Background Growth hormone (GH) treatment is currently recommended in Prader-Willi syndrome (PWS) patients.
Objectives To evaluate the impact (efficacy and safety) of the use of recombinant human GH (rhGH) as a treatment for PWS.
Method We performed a systematic review and, where possible, meta-analysis for the following outcomes: growth, body mass index, body composition, cognitive function, quality of life, head circumference, motor development/strength, behaviour and adverse effects. We included all PWS patients, with all types of genetic defects and with or without GH deficiency, who participated in rhGH studies performed in infancy, childhood and adolescence, that were either randomised controlled trials (RCTs) (double-blinded or not) or non-randomised controlled trials (NRCTs) (cohort and before and after studies). The databases used were MEDLINE, Embase and Cochrane Central.
Results In 16 RCTs and 20 NRCTs selected, the treated group had an improvement in height (1.67 SD scores (SDS); 1.54 to 1.81); body mass index z-scores (−0.67 SDS; −0.87 to −0.47) and fat mass proportion (−6.5% SDS; −8.46 to −4.54) compared with the control group. Data about cognition could not be aggregated.
Based on high quality evidence, rhGH treatment favoured an improvement of stature, body composition and body mass index, modifying the disease’s natural history; rhGH treatment may also be implicated in improved cognition and motor development in PWS patients at a young age.
Ethics and dissemination The current review was approved by the ethical committee of our institution. The results will be disseminated through conference presentations and publications in peer-reviewed journals.
PROSPERO registration number CRD42019140295
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Contributors All authors contributed to the focus of the systematic review. CP and WB drafted the initial protocol, with guidance and feedback at all stages from SI, RF, ET, DD and MP. CP, SI and RF collected data from the articles. CP, WB, DD and MP interpreted the results. All authors have revised and reviewed the results and have approved the final manuscript.
Funding The authors have not declared a specific grant for this research from any funding agency in the public, commercial or not-for-profit sectors.
Competing interests No, there are no competing interests.
Patient consent for publication Not required.
Provenance and peer review Not commissioned; externally peer reviewed.
Data availability statement Data are available upon reasonable request. All data relevant to the study are included in the article or uploaded as supplementary information. Data from published studies were analysed.