Clinical trial readiness in non-ambulatory boys and men with duchenne muscular dystrophy: MDA-DMD network follow-up
ABSTRACT Introduction: Outcomes sensitive to change over time in non‐ambulatory boys/men with Duchenne muscular dystrophy (DMD) are not well‐established. Methods: Subjects (n = 91; 16.8 ± 4.5 years old) were assessed at baseline and 6‐month intervals for 2 years. We analyzed all subjects using an in...
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| Veröffentlicht in: | Muscle & nerve 2016-10, Vol.54 (4), p.681-689 |
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| creator | Connolly, Anne M. Florence, Julaine M. Zaidman, Craig M. Golumbek, Paul T. Mendell, Jerry R. Flanigan, Kevin M. Karachunski, Peter I. Day, John W. McDonald, Craig M. Darras, Basil T. Kang, Peter B. Siener, Catherine A. Gadeken, Rebecca K. Anand, Pallavi Schierbecker, Jeanine R. Malkus, Elizabeth C. Lowes, Linda P. Alfano, Lindsay N. Johnson, Linda Nicorici, Alina Kelecic, Jason M. Quigley, Janet Pasternak, Amy E. Miller, J. Philip |
| description | ABSTRACT
Introduction: Outcomes sensitive to change over time in non‐ambulatory boys/men with Duchenne muscular dystrophy (DMD) are not well‐established. Methods: Subjects (n = 91; 16.8 ± 4.5 years old) were assessed at baseline and 6‐month intervals for 2 years. We analyzed all subjects using an intent‐to‐treat model and a subset of stronger subjects with Brooke Scale score ≤4, using repeated measures. Results: Eight patients (12–33 years old) died during the study. Sixty‐six completed 12‐month follow‐up, and 51 completed 24‐month follow‐up. Those taking corticosteroids performed better at baseline, but rates of decline were similar. Forced vital capacity percent predicted (FVC% predicted) declined significantly only after 2 years. However, Brooke and Egen Klassifikation (EK) Scale scores, elbow flexion, and grip strength declined significantly over both 1 and 2 years. Conclusion: Brooke and EK Scale scores, elbow flexion, and grip strength were outcomes most responsive to change. FVC% predicted was responsive to change over 2 years. Corticosteroids benefited non‐ambulatory DMD subjects but did not affect decline rates of measures tested here. Muscle Nerve 54: 681–689, 2016 |
| doi_str_mv | 10.1002/mus.25089 |
| format | Article |
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Introduction: Outcomes sensitive to change over time in non‐ambulatory boys/men with Duchenne muscular dystrophy (DMD) are not well‐established. Methods: Subjects (n = 91; 16.8 ± 4.5 years old) were assessed at baseline and 6‐month intervals for 2 years. We analyzed all subjects using an intent‐to‐treat model and a subset of stronger subjects with Brooke Scale score ≤4, using repeated measures. Results: Eight patients (12–33 years old) died during the study. Sixty‐six completed 12‐month follow‐up, and 51 completed 24‐month follow‐up. Those taking corticosteroids performed better at baseline, but rates of decline were similar. Forced vital capacity percent predicted (FVC% predicted) declined significantly only after 2 years. However, Brooke and Egen Klassifikation (EK) Scale scores, elbow flexion, and grip strength declined significantly over both 1 and 2 years. Conclusion: Brooke and EK Scale scores, elbow flexion, and grip strength were outcomes most responsive to change. FVC% predicted was responsive to change over 2 years. Corticosteroids benefited non‐ambulatory DMD subjects but did not affect decline rates of measures tested here. Muscle Nerve 54: 681–689, 2016</description><identifier>ISSN: 0148-639X</identifier><identifier>EISSN: 1097-4598</identifier><identifier>DOI: 10.1002/mus.25089</identifier><identifier>PMID: 26930423</identifier><identifier>CODEN: MUNEDE</identifier><language>eng</language><publisher>United States: Blackwell Publishing Ltd</publisher><subject>Adolescent ; Adrenal Cortex Hormones - therapeutic use ; Adult ; Brooke Scale ; Child ; clinical trial ; Duchenne muscular dystrophy ; Egen Klassifikation Scale ; Elbow ; Follow-Up Studies ; force vital capacity ; Hand Strength - physiology ; Humans ; Male ; Mobility Limitation ; Muscular dystrophy ; Muscular Dystrophy, Duchenne - diagnosis ; Muscular Dystrophy, Duchenne - drug therapy ; Muscular Dystrophy, Duchenne - physiopathology ; non-ambulatory ; outcomes ; Patient Participation - methods ; Range of Motion, Articular - physiology ; Vital Capacity - physiology ; Young Adult</subject><ispartof>Muscle & nerve, 2016-10, Vol.54 (4), p.681-689</ispartof><rights>2016 Wiley Periodicals, Inc.</rights><lds50>peer_reviewed</lds50><woscitedreferencessubscribed>false</woscitedreferencessubscribed><citedby>FETCH-LOGICAL-c4609-82269d5887172654755fedf83a73f739c661767221bdf7ea09b49aacb7e5c453</citedby></display><links><openurl>$$Topenurl_article</openurl><openurlfulltext>$$Topenurlfull_article</openurlfulltext><thumbnail>$$Tsyndetics_thumb_exl</thumbnail><linktopdf>$$Uhttps://onlinelibrary.wiley.com/doi/pdf/10.1002%2Fmus.25089$$EPDF$$P50$$Gwiley$$H</linktopdf><linktohtml>$$Uhttps://onlinelibrary.wiley.com/doi/full/10.1002%2Fmus.25089$$EHTML$$P50$$Gwiley$$H</linktohtml><link.rule.ids>314,776,780,1411,27901,27902,45550,45551</link.rule.ids><backlink>$$Uhttps://www.ncbi.nlm.nih.gov/pubmed/26930423$$D View this record in MEDLINE/PubMed$$Hfree_for_read</backlink></links><search><creatorcontrib>Connolly, Anne M.</creatorcontrib><creatorcontrib>Florence, Julaine M.</creatorcontrib><creatorcontrib>Zaidman, Craig M.</creatorcontrib><creatorcontrib>Golumbek, Paul T.</creatorcontrib><creatorcontrib>Mendell, Jerry R.</creatorcontrib><creatorcontrib>Flanigan, Kevin M.</creatorcontrib><creatorcontrib>Karachunski, Peter I.</creatorcontrib><creatorcontrib>Day, John W.</creatorcontrib><creatorcontrib>McDonald, Craig M.</creatorcontrib><creatorcontrib>Darras, Basil T.</creatorcontrib><creatorcontrib>Kang, Peter B.</creatorcontrib><creatorcontrib>Siener, Catherine A.</creatorcontrib><creatorcontrib>Gadeken, Rebecca K.</creatorcontrib><creatorcontrib>Anand, Pallavi</creatorcontrib><creatorcontrib>Schierbecker, Jeanine R.</creatorcontrib><creatorcontrib>Malkus, Elizabeth C.</creatorcontrib><creatorcontrib>Lowes, Linda P.</creatorcontrib><creatorcontrib>Alfano, Lindsay N.</creatorcontrib><creatorcontrib>Johnson, Linda</creatorcontrib><creatorcontrib>Nicorici, Alina</creatorcontrib><creatorcontrib>Kelecic, Jason M.</creatorcontrib><creatorcontrib>Quigley, Janet</creatorcontrib><creatorcontrib>Pasternak, Amy E.</creatorcontrib><creatorcontrib>Miller, J. Philip</creatorcontrib><creatorcontrib>MDA-DMD Clinical Research Network</creatorcontrib><title>Clinical trial readiness in non-ambulatory boys and men with duchenne muscular dystrophy: MDA-DMD network follow-up</title><title>Muscle & nerve</title><addtitle>Muscle Nerve</addtitle><description>ABSTRACT
Introduction: Outcomes sensitive to change over time in non‐ambulatory boys/men with Duchenne muscular dystrophy (DMD) are not well‐established. Methods: Subjects (n = 91; 16.8 ± 4.5 years old) were assessed at baseline and 6‐month intervals for 2 years. We analyzed all subjects using an intent‐to‐treat model and a subset of stronger subjects with Brooke Scale score ≤4, using repeated measures. Results: Eight patients (12–33 years old) died during the study. Sixty‐six completed 12‐month follow‐up, and 51 completed 24‐month follow‐up. Those taking corticosteroids performed better at baseline, but rates of decline were similar. Forced vital capacity percent predicted (FVC% predicted) declined significantly only after 2 years. However, Brooke and Egen Klassifikation (EK) Scale scores, elbow flexion, and grip strength declined significantly over both 1 and 2 years. Conclusion: Brooke and EK Scale scores, elbow flexion, and grip strength were outcomes most responsive to change. FVC% predicted was responsive to change over 2 years. Corticosteroids benefited non‐ambulatory DMD subjects but did not affect decline rates of measures tested here. Muscle Nerve 54: 681–689, 2016</description><subject>Adolescent</subject><subject>Adrenal Cortex Hormones - therapeutic use</subject><subject>Adult</subject><subject>Brooke Scale</subject><subject>Child</subject><subject>clinical trial</subject><subject>Duchenne muscular dystrophy</subject><subject>Egen Klassifikation Scale</subject><subject>Elbow</subject><subject>Follow-Up Studies</subject><subject>force vital capacity</subject><subject>Hand Strength - physiology</subject><subject>Humans</subject><subject>Male</subject><subject>Mobility Limitation</subject><subject>Muscular dystrophy</subject><subject>Muscular Dystrophy, Duchenne - diagnosis</subject><subject>Muscular Dystrophy, Duchenne - drug therapy</subject><subject>Muscular Dystrophy, Duchenne - physiopathology</subject><subject>non-ambulatory</subject><subject>outcomes</subject><subject>Patient Participation - methods</subject><subject>Range of Motion, Articular - physiology</subject><subject>Vital Capacity - physiology</subject><subject>Young Adult</subject><issn>0148-639X</issn><issn>1097-4598</issn><fulltext>true</fulltext><rsrctype>article</rsrctype><creationdate>2016</creationdate><recordtype>article</recordtype><sourceid>EIF</sourceid><recordid>eNqNkT1v2zAQhomiReOkGfoHCgJduijhl_jRLbATt0CcDkmdbgQlUTATiXRJCY7-fek4zdCpy90B97yH9-4A-IjRGUaInPdjOiMlkuoNmGGkRMFKJd-CGcJMFpyqX0fgOKUHhBCWXLwHR4QrihihM5DmnfOuNh0cossxWtM4b1OCzkMffGH6auzMEOIEqzAlaHwDe-vhzg0b2Iz1xnpvYXZQZyzCZkpDDNvN9BWuFhfFYrWA3g67EB9hG7ou7Ipx-wG8a02X7OlLPgF3V5d382_F9Y_l9_nFdVEzjlQhSbbZlFIKLAgvmSjL1jatpEbQVlBVc44FF4TgqmmFNUhVTBlTV8KWNSvpCfhyGLuN4fdo06B7l2rbdcbbMCaNJREKUSLYf6BYUoaV2E_9_A_6EMbo8x57Sojsk_NMfXqhxqq3jd5G15s46b-Hz8D5Adi5zk6vfYz0_qM631M_f1Svft4-F1lRHBQuDfbpVWHio-aCilLf3yz1cs1u1ovblV7TP8E8oXc</recordid><startdate>201610</startdate><enddate>201610</enddate><creator>Connolly, Anne M.</creator><creator>Florence, Julaine M.</creator><creator>Zaidman, Craig M.</creator><creator>Golumbek, Paul T.</creator><creator>Mendell, Jerry R.</creator><creator>Flanigan, Kevin M.</creator><creator>Karachunski, Peter I.</creator><creator>Day, John W.</creator><creator>McDonald, Craig M.</creator><creator>Darras, Basil T.</creator><creator>Kang, Peter B.</creator><creator>Siener, Catherine A.</creator><creator>Gadeken, Rebecca K.</creator><creator>Anand, Pallavi</creator><creator>Schierbecker, Jeanine R.</creator><creator>Malkus, Elizabeth C.</creator><creator>Lowes, Linda P.</creator><creator>Alfano, Lindsay N.</creator><creator>Johnson, Linda</creator><creator>Nicorici, Alina</creator><creator>Kelecic, Jason M.</creator><creator>Quigley, Janet</creator><creator>Pasternak, Amy E.</creator><creator>Miller, J. Philip</creator><general>Blackwell Publishing Ltd</general><general>Wiley Subscription Services, Inc</general><scope>BSCLL</scope><scope>CGR</scope><scope>CUY</scope><scope>CVF</scope><scope>ECM</scope><scope>EIF</scope><scope>NPM</scope><scope>7T5</scope><scope>7TK</scope><scope>7TM</scope><scope>7TS</scope><scope>7U7</scope><scope>7U9</scope><scope>C1K</scope><scope>H94</scope><scope>K9.</scope><scope>NAPCQ</scope><scope>7X8</scope></search><sort><creationdate>201610</creationdate><title>Clinical trial readiness in non-ambulatory boys and men with duchenne muscular dystrophy: MDA-DMD network follow-up</title><author>Connolly, Anne M. ; Florence, Julaine M. ; Zaidman, Craig M. ; Golumbek, Paul T. ; Mendell, Jerry R. ; Flanigan, Kevin M. ; Karachunski, Peter I. ; Day, John W. ; McDonald, Craig M. ; Darras, Basil T. ; Kang, Peter B. ; Siener, Catherine A. ; Gadeken, Rebecca K. ; Anand, Pallavi ; Schierbecker, Jeanine R. ; Malkus, Elizabeth C. ; Lowes, Linda P. ; Alfano, Lindsay N. ; Johnson, Linda ; Nicorici, Alina ; Kelecic, Jason M. ; Quigley, Janet ; Pasternak, Amy E. ; Miller, J. Philip</author></sort><facets><frbrtype>5</frbrtype><frbrgroupid>cdi_FETCH-LOGICAL-c4609-82269d5887172654755fedf83a73f739c661767221bdf7ea09b49aacb7e5c453</frbrgroupid><rsrctype>articles</rsrctype><prefilter>articles</prefilter><language>eng</language><creationdate>2016</creationdate><topic>Adolescent</topic><topic>Adrenal Cortex Hormones - therapeutic use</topic><topic>Adult</topic><topic>Brooke Scale</topic><topic>Child</topic><topic>clinical trial</topic><topic>Duchenne muscular dystrophy</topic><topic>Egen Klassifikation Scale</topic><topic>Elbow</topic><topic>Follow-Up Studies</topic><topic>force vital capacity</topic><topic>Hand Strength - physiology</topic><topic>Humans</topic><topic>Male</topic><topic>Mobility Limitation</topic><topic>Muscular dystrophy</topic><topic>Muscular Dystrophy, Duchenne - diagnosis</topic><topic>Muscular Dystrophy, Duchenne - drug therapy</topic><topic>Muscular Dystrophy, Duchenne - physiopathology</topic><topic>non-ambulatory</topic><topic>outcomes</topic><topic>Patient Participation - methods</topic><topic>Range of Motion, Articular - physiology</topic><topic>Vital Capacity - physiology</topic><topic>Young Adult</topic><toplevel>peer_reviewed</toplevel><toplevel>online_resources</toplevel><creatorcontrib>Connolly, Anne M.</creatorcontrib><creatorcontrib>Florence, Julaine M.</creatorcontrib><creatorcontrib>Zaidman, Craig M.</creatorcontrib><creatorcontrib>Golumbek, Paul T.</creatorcontrib><creatorcontrib>Mendell, Jerry R.</creatorcontrib><creatorcontrib>Flanigan, Kevin M.</creatorcontrib><creatorcontrib>Karachunski, Peter I.</creatorcontrib><creatorcontrib>Day, John W.</creatorcontrib><creatorcontrib>McDonald, Craig M.</creatorcontrib><creatorcontrib>Darras, Basil T.</creatorcontrib><creatorcontrib>Kang, Peter B.</creatorcontrib><creatorcontrib>Siener, Catherine A.</creatorcontrib><creatorcontrib>Gadeken, Rebecca K.</creatorcontrib><creatorcontrib>Anand, Pallavi</creatorcontrib><creatorcontrib>Schierbecker, Jeanine R.</creatorcontrib><creatorcontrib>Malkus, Elizabeth C.</creatorcontrib><creatorcontrib>Lowes, Linda P.</creatorcontrib><creatorcontrib>Alfano, Lindsay N.</creatorcontrib><creatorcontrib>Johnson, Linda</creatorcontrib><creatorcontrib>Nicorici, Alina</creatorcontrib><creatorcontrib>Kelecic, Jason M.</creatorcontrib><creatorcontrib>Quigley, Janet</creatorcontrib><creatorcontrib>Pasternak, Amy E.</creatorcontrib><creatorcontrib>Miller, J. Philip</creatorcontrib><creatorcontrib>MDA-DMD Clinical Research Network</creatorcontrib><collection>Istex</collection><collection>Medline</collection><collection>MEDLINE</collection><collection>MEDLINE (Ovid)</collection><collection>MEDLINE</collection><collection>MEDLINE</collection><collection>PubMed</collection><collection>Immunology Abstracts</collection><collection>Neurosciences Abstracts</collection><collection>Nucleic Acids Abstracts</collection><collection>Physical Education Index</collection><collection>Toxicology Abstracts</collection><collection>Virology and AIDS Abstracts</collection><collection>Environmental Sciences and Pollution Management</collection><collection>AIDS and Cancer Research Abstracts</collection><collection>ProQuest Health & Medical Complete (Alumni)</collection><collection>Nursing & Allied Health Premium</collection><collection>MEDLINE - Academic</collection><jtitle>Muscle & nerve</jtitle></facets><delivery><delcategory>Remote Search Resource</delcategory><fulltext>fulltext</fulltext></delivery><addata><au>Connolly, Anne M.</au><au>Florence, Julaine M.</au><au>Zaidman, Craig M.</au><au>Golumbek, Paul T.</au><au>Mendell, Jerry R.</au><au>Flanigan, Kevin M.</au><au>Karachunski, Peter I.</au><au>Day, John W.</au><au>McDonald, Craig M.</au><au>Darras, Basil T.</au><au>Kang, Peter B.</au><au>Siener, Catherine A.</au><au>Gadeken, Rebecca K.</au><au>Anand, Pallavi</au><au>Schierbecker, Jeanine R.</au><au>Malkus, Elizabeth C.</au><au>Lowes, Linda P.</au><au>Alfano, Lindsay N.</au><au>Johnson, Linda</au><au>Nicorici, Alina</au><au>Kelecic, Jason M.</au><au>Quigley, Janet</au><au>Pasternak, Amy E.</au><au>Miller, J. Philip</au><aucorp>MDA-DMD Clinical Research Network</aucorp><format>journal</format><genre>article</genre><ristype>JOUR</ristype><atitle>Clinical trial readiness in non-ambulatory boys and men with duchenne muscular dystrophy: MDA-DMD network follow-up</atitle><jtitle>Muscle & nerve</jtitle><addtitle>Muscle Nerve</addtitle><date>2016-10</date><risdate>2016</risdate><volume>54</volume><issue>4</issue><spage>681</spage><epage>689</epage><pages>681-689</pages><issn>0148-639X</issn><eissn>1097-4598</eissn><coden>MUNEDE</coden><abstract>ABSTRACT
Introduction: Outcomes sensitive to change over time in non‐ambulatory boys/men with Duchenne muscular dystrophy (DMD) are not well‐established. Methods: Subjects (n = 91; 16.8 ± 4.5 years old) were assessed at baseline and 6‐month intervals for 2 years. We analyzed all subjects using an intent‐to‐treat model and a subset of stronger subjects with Brooke Scale score ≤4, using repeated measures. Results: Eight patients (12–33 years old) died during the study. Sixty‐six completed 12‐month follow‐up, and 51 completed 24‐month follow‐up. Those taking corticosteroids performed better at baseline, but rates of decline were similar. Forced vital capacity percent predicted (FVC% predicted) declined significantly only after 2 years. However, Brooke and Egen Klassifikation (EK) Scale scores, elbow flexion, and grip strength declined significantly over both 1 and 2 years. Conclusion: Brooke and EK Scale scores, elbow flexion, and grip strength were outcomes most responsive to change. FVC% predicted was responsive to change over 2 years. Corticosteroids benefited non‐ambulatory DMD subjects but did not affect decline rates of measures tested here. Muscle Nerve 54: 681–689, 2016</abstract><cop>United States</cop><pub>Blackwell Publishing Ltd</pub><pmid>26930423</pmid><doi>10.1002/mus.25089</doi><tpages>9</tpages></addata></record> |
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| ispartof | Muscle & nerve, 2016-10, Vol.54 (4), p.681-689 |
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| subjects | Adolescent Adrenal Cortex Hormones - therapeutic use Adult Brooke Scale Child clinical trial Duchenne muscular dystrophy Egen Klassifikation Scale Elbow Follow-Up Studies force vital capacity Hand Strength - physiology Humans Male Mobility Limitation Muscular dystrophy Muscular Dystrophy, Duchenne - diagnosis Muscular Dystrophy, Duchenne - drug therapy Muscular Dystrophy, Duchenne - physiopathology non-ambulatory outcomes Patient Participation - methods Range of Motion, Articular - physiology Vital Capacity - physiology Young Adult |
| title | Clinical trial readiness in non-ambulatory boys and men with duchenne muscular dystrophy: MDA-DMD network follow-up |
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