Facioscapulohumeral muscular dystrophy functional composite outcome measure
ABSTRACT Introduction: We developed an evaluator‐administered functional facioscapulohumeral muscular dystrophy composite outcome measure (FSHD‐COM) comprising patient‐identified areas of functional burden for future clinical trials. Methods: We performed a prospective observational study of 41 pati...
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| Veröffentlicht in: | Muscle & nerve 2018-07, Vol.58 (1), p.72-78 |
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| creator | Eichinger, Katy Heatwole, Chad Iyadurai, Stanley King, Wendy Baker, Lindsay Heininger, Susanne Bartlett, Amy Dilek, Nuran Martens, William B. Mcdermott, Michael Kissel, John T. Tawil, Rabi Statland, Jeffrey M. |
| description | ABSTRACT
Introduction: We developed an evaluator‐administered functional facioscapulohumeral muscular dystrophy composite outcome measure (FSHD‐COM) comprising patient‐identified areas of functional burden for future clinical trials. Methods: We performed a prospective observational study of 41 patients with FSHD at 2 sites. The FSHD‐COM includes functional assessment of the legs, shoulders and arms, trunk, hands, and balance/mobility. We determined the test‐retest reliability and convergent validity compared to established FSHD disease metrics. Results: The FSHD‐COM demonstrated excellent test‐retest reliability (intraclass correlation coefficient [ICC] 0.96; subscale ICC range, 0.90–0.94). Cross‐sectional associations between the FSHD‐COM and disease duration, clinical severity, and strength were moderate to strong (Pearson correlation coefficient range |0.51–0.92|). Discussion: The FSHD‐COM is a disease‐relevant, functional composite outcome measure suitable for future FSHD clinical trials that shows excellent test‐retest reliability and cross‐sectional associations to disease measures. Future directions include determining multisite reliability, sensitivity to change, and the minimal clinically important change in the FSHD‐COM. Muscle Nerve 58: 72–78, 2018 |
| doi_str_mv | 10.1002/mus.26088 |
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Introduction: We developed an evaluator‐administered functional facioscapulohumeral muscular dystrophy composite outcome measure (FSHD‐COM) comprising patient‐identified areas of functional burden for future clinical trials. Methods: We performed a prospective observational study of 41 patients with FSHD at 2 sites. The FSHD‐COM includes functional assessment of the legs, shoulders and arms, trunk, hands, and balance/mobility. We determined the test‐retest reliability and convergent validity compared to established FSHD disease metrics. Results: The FSHD‐COM demonstrated excellent test‐retest reliability (intraclass correlation coefficient [ICC] 0.96; subscale ICC range, 0.90–0.94). Cross‐sectional associations between the FSHD‐COM and disease duration, clinical severity, and strength were moderate to strong (Pearson correlation coefficient range |0.51–0.92|). Discussion: The FSHD‐COM is a disease‐relevant, functional composite outcome measure suitable for future FSHD clinical trials that shows excellent test‐retest reliability and cross‐sectional associations to disease measures. Future directions include determining multisite reliability, sensitivity to change, and the minimal clinically important change in the FSHD‐COM. Muscle Nerve 58: 72–78, 2018</description><identifier>ISSN: 0148-639X</identifier><identifier>EISSN: 1097-4598</identifier><identifier>DOI: 10.1002/mus.26088</identifier><identifier>PMID: 29381807</identifier><language>eng</language><publisher>United States: Wiley Subscription Services, Inc</publisher><subject>Clinical trials ; composite measures ; Correlation coefficient ; Correlation coefficients ; Dystrophy ; facioscapulohumeral muscular dystrophy ; functional testing ; Hand (anatomy) ; Identification methods ; Medical research ; Muscles ; Muscular dystrophy ; outcome measures ; Reliability</subject><ispartof>Muscle & nerve, 2018-07, Vol.58 (1), p.72-78</ispartof><rights>2018 Wiley Periodicals, Inc.</rights><lds50>peer_reviewed</lds50><oa>free_for_read</oa><woscitedreferencessubscribed>false</woscitedreferencessubscribed><citedby>FETCH-LOGICAL-c4438-d90741f40d789526cbbd7c8095faf6233968d3cd2b11030824af069d966019e03</citedby><cites>FETCH-LOGICAL-c4438-d90741f40d789526cbbd7c8095faf6233968d3cd2b11030824af069d966019e03</cites><orcidid>0000-0003-0790-5315</orcidid></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.26088$$EPDF$$P50$$Gwiley$$H</linktopdf><linktohtml>$$Uhttps://onlinelibrary.wiley.com/doi/full/10.1002%2Fmus.26088$$EHTML$$P50$$Gwiley$$H</linktohtml><link.rule.ids>230,314,776,780,881,1411,27901,27902,45550,45551</link.rule.ids><backlink>$$Uhttps://www.ncbi.nlm.nih.gov/pubmed/29381807$$D View this record in MEDLINE/PubMed$$Hfree_for_read</backlink></links><search><creatorcontrib>Eichinger, Katy</creatorcontrib><creatorcontrib>Heatwole, Chad</creatorcontrib><creatorcontrib>Iyadurai, Stanley</creatorcontrib><creatorcontrib>King, Wendy</creatorcontrib><creatorcontrib>Baker, Lindsay</creatorcontrib><creatorcontrib>Heininger, Susanne</creatorcontrib><creatorcontrib>Bartlett, Amy</creatorcontrib><creatorcontrib>Dilek, Nuran</creatorcontrib><creatorcontrib>Martens, William B.</creatorcontrib><creatorcontrib>Mcdermott, Michael</creatorcontrib><creatorcontrib>Kissel, John T.</creatorcontrib><creatorcontrib>Tawil, Rabi</creatorcontrib><creatorcontrib>Statland, Jeffrey M.</creatorcontrib><title>Facioscapulohumeral muscular dystrophy functional composite outcome measure</title><title>Muscle & nerve</title><addtitle>Muscle Nerve</addtitle><description>ABSTRACT
Introduction: We developed an evaluator‐administered functional facioscapulohumeral muscular dystrophy composite outcome measure (FSHD‐COM) comprising patient‐identified areas of functional burden for future clinical trials. Methods: We performed a prospective observational study of 41 patients with FSHD at 2 sites. The FSHD‐COM includes functional assessment of the legs, shoulders and arms, trunk, hands, and balance/mobility. We determined the test‐retest reliability and convergent validity compared to established FSHD disease metrics. Results: The FSHD‐COM demonstrated excellent test‐retest reliability (intraclass correlation coefficient [ICC] 0.96; subscale ICC range, 0.90–0.94). Cross‐sectional associations between the FSHD‐COM and disease duration, clinical severity, and strength were moderate to strong (Pearson correlation coefficient range |0.51–0.92|). Discussion: The FSHD‐COM is a disease‐relevant, functional composite outcome measure suitable for future FSHD clinical trials that shows excellent test‐retest reliability and cross‐sectional associations to disease measures. Future directions include determining multisite reliability, sensitivity to change, and the minimal clinically important change in the FSHD‐COM. Muscle Nerve 58: 72–78, 2018</description><subject>Clinical trials</subject><subject>composite measures</subject><subject>Correlation coefficient</subject><subject>Correlation coefficients</subject><subject>Dystrophy</subject><subject>facioscapulohumeral muscular dystrophy</subject><subject>functional testing</subject><subject>Hand (anatomy)</subject><subject>Identification methods</subject><subject>Medical research</subject><subject>Muscles</subject><subject>Muscular dystrophy</subject><subject>outcome measures</subject><subject>Reliability</subject><issn>0148-639X</issn><issn>1097-4598</issn><fulltext>true</fulltext><rsrctype>article</rsrctype><creationdate>2018</creationdate><recordtype>article</recordtype><recordid>eNp1kcFKxDAQhoMouq4efAEpeNFDdydNNk0ugiyuiooHFbyFbJq6lbapSaPs2xtdFRU8DcN8fPzMj9AehhEGyMZN8KOMAedraIBB5CmdCL6OBoApTxkRD1to2_snAMCc5ZtoKxOEYw75AF3OlK6s16oLtV2ExjhVJ9GnQ61cUix972y3WCZlaHVf2TZetW0666veJDb0cTFJY5QPzuygjVLV3ux-ziG6n53eTc_Tq5uzi-nJVaopJTwtBOQUlxSKnItJxvR8XuSag5iUqmQZIYLxgugim2MMBHhGVQlMFIIxwMIAGaLjlbcL88YU2rR9TC07VzXKLaVVlfx9aauFfLQvkgFjlNEoOPwUOPscjO9lU3lt6lq1xgYvsRAEMMETEtGDP-iTDS6-wcsM8phHAMeROlpR2lnvnSm_w2CQ7xXJ-FL5UVFk93-m_ya_OonAeAW8VrVZ_m-S1_e3K-UbDluccw</recordid><startdate>201807</startdate><enddate>201807</enddate><creator>Eichinger, Katy</creator><creator>Heatwole, Chad</creator><creator>Iyadurai, Stanley</creator><creator>King, Wendy</creator><creator>Baker, Lindsay</creator><creator>Heininger, Susanne</creator><creator>Bartlett, Amy</creator><creator>Dilek, Nuran</creator><creator>Martens, William B.</creator><creator>Mcdermott, Michael</creator><creator>Kissel, John T.</creator><creator>Tawil, Rabi</creator><creator>Statland, Jeffrey M.</creator><general>Wiley Subscription Services, Inc</general><scope>NPM</scope><scope>AAYXX</scope><scope>CITATION</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><scope>5PM</scope><orcidid>https://orcid.org/0000-0003-0790-5315</orcidid></search><sort><creationdate>201807</creationdate><title>Facioscapulohumeral muscular dystrophy functional composite outcome measure</title><author>Eichinger, Katy ; Heatwole, Chad ; Iyadurai, Stanley ; King, Wendy ; Baker, Lindsay ; Heininger, Susanne ; Bartlett, Amy ; Dilek, Nuran ; Martens, William B. ; Mcdermott, Michael ; Kissel, John T. ; Tawil, Rabi ; Statland, Jeffrey M.</author></sort><facets><frbrtype>5</frbrtype><frbrgroupid>cdi_FETCH-LOGICAL-c4438-d90741f40d789526cbbd7c8095faf6233968d3cd2b11030824af069d966019e03</frbrgroupid><rsrctype>articles</rsrctype><prefilter>articles</prefilter><language>eng</language><creationdate>2018</creationdate><topic>Clinical trials</topic><topic>composite measures</topic><topic>Correlation coefficient</topic><topic>Correlation coefficients</topic><topic>Dystrophy</topic><topic>facioscapulohumeral muscular dystrophy</topic><topic>functional testing</topic><topic>Hand (anatomy)</topic><topic>Identification methods</topic><topic>Medical research</topic><topic>Muscles</topic><topic>Muscular dystrophy</topic><topic>outcome measures</topic><topic>Reliability</topic><toplevel>peer_reviewed</toplevel><toplevel>online_resources</toplevel><creatorcontrib>Eichinger, Katy</creatorcontrib><creatorcontrib>Heatwole, Chad</creatorcontrib><creatorcontrib>Iyadurai, Stanley</creatorcontrib><creatorcontrib>King, Wendy</creatorcontrib><creatorcontrib>Baker, Lindsay</creatorcontrib><creatorcontrib>Heininger, Susanne</creatorcontrib><creatorcontrib>Bartlett, Amy</creatorcontrib><creatorcontrib>Dilek, Nuran</creatorcontrib><creatorcontrib>Martens, William B.</creatorcontrib><creatorcontrib>Mcdermott, Michael</creatorcontrib><creatorcontrib>Kissel, John T.</creatorcontrib><creatorcontrib>Tawil, Rabi</creatorcontrib><creatorcontrib>Statland, Jeffrey M.</creatorcontrib><collection>PubMed</collection><collection>CrossRef</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><collection>PubMed Central (Full Participant titles)</collection><jtitle>Muscle & nerve</jtitle></facets><delivery><delcategory>Remote Search Resource</delcategory><fulltext>fulltext</fulltext></delivery><addata><au>Eichinger, Katy</au><au>Heatwole, Chad</au><au>Iyadurai, Stanley</au><au>King, Wendy</au><au>Baker, Lindsay</au><au>Heininger, Susanne</au><au>Bartlett, Amy</au><au>Dilek, Nuran</au><au>Martens, William B.</au><au>Mcdermott, Michael</au><au>Kissel, John T.</au><au>Tawil, Rabi</au><au>Statland, Jeffrey M.</au><format>journal</format><genre>article</genre><ristype>JOUR</ristype><atitle>Facioscapulohumeral muscular dystrophy functional composite outcome measure</atitle><jtitle>Muscle & nerve</jtitle><addtitle>Muscle Nerve</addtitle><date>2018-07</date><risdate>2018</risdate><volume>58</volume><issue>1</issue><spage>72</spage><epage>78</epage><pages>72-78</pages><issn>0148-639X</issn><eissn>1097-4598</eissn><abstract>ABSTRACT
Introduction: We developed an evaluator‐administered functional facioscapulohumeral muscular dystrophy composite outcome measure (FSHD‐COM) comprising patient‐identified areas of functional burden for future clinical trials. Methods: We performed a prospective observational study of 41 patients with FSHD at 2 sites. The FSHD‐COM includes functional assessment of the legs, shoulders and arms, trunk, hands, and balance/mobility. We determined the test‐retest reliability and convergent validity compared to established FSHD disease metrics. Results: The FSHD‐COM demonstrated excellent test‐retest reliability (intraclass correlation coefficient [ICC] 0.96; subscale ICC range, 0.90–0.94). Cross‐sectional associations between the FSHD‐COM and disease duration, clinical severity, and strength were moderate to strong (Pearson correlation coefficient range |0.51–0.92|). Discussion: The FSHD‐COM is a disease‐relevant, functional composite outcome measure suitable for future FSHD clinical trials that shows excellent test‐retest reliability and cross‐sectional associations to disease measures. Future directions include determining multisite reliability, sensitivity to change, and the minimal clinically important change in the FSHD‐COM. Muscle Nerve 58: 72–78, 2018</abstract><cop>United States</cop><pub>Wiley Subscription Services, Inc</pub><pmid>29381807</pmid><doi>10.1002/mus.26088</doi><tpages>7</tpages><orcidid>https://orcid.org/0000-0003-0790-5315</orcidid><oa>free_for_read</oa></addata></record> |
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| subjects | Clinical trials composite measures Correlation coefficient Correlation coefficients Dystrophy facioscapulohumeral muscular dystrophy functional testing Hand (anatomy) Identification methods Medical research Muscles Muscular dystrophy outcome measures Reliability |
| title | Facioscapulohumeral muscular dystrophy functional composite outcome measure |
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