Growth Hormone Treatment Clinical Trial
Official title:
Effectiveness of Recombinant Human Growth Hormone Therapy for Children With Phelan-McDermid Syndrome: An Open-label, Cross-over, Preliminary Study
In summary, this piot study with 6 participants shown that recombinant human growth hormone (rhGH) has a positive effect on the treatment with PMS. In addition, This study indicated that rhGH can improve PMS symptoms via increase the level of serum insulin-like growth factor-1 (IGF-1) and insulin-like growth factor binding protein 3 (IGFBP-3). RhGH may be low cost, more accessible, alternative treatment for PMS.
Compared with the placebo, rhGH treatment significantly decreased the total scores and subscale scores of GDS (P <0.05), while the total scores and subscale scores of SC-ABC significantly decreased (P < 0.05) following three-months rhGH treatment. The similar results were also observed in comparison with baseline. Compared with the baseline, the level of serum IGF-1 and IGFBP-3 increased significantly (P < 0.05) following three-months rhGH treatment, while the placebo group had no significant impact on serum IGF-1 and IGFBP-3 (P > 0.05). One participant developed skin allergy the day after the first rhGH treatment, which were resolved later. ;
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