A 4-Year, Open-Label, Multicenter, Randomized Trial of Genotropin® Growth Hormone in Patients with Idiopathic Short Stature: Analysis of 4-Year Data Comparing Efficacy, Efficiency, and Safety between an Individualized, Target-Driven Regimen and Standard Dosing.

Background/aims: Growth hormone (GH) treatment regimens for children with non-GH-deficient, idiopathic short stature (ISS) have not been optimized. To compare the efficacy, efficiency, and safety of an individualized, target-driven GH regimen with standard weight-based dosing after 4 years of treatment.

Methods: This is a 4-year, open-label, multicenter, randomized trial comparing individualized, formula-based dosing of Genotropin® versus a widely used ISS dose of Genotropin®.

Neuropsychological functioning in girls with premature adrenarche.

Contemporary research indicates that brain development occurs during childhood and into early adulthood, particularly in certain regions. A critical question is whether premature or atypical hormone exposures impact brain development (e.g.

Duration of suppression of adrenal steroids after glucocorticoid administration.

Hydrocortisone has long been the treatment of choice for congenital adrenal hyperplasia (CAH). However, treatment with this medication remains problematic. Patients with 21-hydroxylase deficiency CAH have significant diurnal variation in the secretion of 17-hydroxyprogesterone (17OHP).

Treatment of precocious puberty in a female with Prader-Willi syndrome.

Prader-Willi syndrome (PWS) is characterized by neonatal hypotonia, hyperphagia, childhood obesity at an early age, short stature, varying degrees of mental deficiency, and hypogonadism. In PWS, puberty is usually delayed and fails to complete, with most females never having regular menstrual cycles. We report a female patient with uniparental disomy, who experienced precocious puberty with menarche at age 8 years.

Congenital neonatal hyperthyroidism caused by germline mutations in the TSH receptor gene.

Neonatal hyperthyroidism, a rare and serious disorder, occurs in two forms. An autoimmune form associated with maternal Graves' disease, resulting from transplacental passage of maternal thyroid-stimulating antibodies, and a non-autoimmune form, resulting from mutations in the stimulatory G protein or the thyrotropin receptor (TSHR) causing constitutive activation of intracellular signaling cascades. To date, 29 separate cases of thyrotoxicosis caused by germline mutations of the TSHR have been documented.

Differences in endocrine parameters and psychopathology in girls with premature adrenarche versus on-time adrenarche.

Girls with premature adrenarche (PA) are at risk for multiple problems related to exaggerated androgen synthesis. Whether PA carries a risk of psychopathology remains unknown. This study examined group differences in: (a) anthropometric and endocrine parameters, and (b) mood and behavior problems, in 6-8 year-old girls with PA (n = 40) compared to on-time adrenarche girls (n = 36).

Treatment of precocious puberty in two patients with Turner mosaicism.

Turner's syndrome (TS) is clinically characterized by reduced growth, ovarian dysgenesis and infertility. The majority of patients with TS do not undergo spontaneous pubertal development. We report two patients with mosaic Turner karyotype who experienced precocious pubertal development.

Early puberty in girls: the case of premature adrenarche.

In this article we examine the issue of early puberty in girls. First, a brief overview of normal pubertal development is provided, including the two endocrine components of puberty: gonadarche and adrenarche. Second, we critically discuss the controversy regarding whether puberty truly is occurring earlier in girls.

Treatment to near adult stature of patients with myelomeningocele with recombinant human growth hormone.

Unlabelled: We evaluated near adult statural outcome of treating growth hormone (GH) deficient children (peak GH values <10 ng/ml) with myelomeningocele (MMC) with 0.3 mg/kg/week of recombinant human GH. Retrospective evaluation of 20 patients (12 males) who were consistently measured using recumbent length and who had achieved more than 90% of their adult stature on GH treatment were included.

Circulating plasma leptin and IGF-1 levels in girls with premature adrenarche: potential implications of a preliminary study.

Premature adrenarche is a condition characterized by precocious development of pubic and/or axillary hair, due to early onset of adrenal androgen secretion. Girls with premature adrenarche may later develop menstrual irregularities, hyperandrogenism, and the classic polycystic ovary syndrome. As leptin is thought to modulate the onset of pubertal development, we measured plasma leptin levels in 7 girls with premature adrenarche, and 8 age-matched comparison girls.

Biopsychological and cognitive differences in children with premature vs. on-time adrenarche.

Background: Puberty consists of 2 components: gonadarche and adrenarche. Both components have distinct endocrine changes. Adrenarche has virtually been ignored with respect to examining hormone-behavior relations.

Growth hormone treatment of children with myelomeningocele.

From the National Cooperative Growth Study database 106 patients (53 boys) with myelomeningocele who were treated with recombinant human growth hormone (GH) at 56 centers were identified. Eighty-one patients (41 boys) were prepubertal at enrollment. The mean pretreatment growth rate (GR) in these prepubertal patients was 4.

Growth hormone treatment of children with neural tube defects: results from 6 months to 6 years.

Objective: Patients with neural tube defects (myelomeningocele) have severe growth retardation, and treatment with recombinant human growth hormone (rHGH) for 6 months accelerates growth velocity. We examined patients treated for longer periods to determine whether accelerated growth persists, and whether patients demonstrated to be growth hormone deficient have a greater response to rHGH therapy.

Methods: We retrospectively evaluated the growth rate and length standard deviation score (SDS) of 22 patients in response to treatment with 0.

Growth of growth hormone-treated and nontreated children before and after tethered spinal cord release.

Children with myelomeningocele (MM) are very short and respond to treatment with recombinant human growth hormone (RHGH) with an acceleration in growth. Following primary closure of the MM, a tethered spinal cord may produce neurologic and orthopedic deformities. We compared the short-term growth rate and length standard deviation score (L-SDS) for stature of 13 prepubertal children with MM (7 males, 6 females, mean age 6.

Adult stature and anthropomorphic measurements of patients with myelomeningocele.

Unlabelled: Children with myelomeningocele are extremely short, yet little data exists on adult stature and anthropomorphic measurements. We measured the recumbent length, weight, arm length, sitting height and calculated body mass index of 54 adults with myelomeningocele. Mid parental height was also calculated.

Change in spinal curvature following release of tethered spinal cord associated with spina bifida.

Changes in spinal curvature, scoliosis, kyphosis and lordosis are associated with the growth of patients with myelomeningocele. Previous investigators have stated that progressive developmental scoliosis is related to tethered spinal cord. In order to investigate the relationship of tethered spinal cord release to progression of spinal curvature, we surveyed the medical records of 262 patients with a history of one or more tethered spinal cord release.

Palliative treatment of hyperinsulinism with cyproheptadine and diazoxide.

Treatment for hyperinsulinism in infants and children can be difficult and has included numerous treatment modalities. This paper reports 16 months of palliative treatment with cyproheptadine and diazoxide in a child with hyperinsulinism initially diagnosed at 6 months of age (her insulin level was 80 microU/mL while her glucose level was 38 mg/dL). She continued to have episodes of staring and alteration in level of consciousness while receiving her usual doses of diazoxide (12 mg/kg) alone.