Phrenic nerve study as outcome in clinical trials for amyotrophic lateral sclerosis

Detalhes bibliográficos
Autor(a) principal: Pinto, Susana
Data de Publicação: 2021
Outros Autores: Carvalho, Mamede
Tipo de documento: Artigo
Idioma: eng
Título da fonte: Repositório Científico de Acesso Aberto de Portugal (Repositórios Cientìficos)
Texto Completo: http://hdl.handle.net/10451/50203
Resumo: © 2021 The Author(s). Published by Informa UK Limited trading as Taylor & Francis Group on behalf of the Montreal Neurological Institute-Hospital 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 cited.
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spelling Phrenic nerve study as outcome in clinical trials for amyotrophic lateral sclerosisAmyotrophic lateral sclerosisClinical trialsPhrenic nerve studySample sizeSlow vital capacity© 2021 The Author(s). Published by Informa UK Limited trading as Taylor & Francis Group on behalf of the Montreal Neurological Institute-Hospital 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 cited.Introduction: Respiratory tests are fundamental for monitoring respiratory function in ALS, and essential in clinical trials. Slow vital capacity (SVC) was canceled in some countries to prevent COVID-19 transmission. We aimed to test phrenic nerve motor responses as an option to SVC in clinical trials. Methodology: Patients followed-up in our unit were selected respecting inclusion criteria used elsewhere: possible/probable/definite disease; onset-age 18-80years; disease duration from disease duration ≤24months; body mass index (BMI)>20kg/m2; respiratory subscore of the revised ALS functional rating scale (ALSFRS-R)≥11; upright SVC ≥ 70%. We added normal phrenic responses (meanPhrenAmpl, ≥0.4mV). All patients were on riluzole. SVC and meanPhrenAmpl were recorded at study entry (T0) and 24 weeks later (T1). Decays were determined. Sample size was calculated for a treatment effect of 30% on the decay rate. Results: We included 317 ALS patients (191 males, 225 spinal-onset), mean onset-age 59.9 ± 10.7 (31-80)years, mean onset BMI 25.48 ± 3.2 (20.1-35)kg/m2, mean disease duration 10.5 ± 5.6 (1-24)months, mean ALSFRS-R 41.54 ± 4.3 (22-47) and respiratory subscore 11.83 ± 0.38 (11-12). MeanPhrenAmpl and SVC were weakly but significantly correlated at T0 and T1. At T1, MeanPhrenAmpl decayed 16.94 ± 16.45% and SVC 13.5 ± 16.86%. For the proposed drug effect, 174 and 272 patients would be needed to recruit using respectively meanPhrenAmpl and SVC decline as the primary outcome measurement (accepting no dropouts). Discussion: Contrary to SVC, meanPhrenAmpl is non-volitional and not associated with aerosolization risk. Lower recruitment number (98 patients less) would be needed, translating shorter inclusion period, trial length and costs, and probable lower missed data rate. MeanPhrenAmp is an alternative test in ALS clinical trials.This work was funded by Comprehensive evaluation of circulating MicroRNA as diagnostic and prognostic biomarkers in Amyotrophic Lateral Sclerosis (PTDC/MEC-NEU/31195/2017).Taylor & FrancisRepositório da Universidade de LisboaPinto, SusanaCarvalho, Mamede2021-11-29T16:23:02Z20212021-01-01T00:00:00Zinfo:eu-repo/semantics/publishedVersioninfo:eu-repo/semantics/articleapplication/pdfhttp://hdl.handle.net/10451/50203engAmyotroph Lateral Scler Frontotemporal Degener. 2021;22(sup1):9-1310.1080/21678421.2021.18958422167-9223info:eu-repo/semantics/openAccessreponame:Repositório Científico de Acesso Aberto de Portugal (Repositórios Cientìficos)instname:Agência para a Sociedade do Conhecimento (UMIC) - FCT - Sociedade da Informaçãoinstacron:RCAAP2023-11-08T16:54:09Zoai:repositorio.ul.pt:10451/50203Portal AgregadorONGhttps://www.rcaap.pt/oai/openaireopendoar:71602024-03-19T22:01:36.730082Repositório Científico de Acesso Aberto de Portugal (Repositórios Cientìficos) - Agência para a Sociedade do Conhecimento (UMIC) - FCT - Sociedade da Informaçãofalse
dc.title.none.fl_str_mv Phrenic nerve study as outcome in clinical trials for amyotrophic lateral sclerosis
title Phrenic nerve study as outcome in clinical trials for amyotrophic lateral sclerosis
spellingShingle Phrenic nerve study as outcome in clinical trials for amyotrophic lateral sclerosis
Pinto, Susana
Amyotrophic lateral sclerosis
Clinical trials
Phrenic nerve study
Sample size
Slow vital capacity
title_short Phrenic nerve study as outcome in clinical trials for amyotrophic lateral sclerosis
title_full Phrenic nerve study as outcome in clinical trials for amyotrophic lateral sclerosis
title_fullStr Phrenic nerve study as outcome in clinical trials for amyotrophic lateral sclerosis
title_full_unstemmed Phrenic nerve study as outcome in clinical trials for amyotrophic lateral sclerosis
title_sort Phrenic nerve study as outcome in clinical trials for amyotrophic lateral sclerosis
author Pinto, Susana
author_facet Pinto, Susana
Carvalho, Mamede
author_role author
author2 Carvalho, Mamede
author2_role author
dc.contributor.none.fl_str_mv Repositório da Universidade de Lisboa
dc.contributor.author.fl_str_mv Pinto, Susana
Carvalho, Mamede
dc.subject.por.fl_str_mv Amyotrophic lateral sclerosis
Clinical trials
Phrenic nerve study
Sample size
Slow vital capacity
topic Amyotrophic lateral sclerosis
Clinical trials
Phrenic nerve study
Sample size
Slow vital capacity
description © 2021 The Author(s). Published by Informa UK Limited trading as Taylor & Francis Group on behalf of the Montreal Neurological Institute-Hospital 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 cited.
publishDate 2021
dc.date.none.fl_str_mv 2021-11-29T16:23:02Z
2021
2021-01-01T00:00:00Z
dc.type.status.fl_str_mv info:eu-repo/semantics/publishedVersion
dc.type.driver.fl_str_mv info:eu-repo/semantics/article
format article
status_str publishedVersion
dc.identifier.uri.fl_str_mv http://hdl.handle.net/10451/50203
url http://hdl.handle.net/10451/50203
dc.language.iso.fl_str_mv eng
language eng
dc.relation.none.fl_str_mv Amyotroph Lateral Scler Frontotemporal Degener. 2021;22(sup1):9-13
10.1080/21678421.2021.1895842
2167-9223
dc.rights.driver.fl_str_mv info:eu-repo/semantics/openAccess
eu_rights_str_mv openAccess
dc.format.none.fl_str_mv application/pdf
dc.publisher.none.fl_str_mv Taylor & Francis
publisher.none.fl_str_mv Taylor & Francis
dc.source.none.fl_str_mv reponame:Repositório Científico de Acesso Aberto de Portugal (Repositórios Cientìficos)
instname:Agência para a Sociedade do Conhecimento (UMIC) - FCT - Sociedade da Informação
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collection Repositório Científico de Acesso Aberto de Portugal (Repositórios Cientìficos)
repository.name.fl_str_mv Repositório Científico de Acesso Aberto de Portugal (Repositórios Cientìficos) - Agência para a Sociedade do Conhecimento (UMIC) - FCT - Sociedade da Informação
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