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KMID : 0941820220320040336
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Korean Journal of Clinical Pharmacy
2022 Volume.32 No. 4 p.336 ~ p.351
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Evaluating the Efficacy of Pharmacological Therapy for Prader-Willi Syndrome: A Systematic Review and Meta-analysis
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Yoo A-Lim
Park So-Hyeon
Lee Hee-Young
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Abstract
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Background: Prader-Willi Syndrome (PWS) is a rare genetic disorder. To improve the health deterioration of PWS, investigatingoptimal treatment options for PWS is required. Thus, we aimed to evaluate the efficacy of pharmacotherapies compared withsupportive care or placebos in patients with PWS.
Methods: PubMed and EMBASE databases were used to search for randomizedcontrolled trials (RCTs) evaluating the efficacy of pharmacotherapy in PWS patients. Only RCTs that evaluating the efficacy ofpharmacotherapy in PWS patients were retrieved.
Results: A total of 26 studies were included to evaluate body composition,hormones, glucose levels and hyperphagia behavioral status. Pharmacological treatment group showed a significant decrease ofbody fat (mean difference (MD): -6.32, 95% confidence interval (CI): -10.58 to -2.06, p=0.004), a significant increase of lean bodymass (LBM) (MD: 1.86, 95% CI: 1.43 to 2.30, p<0.00001) and insulin-like growth factor 1 (IGF-1) levels (MD: 241.62, 95% CI:68.59 to 414.64, p=0.006) compared with the control group. Nevertheless, based on other outcomes evaluated by the currentsystematic review, pharmacological options showed different efficacy in treating PWS.
Conclusion: Pharmacological therapies wereeffective to decrease significantly in body fat and increase significantly on LBM and IGF-1 levels in patients with PWS. However,still, individualized therapies should be considered in real-world practice in PWS treatment.
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KEYWORD
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Prader-Willi syndrome, pharmacological therapy, systematic review, meta-analysis, efficacy
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