A novel treatment for height growth in patients with growth hormone insensitivity syndrome by cyproheptadine hydrochloride.

BACKGROUND: Cyproheptadine HCl (CyproH) is an appetite-stimulating drug and while it was prescribed for a patient with growth hormone insensitivity syndrome (GHIS) for increasing appetite, his height growth was surprisingly increased. Therefore, the aim of this study was to investigate the effect of CyproH on growth parameters of the patients with GHIS.

PATIENTS AND DESIGN: Twenty patients were enrolled in two prospective cohorts at two different times. Fifteen cases were observed for 1.17 ± 1.3 years without treatment (observation period, OP). Then, CyproH was administered for 2.2 ± 2.7 years (treatment period, TP), and growth parameters were compared within these two periods. Five patients who did not receive any treatment for 1-8.24 years (4 ± 2.9) were the control group.

RESULTS: Height velocity (HV) increased from 1.88 ± 0.7 to 6.1 ± 0.8 cm/year and HV-SDS reached from -4.5 ± 0.74 to -0.21 ± 1.2 in OP and TP, respectively (P < .001), whereas HV and HV-SDS were 2.2 ± 1.1 cm/yr and -4.2 ± 1.2, respectively, in controls (P < .001). Height SDS was -7.0 ± 1.7 and increased to -6 ± 2.2 after treatment (P = .002). Gain in height was 2.3 ± 0.6 SDS in 5 patients who were treated for 5.4 ± 2.8 years. BMI-SDS was not significantly changed within two time periods and also in cases and controls.

CONCLUSION: CyproH caused height growth in the patients with GHIS, and therefore, this treatment can be considered as an alternative option to IGF-I injection.