Efficacy and safety of recombinant growth hormone treatment in children with growth retardation related to long-term glucocorticosteroid therapy

Abstract

ObjectiveTo evaluate safety and efficacy of recombinant human growth hormone treatment in children on long-term glucocorticoid therapy. MethodsA 5-year prospective open-label study included children on glucocorticoid therapy with either standard deviation score (SDS)<−2 for height for chronological age (CA) if naïve to growth hormone treatment, or annual growth rate≥0 SDS for CA if currently receiving growth hormone. ResultsNinety-eight patients began treatment, 63 discontinued; 59 were analyzed for safety and 58 for efficacy. There was male predominance (78.0%). Median age was 13.0 years. Median height screening was 136.0cm (range, 95.1–159.7cm). Mean SDS for height for CA in the efficacy analysis set was −2.91±1.19 (range, −7.49 to −0.96). Mean growth hormone dose was 0.4, 0.4, 0.4 and 0.3mg/kg/week at month 0, M12, M24, and M36, respectively. Primary analysis of change in SDS for height for CA from baseline to M36 showed a significant increase of 0.80±1.03. Twenty patients in the safety analysis set had≥1 treatment-emergent adverse event (TEAE) related to study treatment. Two patients experienced serious treatment-related TEAEs: 1 case of poor compliance, and 1 of mild hyperglycemia, both already observed under growth hormone treatment. ConclusionThis study suggests that growth hormone treatment could be effective in increasing height in children on long-term glucocorticoid treatment with a safety profile comparable to that in approved rhGH treatment indications. Clinical trial registrationNCT00163189.