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A Multicenter Study of the Efficacy and Safety of Sustained Release GH in the Treatment of Naive Pediatric Patients with GH Deficiency

Baystate Medical Center Children’s Hospital, Tufts University School of Medicine (E.O.R.), Springfield, Massachusetts 01199; Genentech, Inc. (K.M.A.), South San Francisco, California 94080; Children’s Hospital of Philadelphia (T.M.), Philadelphia, Pennsylvania 19104; Children’s Memorial Hospital (B.L.S.), Chicago, Illinois 60614; Arkansas Children’s Hospital (S.F.K.), Little Rock, Arkansas 72202; Alkermes, Inc. (R.B.N., K.M.F.), Cambridge, Massachusetts 02139; and Montefiore Medical Center (P.S.), Bronx, New York 10467

Address all correspondence and requests for reprints to: Edward O. Reiter, M.D., Department of Pediatrics, Baystate Medical Center Children’s Hospital, Tufts University School of Medicine, Springfield, Massachusetts 01199. E-mail: edward.reiter{at}bhs.org

Abstract

Treatment of naive children with GH deficiency has relied upon long-term replacement therapy with daily injections of GH. The daily schedule may be inconvenient for patients and their caregivers, possibly promoting nonadherence with the treatment regimen or premature termination of treatment. We studied a new sustained release GH formulation, administered once or twice monthly, to determine its efficacy and safety in this population.

Seventy-four prepubertal patients with documented GH deficiency were randomized to receive sustained release recombinant human GH at either 1.5 mg/kg once monthly or 0.75 mg/kg twice monthly by sc injection in a 6-month open-label study. Efficacy was determined by growth data from 69 patients completing 6 months and 56 patients completing 12 months in an extension study.

Growth rates were significantly increased over baseline and were similar for the two dosage groups. The mean (±SD) annualized growth rate (pooled data) was 8.4 ± 2.1 cm/yr at 6 months, and the growth rate was 7.8 ± 1.8 at 12 months compared with 4.5 ± 2.3 at baseline. Standardized height, bone age, and predicted adult height assessments demonstrated catch-up growth without excessive skeletal maturation. Injection site-related events (including pain, erythema, and nodules) were the most commonly reported adverse events; no serious adverse events related to treatment were reported. Laboratory studies documented no accumulation of trough GH or IGF-I levels during treatment, nor did glucose intolerance or persistent hyperinsulinism develop.

Sustained release recombinant human GH is safe and effective for long-term GH replacement in children with GH deficiency. Patients achieved similar growth velocities when sustained release GH was given once or twice monthly. The enhanced convenience of this dosage form may result in greater long-term adherence to the treatment regimen.

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