Efficacy and safety of long-acting growth hormone in children with short stature: a systematic review and meta-analysis

Purpose Long-acting growth hormone (GH) has been developed to address the noncompliance and decreased efficacy associated with daily GH injections. We aimed to evaluate the efficacy and safety of long-acting GH replacement therapy in children with short stature. Methods Randomized controlled trials (RCTs) that investigated the efficacy and safety of long-acting GH therapy in children with short stature in comparison with daily GH injections were searched in Medline, Embase, and the Cochrane Central Register of Controlled Trials. A random-effect model was used to pool data using mean difference and odds ratios (OR). (PROSPERO registration number: CRD42018111105). Results Seven relevant studies were finally included. Meta-analysis found there was no significant difference between high-dose long-acting GH and daily GH in terms of height velocity (HV) (mean difference (MD) = −0.10, 95% CI, −0.79 to 0.60, P = 0.79). Moreover, no significant difference was observed in height standard deviation scores (Ht SDS) between high-dose long-acting GH and daily GH (MD = −0.07, 95% CI, −0.18 to 0.03, P = 0.17). Treatment with high-dose long-acting GH significantly increased IGF-1 SDS when compared with daily GH (MD = 0.31, 95% CI, 0.06–0.56, P = 0.02). In safety assessment, no significant difference was observed in the incidence of adverse events between high-dose long-acting GH and daily GH (OR 1.42, 95% CI, 0.65–3.11, P = 0.38). Conclusions There is no evidence to support differences in the effects of long-acting GH compared with those of daily GH. More RCTs that focus on the safety of high-dose long-acting GH treatment, especially the detection of adverse events caused by elevated levels of serum IGF-1, are needed in the future.