PMID- 15899952 OWN - NLM STAT- MEDLINE DCOM- 20051006 LR - 20220309 IS - 0021-972X (Print) IS - 0021-972X (Linking) VI - 90 IP - 9 DP - 2005 Sep TI - Safety of growth hormone treatment in pediatric patients with idiopathic short stature. PG - 5188-96 AB - CONTEXT: Recombinant human GH was approved by the United States Food and Drug Administration in 2003 for the treatment of idiopathic short stature (ISS). However, to date, the safety of GH in this patient population has not been rigorously studied. OBJECTIVE: The objective of this study was to address the safety of GH treatment in children with ISS compared with GH safety in patient populations for which GH has been approved previously: Turner syndrome (TS) and GH deficiency (GHD). DESIGN/SETTING: The rates of serious adverse events (SAEs) and adverse events (AEs) of particular relevance to GH-treated populations were compared across the three patient populations among five multicenter GH registration studies. PATIENTS: Children with ISS, TS, or GHD were studied. INTERVENTION: Treatment consisted of GH doses ranging from 0.18-0.37 mg/kg.wk. MAIN OUTCOME MEASURES: The main outcome measures were rates of SAEs and AEs of special relevance to patients receiving GH. Laboratory measures of carbohydrate metabolism were used as outcome measures for the ISS studies. RESULTS: Within the ISS studies, comprising one double-blind, placebo-controlled study and one open-label, dose-response study, SAEs (mainly hospitalizations for accidental injury or acute illness unrelated to GH exposure) were reported for 13-14% of GH-treated patients. Overall AE rates (serious and nonserious) as well as rates of potentially GH-related AEs were similar in the GHD, TS, and ISS studies (for ISS studies combined: otitis media, 8%; scoliosis, 3%; hypothyroidism, 0.7%; changes in carbohydrate metabolism, 0.7%; hypertension, 0.4%). Measures of carbohydrate metabolism were not affected by GH treatment in patients with ISS. There was no significant GH effect on fasting blood glucose in either study (GH dose range, 0.22-0.37 mg/kg.wk) or on insulin sensitivity (placebo-controlled study only). CONCLUSION: GH appears safe in ISS; however, the studies were not powered to assess the frequency of rare GH-related events, and longer-term follow-up studies of GH-treated patients with ISS are warranted. FAU - Quigley, Charmian A AU - Quigley CA AD - Lilly Research Laboratories, D/C 5015, Indianapolis, Indiana 46285, USA. qac@lilly.com FAU - Gill, Anne M AU - Gill AM FAU - Crowe, Brenda J AU - Crowe BJ FAU - Robling, Kristen AU - Robling K FAU - Chipman, John J AU - Chipman JJ FAU - Rose, Susan R AU - Rose SR FAU - Ross, Judith L AU - Ross JL FAU - Cassorla, Fernando G AU - Cassorla FG FAU - Wolka, Anne M AU - Wolka AM FAU - Wit, Jan M AU - Wit JM FAU - Rekers-Mombarg, Lyset T M AU - Rekers-Mombarg LT FAU - Cutler, Gordon B Jr AU - Cutler GB Jr LA - eng PT - Comparative Study PT - Journal Article PT - Research Support, Non-U.S. Gov't DEP - 20050517 PL - United States TA - J Clin Endocrinol Metab JT - The Journal of clinical endocrinology and metabolism JID - 0375362 RN - 0 (Lipids) RN - 12629-01-5 (Human Growth Hormone) RN - 67763-96-6 (Insulin-Like Growth Factor I) SB - IM CIN - J Clin Endocrinol Metab. 2005 Sep;90(9):5502-4. PMID: 16148349 MH - Adolescent MH - *Body Height MH - Carbohydrate Metabolism MH - Child MH - Child, Preschool MH - Female MH - Growth Disorders/*drug therapy/physiopathology MH - Human Growth Hormone/*adverse effects/deficiency/therapeutic use MH - Humans MH - Insulin-Like Growth Factor I/metabolism MH - Lipids/blood MH - Male MH - Multicenter Studies as Topic MH - Randomized Controlled Trials as Topic MH - Steroid Metabolism, Inborn Errors/drug therapy/physiopathology MH - Thyroid Gland/drug effects/physiopathology MH - Turner Syndrome/drug therapy/physiopathology EDAT- 2005/05/19 09:00 MHDA- 2005/10/07 09:00 CRDT- 2005/05/19 09:00 PHST- 2005/05/19 09:00 [pubmed] PHST- 2005/10/07 09:00 [medline] PHST- 2005/05/19 09:00 [entrez] AID - jc.2004-2543 [pii] AID - 10.1210/jc.2004-2543 [doi] PST - ppublish SO - J Clin Endocrinol Metab. 2005 Sep;90(9):5188-96. doi: 10.1210/jc.2004-2543. Epub 2005 May 17.