Objective: To evaluate safety and efficacy of recombinant human growth hormone treatment in children on long-term glucocorticoid therapy.
Methods: A 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.
Results: Ninety-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.
Conclusion: This 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 registration: NCT00163189.
Keywords: Croissance; Glucocorticosteroid; Glucocorticoïde; Growth; Growth retardation; Hormone de croissance recombinante; Recombinant growth hormone; Retard de croissance; Traitement; Treatment.
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