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

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dc.contributor.authorPinto, Susana
dc.contributor.authorCarvalho, Mamede
dc.date.accessioned2021-11-29T16:23:02Z
dc.date.available2021-11-29T16:23:02Z
dc.date.issued2021
dc.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.pt_PT
dc.description.abstractIntroduction: 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.pt_PT
dc.description.sponsorshipThis work was funded by Comprehensive evaluation of circulating MicroRNA as diagnostic and prognostic biomarkers in Amyotrophic Lateral Sclerosis (PTDC/MEC-NEU/31195/2017).pt_PT
dc.description.versioninfo:eu-repo/semantics/publishedVersionpt_PT
dc.identifier.citationAmyotroph Lateral Scler Frontotemporal Degener. 2021;22(sup1):9-13pt_PT
dc.identifier.doi10.1080/21678421.2021.1895842pt_PT
dc.identifier.eissn2167-9223
dc.identifier.urihttp://hdl.handle.net/10451/50203
dc.language.isoengpt_PT
dc.peerreviewedyespt_PT
dc.publisherTaylor & Francispt_PT
dc.relationComprehensive Evaluation of Circulating MicroRNA as Diagnostic and Prognostic Biomarkers in Amyotrophic Lateral Sclerosis
dc.relation.publisherversionhttps://www.tandfonline.com/toc/iafd20/currentpt_PT
dc.rights.urihttp://creativecommons.org/licenses/by/4.0/pt_PT
dc.subjectAmyotrophic lateral sclerosispt_PT
dc.subjectClinical trialspt_PT
dc.subjectPhrenic nerve studypt_PT
dc.subjectSample sizept_PT
dc.subjectSlow vital capacitypt_PT
dc.titlePhrenic nerve study as outcome in clinical trials for amyotrophic lateral sclerosispt_PT
dc.typejournal article
dspace.entity.typePublication
oaire.awardNumberPTDC/MEC-NEU/31195/2017
oaire.awardTitleComprehensive Evaluation of Circulating MicroRNA as Diagnostic and Prognostic Biomarkers in Amyotrophic Lateral Sclerosis
oaire.awardURIinfo:eu-repo/grantAgreement/FCT/3599-PPCDT/PTDC%2FMEC-NEU%2F31195%2F2017/PT
oaire.citation.endPage13pt_PT
oaire.citation.issuesup1pt_PT
oaire.citation.startPage9pt_PT
oaire.citation.titleAmyotrophic Lateral Sclerosis and Frontotemporal Degenerationpt_PT
oaire.citation.volume22pt_PT
oaire.fundingStream3599-PPCDT
person.familyNamePinto
person.familyNamede Carvalho
person.givenNameSusana
person.givenNameMamede
person.identifier593760
person.identifier.ciencia-idD31B-E89C-CB94
person.identifier.orcid0000-0002-0727-5897
person.identifier.orcid0000-0001-7556-0158
person.identifier.ridL-8394-2019
person.identifier.scopus-author-id23397978500
person.identifier.scopus-author-id7101893769
project.funder.identifierhttp://doi.org/10.13039/501100001871
project.funder.nameFundação para a Ciência e a Tecnologia
rcaap.rightsopenAccesspt_PT
rcaap.typearticlept_PT
relation.isAuthorOfPublication3fa09e71-c9b7-4536-a359-f7e3576dca09
relation.isAuthorOfPublicationdd7f55d4-c2b5-4fd2-9bd1-a9542a62f58f
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