A meta-analysis of randomized double-blind clinical trials in CMT1A to assess the change from baseline in CMTNS and ONLS scales after one year of treatmentReportar como inadecuado




A meta-analysis of randomized double-blind clinical trials in CMT1A to assess the change from baseline in CMTNS and ONLS scales after one year of treatment - Descarga este documento en PDF. Documentación en PDF para descargar gratis. Disponible también para leer online.

Orphanet Journal of Rare Diseases

, 10:74

First Online: 13 June 2015Received: 24 April 2015Accepted: 04 June 2015DOI: 10.1186-s13023-015-0293-y

Cite this article as: Mandel, J., Bertrand, V., Lehert, P. et al. Orphanet J Rare Dis 2015 10: 74. doi:10.1186-s13023-015-0293-y

Abstract

CMT1A is the most common inherited peripheral neuropathy. There is currently no approved treatment. We performed a meta-analysis including four randomized, double-blind, Placebo-controlled clinical trials to assess the disease progression after one year under Placebo, Ascorbic Acid AA or PXT3003, a combination of three repurposed drugs. We observed a weak deterioration in patients under Placebo, well below the reported natural disease progression. Patients treated with AA were stable after one year but not significantly different from Placebo. Patients undergoing PXT3003 treatment showed an improvement in CMTNS and ONLS, statistically significant versus Placebo and potentially precursory of a meaningful change in the disease course.

KeywordsCharcot-Marie-Tooth CMT1A CMTNS ONLS PXT3003 Meta-analysis Ascorbic acid Clinical trials Randomized Double blind AbbreviationsCMT1ACharcot-Marie-Tooth disease type 1A

CMTNSCharcot-Marie-Tooth neuropathy score

ONLSOverall neuropathy limitation scale

AAAscorbic acid

SDStandard deviation

Jonas Mandel and Mickael Guedj contributed equally to this work.

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Autor: Jonas Mandel - Viviane Bertrand - Philippe Lehert - Shahram Attarian - Laurent Magy - Joëlle Micallef - Ilya Chumakov - Ca

Fuente: https://link.springer.com/



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