Efficacy and safety of rhGH in juvenile dermatomyositis children with glucocorticoid-related growth failure.

OBJECTIVE

To evaluate the efficacy and safety of recombinant human growth hormone (rhGH) in children with JDM experiencing glucocorticoid (GC)-induced growth failure.

METHODS

A retrospective cohort study included 16 refractory JDM patients treated with rhGH (0.10-0.15 IU/kg/day) for 6-24 months. Outcomes including height Z-score, growth velocity (GV), biochemical markers, disease activity and adverse events were recorded and analysed.

RESULTS

After 6 months of rhGH treatment, the height Z-score improved from -2.37 ± 1.15 at baseline to -1.73 ± 1.06 (P < 0.001), and GV increased from 2.74 ± 1.53 cm/year to 13.05 ± 3.64 cm/year (P < 0.001). Serum insulin-like growth factor 1 and alkaline phosphatase levels rose significantly (from 264.13 ± 81.24-351.59 ± 123.41 ng/ml and 242.40 ± 42.25-318.70 ± 86.12 U/l, respectively; P < 0.05). Creatine kinase levels increased but remained within the normal range (from 79.33 ± 27.56-111.60 ± 42.84 U/l; P < 0.01). Only one patient (1/16, 6.25%) experienced JDM relapse, a rate lower than the reported recurrence rate (31.6%) in JDM patients not receiving rhGH treatment‌. No adverse metabolic or endocrine effects were observed.

CONCLUSION

In JDM patients with growth failure, rhGH therapy may be considered as a potential treatment option, supported by preliminary evidence. Our study suggests an acceptable safety profile and potential efficacy in improving growth parameters, even in refractory cases. Further larger-scale multicentre studies will be necessary to confirm its application value in JDM.