Elisa Frisaldi1, Aziz Shaibani2, Jan Vollert3,4, Bruno Ferrero1, Roberta Carrino1, Hayan Dhamer Ibraheem2, Lene Vase5, Fabrizio Benedetti1,6. 1. Department of Neuroscience, University of Turin Medical School, Corso Raffaello 30, 10125 Turin, Italy. 2. Nerve & Muscle Center, Houston, Texas, USA. 3. Pain Research, Faculty of Medicine, Department of Surgery & Cancer, Imperial College London, London, United Kingdom. 4. Center of Biomedicine and Medical Technology Mannheim CBTM, Medical Faculty Mannheim, Heidelberg University, Germany. 5. Department of Psychology and Behavioural Sciences, School of Business and Social Sciences, Aarhus University, Aarhus, Denmark. 6. Plateau Rosà Laboratories, Plateau Rosà, Italy/Switzerland.
Abstract
INTRODUCTION: This meta-analysis investigates the placebo response in generalized myasthenia gravis (MG) trials by means of Quantitative Myasthenia Gravis (QMG) scores. METHODS: PubMed, Scopus, Web of Science, Cochrane Controlled Trial Register, and EMBASE were searched. QMG score, dropouts rate, adverse events (AEs), and AEs responsible for dropouts were examined, together with treatment moderators. RESULTS: The magnitude of placebo response showed an effect size of 0.24, which was significantly lower than 0.67 of the drug response (P = 0.019). Furthermore, the forest plot revealed that, overall, active treatments showed a significantly higher impact on QMG scores than placebos. CONCLUSIONS: Placebo and drug responses in MG trials are small and moderate, respectively. The lack of MG trials with a pure placebo arm or a no-treatment control arm made it impossible to disentangle improvements due to the placebo psychological effect from other effects such as natural history and/or regression to the mean. Muscle Nerve 59:671-678, 2019.
INTRODUCTION: This meta-analysis investigates the placebo response in generalized myasthenia gravis (MG) trials by means of Quantitative Myasthenia Gravis (QMG) scores. METHODS: PubMed, Scopus, Web of Science, Cochrane Controlled Trial Register, and EMBASE were searched. QMG score, dropouts rate, adverse events (AEs), and AEs responsible for dropouts were examined, together with treatment moderators. RESULTS: The magnitude of placebo response showed an effect size of 0.24, which was significantly lower than 0.67 of the drug response (P = 0.019). Furthermore, the forest plot revealed that, overall, active treatments showed a significantly higher impact on QMG scores than placebos. CONCLUSIONS: Placebo and drug responses in MG trials are small and moderate, respectively. The lack of MG trials with a pure placebo arm or a no-treatment control arm made it impossible to disentangle improvements due to the placebo psychological effect from other effects such as natural history and/or regression to the mean. Muscle Nerve 59:671-678, 2019.
Authors: Carlos Capella-Peris; Mary M Cosgrove; Irene C Chrismer; M Sonia Razaqyar; Jeffrey S Elliott; Anna Kuo; Magalie Emile-Backer; Katherine G Meilleur Journal: Patient Date: 2020-08 Impact factor: 3.883