Self-management and spina bifida: A systematic review of the literature.

Background: Self-management is critical to optimizing the health of individuals with a chronic condition or disability and is, therefore, a central concept in individual and family-centered healthcare delivery. The purpose of this review is to report the state of the science of self-management for individuals with spina bifida (SB) from a lifespan perspective.

Objective: This review will summarize the (a) development and use of self-management skills and behaviors across the life span, (b) factors related to self-management behaviors, (c) development of generic or condition-specific measures of self-management used with a spina bifida population, and (d) development and/or outcomes of interventions to improve self-management in SB.

The longitudinal relationship between patient-reported outcomes and clinical characteristics among patients with focal segmental glomerulosclerosis in the Nephrotic Syndrome Study Network.

Background: Understanding the relationship between clinical and patient-reported outcomes (PROs) will help support clinical care and future clinical trial design of novel therapies for focal segmental glomerulosclerosis (FSGS).

Methods: FSGS patients ≥8 years of age enrolled in the Nephrotic Syndrome Study Network completed Patient-Reported Outcomes Measurement Information System PRO measures of health-related quality of life (HRQoL) (children: global health, mobility, fatigue, pain interference, depression, anxiety, stress and peer relationships; adults: physical functioning, fatigue, pain interference, sleep impairment, mental health, depression, anxiety and social satisfaction) at baseline and during longitudinal follow-up for a maximum of 5 years. Linear mixed-effects models were used to determine which demographic, clinical and laboratory features were associated with PROs for each of the eight children and eight adults studied.

Management and Outcomes of Transvenous Pacing Leads in Patients Undergoing Transcatheter Tricuspid Valve Replacement.

Objectives: The aim of this study was to determine the prevalence of pacemaker lead-related complications following transcatheter tricuspid valve replacement (TTVR).

Background: The rate of permanent pacemaker implantation following tricuspid valve (TV) surgery is high, and many patients have transvenous leads. The feasibility, safety, and outcomes of subsequently performing TTVR in the setting of transvenous pacemaker leads have not been established.

Willingness to Vaccinate Children against Influenza after the Coronavirus Disease 2019 Pandemic.

Objectives: To determine factors associated with parents who plan to vaccinate their children against influenza next year, especially those who did not vaccinate against influenza last year using a global survey.

Study Design: A survey of caregivers accompanying their children aged 1-19 years old in 17 pediatric emergency departments in 6 countries at the peak of the coronavirus disease 2019 (COVID-19) pandemic. Anonymous online survey included caregiver and child demographic information, vaccination history and future intentions, and concern about the child and caregiver having COVID-19 at the time of emergency department visit.

The impact of increased awareness of acute kidney injury in the Neonatal Intensive Care Unit on acute kidney injury incidence and reporting: results of a retrospective cohort study.

Objective: To evaluate the impact of nephrology integration in the NICU on acute kidney injury (AKI) incidence, provider reporting, and nephrology referral.

Study Design: Cohort study in a single-center NICU from January 2012 to December 2017 (n = 1464). We assessed the impact of clinical practice changes including neonatal-nephrology rounds on the incidence of AKI.

Reversal of severe vergence anomaly associated with convergence insufficiency.

Central disruption of fusion refers to a subject's inability to fuse images, resulting in constant diplopia. Severely reduced vergences, or vergence anomalies, producing markedly decreased fusional amplitudes resembling fusional disruption have not been reported previously with convergence insufficiency. We report 3 patients with severe vergence anomalies in the setting of convergence insufficiency.

Severe Acute Kidney Injury Is Associated With Increased Risk of Death and New Morbidity After Pediatric Septic Shock.

Objectives: Acute kidney injury is common in critically ill children; however, the frequency of septic shock-associated acute kidney injury and impact on functional status are unknown. We evaluated functional outcomes of children with septic shock-associated acute kidney injury.

Design: Secondary analysis of patients with septic shock from the prospective Life after Pediatric Sepsis Evaluation study.

The Low Medial Horizontal Osteotomy in Patients With Atypical Ramus Morphology Undergoing Sagittal Split Osteotomy.

Purpose: Sagittal split osteotomy (SSO) of rami with fused cortices or minimal marrow space above the lingula can increase the risk of an unfavorable osteotomy split of the proximal ramus. To reduce the risk of a proximal segment fracture, a recent modification of the SSO places the medial horizontal osteotomy below the lingula. The purpose of the present study was to evaluate the outcomes of SSOs utilizing a low medial horizontal osteotomy in patients with atypical ramus morphologies.

Single-access ultrasound-guided tunneled femoral lines in critically ill pediatric patients.

Central venous access is an essential aspect of critical care for pediatric patients. In the critically ill pediatric population, image-guided procedures performed at the bedside expedite care and may reduce risks and logistical challenges associated with patient transport to a remote procedure suite such as interventional radiology. We describe our institutional technique for ultrasound-guided tunneled femoral venous access in neonates and infants and provide technical pearls from our experience, with an intended audience including specialists performing point-of-care ultrasound-guided procedures as well as interventional radiologist making their services available in the intensive care unit.

Transforaminal intrathecal delivery of nusinersen for older children and adults with spinal muscular atrophy and complex spinal anatomy: an analysis of 200 consecutive injections.

Background: Nusinersen is the only approved treatment for all spinal muscular atrophy (SMA) subtypes and is delivered intrathecally. Distorted spinal anatomy and instrumentation preclude standard approaches for intrathecal access, necessitating alternative techniques for delivery. The purpose of this study is to report technical success and adverse events of transforaminal intrathecal delivery of nusinersen.

Submucosal Supernumerary Smooth Muscle Coat: A Common Histologic Finding in Mowat-Wilson Syndrome With or Without Hirschsprung Disease.

Background: Mowat-Wilson syndrome (MWS) is a multiorgan system disorder caused by (zinc finger E-box-binding homeobox 2) mutations or deletions. One common manifestation is constipation, and approximately half of the patients have Hirschsprung disease (HSCR). In addition to classic histologic features of HSCR, an unusual supernumerary intestinal muscle coat was recently reported in a patient of MWS with HSCR.

Imaging of Nontraumatic Orbital and Neuro-ophthalmological Emergencies.

Emergency department (ED) visits for eye and vision-related concerns are common. In most cases, accurate diagnosis is made using clinical history and physical exam findings without the need for emergent imaging. When orbital imaging is performed in the ED, it is often in patients with orbital trauma where CT is used to assess for orbital fractures and associated injuries.

Irinotecan, Temozolomide, and Dinutuximab With GM-CSF in Children With Refractory or Relapsed Neuroblastoma: A Report From the Children's Oncology Group.

Purpose: The combination of irinotecan, temozolomide, dintuximab, and granulocyte-macrophage colony-stimulating factor (I/T/DIN/GM-CSF) demonstrated activity in patients with relapsed/refractory neuroblastoma in the randomized Children's Oncology Group ANBL1221 trial. To more accurately assess response rate and toxicity, an expanded cohort was nonrandomly assigned to I/T/DIN/GM-CSF.

Patients And Methods: Patients were eligible at first relapse or first designation of refractory disease.

Longer upper airway lengths in Robin Sequence: A case-control study using computed tomography.

Background: Direct laryngoscopy and intubation are often difficult in children with Robin Sequence. Previous research characterizing anatomic airway differences has focused on parameters influencing airway patency; there is a paucity of data pertaining to intubation trajectories and depth. Such information could impact airway management approaches and decrease the incidence of endotracheal tube malpositioning.

Predicting Mortality in Children With Pediatric Acute Respiratory Distress Syndrome: A Pediatric Acute Respiratory Distress Syndrome Incidence and Epidemiology Study.

Objectives: Pediatric acute respiratory distress syndrome is heterogeneous, with a paucity of risk stratification tools to assist with trial design. We aimed to develop and validate mortality prediction models for patients with pediatric acute respiratory distress syndrome.

Design: Leveraging additional data collection from a preplanned ancillary study (Version 1) of the multinational Pediatric Acute Respiratory Distress syndrome Incidence and Epidemiology study, we identified predictors of mortality.

Development of a prenatal clinical care pathway for uncomplicated gastroschisis and literature review.

Background: Gastroschisis is an abdominal wall defect wherein the bowel is herniated into the amniotic fluid. Controversy exists regarding optimal prenatal surveillance strategies that predict fetal well-being and help guide timing of delivery. Our objective was to develop a clinical care pathway for prenatal management of uncomplicated gastroschisis at our institution.

Exploring Longitudinal Mechanisms of Irritability in Children: Implications for Cognitive-Behavioral Intervention.

Severe irritability is a common and clinically important problem longitudinally associated with internalizing and externalizing problems in children. To better understand these mechanisms and to inform treatment research, we tested cognitive-behavioral processes as candidate mediators in the paths from irritability to later problems. Methods: A school sample (N = 238, 48% female, ages 8-10) was assessed at ~6-month intervals in fall (T1) and spring (T2) of third to fourth grade, and again the following fall (T3).

Early Use of Adjunctive Therapies for Pediatric Acute Respiratory Distress Syndrome: A PARDIE Study.

Few data exist to guide early adjunctive therapy use in pediatric acute respiratory distress syndrome (PARDS). To describe contemporary use of adjunctive therapies for early PARDS as a framework for future investigations. This was a preplanned substudy of a prospective, international, cross-sectional observational study of children with PARDS from 100 centers over 10 study weeks.

Dose-Ranging Effect of Adjunctive Oral Cannabidiol vs Placebo on Convulsive Seizure Frequency in Dravet Syndrome: A Randomized Clinical Trial.

Importance: Clinical evidence supports effectiveness of cannabidiol for treatment-resistant seizures in Dravet syndrome, but this trial is the first to evaluate the 10-mg/kg/d dose.

Objective: To evaluate the efficacy and safety of a pharmaceutical formulation of cannabidiol, 10 and 20 mg/kg/d, vs placebo for adjunctive treatment of convulsive seizures in patients with Dravet syndrome.

Design, Setting, And Participants: This double-blind, placebo-controlled, randomized clinical trial (GWPCARE2) recruited patients from April 13, 2015, to November 10, 2017, with follow-up completed on April 9, 2018.