Functional abilities, respiratory and cardiac function in a large cohort of adults with Duchenne muscular dystrophy treated with glucocorticoids
Background and purpose The transition to adult services, and subsequent glucocorticoid management, is critical in adults with Duchenne muscular dystrophy. This study aims (1) to describe treatment, functional abilities, respiratory and cardiac status during transition to adulthood and adult stages;...
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| Veröffentlicht in: | European journal of neurology 2024-06, Vol.31 (6), p.e16267-n/a |
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| creator | Schiava, Marianela Lofra, Robert Muni Bourke, John P. Díaz‐Manera, Jordi James, Meredith K. Elseed, Maha A. Malinova, Monika Michel‐Sodhi, Jassi Moat, Dionne Ghimenton, Elisabetta Mccallum, Michelle Díaz, Carla Florencia Bolaño Mayhew, Anna Wong, Karen Richardson, Mark Tasca, Giorgio Eglon, Gail Eagle, Michelle Turner, Cathy Heslop, Emma Straub, Volker Bettolo, Chiara Marini Guglieri, Michela |
| description | Background and purpose
The transition to adult services, and subsequent glucocorticoid management, is critical in adults with Duchenne muscular dystrophy. This study aims (1) to describe treatment, functional abilities, respiratory and cardiac status during transition to adulthood and adult stages; and (2) to explore the association between glucocorticoid treatment after loss of ambulation (LOA) and late‐stage clinical outcomes.
Methods
This was a retrospective single‐centre study on individuals with Duchenne muscular dystrophy (≥16 years old) between 1986 and 2022. Logistic regression, Cox proportional hazards models and survival analyses were conducted utilizing data from clinical records.
Results
In all, 112 individuals were included. Mean age was 23.4 ± 5.2 years and mean follow‐up was 18.5 ± 5.5 years. At last assessment, 47.2% were on glucocorticoids; the mean dose of prednisone was 0.38 ± 0.13 mg/kg/day and of deflazacort 0.43 ± 0.16 mg/kg/day. At age 16 years, motor function limitations included using a manual wheelchair (89.7%), standing (87.9%), transferring from a wheelchair (86.2%) and turning in bed (53.4%); 77.5% had a peak cough flow |
| doi_str_mv | 10.1111/ene.16267 |
| format | Article |
| fullrecord | <record><control><sourceid>proquest_cross</sourceid><recordid>TN_cdi_proquest_miscellaneous_3029816908</recordid><sourceformat>XML</sourceformat><sourcesystem>PC</sourcesystem><sourcerecordid>3029816908</sourcerecordid><originalsourceid>FETCH-LOGICAL-c3487-ec65bad973ebf57ef46558cacb898efda56d1462b727320229d2676317f431443</originalsourceid><addsrcrecordid>eNp10ctu1DAYBWALgegFFrwAssQGJNL6EjvOEpUpIFXtBtaRY__puPLEgy-q8hY8ct1mYIFUb34vPp3FOQi9o-SM1ncOM5xRyWT3Ah3TVqqGck5f1j8XtBGU0CN0ktIdIYR1jLxGR1wJIVXPj9GfyzKb7MKsPdaj8y47SJ9xhLR3UecQF6xni42O1mmDp4PGbsYaex1vAZuwDTHjMGFti88J37u8xV-L2cI8A96VZEqV2C4px7DfLjhH0BnsCm99McHUBGeCs-kNejVpn-Dt4Z6iX5ebnxffm6ubbz8uvlw1hreqa8BIMWrbdxzGSXQwtVIIZbQZVa9gslpIW6tgY8c6zghjva39SE67qeW0bfkp-rjm7mP4XSDlYeeSAe_1DKGkgRPWKyp7oir98B-9CyXWxh6VoIoLplhVn1ZlYkgpwjTso9vpuAyUDI8zDXWm4Wmmat8fEsu4A_tP_t2lgvMV3DsPy_NJw-Z6s0Y-AK6fnkQ</addsrcrecordid><sourcetype>Aggregation Database</sourcetype><iscdi>true</iscdi><recordtype>article</recordtype><pqid>3051835282</pqid></control><display><type>article</type><title>Functional abilities, respiratory and cardiac function in a large cohort of adults with Duchenne muscular dystrophy treated with glucocorticoids</title><source>MEDLINE</source><source>Wiley Online Library Open Access</source><source>Wiley Online Library Journals Frontfile Complete</source><source>PubMed Central</source><creator>Schiava, Marianela ; Lofra, Robert Muni ; Bourke, John P. ; Díaz‐Manera, Jordi ; James, Meredith K. ; Elseed, Maha A. ; Malinova, Monika ; Michel‐Sodhi, Jassi ; Moat, Dionne ; Ghimenton, Elisabetta ; Mccallum, Michelle ; Díaz, Carla Florencia Bolaño ; Mayhew, Anna ; Wong, Karen ; Richardson, Mark ; Tasca, Giorgio ; Eglon, Gail ; Eagle, Michelle ; Turner, Cathy ; Heslop, Emma ; Straub, Volker ; Bettolo, Chiara Marini ; Guglieri, Michela</creator><creatorcontrib>Schiava, Marianela ; Lofra, Robert Muni ; Bourke, John P. ; Díaz‐Manera, Jordi ; James, Meredith K. ; Elseed, Maha A. ; Malinova, Monika ; Michel‐Sodhi, Jassi ; Moat, Dionne ; Ghimenton, Elisabetta ; Mccallum, Michelle ; Díaz, Carla Florencia Bolaño ; Mayhew, Anna ; Wong, Karen ; Richardson, Mark ; Tasca, Giorgio ; Eglon, Gail ; Eagle, Michelle ; Turner, Cathy ; Heslop, Emma ; Straub, Volker ; Bettolo, Chiara Marini ; Guglieri, Michela</creatorcontrib><description><![CDATA[Background and purpose
The transition to adult services, and subsequent glucocorticoid management, is critical in adults with Duchenne muscular dystrophy. This study aims (1) to describe treatment, functional abilities, respiratory and cardiac status during transition to adulthood and adult stages; and (2) to explore the association between glucocorticoid treatment after loss of ambulation (LOA) and late‐stage clinical outcomes.
Methods
This was a retrospective single‐centre study on individuals with Duchenne muscular dystrophy (≥16 years old) between 1986 and 2022. Logistic regression, Cox proportional hazards models and survival analyses were conducted utilizing data from clinical records.
Results
In all, 112 individuals were included. Mean age was 23.4 ± 5.2 years and mean follow‐up was 18.5 ± 5.5 years. At last assessment, 47.2% were on glucocorticoids; the mean dose of prednisone was 0.38 ± 0.13 mg/kg/day and of deflazacort 0.43 ± 0.16 mg/kg/day. At age 16 years, motor function limitations included using a manual wheelchair (89.7%), standing (87.9%), transferring from a wheelchair (86.2%) and turning in bed (53.4%); 77.5% had a peak cough flow <270 L/min, 53.3% a forced vital capacity percentage of predicted <50% and 40.3% a left ventricular ejection fraction <50%. Glucocorticoids after LOA reduced the risk and delayed the time to difficulties balancing in the wheelchair, loss of hand to mouth function, forced vital capacity percentage of predicted <30% and forced vital capacity <1 L and were associated with lower frequency of left ventricular ejection fraction <50%, without differences between prednisone and deflazacort. Glucocorticoid dose did not differ by functional, respiratory or cardiac status.
Conclusion
Glucocorticoids after LOA preserve late‐stage functional abilities, respiratory and cardiac function. It is suggested using functional abilities, respiratory and cardiac status at transition stages for adult services planning.]]></description><identifier>ISSN: 1351-5101</identifier><identifier>ISSN: 1468-1331</identifier><identifier>EISSN: 1468-1331</identifier><identifier>DOI: 10.1111/ene.16267</identifier><identifier>PMID: 38556893</identifier><language>eng</language><publisher>England: John Wiley & Sons, Inc</publisher><subject>Adolescent ; Adult ; Adults ; Cardiac function ; cardiac function in adults with DMD ; Cohort Studies ; Cough ; Duchenne's muscular dystrophy ; Dystrophy ; Ejection fraction ; EK scale in adults with DMD ; Female ; glucocorticoid dose in adults with DMD ; Glucocorticoids ; Glucocorticoids - therapeutic use ; Heart ; Heart - drug effects ; Heart - physiopathology ; Humans ; Male ; Mobility Limitation ; Muscular dystrophy ; Muscular Dystrophy, Duchenne - drug therapy ; Muscular Dystrophy, Duchenne - physiopathology ; Prednisone ; Prednisone - therapeutic use ; Pregnenediones - therapeutic use ; Regression analysis ; respiratory function in adults with DMD ; Retrospective Studies ; Risk reduction ; Statistical models ; transition to adulthood in DMD ; Ventricle ; Wheelchairs ; Young Adult</subject><ispartof>European journal of neurology, 2024-06, Vol.31 (6), p.e16267-n/a</ispartof><rights>2024 The Authors. published by John Wiley & Sons Ltd on behalf of European Academy of Neurology.</rights><rights>2024 The Authors. European Journal of Neurology published by John Wiley & Sons Ltd on behalf of European Academy of Neurology.</rights><rights>2024. This article is published under http://creativecommons.org/licenses/by-nc-nd/4.0/ (the “License”). Notwithstanding the ProQuest Terms and Conditions, you may use this content in accordance with the terms of the License.</rights><lds50>peer_reviewed</lds50><oa>free_for_read</oa><woscitedreferencessubscribed>false</woscitedreferencessubscribed><cites>FETCH-LOGICAL-c3487-ec65bad973ebf57ef46558cacb898efda56d1462b727320229d2676317f431443</cites><orcidid>0000-0001-6674-6970 ; 0000-0002-2709-265X ; 0000-0002-8455-0637 ; 0000-0003-2941-7988</orcidid></display><links><openurl>$$Topenurl_article</openurl><openurlfulltext>$$Topenurlfull_article</openurlfulltext><thumbnail>$$Tsyndetics_thumb_exl</thumbnail><linktopdf>$$Uhttps://onlinelibrary.wiley.com/doi/pdf/10.1111%2Fene.16267$$EPDF$$P50$$Gwiley$$Hfree_for_read</linktopdf><linktohtml>$$Uhttps://onlinelibrary.wiley.com/doi/full/10.1111%2Fene.16267$$EHTML$$P50$$Gwiley$$Hfree_for_read</linktohtml><link.rule.ids>314,776,780,1411,11541,27901,27902,45550,45551,46027,46451</link.rule.ids><backlink>$$Uhttps://www.ncbi.nlm.nih.gov/pubmed/38556893$$D View this record in MEDLINE/PubMed$$Hfree_for_read</backlink></links><search><creatorcontrib>Schiava, Marianela</creatorcontrib><creatorcontrib>Lofra, Robert Muni</creatorcontrib><creatorcontrib>Bourke, John P.</creatorcontrib><creatorcontrib>Díaz‐Manera, Jordi</creatorcontrib><creatorcontrib>James, Meredith K.</creatorcontrib><creatorcontrib>Elseed, Maha A.</creatorcontrib><creatorcontrib>Malinova, Monika</creatorcontrib><creatorcontrib>Michel‐Sodhi, Jassi</creatorcontrib><creatorcontrib>Moat, Dionne</creatorcontrib><creatorcontrib>Ghimenton, Elisabetta</creatorcontrib><creatorcontrib>Mccallum, Michelle</creatorcontrib><creatorcontrib>Díaz, Carla Florencia Bolaño</creatorcontrib><creatorcontrib>Mayhew, Anna</creatorcontrib><creatorcontrib>Wong, Karen</creatorcontrib><creatorcontrib>Richardson, Mark</creatorcontrib><creatorcontrib>Tasca, Giorgio</creatorcontrib><creatorcontrib>Eglon, Gail</creatorcontrib><creatorcontrib>Eagle, Michelle</creatorcontrib><creatorcontrib>Turner, Cathy</creatorcontrib><creatorcontrib>Heslop, Emma</creatorcontrib><creatorcontrib>Straub, Volker</creatorcontrib><creatorcontrib>Bettolo, Chiara Marini</creatorcontrib><creatorcontrib>Guglieri, Michela</creatorcontrib><title>Functional abilities, respiratory and cardiac function in a large cohort of adults with Duchenne muscular dystrophy treated with glucocorticoids</title><title>European journal of neurology</title><addtitle>Eur J Neurol</addtitle><description><![CDATA[Background and purpose
The transition to adult services, and subsequent glucocorticoid management, is critical in adults with Duchenne muscular dystrophy. This study aims (1) to describe treatment, functional abilities, respiratory and cardiac status during transition to adulthood and adult stages; and (2) to explore the association between glucocorticoid treatment after loss of ambulation (LOA) and late‐stage clinical outcomes.
Methods
This was a retrospective single‐centre study on individuals with Duchenne muscular dystrophy (≥16 years old) between 1986 and 2022. Logistic regression, Cox proportional hazards models and survival analyses were conducted utilizing data from clinical records.
Results
In all, 112 individuals were included. Mean age was 23.4 ± 5.2 years and mean follow‐up was 18.5 ± 5.5 years. At last assessment, 47.2% were on glucocorticoids; the mean dose of prednisone was 0.38 ± 0.13 mg/kg/day and of deflazacort 0.43 ± 0.16 mg/kg/day. At age 16 years, motor function limitations included using a manual wheelchair (89.7%), standing (87.9%), transferring from a wheelchair (86.2%) and turning in bed (53.4%); 77.5% had a peak cough flow <270 L/min, 53.3% a forced vital capacity percentage of predicted <50% and 40.3% a left ventricular ejection fraction <50%. Glucocorticoids after LOA reduced the risk and delayed the time to difficulties balancing in the wheelchair, loss of hand to mouth function, forced vital capacity percentage of predicted <30% and forced vital capacity <1 L and were associated with lower frequency of left ventricular ejection fraction <50%, without differences between prednisone and deflazacort. Glucocorticoid dose did not differ by functional, respiratory or cardiac status.
Conclusion
Glucocorticoids after LOA preserve late‐stage functional abilities, respiratory and cardiac function. It is suggested using functional abilities, respiratory and cardiac status at transition stages for adult services planning.]]></description><subject>Adolescent</subject><subject>Adult</subject><subject>Adults</subject><subject>Cardiac function</subject><subject>cardiac function in adults with DMD</subject><subject>Cohort Studies</subject><subject>Cough</subject><subject>Duchenne's muscular dystrophy</subject><subject>Dystrophy</subject><subject>Ejection fraction</subject><subject>EK scale in adults with DMD</subject><subject>Female</subject><subject>glucocorticoid dose in adults with DMD</subject><subject>Glucocorticoids</subject><subject>Glucocorticoids - therapeutic use</subject><subject>Heart</subject><subject>Heart - drug effects</subject><subject>Heart - physiopathology</subject><subject>Humans</subject><subject>Male</subject><subject>Mobility Limitation</subject><subject>Muscular dystrophy</subject><subject>Muscular Dystrophy, Duchenne - drug therapy</subject><subject>Muscular Dystrophy, Duchenne - physiopathology</subject><subject>Prednisone</subject><subject>Prednisone - therapeutic use</subject><subject>Pregnenediones - therapeutic use</subject><subject>Regression analysis</subject><subject>respiratory function in adults with DMD</subject><subject>Retrospective Studies</subject><subject>Risk reduction</subject><subject>Statistical models</subject><subject>transition to adulthood in DMD</subject><subject>Ventricle</subject><subject>Wheelchairs</subject><subject>Young Adult</subject><issn>1351-5101</issn><issn>1468-1331</issn><issn>1468-1331</issn><fulltext>true</fulltext><rsrctype>article</rsrctype><creationdate>2024</creationdate><recordtype>article</recordtype><sourceid>24P</sourceid><sourceid>EIF</sourceid><recordid>eNp10ctu1DAYBWALgegFFrwAssQGJNL6EjvOEpUpIFXtBtaRY__puPLEgy-q8hY8ct1mYIFUb34vPp3FOQi9o-SM1ncOM5xRyWT3Ah3TVqqGck5f1j8XtBGU0CN0ktIdIYR1jLxGR1wJIVXPj9GfyzKb7MKsPdaj8y47SJ9xhLR3UecQF6xni42O1mmDp4PGbsYaex1vAZuwDTHjMGFti88J37u8xV-L2cI8A96VZEqV2C4px7DfLjhH0BnsCm99McHUBGeCs-kNejVpn-Dt4Z6iX5ebnxffm6ubbz8uvlw1hreqa8BIMWrbdxzGSXQwtVIIZbQZVa9gslpIW6tgY8c6zghjva39SE67qeW0bfkp-rjm7mP4XSDlYeeSAe_1DKGkgRPWKyp7oir98B-9CyXWxh6VoIoLplhVn1ZlYkgpwjTso9vpuAyUDI8zDXWm4Wmmat8fEsu4A_tP_t2lgvMV3DsPy_NJw-Z6s0Y-AK6fnkQ</recordid><startdate>202406</startdate><enddate>202406</enddate><creator>Schiava, Marianela</creator><creator>Lofra, Robert Muni</creator><creator>Bourke, John P.</creator><creator>Díaz‐Manera, Jordi</creator><creator>James, Meredith K.</creator><creator>Elseed, Maha A.</creator><creator>Malinova, Monika</creator><creator>Michel‐Sodhi, Jassi</creator><creator>Moat, Dionne</creator><creator>Ghimenton, Elisabetta</creator><creator>Mccallum, Michelle</creator><creator>Díaz, Carla Florencia Bolaño</creator><creator>Mayhew, Anna</creator><creator>Wong, Karen</creator><creator>Richardson, Mark</creator><creator>Tasca, Giorgio</creator><creator>Eglon, Gail</creator><creator>Eagle, Michelle</creator><creator>Turner, Cathy</creator><creator>Heslop, Emma</creator><creator>Straub, Volker</creator><creator>Bettolo, Chiara Marini</creator><creator>Guglieri, Michela</creator><general>John Wiley & Sons, Inc</general><scope>24P</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>7TK</scope><scope>7U7</scope><scope>C1K</scope><scope>K9.</scope><scope>7X8</scope><orcidid>https://orcid.org/0000-0001-6674-6970</orcidid><orcidid>https://orcid.org/0000-0002-2709-265X</orcidid><orcidid>https://orcid.org/0000-0002-8455-0637</orcidid><orcidid>https://orcid.org/0000-0003-2941-7988</orcidid></search><sort><creationdate>202406</creationdate><title>Functional abilities, respiratory and cardiac function in a large cohort of adults with Duchenne muscular dystrophy treated with glucocorticoids</title><author>Schiava, Marianela ; Lofra, Robert Muni ; Bourke, John P. ; Díaz‐Manera, Jordi ; James, Meredith K. ; Elseed, Maha A. ; Malinova, Monika ; Michel‐Sodhi, Jassi ; Moat, Dionne ; Ghimenton, Elisabetta ; Mccallum, Michelle ; Díaz, Carla Florencia Bolaño ; Mayhew, Anna ; Wong, Karen ; Richardson, Mark ; Tasca, Giorgio ; Eglon, Gail ; Eagle, Michelle ; Turner, Cathy ; Heslop, Emma ; Straub, Volker ; Bettolo, Chiara Marini ; Guglieri, Michela</author></sort><facets><frbrtype>5</frbrtype><frbrgroupid>cdi_FETCH-LOGICAL-c3487-ec65bad973ebf57ef46558cacb898efda56d1462b727320229d2676317f431443</frbrgroupid><rsrctype>articles</rsrctype><prefilter>articles</prefilter><language>eng</language><creationdate>2024</creationdate><topic>Adolescent</topic><topic>Adult</topic><topic>Adults</topic><topic>Cardiac function</topic><topic>cardiac function in adults with DMD</topic><topic>Cohort Studies</topic><topic>Cough</topic><topic>Duchenne's muscular dystrophy</topic><topic>Dystrophy</topic><topic>Ejection fraction</topic><topic>EK scale in adults with DMD</topic><topic>Female</topic><topic>glucocorticoid dose in adults with DMD</topic><topic>Glucocorticoids</topic><topic>Glucocorticoids - therapeutic use</topic><topic>Heart</topic><topic>Heart - drug effects</topic><topic>Heart - physiopathology</topic><topic>Humans</topic><topic>Male</topic><topic>Mobility Limitation</topic><topic>Muscular dystrophy</topic><topic>Muscular Dystrophy, Duchenne - drug therapy</topic><topic>Muscular Dystrophy, Duchenne - physiopathology</topic><topic>Prednisone</topic><topic>Prednisone - therapeutic use</topic><topic>Pregnenediones - therapeutic use</topic><topic>Regression analysis</topic><topic>respiratory function in adults with DMD</topic><topic>Retrospective Studies</topic><topic>Risk reduction</topic><topic>Statistical models</topic><topic>transition to adulthood in DMD</topic><topic>Ventricle</topic><topic>Wheelchairs</topic><topic>Young Adult</topic><toplevel>peer_reviewed</toplevel><toplevel>online_resources</toplevel><creatorcontrib>Schiava, Marianela</creatorcontrib><creatorcontrib>Lofra, Robert Muni</creatorcontrib><creatorcontrib>Bourke, John P.</creatorcontrib><creatorcontrib>Díaz‐Manera, Jordi</creatorcontrib><creatorcontrib>James, Meredith K.</creatorcontrib><creatorcontrib>Elseed, Maha A.</creatorcontrib><creatorcontrib>Malinova, Monika</creatorcontrib><creatorcontrib>Michel‐Sodhi, Jassi</creatorcontrib><creatorcontrib>Moat, Dionne</creatorcontrib><creatorcontrib>Ghimenton, Elisabetta</creatorcontrib><creatorcontrib>Mccallum, Michelle</creatorcontrib><creatorcontrib>Díaz, Carla Florencia Bolaño</creatorcontrib><creatorcontrib>Mayhew, Anna</creatorcontrib><creatorcontrib>Wong, Karen</creatorcontrib><creatorcontrib>Richardson, Mark</creatorcontrib><creatorcontrib>Tasca, Giorgio</creatorcontrib><creatorcontrib>Eglon, Gail</creatorcontrib><creatorcontrib>Eagle, Michelle</creatorcontrib><creatorcontrib>Turner, Cathy</creatorcontrib><creatorcontrib>Heslop, Emma</creatorcontrib><creatorcontrib>Straub, Volker</creatorcontrib><creatorcontrib>Bettolo, Chiara Marini</creatorcontrib><creatorcontrib>Guglieri, Michela</creatorcontrib><collection>Wiley Online Library 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>Neurosciences Abstracts</collection><collection>Toxicology Abstracts</collection><collection>Environmental Sciences and Pollution Management</collection><collection>ProQuest Health & Medical Complete (Alumni)</collection><collection>MEDLINE - Academic</collection><jtitle>European journal of neurology</jtitle></facets><delivery><delcategory>Remote Search Resource</delcategory><fulltext>fulltext</fulltext></delivery><addata><au>Schiava, Marianela</au><au>Lofra, Robert Muni</au><au>Bourke, John P.</au><au>Díaz‐Manera, Jordi</au><au>James, Meredith K.</au><au>Elseed, Maha A.</au><au>Malinova, Monika</au><au>Michel‐Sodhi, Jassi</au><au>Moat, Dionne</au><au>Ghimenton, Elisabetta</au><au>Mccallum, Michelle</au><au>Díaz, Carla Florencia Bolaño</au><au>Mayhew, Anna</au><au>Wong, Karen</au><au>Richardson, Mark</au><au>Tasca, Giorgio</au><au>Eglon, Gail</au><au>Eagle, Michelle</au><au>Turner, Cathy</au><au>Heslop, Emma</au><au>Straub, Volker</au><au>Bettolo, Chiara Marini</au><au>Guglieri, Michela</au><format>journal</format><genre>article</genre><ristype>JOUR</ristype><atitle>Functional abilities, respiratory and cardiac function in a large cohort of adults with Duchenne muscular dystrophy treated with glucocorticoids</atitle><jtitle>European journal of neurology</jtitle><addtitle>Eur J Neurol</addtitle><date>2024-06</date><risdate>2024</risdate><volume>31</volume><issue>6</issue><spage>e16267</spage><epage>n/a</epage><pages>e16267-n/a</pages><issn>1351-5101</issn><issn>1468-1331</issn><eissn>1468-1331</eissn><abstract><![CDATA[Background and purpose
The transition to adult services, and subsequent glucocorticoid management, is critical in adults with Duchenne muscular dystrophy. This study aims (1) to describe treatment, functional abilities, respiratory and cardiac status during transition to adulthood and adult stages; and (2) to explore the association between glucocorticoid treatment after loss of ambulation (LOA) and late‐stage clinical outcomes.
Methods
This was a retrospective single‐centre study on individuals with Duchenne muscular dystrophy (≥16 years old) between 1986 and 2022. Logistic regression, Cox proportional hazards models and survival analyses were conducted utilizing data from clinical records.
Results
In all, 112 individuals were included. Mean age was 23.4 ± 5.2 years and mean follow‐up was 18.5 ± 5.5 years. At last assessment, 47.2% were on glucocorticoids; the mean dose of prednisone was 0.38 ± 0.13 mg/kg/day and of deflazacort 0.43 ± 0.16 mg/kg/day. At age 16 years, motor function limitations included using a manual wheelchair (89.7%), standing (87.9%), transferring from a wheelchair (86.2%) and turning in bed (53.4%); 77.5% had a peak cough flow <270 L/min, 53.3% a forced vital capacity percentage of predicted <50% and 40.3% a left ventricular ejection fraction <50%. Glucocorticoids after LOA reduced the risk and delayed the time to difficulties balancing in the wheelchair, loss of hand to mouth function, forced vital capacity percentage of predicted <30% and forced vital capacity <1 L and were associated with lower frequency of left ventricular ejection fraction <50%, without differences between prednisone and deflazacort. Glucocorticoid dose did not differ by functional, respiratory or cardiac status.
Conclusion
Glucocorticoids after LOA preserve late‐stage functional abilities, respiratory and cardiac function. It is suggested using functional abilities, respiratory and cardiac status at transition stages for adult services planning.]]></abstract><cop>England</cop><pub>John Wiley & Sons, Inc</pub><pmid>38556893</pmid><doi>10.1111/ene.16267</doi><tpages>17</tpages><orcidid>https://orcid.org/0000-0001-6674-6970</orcidid><orcidid>https://orcid.org/0000-0002-2709-265X</orcidid><orcidid>https://orcid.org/0000-0002-8455-0637</orcidid><orcidid>https://orcid.org/0000-0003-2941-7988</orcidid><oa>free_for_read</oa></addata></record> |
| fulltext | fulltext |
| identifier | ISSN: 1351-5101 |
| ispartof | European journal of neurology, 2024-06, Vol.31 (6), p.e16267-n/a |
| issn | 1351-5101 1468-1331 1468-1331 |
| language | eng |
| recordid | cdi_proquest_miscellaneous_3029816908 |
| source | MEDLINE; Wiley Online Library Open Access; Wiley Online Library Journals Frontfile Complete; PubMed Central |
| subjects | Adolescent Adult Adults Cardiac function cardiac function in adults with DMD Cohort Studies Cough Duchenne's muscular dystrophy Dystrophy Ejection fraction EK scale in adults with DMD Female glucocorticoid dose in adults with DMD Glucocorticoids Glucocorticoids - therapeutic use Heart Heart - drug effects Heart - physiopathology Humans Male Mobility Limitation Muscular dystrophy Muscular Dystrophy, Duchenne - drug therapy Muscular Dystrophy, Duchenne - physiopathology Prednisone Prednisone - therapeutic use Pregnenediones - therapeutic use Regression analysis respiratory function in adults with DMD Retrospective Studies Risk reduction Statistical models transition to adulthood in DMD Ventricle Wheelchairs Young Adult |
| title | Functional abilities, respiratory and cardiac function in a large cohort of adults with Duchenne muscular dystrophy treated with glucocorticoids |
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