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

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,...

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Autores principales: Mandel, Jonas, Bertrand, Viviane, Lehert, Philippe, Attarian, Shahram, Magy, Laurent, Micallef, Joëlle, Chumakov, Ilya, Scart-Grès, Catherine, Guedj, Mickael, Cohen, Daniel
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BioMed Central 2015
Materias:
Letter to the Editor
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4482281/
https://www.ncbi.nlm.nih.gov/pubmed/26070802
http://dx.doi.org/10.1186/s13023-015-0293-y
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author Mandel, Jonas
Bertrand, Viviane
Lehert, Philippe
Attarian, Shahram
Magy, Laurent
Micallef, Joëlle
Chumakov, Ilya
Scart-Grès, Catherine
Guedj, Mickael
Cohen, Daniel
author_facet Mandel, Jonas
Bertrand, Viviane
Lehert, Philippe
Attarian, Shahram
Magy, Laurent
Micallef, Joëlle
Chumakov, Ilya
Scart-Grès, Catherine
Guedj, Mickael
Cohen, Daniel
author_sort Mandel, Jonas
collection PubMed
description 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.
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spelling pubmed-44822812015-06-27 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 Mandel, Jonas Bertrand, Viviane Lehert, Philippe Attarian, Shahram Magy, Laurent Micallef, Joëlle Chumakov, Ilya Scart-Grès, Catherine Guedj, Mickael Cohen, Daniel Orphanet J Rare Dis Letter to the Editor 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. BioMed Central 2015-06-13 /pmc/articles/PMC4482281/ /pubmed/26070802 http://dx.doi.org/10.1186/s13023-015-0293-y Text en © Mandel et al. 2015 This is an Open Access article distributed under the terms of the Creative Commons Attribution License (http://creativecommons.org/licenses/by/4.0), which permits unrestricted use, distribution, and reproduction in any medium, provided the original work is properly credited. The Creative Commons Public Domain Dedication waiver (http://creativecommons.org/publicdomain/zero/1.0/) applies to the data made available in this article, unless otherwise stated.
spellingShingle Letter to the Editor
Mandel, Jonas
Bertrand, Viviane
Lehert, Philippe
Attarian, Shahram
Magy, Laurent
Micallef, Joëlle
Chumakov, Ilya
Scart-Grès, Catherine
Guedj, Mickael
Cohen, Daniel
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
title 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
title_full 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
title_fullStr 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
title_full_unstemmed 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
title_short 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
title_sort 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
topic Letter to the Editor
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4482281/
https://www.ncbi.nlm.nih.gov/pubmed/26070802
http://dx.doi.org/10.1186/s13023-015-0293-y
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