Gaboxadol in angelman syndrome: A double-blind, parallel-group, randomized placebo-controlled phase 3 study.

Purpose: To evaluate efficacy and safety of gaboxadol for treatment of children with Angelman syndrome (AS).

Method: In this international, double-blind, phase 3 trial, we randomized children 4-12 years old with a molecular diagnosis of AS and a Clinical Global Impression (CGI)-severity score ≥3 to either daily administration of weight-based gaboxadol or matching placebo for 12 weeks. The primary endpoint was the CGI-Improvement-AS (CGI-I-AS) score at week 12.

Effect of a Pharmacist-Led Discharge Counseling Service at a Children's Hospital.

Objective: To evaluate the effect of a pharmacist-led discharge counseling service at a pediatric hospital.

Methods: This was a prospective observational cohort study. Patients in the pre-implementation phase were identified by the pharmacist at the time of admission medication reconciliation, whereas patients in the pos-timplementation phase were identified at the time of pharmacist discharge medication counselling.

Effect of a Pharmacist Admission Medication Reconciliation Service at a Children's Hospital.

Objective: To evaluate the clinical effect and estimate cost avoidance attributed to a pharmacist-led admission medication reconciliation service at a children's hospital.

Methods: This was a prospective observational cohort study that measured pharmacist interventions for pediatric patients over a 90-day period. Pharmacists logged all interventions identified during medication reconciliation in real time.

Implementation of a Pharmacist-Driven Vancomycin and Aminoglycoside Dosing Service in a Pediatric Hospital.

Objective: Pharmacy-driven antibiotic dosing services have been shown to improve clinical outcomes in adult patients. This study evaluated the effect of a pharmacist-driven antimicrobial dosing service on the percentage of therapeutic serum concentrations achieved following initial vancomycin or aminoglycoside dosing regimens. A secondary objective was to determine the effect of the dosing service on nephrotoxicity in pediatric patients.

Improving Timeliness of Insulin Administration by Using an Insulin Dose Calculator.

Objectives: Insulin is a high-risk medication, and its dosing depends on the individualized clinical and nutritional needs of each patient. Our hospital implemented an insulin dose calculator (IDC) imbedded in the electronic medical record with the goal of decreasing average wait times in inpatient insulin ordering and administration. In this study, we evaluated whether implementation of an IDC decreased the average wait time for insulin administration for hospitalized pediatric patients.

Validation of the MediByte Portable Monitor for the Diagnosis of Sleep Apnea in Pediatric Patients.

Study Objectives: Polysomnography (PSG) is considered the gold standard in the diagnosis of sleep apnea. In pediatric patients, because of limited availability and access to laboratory-based PSG, there can be significant delays in the diagnosis and management of sleep apnea that can result in progressive associated comorbidities. The main objective of the current study was to test the diagnostic value of a portable sleep monitor (PM), the MediByte, in comparison with laboratory PSG in pediatric patients wearing both setups simultaneously.

Sleep and Delirium in Pediatric Critical Illness: What Is the Relationship?

With growing recognition of pediatric delirium in pediatric critical illness there has also been increased investigation into improving recognition and determining potential risk factors. Disturbed sleep has been assumed to be one of the key risk factors leading to delirium and is commonplace in the pediatric critical care setting as the nature of intensive care requires frequent and invasive monitoring and interventions. However, this relationship between sleep and delirium in pediatric critical illness has not been definitively established and may, instead, reflect significant overlap in risk factors and consequences of underlying neurologic dysfunction.

Early Closure of Gastroschisis After Silo Placement Correlates with Earlier Enteral Feeding.

Objectives: Gastroschisis is a congenital anomaly affecting 2.3-4.4/10,000 births.

Managing hypertension in the newborn infants.

Hypertension in newborn infants, particularly those requiring intensive care, is becoming increasingly recognized, with prevalence of 0.2-3%. Recent studies have established normative tables for blood pressure (BP) in both term and pre-term infants based on the gestational age, postnatal age, gender, weight and height, identifying the neonates at increased risk for early-onset cardiovascular disease.

Strategies to reduce pitfalls in measuring blood pressure.

Errors in blood pressure (BP) measurement are common in the clinical practice. Inaccurate measurements of BP may lead to misdiagnosis and inappropriate treatment of hypertension. The preferred method of BP measurement in the clinical setting is auscultation, using the first and the fifth Korotkoff sounds.

Tobacco exposure in children and adolescents with chronic kidney disease: parental behavior and knowledge. A study from the Midwest Pediatric Nephrology Consortium.

Aim: The incidence of cardiovascular disease (CVD) in children with chronic kidney disease (CKD) is high. Exposure to second hand smoke (SHS) is a known risk factor for CVD. Due to a recent report of high incidence of SHS in children with CKD, we sought to investigate via questionnaire the smoking behaviors of caregivers of children with CKD.

Considerations in individualizing prophylaxis in patients with haemophilia A.

Prophylaxis is considered optimal care for children and adults with severe haemophilia A because of its proven ability to reduce joint and other bleeding episodes. However, a 'one size fits all' approach to prophylaxis is not ideal, potentially leading to over-treatment in some individuals and under-treatment in others. Moreover, a generic plan fails to take into account a patient's lifestyle and personal preferences.

Establishing quality review of cardiac and respiratory arrest in a pediatric intensive care unit.

Cardiac arrest in children is a rare event; however, the outcomes following resuscitation are universally disappointing. Despite widespread recognition of its importance, there is no standard approach to conducting reviews surrounding critical resuscitation events. A standardized approach to the review of respiratory and cardiac arrests occurring in the pediatric intensive care unit focusing on processes of care and team performance was undertaken at a single pediatric academic medical center.

Exploring the biological basis of haemophilic joint disease: experimental studies.

Haemophilia has been recognized as the most severe among the inherited disorders of blood coagulation since the beginning of the first millennium. Joint damage is the hallmark of the disease. Despite its frequency and severity, the pathobiology of blood-induced joint disease remains obscure.

Blood-induced joint disease: the pathophysiology of hemophilic arthropathy.

Arthropathy is a frequent and serious complication of repeated joint bleeding in patients with hemophilia, resulting in pain, deformity, and disability. Although the pathogenesis of hemophilic arthropathy has not been fully elucidated, it appears to have similarities with the degenerative joint damage that occurs in osteoarthritis and the inflammatory processes associated with rheumatoid arthritis. This article reviews the potential actions of various blood constituents on joint components that culminate in the development of hemophilic arthropathy.

Control of disseminated intravascular coagulation in Klippel-Trenaunay-Weber syndrome using enoxaparin and recombinant activated factor VIIa: a case report.

Introduction: Vascular malformation is associated with coagulopathies, especially when hemostasis is challenged.

Case Presentation: We present the case of an 11-year-old Hispanic girl with Klippel-Trenaunay-Weber syndrome that developed disseminated intravascular coagulation after minor surgery, which was controlled by blood product transfusions and enoxaparin to address an ongoing consumptive coagulopathy. The patient, however, developed bacteremia and liver trauma that resulted in severe bleeding.

HTR2A variation and sudden infant death syndrome: a case-control analysis.

Aim: The serotonergic (5-HT) system functions in central autonomic regulation with homeostatic roles in cardiorespiratory control, thermoregulation, arousal and sleep-wake cycling. Altered function and development of this system in cases of sudden infant death syndrome (SIDS) have been established, but the aetiology of these disturbances remains unclear. The serotonin receptor, HTR2A, functions within this system with roles in the homeostatic response to hypoxia including excitatory effects on respiration, gasping and rhythm generation, all functions potentially compromised in SIDS.

Blood-induced joint disease: the confluence of dysregulated oncogenes, inflammatory signals, and angiogenic cues.

Hemophilia is a congenital disorder that commonly results in musculoskeletal bleeding and orthopedic complications. After an acute joint hemorrhage, there is pain, swelling, and limited motion due to an increase in intra-articular pressure and inflammation. Increases in intra-articular pressure induce mechanical signals that lead to cartilage cell apoptosis.

Relation of left ventricular hypertrophy to microalbuminuria and C-reactive protein in children and adolescents with essential hypertension.

Microalbuminuria (MA) and C-reactive protein (CRP) levels are predictors of increased risk for left ventricular hypertrophy (LVH). Whether the strength of association between CRP and LVH is comparable to that of MA in hypertensive children is unknown. CRP and MA were measured in 64 children and adolescents with essential hypertension (HTN).

Sudden infant death syndrome: rare mutation in the serotonin system FEV gene.

Recent studies have identified abnormalities in the development and function of medullary serotonin (5-HT) pathways in postmortem brain from sudden infant death syndrome (SIDS) cases, suggesting 5-HT-mediated dysregulation of the autonomic nervous system (ANS) in SIDS. The human fifth Ewing variant (FEV) gene is specifically expressed in central 5-HT neurons in the brain, with a predicted role in specification and maintenance of serotonergic neuronal phenotype. We hypothesized that variations of FEV may underlie abnormalities of the 5-HT system in SIDS cases and thus may be associated with SIDS risk.

Effect of microalbuminuria lowering on regression of left ventricular hypertrophy in children and adolescents with essential hypertension.

Microalbuminuria (MA) is associated with increased cardiovascular risk in adult hypertensive patients, but no study has specifically examined the effects of MA lowering on regression of left ventricular hypertrophy (LVH) among pediatric patients with hypertension. Fifty-five patients with essential hypertension, 11-19 years old, were prospectively studied. All patients received concomitant therapy of hydrochlorothiazide and angiotensin-converting enzyme inhibitor.

Histological changes in murine haemophilic synovitis: a quantitative grading system to assess blood-induced synovitis.

Haemophilia is a congenital disorder that results in frequent bleeding into joints, in which a chronic and debilitating arthritis develops. The presence of blood evokes an inflammatory and proliferative synovial reaction. Although the molecular mechanisms and biochemical pathways which underlie this disorder are not known, significant advances have been made by studying a murine model of human haemophilic synovitis.