Purpose: The objectives of the ongoing, Phase 3, open-label extension trial enliGHten are to assess the long-term safety and efficacy of weekly administered long-acting growth hormone lonapegsomatropin in children with growth hormone deficiency.
Methods: Eligible subjects completing a prior Phase 3 lonapegsomatropin parent trial (heiGHt or fliGHt) were invited to participate. All subjects were treated with lonapegsomatropin.
Introduction: The phase 3 fliGHt Trial evaluated the safety and tolerability of once-weekly lonapegsomatropin, a long-acting prodrug, in children with growth hormone deficiency (GHD) who switched from daily somatropin therapy to lonapegsomatropin.
Methods: This multicenter, open-label, 26-week phase 3 trial took place at 28 sites across 4 countries (Australia, Canada, New Zealand, and the USA). The trial enrolled 146 children with GHD, 143 of which were previously treated with daily somatropin.
Background: Type 1 diabetes is associated with significant mortality and economic cost. Management of type 1 diabetes involves completing multiple daily adherence behaviors, and many adolescents struggle with self-management and show poor glycemic control.
Purpose: The purpose was to conduct an unblinded pilot randomized controlled parallel-group study of a web-delivered multicomponent intervention targeting self-monitoring of blood glucose, working memory, and parent supervision of diabetes care among adolescents with type 1 diabetes.
Objective: The case report details an unusual presentation of a teenage patient with hypophosphatasia.
Patient And Methods: A 17 year-old female patient presented to endocrinology for the evaluation of fatigue and possible adrenal insufficiency. In the course of her clinical evaluation she was noted to have a low serum alkaline phosphatase activity.
Objective. The purpose of this study was to examine the feasibility and effectiveness of a web-delivered multicomponent behavioral and family-based intervention targeting self-regulation and self-monitoring of blood glucose levels (SMBG) and glycemic control (HbA1c) in teens with type 1 diabetes (T1DM) living in rural US. Methods.
View Article and Find Full Text PDFPurpose Of Review: This review focuses on recently published literature on pituitary tumors in children with special focus on craniopharyngioma and prolactinoma. Although most pituitary masses are benign neoplasms, they are associated with high morbidity and mortality related to their proximity to vital neurologic structures including the optic chiasm, hypophysis and hypothalamus, resulting in disruption of pituitary hormone secretion.
Recent Findings: The role of Wingless/β-beta catenin signaling and aryl hydrocarbon receptor-interacting protein mutations in pituitary tumor formation has been recognized.
Objective: The purpose of this study was to determine the effect of cinnamon on glycemic control in adolescents with type 1 diabetes.
Research Design And Methods: Using a prospective, double-blind, placebo-controlled design, 72 adolescent type 1 diabetic subjects were treated in an outpatient setting with cinnamon (1 g/day) or an equivalent-appearing placebo for 90 days. A1C, total daily insulin intake, and adverse events were recorded and compared between groups.
Diabetes care has become more complex as we strive to help children with diabetes achieve and maintain tight glycemic control. The technologic advances described herein have provided the means for safer, more convenient, and more effective diabetes care.
View Article and Find Full Text PDFActivation of vitamin D requires hepatic 25-hydroxylation and renal 1alpha-hydroxylation. Defects in renal P450c1alpha are well-described, but few patients with defective vitamin D 25-hydroxylation are reported. The cytochrome P450 enzymes CYP2D6 and CYP27 are potential 25-hydroxylases.
View Article and Find Full Text PDFObjectives: To identify and study adults (21 years or older) who have a 46,XY karyotype and presented as infants or children with genital ambiguity, including a small phallus and perineoscrotal hypospadias, reared male or female.
Methods: Participants were classified according to the cause underlying their intersex condition based on review of medical and surgical records. Long-term medical and surgical outcome was assessed with a written questionnaire and physical examination.