and Young Adults With Tumors Harboring Activating Mitogen-Activated Protein Kinase Pathway Genetic Alterations: Arm E of the NCI-COG Pediatric MATCH Trial.

Purpose: The NCI-COG Pediatric MATCH trial assigns patients age 1-21 years with relapsed or refractory solid tumors, lymphomas, and histiocytic disorders to phase II studies of molecularly targeted therapies on the basis of detection of predefined genetic alterations. Patients with tumors harboring mutations or fusions driving activation of the mitogen-activated protein kinase (MAPK) pathway were treated with the MEK inhibitor selumetinib.

Methods: Patients received selumetinib twice daily for 28-day cycles until disease progression or intolerable toxicity.

Actionable Tumor Alterations and Treatment Protocol Enrollment of Pediatric and Young Adult Patients With Refractory Cancers in the National Cancer Institute-Children's Oncology Group Pediatric MATCH Trial.

Purpose: The National Cancer Institute-Children's Oncology Group Pediatric MATCH trial aimed to facilitate evaluation of molecular-targeted therapies in biomarker-selected cohorts of childhood and young adult patients with cancer by screening tumors for actionable alterations.

Patients And Methods: Tumors from patients age 1-21 years with refractory solid tumors, lymphomas, or histiocytic disorders were subjected to cancer gene panel sequencing and limited immunohistochemistry to identify actionable alterations for assignment to phase II treatment arms. The rates of treatment arm assignment and enrollment were compared between clinical and demographic groups.

Lean methodology in quality improvement.

Measurement of quality and improvement in medicine has existed since Florence Nightingale's time. In modern times, medicine has sought to learn from other high-reliability industries such as aviation and nuclear power, where errors can result in catastrophic outcomes. Lean is a unique quality improvement strategy that seeks to improve both quality and safety by driving out waste and, where possible, standardizing work practices.

Pulmonary and Critical Care Considerations for e-Cigarette, or Vaping, Product Use-Associated Lung Injury.

Background: In 2019, the United States experienced a nationwide outbreak of e-cigarette, or vaping, product use-associated lung injury (EVALI). More than one-half of these patients required admission to an ICU.

Research Question: What are the recent literature and expert opinions which inform the diagnosis and management of patients with critical illness with EVALI?

Study Design And Methods: To synthesize information critical to pulmonary/critical care specialists in the care of patients with EVALI, this study examined data available from patients hospitalized with EVALI between August 2019 and January 2020; reviewed the clinical course and critical care experience with those patients admitted to the ICU; and compiled opinion of national experts.