Meta-analyses of deflazacort versus prednisone/prednisolone in patients with nonsense mutation Duchenne muscular dystrophy

Compare efficacies of deflazacort and prednisone/prednisolone in providing clinically meaningful delays in loss of physical milestones in patients with nonsense mutation Duchenne muscular dystrophy. Placebo data from Phase IIb (ClinicalTrials.gov Identifier: NCT00592553) and ACT DMD (ClinicalTrials....

Full description

Saved in:
Bibliographic Details
Published in:Journal of comparative effectiveness research 2021-12, Vol.10 (18), p.1337-1347
Main Authors: Shieh, Perry B, Elfring, Gary, Trifillis, Panayiota, Santos, Claudio, Peltz, Stuart W, Parsons, Julie A, Apkon, Susan, Darras, Basil T, Campbell, Craig, McDonald, Craig M
Format: Article
Language:English
Subjects:
Codon, Nonsense
deflazacort
Duchenne muscular dystrophy
Humans
meta-analysis
Muscular Dystrophy, Duchenne - drug therapy
Muscular Dystrophy, Duchenne - genetics
nonsense mutation Duchenne muscular dystrophy
prednisolone
Prednisolone - therapeutic use
prednisone
Prednisone - therapeutic use
Pregnenediones
Retrospective Studies
Citations: Items that this one cites
Items that cite this one
Online Access:Get full text
Tags: Add Tag
No Tags, Be the first to tag this record!
cited_by cdi_FETCH-LOGICAL-c420t-deb3942478528fc7fa8a7ab6faee976aa55fefc1554e25f0214ad4fc80c6484b3
cites cdi_FETCH-LOGICAL-c420t-deb3942478528fc7fa8a7ab6faee976aa55fefc1554e25f0214ad4fc80c6484b3
container_end_page 1347
container_issue 18
container_start_page 1337
container_title Journal of comparative effectiveness research
container_volume 10
creator Shieh, Perry B
Elfring, Gary
Trifillis, Panayiota
Santos, Claudio
Peltz, Stuart W
Parsons, Julie A
Apkon, Susan
Darras, Basil T
Campbell, Craig
McDonald, Craig M
description Compare efficacies of deflazacort and prednisone/prednisolone in providing clinically meaningful delays in loss of physical milestones in patients with nonsense mutation Duchenne muscular dystrophy. Placebo data from Phase IIb (ClinicalTrials.gov Identifier: NCT00592553) and ACT DMD (ClinicalTrials.gov Identifier: NCT01826487) ataluren nonsense mutation Duchenne muscular dystrophy clinical trials were retrospectively combined in meta-analyses (intent-to-treat population; for change from baseline to week 48 in 6-min walk distance [6MWD] and timed function tests). Significant improvements in change in 6-min walk distance with deflazacort versus prednisone/prednisolone (least-squares mean difference 39.54 m [95% CI: 13.799, 65.286; p = 0.0026]). Significant and clinically meaningful improvements in 4-stair climb and 4-stair descend for deflazacort versus prednisone/prednisolone. Deflazacort provides clinically meaningful delays in loss of physical milestones over 48 weeks compared with prednisone/prednisolone for patients with nonsense mutation Duchenne muscular dystrophy.
doi_str_mv 10.2217/cer-2021-0018
format article
fullrecord <record><control><sourceid>proquest_cross</sourceid><recordid>TN_cdi_proquest_miscellaneous_2585930056</recordid><sourceformat>XML</sourceformat><sourcesystem>PC</sourcesystem><sourcerecordid>2585930056</sourcerecordid><originalsourceid>FETCH-LOGICAL-c420t-deb3942478528fc7fa8a7ab6faee976aa55fefc1554e25f0214ad4fc80c6484b3</originalsourceid><addsrcrecordid>eNp1kD1PHDEQhq0oKIfgStrIZZoFr9fejzIiHyCBaKC25rxj3UZ79sZjJzp-PT4d0GGN5Bn7mbd4GLuoxaWUdXdlMVZSyLoSou4_sVMplKzapm4-v_dCr9ia6I8op-3VoOUXtmpUOzSd1Kfs-R4TVOBh3hMSD46P6GZ4Bhti4v8wUia-RBz9RMHj1Vs7l4FPni-QJvSJ-P8pbbkPnrAU3-VUPoLnP7LdoveHF7J5hsjHPaUYlu3-nJ04mAnXr_cZe_r18_H6prp7-H17_f2uskqKVI24aQYlVddr2TvbOeihg03rAHHoWgCtHTpba61QaldsKBiVs72wrerVpjlj3465Swx_M1Iyu4kszjN4DJmM1L0eGiF0W9DqiNoYiCI6s8RpB3FvamEOxk0xbg7GzcF44b--RufNDsd3-s1vAYYj4HLKEckWWRbNcSobk508fhD-AlH6k60</addsrcrecordid><sourcetype>Aggregation Database</sourcetype><iscdi>true</iscdi><recordtype>article</recordtype><pqid>2585930056</pqid></control><display><type>article</type><title>Meta-analyses of deflazacort versus prednisone/prednisolone in patients with nonsense mutation Duchenne muscular dystrophy</title><source>PubMed Central</source><creator>Shieh, Perry B ; Elfring, Gary ; Trifillis, Panayiota ; Santos, Claudio ; Peltz, Stuart W ; Parsons, Julie A ; Apkon, Susan ; Darras, Basil T ; Campbell, Craig ; McDonald, Craig M</creator><creatorcontrib><![CDATA[Shieh, Perry B ; Elfring, Gary ; Trifillis, Panayiota ; Santos, Claudio ; Peltz, Stuart W ; Parsons, Julie A ; Apkon, Susan ; Darras, Basil T ; Campbell, Craig ; McDonald, Craig M ; Members of the Ataluren Phase IIb Study Group<xref ref-type="author-notes" rid="FN1" ptype="Fcer20210018" citart="citart1">†:</xref>, Members of the Ataluren Phase IIb Study Clinical Evaluator Training Group<xref ref-type="author-notes" rid="FN1" ptype="Fcer20210018" citart="citart1">‡:</xref>, Members of the ACT DMD Study Group<xref ref-type="author-notes" rid="FN1" ptype="Fcer20210018" citart="citart1">¶:</xref>, Members of the ACT DMD Clinical Evaluator Training Group<xref ref-type="author-notes" rid="FN1" ptype="Fcer20210018" citart="citart1">§:</xref>]]></creatorcontrib><description>Compare efficacies of deflazacort and prednisone/prednisolone in providing clinically meaningful delays in loss of physical milestones in patients with nonsense mutation Duchenne muscular dystrophy. Placebo data from Phase IIb (ClinicalTrials.gov Identifier: NCT00592553) and ACT DMD (ClinicalTrials.gov Identifier: NCT01826487) ataluren nonsense mutation Duchenne muscular dystrophy clinical trials were retrospectively combined in meta-analyses (intent-to-treat population; for change from baseline to week 48 in 6-min walk distance [6MWD] and timed function tests). Significant improvements in change in 6-min walk distance with deflazacort versus prednisone/prednisolone (least-squares mean difference 39.54 m [95% CI: 13.799, 65.286; p = 0.0026]). Significant and clinically meaningful improvements in 4-stair climb and 4-stair descend for deflazacort versus prednisone/prednisolone. Deflazacort provides clinically meaningful delays in loss of physical milestones over 48 weeks compared with prednisone/prednisolone for patients with nonsense mutation Duchenne muscular dystrophy.</description><identifier>ISSN: 2042-6305</identifier><identifier>EISSN: 2042-6313</identifier><identifier>DOI: 10.2217/cer-2021-0018</identifier><identifier>PMID: 34693725</identifier><language>eng</language><publisher>England: Future Medicine Ltd</publisher><subject>Codon, Nonsense ; deflazacort ; Duchenne muscular dystrophy ; Humans ; meta-analysis ; Muscular Dystrophy, Duchenne - drug therapy ; Muscular Dystrophy, Duchenne - genetics ; nonsense mutation Duchenne muscular dystrophy ; prednisolone ; Prednisolone - therapeutic use ; prednisone ; Prednisone - therapeutic use ; Pregnenediones ; Retrospective Studies</subject><ispartof>Journal of comparative effectiveness research, 2021-12, Vol.10 (18), p.1337-1347</ispartof><rights>2021 Shieh et al.</rights><lds50>peer_reviewed</lds50><oa>free_for_read</oa><woscitedreferencessubscribed>false</woscitedreferencessubscribed><citedby>FETCH-LOGICAL-c420t-deb3942478528fc7fa8a7ab6faee976aa55fefc1554e25f0214ad4fc80c6484b3</citedby><cites>FETCH-LOGICAL-c420t-deb3942478528fc7fa8a7ab6faee976aa55fefc1554e25f0214ad4fc80c6484b3</cites><orcidid>0000-0002-8779-3220 ; 0000-0001-7145-7663</orcidid></display><links><openurl>$$Topenurl_article</openurl><openurlfulltext>$$Topenurlfull_article</openurlfulltext><thumbnail>$$Tsyndetics_thumb_exl</thumbnail><link.rule.ids>314,776,780,27901,27902</link.rule.ids><backlink>$$Uhttps://www.ncbi.nlm.nih.gov/pubmed/34693725$$D View this record in MEDLINE/PubMed$$Hfree_for_read</backlink></links><search><creatorcontrib>Shieh, Perry B</creatorcontrib><creatorcontrib>Elfring, Gary</creatorcontrib><creatorcontrib>Trifillis, Panayiota</creatorcontrib><creatorcontrib>Santos, Claudio</creatorcontrib><creatorcontrib>Peltz, Stuart W</creatorcontrib><creatorcontrib>Parsons, Julie A</creatorcontrib><creatorcontrib>Apkon, Susan</creatorcontrib><creatorcontrib>Darras, Basil T</creatorcontrib><creatorcontrib>Campbell, Craig</creatorcontrib><creatorcontrib>McDonald, Craig M</creatorcontrib><creatorcontrib><![CDATA[Members of the Ataluren Phase IIb Study Group<xref ref-type="author-notes" rid="FN1" ptype="Fcer20210018" citart="citart1">†:</xref>, Members of the Ataluren Phase IIb Study Clinical Evaluator Training Group<xref ref-type="author-notes" rid="FN1" ptype="Fcer20210018" citart="citart1">‡:</xref>, Members of the ACT DMD Study Group<xref ref-type="author-notes" rid="FN1" ptype="Fcer20210018" citart="citart1">¶:</xref>, Members of the ACT DMD Clinical Evaluator Training Group<xref ref-type="author-notes" rid="FN1" ptype="Fcer20210018" citart="citart1">§:</xref>]]></creatorcontrib><title>Meta-analyses of deflazacort versus prednisone/prednisolone in patients with nonsense mutation Duchenne muscular dystrophy</title><title>Journal of comparative effectiveness research</title><addtitle>J Comp Eff Res</addtitle><description>Compare efficacies of deflazacort and prednisone/prednisolone in providing clinically meaningful delays in loss of physical milestones in patients with nonsense mutation Duchenne muscular dystrophy. Placebo data from Phase IIb (ClinicalTrials.gov Identifier: NCT00592553) and ACT DMD (ClinicalTrials.gov Identifier: NCT01826487) ataluren nonsense mutation Duchenne muscular dystrophy clinical trials were retrospectively combined in meta-analyses (intent-to-treat population; for change from baseline to week 48 in 6-min walk distance [6MWD] and timed function tests). Significant improvements in change in 6-min walk distance with deflazacort versus prednisone/prednisolone (least-squares mean difference 39.54 m [95% CI: 13.799, 65.286; p = 0.0026]). Significant and clinically meaningful improvements in 4-stair climb and 4-stair descend for deflazacort versus prednisone/prednisolone. Deflazacort provides clinically meaningful delays in loss of physical milestones over 48 weeks compared with prednisone/prednisolone for patients with nonsense mutation Duchenne muscular dystrophy.</description><subject>Codon, Nonsense</subject><subject>deflazacort</subject><subject>Duchenne muscular dystrophy</subject><subject>Humans</subject><subject>meta-analysis</subject><subject>Muscular Dystrophy, Duchenne - drug therapy</subject><subject>Muscular Dystrophy, Duchenne - genetics</subject><subject>nonsense mutation Duchenne muscular dystrophy</subject><subject>prednisolone</subject><subject>Prednisolone - therapeutic use</subject><subject>prednisone</subject><subject>Prednisone - therapeutic use</subject><subject>Pregnenediones</subject><subject>Retrospective Studies</subject><issn>2042-6305</issn><issn>2042-6313</issn><fulltext>true</fulltext><rsrctype>article</rsrctype><creationdate>2021</creationdate><recordtype>article</recordtype><recordid>eNp1kD1PHDEQhq0oKIfgStrIZZoFr9fejzIiHyCBaKC25rxj3UZ79sZjJzp-PT4d0GGN5Bn7mbd4GLuoxaWUdXdlMVZSyLoSou4_sVMplKzapm4-v_dCr9ia6I8op-3VoOUXtmpUOzSd1Kfs-R4TVOBh3hMSD46P6GZ4Bhti4v8wUia-RBz9RMHj1Vs7l4FPni-QJvSJ-P8pbbkPnrAU3-VUPoLnP7LdoveHF7J5hsjHPaUYlu3-nJ04mAnXr_cZe_r18_H6prp7-H17_f2uskqKVI24aQYlVddr2TvbOeihg03rAHHoWgCtHTpba61QaldsKBiVs72wrerVpjlj3465Swx_M1Iyu4kszjN4DJmM1L0eGiF0W9DqiNoYiCI6s8RpB3FvamEOxk0xbg7GzcF44b--RufNDsd3-s1vAYYj4HLKEckWWRbNcSobk508fhD-AlH6k60</recordid><startdate>20211201</startdate><enddate>20211201</enddate><creator>Shieh, Perry B</creator><creator>Elfring, Gary</creator><creator>Trifillis, Panayiota</creator><creator>Santos, Claudio</creator><creator>Peltz, Stuart W</creator><creator>Parsons, Julie A</creator><creator>Apkon, Susan</creator><creator>Darras, Basil T</creator><creator>Campbell, Craig</creator><creator>McDonald, Craig M</creator><general>Future Medicine Ltd</general><scope>FUMOA</scope><scope>CGR</scope><scope>CUY</scope><scope>CVF</scope><scope>ECM</scope><scope>EIF</scope><scope>NPM</scope><scope>AAYXX</scope><scope>CITATION</scope><scope>7X8</scope><orcidid>https://orcid.org/0000-0002-8779-3220</orcidid><orcidid>https://orcid.org/0000-0001-7145-7663</orcidid></search><sort><creationdate>20211201</creationdate><title>Meta-analyses of deflazacort versus prednisone/prednisolone in patients with nonsense mutation Duchenne muscular dystrophy</title><author>Shieh, Perry B ; Elfring, Gary ; Trifillis, Panayiota ; Santos, Claudio ; Peltz, Stuart W ; Parsons, Julie A ; Apkon, Susan ; Darras, Basil T ; Campbell, Craig ; McDonald, Craig M</author></sort><facets><frbrtype>5</frbrtype><frbrgroupid>cdi_FETCH-LOGICAL-c420t-deb3942478528fc7fa8a7ab6faee976aa55fefc1554e25f0214ad4fc80c6484b3</frbrgroupid><rsrctype>articles</rsrctype><prefilter>articles</prefilter><language>eng</language><creationdate>2021</creationdate><topic>Codon, Nonsense</topic><topic>deflazacort</topic><topic>Duchenne muscular dystrophy</topic><topic>Humans</topic><topic>meta-analysis</topic><topic>Muscular Dystrophy, Duchenne - drug therapy</topic><topic>Muscular Dystrophy, Duchenne - genetics</topic><topic>nonsense mutation Duchenne muscular dystrophy</topic><topic>prednisolone</topic><topic>Prednisolone - therapeutic use</topic><topic>prednisone</topic><topic>Prednisone - therapeutic use</topic><topic>Pregnenediones</topic><topic>Retrospective Studies</topic><toplevel>peer_reviewed</toplevel><toplevel>online_resources</toplevel><creatorcontrib>Shieh, Perry B</creatorcontrib><creatorcontrib>Elfring, Gary</creatorcontrib><creatorcontrib>Trifillis, Panayiota</creatorcontrib><creatorcontrib>Santos, Claudio</creatorcontrib><creatorcontrib>Peltz, Stuart W</creatorcontrib><creatorcontrib>Parsons, Julie A</creatorcontrib><creatorcontrib>Apkon, Susan</creatorcontrib><creatorcontrib>Darras, Basil T</creatorcontrib><creatorcontrib>Campbell, Craig</creatorcontrib><creatorcontrib>McDonald, Craig M</creatorcontrib><creatorcontrib><![CDATA[Members of the Ataluren Phase IIb Study Group<xref ref-type="author-notes" rid="FN1" ptype="Fcer20210018" citart="citart1">†:</xref>, Members of the Ataluren Phase IIb Study Clinical Evaluator Training Group<xref ref-type="author-notes" rid="FN1" ptype="Fcer20210018" citart="citart1">‡:</xref>, Members of the ACT DMD Study Group<xref ref-type="author-notes" rid="FN1" ptype="Fcer20210018" citart="citart1">¶:</xref>, Members of the ACT DMD Clinical Evaluator Training Group<xref ref-type="author-notes" rid="FN1" ptype="Fcer20210018" citart="citart1">§:</xref>]]></creatorcontrib><collection>Future Medicine (Open Access)</collection><collection>Medline</collection><collection>MEDLINE</collection><collection>MEDLINE (Ovid)</collection><collection>MEDLINE</collection><collection>MEDLINE</collection><collection>PubMed</collection><collection>CrossRef</collection><collection>MEDLINE - Academic</collection><jtitle>Journal of comparative effectiveness research</jtitle></facets><delivery><delcategory>Remote Search Resource</delcategory><fulltext>fulltext</fulltext></delivery><addata><au>Shieh, Perry B</au><au>Elfring, Gary</au><au>Trifillis, Panayiota</au><au>Santos, Claudio</au><au>Peltz, Stuart W</au><au>Parsons, Julie A</au><au>Apkon, Susan</au><au>Darras, Basil T</au><au>Campbell, Craig</au><au>McDonald, Craig M</au><aucorp><![CDATA[Members of the Ataluren Phase IIb Study Group<xref ref-type="author-notes" rid="FN1" ptype="Fcer20210018" citart="citart1">†:</xref>, Members of the Ataluren Phase IIb Study Clinical Evaluator Training Group<xref ref-type="author-notes" rid="FN1" ptype="Fcer20210018" citart="citart1">‡:</xref>, Members of the ACT DMD Study Group<xref ref-type="author-notes" rid="FN1" ptype="Fcer20210018" citart="citart1">¶:</xref>, Members of the ACT DMD Clinical Evaluator Training Group<xref ref-type="author-notes" rid="FN1" ptype="Fcer20210018" citart="citart1">§:</xref>]]></aucorp><format>journal</format><genre>article</genre><ristype>JOUR</ristype><atitle>Meta-analyses of deflazacort versus prednisone/prednisolone in patients with nonsense mutation Duchenne muscular dystrophy</atitle><jtitle>Journal of comparative effectiveness research</jtitle><addtitle>J Comp Eff Res</addtitle><date>2021-12-01</date><risdate>2021</risdate><volume>10</volume><issue>18</issue><spage>1337</spage><epage>1347</epage><pages>1337-1347</pages><issn>2042-6305</issn><eissn>2042-6313</eissn><abstract>Compare efficacies of deflazacort and prednisone/prednisolone in providing clinically meaningful delays in loss of physical milestones in patients with nonsense mutation Duchenne muscular dystrophy. Placebo data from Phase IIb (ClinicalTrials.gov Identifier: NCT00592553) and ACT DMD (ClinicalTrials.gov Identifier: NCT01826487) ataluren nonsense mutation Duchenne muscular dystrophy clinical trials were retrospectively combined in meta-analyses (intent-to-treat population; for change from baseline to week 48 in 6-min walk distance [6MWD] and timed function tests). Significant improvements in change in 6-min walk distance with deflazacort versus prednisone/prednisolone (least-squares mean difference 39.54 m [95% CI: 13.799, 65.286; p = 0.0026]). Significant and clinically meaningful improvements in 4-stair climb and 4-stair descend for deflazacort versus prednisone/prednisolone. Deflazacort provides clinically meaningful delays in loss of physical milestones over 48 weeks compared with prednisone/prednisolone for patients with nonsense mutation Duchenne muscular dystrophy.</abstract><cop>England</cop><pub>Future Medicine Ltd</pub><pmid>34693725</pmid><doi>10.2217/cer-2021-0018</doi><tpages>11</tpages><orcidid>https://orcid.org/0000-0002-8779-3220</orcidid><orcidid>https://orcid.org/0000-0001-7145-7663</orcidid><oa>free_for_read</oa></addata></record>
fulltext fulltext
identifier ISSN: 2042-6305
ispartof Journal of comparative effectiveness research, 2021-12, Vol.10 (18), p.1337-1347
issn 2042-6305
2042-6313
language eng
recordid cdi_proquest_miscellaneous_2585930056
source PubMed Central
subjects Codon, Nonsense
deflazacort
Duchenne muscular dystrophy
Humans
meta-analysis
Muscular Dystrophy, Duchenne - drug therapy
Muscular Dystrophy, Duchenne - genetics
nonsense mutation Duchenne muscular dystrophy
prednisolone
Prednisolone - therapeutic use
prednisone
Prednisone - therapeutic use
Pregnenediones
Retrospective Studies
title Meta-analyses of deflazacort versus prednisone/prednisolone in patients with nonsense mutation Duchenne muscular dystrophy
url http://sfxeu10.hosted.exlibrisgroup.com/loughborough?ctx_ver=Z39.88-2004&ctx_enc=info:ofi/enc:UTF-8&ctx_tim=2025-01-30T22%3A49%3A17IST&url_ver=Z39.88-2004&url_ctx_fmt=infofi/fmt:kev:mtx:ctx&rfr_id=info:sid/primo.exlibrisgroup.com:primo3-Article-proquest_cross&rft_val_fmt=info:ofi/fmt:kev:mtx:journal&rft.genre=article&rft.atitle=Meta-analyses%20of%20deflazacort%20versus%20prednisone/prednisolone%20in%20patients%20with%20nonsense%20mutation%20Duchenne%20muscular%20dystrophy&rft.jtitle=Journal%20of%20comparative%20effectiveness%20research&rft.au=Shieh,%20Perry%20B&rft.aucorp=Members%20of%20the%20Ataluren%20Phase%20IIb%20Study%20Group%3Cxref%20ref-type=%22author-notes%22%20rid=%22FN1%22%20ptype=%22Fcer20210018%22%20citart=%22citart1%22%3E%E2%80%A0:%3C/xref%3E,%20Members%20of%20the%20Ataluren%20Phase%20IIb%20Study%20Clinical%20Evaluator%20Training%20Group%3Cxref%20ref-type=%22author-notes%22%20rid=%22FN1%22%20ptype=%22Fcer20210018%22%20citart=%22citart1%22%3E%E2%80%A1:%3C/xref%3E,%20Members%20of%20the%20ACT%20DMD%20Study%20Group%3Cxref%20ref-type=%22author-notes%22%20rid=%22FN1%22%20ptype=%22Fcer20210018%22%20citart=%22citart1%22%3E%C2%B6:%3C/xref%3E,%20Members%20of%20the%20ACT%20DMD%20Clinical%20Evaluator%20Training%20Group%3Cxref%20ref-type=%22author-notes%22%20rid=%22FN1%22%20ptype=%22Fcer20210018%22%20citart=%22citart1%22%3E%C2%A7:%3C/xref%3E&rft.date=2021-12-01&rft.volume=10&rft.issue=18&rft.spage=1337&rft.epage=1347&rft.pages=1337-1347&rft.issn=2042-6305&rft.eissn=2042-6313&rft_id=info:doi/10.2217/cer-2021-0018&rft_dat=%3Cproquest_cross%3E2585930056%3C/proquest_cross%3E%3Cgrp_id%3Ecdi_FETCH-LOGICAL-c420t-deb3942478528fc7fa8a7ab6faee976aa55fefc1554e25f0214ad4fc80c6484b3%3C/grp_id%3E%3Coa%3E%3C/oa%3E%3Curl%3E%3C/url%3E&rft_id=info:oai/&rft_pqid=2585930056&rft_id=info:pmid/34693725&rfr_iscdi=true