National Cooperative Growth Study: 25 Years of Growth Hormone Data, Insights, and Lessons for Future Registries.

Background: The National Cooperative Growth Study (NCGS) data are reviewed from 1985-2010 to report on final demographic, efficacy, and safety findings, and to illustrate the value of long-term, real-world follow-up to physicians and patients.

Methods: The NCGS was a multicenter, open-label, observational, postmarketing surveillance study of Genentech growth hormone (GH) products for the treatment of children with growth failure in North America.

Findings: Data from 65,205 patients representing 240,951 patient-years of experience were collected.

Proceedings from the Turner Resource Network symposium: the crossroads of health care research and health care delivery.

Turner syndrome, a congenital condition that affects ∼1/2,500 births, results from absence or structural alteration of the second sex chromosome. There has been substantial effort by numerous clinical and genetic research groups to delineate the clinical, pathophysiological, cytogenetic, and molecular features of this multisystem condition. Questions about the molecular-genetic and biological basis of many of the clinical features remain unanswered, and health care providers and families seek improved care for affected individuals.

Characteristics of children with the best and poorest first- and second-year growth during rhGH therapy: data from 25 years of the Genentech national cooperative growth study (NCGS).

Background: Models assessing characteristics contributing to response to recombinant human growth hormone (rhGH) response rarely address growth extremes in both years 1 and 2 or examine how children track from year to year. Using National Cooperative Growth Study (NCGS) data, we determined characteristics contributing to responsiveness to rhGH and the pattern of change from years 1 to 2.

Patients And Methods: Height velocity standard deviation score (HV SDS) for 2 years for prepubertal children with idiopathic GH deficiency (IGHD) (n = 1899) and idiopathic short stature (ISS) (n = 1186) treated with similar doses for two years were computed.

Is there "seasonal" variation in height velocity in children treated with growth hormone? Data from the National Cooperative Growth Study.

Background: Growth rate In children is reported to have seasonal variability. There are fewer published data regarding seasonal variability while on growth hormone (GH) therapy, and none analyzing growth rate with respect to number of daylight hours.

Methods: We analyzed 11,587 3-month intervals in 2277 prepubertal children (boys ages 3-14 years, girls ages 3-12 years) with idiopathic GH deficiency from the National Cooperative Growth Study (NCGS) database.

Intracranial hypertension in pediatric patients treated with recombinant human growth hormone: data from 25 years of the Genentech National Cooperative Growth Study.

Intracranial hypertension (IH) is a rare condition in children. However, a relationship between recombinant human growth hormone (rhGH) therapy and IH has been well documented. Risk factors were assessed for 70 rhGH-naive patients enrolled in the National Cooperative Growth Study with reports of IH after treatment initiation.

An observational study to validate the Satisfaction Measure of the Injection of Growth Hormone Therapy (SMIGHTy) questionnaire.

Objective: The objective of this study was to psychometrically evaluate a tool to measure adult caregivers' level of satisfaction with the delivery device used to administer injections of recombinant human growth hormone (rhGH) to a child - the Satisfaction Measure of the Injection of Growth Hormone Therapy (SMIGHTy*) questionnaire.

Research Design And Methods: One hundred caregivers who administer rhGH to a child using an injection device completed the SMIGHTy questionnaire at baseline and 7-14 days later, and also completed other measures of treatment adherence and treatment satisfaction at baseline.

Main Outcome Measures: SMIGHTy reliability (inter-item and test-retest) and external validity (association with other study measures) were assessed.

A multi-center controlled trial of growth hormone treatment in children with cystic fibrosis.

Objectives: We evaluated safety and efficacy of recombinant human growth hormone (rhGH) for improving growth, lean body mass (LBM), pulmonary function, and exercise tolerance in children with cystic fibrosis (CF) and growth restriction.

Study Design: Multicenter, open-label, controlled clinical trial comparing outcomes in prepubertal children <14 years with CF, randomized in a 1:1 ratio to receive daily rhGH (Nutropin AQ) or no treatment (control) for 12 months, followed by a 6-month observation (month 18). Safety was monitored at each visit, including assessments of glucose tolerance.

Treatment of growth hormone-deficient infants with recombinant human growth hormone to near-adult height: patterns of growth.

Background/aims: To determine adult statures and linear growth patterns of children with growth hormone deficiency (GHD) who began treatment with recombinant human growth hormone (rhGH) in infancy.

Methods: Forty-seven patients with GHD in whom administration of rhGH was initiated at or before 2 years of age and who had achieved near-adult heights (NAH) were identified in the database of the Genentech National Cooperative Growth Study.

Results: After beginning treatment at a mean age of 0.

Bioequivalence studies for three formulations of a recombinant human growth hormone: challenges and lessons learned.

Two bioequivalence (BE) studies in healthy volunteers comparing new formulations of the recombinant human growth hormone (rhGH) Nutropin AQ (somatropin [rDNA origin] injection; Genentech, Inc., South San Francisco, CA) with the currently marketed formulation (5 mg/mL) were conducted to extend available dosing options. All formulations were administered by subcutaneous (SC) injection ranging in volume from 0.

First-year response to rhGH therapy in children with CKD: a National Cooperative Growth Study Report.

A clear definition of the appropriate growth response during recombinant human growth hormone (rhGH) treatment has never been established in the pediatric chronic kidney disease (CKD) population. We present here data from Genentech's National Cooperative Growth Study (NCGS) on the first-year growth response in prepubertal children with CKD. Using NCGS data, we constructed response curves for the first year of rhGH therapy in 270 (186 males, 84 females) naïve-to-treatment, prepubertal children with CKD prior to transplant or dialysis.

A stepwise increase in recombinant human growth hormone dosing during puberty achieves improved pubertal growth: a National Cooperative Growth Study report.

Unlabelled: The magnitude of the pubertal growth spurt contributes to adult height. Children treated with increased doses of recombinant human growth hormone (rhGH) during puberty have shown improved near adult height (NAH) outcomes that varied by treatment duration.

Methods: Males, in a single clinic, treated with a prepubertal dose of rhGH (0.

Stimulant medication use and response to growth hormone therapy: an NCGS database analysis.

Background/aims: Determine (1) frequency of attention-deficit hyperactivity disorder (ADHD) treatment and (2) growth responses in growth hormone (GH)-treated children who are receiving ADHD medication versus GH alone.

Methods: Prepubertal children with idiopathic short stature (ISS) or GH deficiency (IGHD) enrolled in Genentech's National Cooperative Growth Study. ADHD treatment was determined by documentation of psycho-stimulant medication use at enrollment.

Growth response, near-adult height, and patterns of growth and puberty in patients with noonan syndrome treated with growth hormone.

Context: Noonan syndrome (NS) is a heterogeneous genetic disorder characterized by short stature.

Setting: The National Cooperative Growth Study (NCGS), a postmarketing observational study of recombinant human GH (rhGH)-treated children, includes a large cohort of children with NS.

Patients: We studied NCGS-enrolled prepubertal and pubertal children with NS.

Growth hormone responsiveness: peak stimulated growth hormone levels and other variables in idiopathic short stature (ISS): data from the National Cooperative Growth Study.

Hypothesis: In children with idiopathic short stature (ISS), growth hormone (GH) response to a provocative test will be inversely related to the first year response to hGH and be a variable accounting for a degree of responsiveness.

Background: Because high levels of GH are a characteristic of GH insensitivity, such as in Laron syndrome, it is possible that a high stimulated GH is associated with a lower first year height velocity among children diagnosed as having ISS.

Methods: We examined the relationship between the peak stimulated GH levels in 3 ISS groups; GH >10 -<25, 25-40, and >40 ng/mL and the first year growth response to rhGH therapy.

Height velocity targets from the national cooperative growth study for first-year growth hormone responses in short children.

Context: Although GH has been used to treat short stature in GH deficiency (GHD) and other conditions for more than 40 yr, criteria for satisfactorily defining targets for GH responsiveness have never been developed.

Objective: The objective of this study was to present the first-year growth expressed as height velocity (HV) for prepubertal boys and girls with idiopathic GHD, organic GHD, idiopathic short stature, or Turner syndrome from Genentech's National Cooperative Growth Study to derive age-specific targets for GH responsiveness for each etiology and gender.

Design And Population: Using data from the National Cooperative Growth Study, we constructed curves of response to GH during the first year of treatment with standard daily doses in naive-to-treatment prepubertal children with idiopathic GHD (2323 males, 842 females), organic GHD (582 males, 387 females), idiopathic short stature (1392 males, 465 females), or Turner syndrome (1367 females).

Long-term safety of recombinant human growth hormone in turner syndrome.

Context: Turner syndrome (TS) affects more than 50,000 girls and women in the United States. The National Cooperative Growth Study (NCGS) has collected efficacy and safety data for 5220 TS children treated with recombinant human GH (rhGH) during the last 20 yr.

Objectives: Our objective was to determine frequencies of specific targeted adverse events (AEs) and additional AEs of interest in TS patients.

Efficacy of a long-acting growth hormone (GH) preparation in patients with adult GH deficiency.

Context: Treatment of adult GH deficiency (AGHD) with daily injections of GH results in decreased adipose mass, increased lean body mass (LBM), increased bone mineral density, and improved quality of life.

Objective: This study seeks to determine whether a depot preparation of GH given every 14 d would lead to comparable decreases in trunk adipose tissue as daily GH.

Design: This open-label, randomized study compares subjects receiving depot GH, daily GH, or no therapy.

Efficacy and safety results of long-term growth hormone treatment of idiopathic short stature.

Context: Small clinical trials of GH treatment of idiopathic short stature (ISS) show variable efficacy.

Objective: The study was an analysis of a large GH registry for efficacy and safety of GH treatment of ISS. There was also a comparison with a specific clinical trial.

Limited efficacy of growth hormone (GH) during transition of GH-deficient patients from adolescence to adulthood: a phase III multicenter, double-blind, randomized two-year trial.

Context: Treatment of GH-deficient adolescents in transition to adulthood remains challenging.

Objective: The objective was to assess the safety and efficacy of GH in GH-deficient adolescents in transition.

Patients: Fifty-eight GH-deficient adolescents (mean age, 15.

Pharmacokinetics and dose finding of a potent aromatase inhibitor, aromasin (exemestane), in young males.

Suppression of estrogen, via estrogen receptor or aromatase blockade, is being investigated in the treatment of different conditions. Exemestane (Aromasin) is a potent and selective irreversible aromatase inhibitor. To characterize its suppression of estrogen and its pharmacokinetic (PK) properties in males, healthy eugonadal subjects (14-26 yr of age) were recruited.