A multicenter study of the efficacy and safety of sustained release GH in the treatment of naive pediatric patients with GH deficiency

Treatment of naive children with GH deficiency has relied upon long-term re placement therapy with daily injections of GH. The daily schedule may be in convenient for patients and their caregivers, possibly promoting nonadheren ce with the treatment regimen or premature termination of treatment. We stu died a new sustained release GH formulation, administered once or twice mon thly, to determine its efficacy and safety in this population. Seventy-four prepubertal patients with documented GH deficiency were random ized to receive sustained release recombinant human GH at either 1.5 mg/kg once monthly or 0.75 mg/kg twice monthly by se injection in a 6-month open- label study. Efficacy was determined by growth data from 69 patients comple ting 6 months and 56 patients completing 12 months in an extension study. Growth rates were significantly increased over baseline and were similar fo r the two dosage groups. The mean (+/-SD) annualized growth rate (pooled da ta) 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, bo ne age, and predicted adult height assessments demonstrated catch-up growth without excessive skeletal maturation. Injection site-related events (incl uding pain, erythema, and nodules) were the most commonly reported adverse events; no serious adverse events related to treatment were reported. Labor atory studies documented no accumulation of trough GH or IGF-I levels durin g treatment, nor did glucose intolerance or persistent hyperinsulinism deve lop. Sustained release recombinant human GH is safe and effective for long-term GH replacement in children with GH deficiency. Patients achieved similar gr owth velocities when sustained release GH was given once or twice monthly. The enhanced convenience of this dosage form may result in greater long-ter m adherence to the treatment regimen.