Spasticity and Dystonia are Underidentified in Young Children at High Risk for Cerebral Palsy.

Background: Early spasticity and dystonia identification in cerebral palsy is critical for guiding diagnostic workup and prompting targeted treatment early when it is most efficacious. However, differentiating spasticity from dystonia is difficult in young children with cerebral palsy.

Methods: We sought to determine spasticity and dystonia underidentification rates in children at high risk for cerebral palsy (following neonatal hypoxic-ischemic encephalopathy) by assessing how often child neurologists identified hypertonia alone versus specifying the hypertonia type as spasticity and/or dystonia by age 5 years.

The Failed Bidirectional Glenn Shunt: Risk Factors for Poor Outcomes and the Role of Early Reoperation.

Bidirectional Glenn shunt (BDG) failure carries high morbidity and mortality but the clinical factors associated with failure and the optimal management strategy are understudied. A total of 217 patients undergoing BDG at our institution between 1989 and 2020 were retrospectively reviewed and categorized as success or failure. Failure was defined as the need for reoperation (BDG takedown, reoperation for correction of cardiac defect, and/or transplantation) at any time postoperatively; operative mortality (death attributable to BDG malfunction occurring during the index hospitalization for BDG or within 30 days of discharge); or late mortality (death directly attributable to BDG malfunction occurring prior to Fontan or next-stage palliation).

Acute Kidney Injury Among Children Admitted With Viral Rhabdomyolysis.

Background: Infectious etiologies cause a large portion of pediatric rhabdomyolysis. Among pediatric patients with rhabdomyolysis, it is unknown who will develop acute kidney injury (AKI). We sought to test the hypothesis that a viral etiology would be associated with less AKI in children admitted with rhabdomyolysis than a nonviral etiology.

Objective and Clinically Feasible Analysis of Diffusion MRI Data can Help Predict Dystonia After Neonatal Brain Injury.

Background: Dystonia in cerebral palsy is debilitating but underdiagnosed precluding targeted treatment that is most effective if instituted early. Deep gray matter injury is associated with dystonic cerebral palsy but is difficult to quantify. Objective and clinically feasible identification of injury preceding dystonia could help determine the children at the highest risk for developing dystonia and thus facilitate early dystonia detection.

Effects of variations in access to care for children with atopic dermatitis.

Background: An estimated 50% of children in the US are Medicaid-insured. Some of these patients have poor health literacy and limited access to medications and specialty care. These factors affect treatment utilization for pediatric patients with atopic dermatitis (AD), the most common inflammatory skin disease in children.

Barriers to Tobacco Cessation for Caregivers of Hospitalized Children: Perspectives of Pediatric Hospitalists.

Objectives: Secondhand smoke exposure is associated with adverse health outcomes in children, yet tobacco cessation efforts for caregivers of hospitalized children are lacking. We sought to explore pediatric hospitalists' attitudes and barriers to providing tobacco cessation for caregivers of hospitalized children.

Methods: We conducted a cross-sectional survey of pediatric hospitalists and fellows at 7 hospitals from November 1, 2018, to November 30, 2019.

A phase 2, open-label study of single-dose dupilumab in children aged 6 months to <6 years with severe uncontrolled atopic dermatitis: pharmacokinetics, safety and efficacy.

Background: Dupilumab has demonstrated efficacy and acceptable safety in adults and children (aged 6-17 years) with moderate-to-severe atopic dermatitis (AD), but effective systemic therapy with a favorable risk-benefit profile in younger children remains a significant unmet need.

Objectives: To determine the pharmacokinetics, safety and efficacy of single-dose dupilumab in children with severe AD aged ≥6 months to <6 years.

Methods: This open-label, multicenter, phase 2, sequential, two-age cohort, two-dose level study (LIBERTY AD PRE-SCHOOL; NCT03346434) included an initial cohort of older children aged ≥2 to <6 years, followed by a younger cohort aged ≥6 months to <2 years.

Frey syndrome-like developmental dysautonomia in a child with PHACE syndrome.

PHACE syndrome classically presents with a large, segmental facial infantile hemangioma (IH) associated with structural and vascular abnormalities involving the head and neck, heart, and eyes. We evaluated an infant who presented with ptosis caused by a clinically subtle, deep right-sided periorbital IH identified on MRI that also incidentally revealed hypoplasia of the right common carotid and right internal carotid arteries, supporting a diagnosis of PHACE syndrome. She subsequently developed acute-onset, transient right-sided facial erythema without anisocoria, triggered by feeding and emotional stress.

Clinically Meaningful Responses to Dupilumab in Adolescents with Uncontrolled Moderate-to-Severe Atopic Dermatitis: Post-hoc Analyses from a Randomized Clinical Trial.

Background: Atopic dermatitis is a chronic inflammatory condition with substantial burden and limited treatment options for adolescents with moderate-to-severe disease. Significantly more patients treated with dupilumab vs. placebo achieved Investigator's Global Assessment 0/1 at week 16.

A Systematic Scoping Literature Review of Publications Supporting Treatment Guidelines for Pediatric Atopic Dermatitis in Contrast to Clinical Practice Patterns.

Introduction: Treatment guidelines endorse a variety of strategies for atopic dermatitis (AD) which may vary from published data and clinical practice patterns. The objective of this review was to quantify the volume of available medical literature supporting pediatric AD treatments and compare these patterns to those recommended by published guidelines and/or clinical practice patterns.

Methods: Searches of Embase (2005-2016) and abstracts from selected meetings (2014-2016) related to AD treatment in patients younger than 17 years of age yielded references that were assessed by study design, primary treatment, age groups, and AD severity.

Developing drugs for treatment of atopic dermatitis in children (≥3 months to <18 years of age): Draft guidance for industry.

Atopic dermatitis is the most common chronic skin disease, and it primarily affects children. Although atopic dermatitis (AD) has the highest effect on burden of skin disease, no high-level studies have defined optimal therapy for severe disease. Corticosteroids have been used to treat AD since the 1950s and remain the only systemic medication with Food and Drug Administration approval for this indication in children, despite published guidelines of care that recommend against this option.

Access to the native atria following conduit total cavopulmonary anastomosis.

Objectives: We describe the use of trans-thoracic and trans-conduit puncture to access the atria and perform interventional procedures in patients who have undergone conduit total cavopulmonary anastomosis.

Background: Catheter access to the atria following intra or extra-cardiac Fontan is desirable when there is a need for trans-atrial interventions.

Methods: Between 2009 and 2014, 5 patients ages 7 to 28 years underwent this approach; three trans-thoracic and 2 trans-conduit punctures.

Topical calcineurin inhibitors and lymphoma risk: evidence update with implications for daily practice.

Topical calcineurin inhibitors (TCIs), commercially available since 2000-2001, are the first and only topical medications approved for chronic treatment of atopic dermatitis (AD) in pediatric patients and remain a welcomed alternative to topical corticosteroids. In January 2006, the US Food and Drug Administration (FDA) issued a boxed warning requirement based on a theoretical risk of malignancy (including lymphoma) with TCI use. However, in the years since, analyses of epidemiologic and clinical data have failed to demonstrate a causal relationship between TCI use and malignancy or lymphoma risk, especially for pimecrolimus cream.

Cardiac surgery in the newborn: improved results in the current era.

Pediatric cardiovascular surgeons can now safely operate on newborns to palliate or completely correct congenital heart defects in babies as small as 1.5-2.0 kilograms.

Pulmonary pathology.

Common causes of neonatal respiratory distress include meconium aspiration, pneumonia, persistent pulmonary hypertension of the newborn, pneumothorax and cystic adenomatoid malformation. Genomics and proteomics have enabled the recent recognition of several additional disorders that lead to neonatal death from respiratory disease. These are broadly classified as disorders of lung homeostasis and have pathological features of proteinosis, interstitial pneumonitis or lipidosis.

Is delayed operative treatment worth the trouble with perforated appendicitis is children?

Background: Approximately 30% to 50% of appendicitis in children is already perforated at presentation. The optimal management of these children remains controversial.

Methods: Ninety-six children (aged 2 to 16 years) were treated for perforated appendicitis.

Pericardial cysts in children: surgical or conservative approach?

The authors present a case of an 11-year-old boy with a history of mild asthma and cough who underwent radiographic examination of the chest. The radiograph showed a round, discrete mass in the right cardiophrenic angle. The patient underwent thoracoscopic excision, and histologic examination found a unilocular, pericardial cyst.

Umbilical cord blood transplantation in Wiskott Aldrich syndrome.

Objective: To report the use of umbilical cord blood (UCB) stem cell transplantation in Wiskott Aldrich syndrome (WAS) when a matched sibling donor was unavailable.

Methods: Three children with WAS received unrelated umbilical cord blood stem cell transplantation after a preparative regimen for the treatment of combined immunodeficiency diseases. The patients ranged in age from 1.

Defective pulmonary development in the absence of heparin-binding vascular endothelial growth factor isoforms.

Development of the airways, alveoli, and the pulmonary vasculature in the fetus is a process that is precisely controlled. One of the growth factors involved, vascular endothelial growth factor (VEGF), is so critical for embryonic development that in the mouse, elimination of just a single allele is lethal. In the early stages of lung development, the mouse VEGF gene expresses three isoforms (120, 164, and 188) in a distinct temporo-spatial pattern, suggesting a specific function for each.

Abdominal wall defects.

Survival for newborns with congenital abdominal wall defects (primarily omphalocele and gastroschisis) has improved, but controversy remains regarding etiology, anatomy and embryology, the role of prenatal diagnosis and mode of delivery, and initial management. A number of recent studies have added to our knowledge and understanding of several of these topics, while several others have raised questions regarding traditional initial management of these infants. Continued improvement in the survival of these infants can be anticipated with further understanding of the in utero and antepartum diagnosis and management of infants with these common congenital abnormalities.

Human umbilical cord blood cells can be induced to express markers for neurons and glia.

Rare cells are present in human umbilical cord blood that do not express the hematopoietic marker CD45 and in culture do not produce cells of hematopoietic lineage. These umbilical cord multipotent stem cells (UC-MC) behave as multilineage progenitor cells (stem cells) and can be expanded in tissue culture. Exposure to basic fibroblast growth factor (bFGF) and human epidermal growth factor (hEGF) for a minimum of 7 days in culture induces expression of neural and glial markers.