Corticosteroids in Duchenne muscular dystrophy: impact on the motor function measure sensitivity to change and implications for clinical trials
Aim To monitor the evolution of the motor function of ambulatory patients with Duchenne muscular dystrophy (DMD) treated by corticosteroids for 2 years in comparison with untreated patients. Method This observational, multicentre cohort study explores the evolution of the motor function measure (MFM...
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| Veröffentlicht in: | Developmental medicine and child neurology 2018-02, Vol.60 (2), p.185-191 |
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| creator | Schreiber, Audrey Brochard, Sylvain Rippert, Pascal Fontaine‐Carbonnel, Stephanie Payan, Christine Poirot, Isabelle Hamroun, Dalil Vuillerot, Carole Peudenier, Sylviane Ropars, Juliette Urtizberea, Andoni Tiffreau, Vincent Puyhaubert, Blandine Fournier Mehouas, Manuella Sacconi, Sabrina Lagrue, Emmanuelle Chabrier, Stéphane |
| description | Aim
To monitor the evolution of the motor function of ambulatory patients with Duchenne muscular dystrophy (DMD) treated by corticosteroids for 2 years in comparison with untreated patients.
Method
This observational, multicentre cohort study explores the evolution of the motor function measure (MFM) over a 24‐month period for 29 ambulant corticosteroids‐treated and 45 ambulant untreated patients with DMD.
Results
Significant differences were found between mean MFM scores in corticosteroids‐treated and untreated groups for domain 1 of the MFM (standing position and transfers; D1), domain 2 of the MFM (axial and proximal motor function; D2), and domain 3 of the MFM (distal motor function; D3). Subscores were between 0 months and 6 months, and 0 months and 24 months. For the D1 subscore specifically, there was a significant increase in the corticosteroids‐treated group (mean±standard deviation [SD] slope of change=12.6±15.5%/y), while a decrease was observed in the untreated group (−17.8±17.7%/y) between 0 months and 6 months (p |
| doi_str_mv | 10.1111/dmcn.13590 |
| format | Article |
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To monitor the evolution of the motor function of ambulatory patients with Duchenne muscular dystrophy (DMD) treated by corticosteroids for 2 years in comparison with untreated patients.
Method
This observational, multicentre cohort study explores the evolution of the motor function measure (MFM) over a 24‐month period for 29 ambulant corticosteroids‐treated and 45 ambulant untreated patients with DMD.
Results
Significant differences were found between mean MFM scores in corticosteroids‐treated and untreated groups for domain 1 of the MFM (standing position and transfers; D1), domain 2 of the MFM (axial and proximal motor function; D2), and domain 3 of the MFM (distal motor function; D3). Subscores were between 0 months and 6 months, and 0 months and 24 months. For the D1 subscore specifically, there was a significant increase in the corticosteroids‐treated group (mean±standard deviation [SD] slope of change=12.6±15.5%/y), while a decrease was observed in the untreated group (−17.8±17.7%/y) between 0 months and 6 months (p<0.001). Sensitivity to change as assessed by standardized response means was high between 12 months and 24 months for D1 of both corticosteroids‐treated and untreated groups (1.0 and 1.2 respectively), and low for D2 and D3 of both treated and untreated groups.
Interpretation
Patients with DMD treated by corticosteroids present a different course of the disease as assessed by MFM, confirming the sensitivity to change of the MFM in this population.
What this paper adds
Corticosteroids have a quantitative impact on muscle strength 6 to 24 months after starting treatment.
Motor function measure is a valid outcome measure in Duchenne muscular dystrophy patients under corticosteroid treatment.
Resumen
Corticoides en distrofia muscular de Duchenne: su impacto en la sensibilidad al cambio de la medición de la función motora e implicancias para los ensayos clínicos
Objetivo
monitorear la evolución de la función motora en pacientes ambulatorios con distrofia muscular de Duchenne (DMD) tratada mediante el uso de corticoides a diferencia de pacientes no tratados.
Método
este estudio observacional, multi‐céntrico de cohorte, estudia la evolución de la medición de la función motora (MFM) durante un periodo de 24 meses en 29 pacientes ambulatorios con DMD tratados con corticoides y 45 pacientes con DMD ambulatorios no tratados.
Resultados
se reflejaron diferencias significativas en los promedios de MFM en grupos tratados y no tratados para el dominio 1 de MFM (bipedestación y transferencia, D1) y dominio 2 de MFM (función motora proximal y de eje, D2) y dominio 3 de MFM (función motora distal, D3). Las escalas de evaluación se realizaron entre los 0 y 6 meses, y entre los 0 y 24 meses. Para la escala especifica D1, hubo un incremento significativo en el grupo de niños tratado (promedio +/‐ desviación estándar de la pendiente del cambio = 12.6 +/‐ 15.5%/ años), mientras que en el grupo de niños no tratado se observo una disminución del (‐17.8 +/‐ 17.7%/y) entre 0 y 6 meses (p<0.001). La sensibilidad del cambio en MFM fue analizada mediante respuesta media estandarizada (SRM), sus resultados fueron: elevada entre 12 y 24 meses para D1 tanto para pacientes tratados como no tratados y baja para D2 y D3 en ambos grupos.
Interpretación
pacientes con DMD tratados con corticoides presentan un curso diferente de la enfermedad evaluado por MFM, confirmado la sensibilidad al cambio del MFM en esta población.
Resumo
Corticoesteróides em distrofia muscular de Duchenne: impacto na função motora, sensibilidade a mudanças e implicações para estudos clínicos
Objetivo
Monitorar a avaliação da função motora de pacientes com distrofia muscular de Duchenne (DMD) tratados com corticoesteróides por 2 anos em comparação com pacientes não tratados.
Método
Este estudo de coorte observacional multicêntrico explora a evolução da Medida da Função Motora (MFM) em um período de 24 meses para 20 pacientes com DMD ambuladores tratados com corticoesteróides e 45 pacientes com DMD ambuladores não tratados.
Resultados
Diferenças significativas foram encontradas entre os escores MFM médios nos grupos tratado e não tratado para o domínio 1 da MFM (posição em pé e transferências; D1), domínio 2 da MFM (função motora axial e proximal; D2), e domínio 3 da MFM (função motora distal; D3). Os sub‐escores estiveram entre 0 e 6 e 0 e 24 meses. Para o sub‐escore D1 especificamente, houve aumento significativo no grupo tratado com corticoesteróides (média ±desvio‐padrão [SD]; curva de mudança= 12,6 ±15,5%/y), enquanto houve redução no grupo não tratado (‐17,8±17,7%/y) entre 0 e 6 meses (p<0,001). A sensibilidade a mudanças avaliada pela media de resposta padronizada (MRP) foi alta entre 12 e 24 meses para D1 tanto para o grupo tratado quanto para o não tratado com corticoesteróides (1,0 e 1,2, respectivamente), e baixa para D2 e D2 para o grupo tratado e para o não tratado.
Interpretação
Pacientes com DMD tratados com corticoesteróides apresentam curso diferente da doença, como avaliado pela MFM, confirmando a sensibilidade a mudanças da MFM para esta população.
What this paper adds
Corticosteroids have a quantitative impact on muscle strength 6 to 24 months after starting treatment.
Motor function measure is a valid outcome measure in Duchenne muscular dystrophy patients under corticosteroid treatment.
This article is commented on by Domingos and Muntoni on page 117 of this issue.
This article's has been translated into Spanish and Portuguese.
Follow the links from the to view the translations.
Video Podcast: https://www.youtube.com/watch?v=qNmTSgJiJco</description><identifier>ISSN: 0012-1622</identifier><identifier>ISSN: 1469-8749</identifier><identifier>EISSN: 1469-8749</identifier><identifier>DOI: 10.1111/dmcn.13590</identifier><identifier>PMID: 28990163</identifier><language>eng</language><publisher>England: Wiley online library</publisher><subject>Adrenal Cortex Hormones - therapeutic use ; Child ; Cohort Studies ; Disability Evaluation ; Female ; Humans ; Life Sciences ; Male ; Movement Disorders - drug therapy ; Movement Disorders - etiology ; Muscle Strength - drug effects ; Muscular Dystrophy, Duchenne - complications ; Muscular Dystrophy, Duchenne - drug therapy ; Sensitivity and Specificity ; Severity of Illness Index ; Statistics, Nonparametric ; Time Factors</subject><ispartof>Developmental medicine and child neurology, 2018-02, Vol.60 (2), p.185-191</ispartof><rights>2017 Mac Keith Press</rights><rights>2017 Mac Keith Press.</rights><rights>Distributed under a Creative Commons Attribution 4.0 International License</rights><lds50>peer_reviewed</lds50><oa>free_for_read</oa><woscitedreferencessubscribed>false</woscitedreferencessubscribed><citedby>FETCH-LOGICAL-c4250-77c102121beec36e4657e7bfe17f41f59ba4572f6be46338e182122493905a7b3</citedby><cites>FETCH-LOGICAL-c4250-77c102121beec36e4657e7bfe17f41f59ba4572f6be46338e182122493905a7b3</cites><orcidid>0000-0003-4995-0202</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%2Fdmcn.13590$$EPDF$$P50$$Gwiley$$H</linktopdf><linktohtml>$$Uhttps://onlinelibrary.wiley.com/doi/full/10.1111%2Fdmcn.13590$$EHTML$$P50$$Gwiley$$H</linktohtml><link.rule.ids>230,314,780,784,885,1416,1432,27922,27923,45572,45573,46407,46831</link.rule.ids><backlink>$$Uhttps://www.ncbi.nlm.nih.gov/pubmed/28990163$$D View this record in MEDLINE/PubMed$$Hfree_for_read</backlink><backlink>$$Uhttps://hal.univ-lille.fr/hal-04477185$$DView record in HAL$$Hfree_for_read</backlink></links><search><creatorcontrib>Schreiber, Audrey</creatorcontrib><creatorcontrib>Brochard, Sylvain</creatorcontrib><creatorcontrib>Rippert, Pascal</creatorcontrib><creatorcontrib>Fontaine‐Carbonnel, Stephanie</creatorcontrib><creatorcontrib>Payan, Christine</creatorcontrib><creatorcontrib>Poirot, Isabelle</creatorcontrib><creatorcontrib>Hamroun, Dalil</creatorcontrib><creatorcontrib>Vuillerot, Carole</creatorcontrib><creatorcontrib>Peudenier, Sylviane</creatorcontrib><creatorcontrib>Ropars, Juliette</creatorcontrib><creatorcontrib>Urtizberea, Andoni</creatorcontrib><creatorcontrib>Tiffreau, Vincent</creatorcontrib><creatorcontrib>Puyhaubert, Blandine</creatorcontrib><creatorcontrib>Fournier Mehouas, Manuella</creatorcontrib><creatorcontrib>Sacconi, Sabrina</creatorcontrib><creatorcontrib>Lagrue, Emmanuelle</creatorcontrib><creatorcontrib>Chabrier, Stéphane</creatorcontrib><creatorcontrib>MFM DMD Corticosteroids Group</creatorcontrib><creatorcontrib>MFM DMD Corticosteroids Group</creatorcontrib><title>Corticosteroids in Duchenne muscular dystrophy: impact on the motor function measure sensitivity to change and implications for clinical trials</title><title>Developmental medicine and child neurology</title><addtitle>Dev Med Child Neurol</addtitle><description>Aim
To monitor the evolution of the motor function of ambulatory patients with Duchenne muscular dystrophy (DMD) treated by corticosteroids for 2 years in comparison with untreated patients.
Method
This observational, multicentre cohort study explores the evolution of the motor function measure (MFM) over a 24‐month period for 29 ambulant corticosteroids‐treated and 45 ambulant untreated patients with DMD.
Results
Significant differences were found between mean MFM scores in corticosteroids‐treated and untreated groups for domain 1 of the MFM (standing position and transfers; D1), domain 2 of the MFM (axial and proximal motor function; D2), and domain 3 of the MFM (distal motor function; D3). Subscores were between 0 months and 6 months, and 0 months and 24 months. For the D1 subscore specifically, there was a significant increase in the corticosteroids‐treated group (mean±standard deviation [SD] slope of change=12.6±15.5%/y), while a decrease was observed in the untreated group (−17.8±17.7%/y) between 0 months and 6 months (p<0.001). Sensitivity to change as assessed by standardized response means was high between 12 months and 24 months for D1 of both corticosteroids‐treated and untreated groups (1.0 and 1.2 respectively), and low for D2 and D3 of both treated and untreated groups.
Interpretation
Patients with DMD treated by corticosteroids present a different course of the disease as assessed by MFM, confirming the sensitivity to change of the MFM in this population.
What this paper adds
Corticosteroids have a quantitative impact on muscle strength 6 to 24 months after starting treatment.
Motor function measure is a valid outcome measure in Duchenne muscular dystrophy patients under corticosteroid treatment.
Resumen
Corticoides en distrofia muscular de Duchenne: su impacto en la sensibilidad al cambio de la medición de la función motora e implicancias para los ensayos clínicos
Objetivo
monitorear la evolución de la función motora en pacientes ambulatorios con distrofia muscular de Duchenne (DMD) tratada mediante el uso de corticoides a diferencia de pacientes no tratados.
Método
este estudio observacional, multi‐céntrico de cohorte, estudia la evolución de la medición de la función motora (MFM) durante un periodo de 24 meses en 29 pacientes ambulatorios con DMD tratados con corticoides y 45 pacientes con DMD ambulatorios no tratados.
Resultados
se reflejaron diferencias significativas en los promedios de MFM en grupos tratados y no tratados para el dominio 1 de MFM (bipedestación y transferencia, D1) y dominio 2 de MFM (función motora proximal y de eje, D2) y dominio 3 de MFM (función motora distal, D3). Las escalas de evaluación se realizaron entre los 0 y 6 meses, y entre los 0 y 24 meses. Para la escala especifica D1, hubo un incremento significativo en el grupo de niños tratado (promedio +/‐ desviación estándar de la pendiente del cambio = 12.6 +/‐ 15.5%/ años), mientras que en el grupo de niños no tratado se observo una disminución del (‐17.8 +/‐ 17.7%/y) entre 0 y 6 meses (p<0.001). La sensibilidad del cambio en MFM fue analizada mediante respuesta media estandarizada (SRM), sus resultados fueron: elevada entre 12 y 24 meses para D1 tanto para pacientes tratados como no tratados y baja para D2 y D3 en ambos grupos.
Interpretación
pacientes con DMD tratados con corticoides presentan un curso diferente de la enfermedad evaluado por MFM, confirmado la sensibilidad al cambio del MFM en esta población.
Resumo
Corticoesteróides em distrofia muscular de Duchenne: impacto na função motora, sensibilidade a mudanças e implicações para estudos clínicos
Objetivo
Monitorar a avaliação da função motora de pacientes com distrofia muscular de Duchenne (DMD) tratados com corticoesteróides por 2 anos em comparação com pacientes não tratados.
Método
Este estudo de coorte observacional multicêntrico explora a evolução da Medida da Função Motora (MFM) em um período de 24 meses para 20 pacientes com DMD ambuladores tratados com corticoesteróides e 45 pacientes com DMD ambuladores não tratados.
Resultados
Diferenças significativas foram encontradas entre os escores MFM médios nos grupos tratado e não tratado para o domínio 1 da MFM (posição em pé e transferências; D1), domínio 2 da MFM (função motora axial e proximal; D2), e domínio 3 da MFM (função motora distal; D3). Os sub‐escores estiveram entre 0 e 6 e 0 e 24 meses. Para o sub‐escore D1 especificamente, houve aumento significativo no grupo tratado com corticoesteróides (média ±desvio‐padrão [SD]; curva de mudança= 12,6 ±15,5%/y), enquanto houve redução no grupo não tratado (‐17,8±17,7%/y) entre 0 e 6 meses (p<0,001). A sensibilidade a mudanças avaliada pela media de resposta padronizada (MRP) foi alta entre 12 e 24 meses para D1 tanto para o grupo tratado quanto para o não tratado com corticoesteróides (1,0 e 1,2, respectivamente), e baixa para D2 e D2 para o grupo tratado e para o não tratado.
Interpretação
Pacientes com DMD tratados com corticoesteróides apresentam curso diferente da doença, como avaliado pela MFM, confirmando a sensibilidade a mudanças da MFM para esta população.
What this paper adds
Corticosteroids have a quantitative impact on muscle strength 6 to 24 months after starting treatment.
Motor function measure is a valid outcome measure in Duchenne muscular dystrophy patients under corticosteroid treatment.
This article is commented on by Domingos and Muntoni on page 117 of this issue.
This article's has been translated into Spanish and Portuguese.
Follow the links from the to view the translations.
Video Podcast: https://www.youtube.com/watch?v=qNmTSgJiJco</description><subject>Adrenal Cortex Hormones - therapeutic use</subject><subject>Child</subject><subject>Cohort Studies</subject><subject>Disability Evaluation</subject><subject>Female</subject><subject>Humans</subject><subject>Life Sciences</subject><subject>Male</subject><subject>Movement Disorders - drug therapy</subject><subject>Movement Disorders - etiology</subject><subject>Muscle Strength - drug effects</subject><subject>Muscular Dystrophy, Duchenne - complications</subject><subject>Muscular Dystrophy, Duchenne - drug therapy</subject><subject>Sensitivity and Specificity</subject><subject>Severity of Illness Index</subject><subject>Statistics, Nonparametric</subject><subject>Time Factors</subject><issn>0012-1622</issn><issn>1469-8749</issn><issn>1469-8749</issn><fulltext>true</fulltext><rsrctype>article</rsrctype><creationdate>2018</creationdate><recordtype>article</recordtype><sourceid>EIF</sourceid><recordid>eNp90cFu1DAQBmALgei2cOEBkI9QKa3tOHHMrdq2FGmBC5wtx5kQo8RebKcoT8Er1yGlR3wZafzNf_kRekPJBc3vspuMu6BlJckztKO8lkUjuHyOdoRQVtCasRN0GuNPQkhZV_wlOmGNlITW5Q792fuQrPExQfC2i9g6fD2bAZwDPM3RzKMOuFtiCv44LB-wnY7aJOwdTkMWPvmA-9mZZPNqAh3nADiCizbZe5sWnDw2g3Y_AGvXreejNXrVEff51ozW5cWIU7B6jK_Qiz4PeP04z9D325tv-7vi8PXjp_3VoTCcVaQQwlDCKKMtgClr4HUlQLQ9UNFz2ley1bwSrK_b_FWWDdAma8ZlKUmlRVueofdb7qBHdQx20mFRXlt1d3VQ645wLgRtqnua7bvNHoP_NUNMarLRwDhqB36OikouSVMT0WR6vlETfIwB-qdsStRallrLUn_LyvjtY-7cTtA90X_tZEA38NuOsPwnSl1_3n_ZQh8Al2ehJw</recordid><startdate>201802</startdate><enddate>201802</enddate><creator>Schreiber, Audrey</creator><creator>Brochard, Sylvain</creator><creator>Rippert, Pascal</creator><creator>Fontaine‐Carbonnel, Stephanie</creator><creator>Payan, Christine</creator><creator>Poirot, Isabelle</creator><creator>Hamroun, Dalil</creator><creator>Vuillerot, Carole</creator><creator>Peudenier, Sylviane</creator><creator>Ropars, Juliette</creator><creator>Urtizberea, Andoni</creator><creator>Tiffreau, Vincent</creator><creator>Puyhaubert, Blandine</creator><creator>Fournier Mehouas, Manuella</creator><creator>Sacconi, Sabrina</creator><creator>Lagrue, Emmanuelle</creator><creator>Chabrier, Stéphane</creator><general>Wiley online library</general><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><scope>1XC</scope><scope>VOOES</scope><orcidid>https://orcid.org/0000-0003-4995-0202</orcidid></search><sort><creationdate>201802</creationdate><title>Corticosteroids in Duchenne muscular dystrophy: impact on the motor function measure sensitivity to change and implications for clinical trials</title><author>Schreiber, Audrey ; Brochard, Sylvain ; Rippert, Pascal ; Fontaine‐Carbonnel, Stephanie ; Payan, Christine ; Poirot, Isabelle ; Hamroun, Dalil ; Vuillerot, Carole ; Peudenier, Sylviane ; Ropars, Juliette ; Urtizberea, Andoni ; Tiffreau, Vincent ; Puyhaubert, Blandine ; Fournier Mehouas, Manuella ; Sacconi, Sabrina ; Lagrue, Emmanuelle ; Chabrier, Stéphane</author></sort><facets><frbrtype>5</frbrtype><frbrgroupid>cdi_FETCH-LOGICAL-c4250-77c102121beec36e4657e7bfe17f41f59ba4572f6be46338e182122493905a7b3</frbrgroupid><rsrctype>articles</rsrctype><prefilter>articles</prefilter><language>eng</language><creationdate>2018</creationdate><topic>Adrenal Cortex Hormones - therapeutic use</topic><topic>Child</topic><topic>Cohort Studies</topic><topic>Disability Evaluation</topic><topic>Female</topic><topic>Humans</topic><topic>Life Sciences</topic><topic>Male</topic><topic>Movement Disorders - drug therapy</topic><topic>Movement Disorders - etiology</topic><topic>Muscle Strength - drug effects</topic><topic>Muscular Dystrophy, Duchenne - complications</topic><topic>Muscular Dystrophy, Duchenne - drug therapy</topic><topic>Sensitivity and Specificity</topic><topic>Severity of Illness Index</topic><topic>Statistics, Nonparametric</topic><topic>Time Factors</topic><toplevel>peer_reviewed</toplevel><toplevel>online_resources</toplevel><creatorcontrib>Schreiber, Audrey</creatorcontrib><creatorcontrib>Brochard, Sylvain</creatorcontrib><creatorcontrib>Rippert, Pascal</creatorcontrib><creatorcontrib>Fontaine‐Carbonnel, Stephanie</creatorcontrib><creatorcontrib>Payan, Christine</creatorcontrib><creatorcontrib>Poirot, Isabelle</creatorcontrib><creatorcontrib>Hamroun, Dalil</creatorcontrib><creatorcontrib>Vuillerot, Carole</creatorcontrib><creatorcontrib>Peudenier, Sylviane</creatorcontrib><creatorcontrib>Ropars, Juliette</creatorcontrib><creatorcontrib>Urtizberea, Andoni</creatorcontrib><creatorcontrib>Tiffreau, Vincent</creatorcontrib><creatorcontrib>Puyhaubert, Blandine</creatorcontrib><creatorcontrib>Fournier Mehouas, Manuella</creatorcontrib><creatorcontrib>Sacconi, Sabrina</creatorcontrib><creatorcontrib>Lagrue, Emmanuelle</creatorcontrib><creatorcontrib>Chabrier, Stéphane</creatorcontrib><creatorcontrib>MFM DMD Corticosteroids Group</creatorcontrib><creatorcontrib>MFM DMD Corticosteroids Group</creatorcontrib><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><collection>Hyper Article en Ligne (HAL)</collection><collection>Hyper Article en Ligne (HAL) (Open Access)</collection><jtitle>Developmental medicine and child neurology</jtitle></facets><delivery><delcategory>Remote Search Resource</delcategory><fulltext>fulltext</fulltext></delivery><addata><au>Schreiber, Audrey</au><au>Brochard, Sylvain</au><au>Rippert, Pascal</au><au>Fontaine‐Carbonnel, Stephanie</au><au>Payan, Christine</au><au>Poirot, Isabelle</au><au>Hamroun, Dalil</au><au>Vuillerot, Carole</au><au>Peudenier, Sylviane</au><au>Ropars, Juliette</au><au>Urtizberea, Andoni</au><au>Tiffreau, Vincent</au><au>Puyhaubert, Blandine</au><au>Fournier Mehouas, Manuella</au><au>Sacconi, Sabrina</au><au>Lagrue, Emmanuelle</au><au>Chabrier, Stéphane</au><aucorp>MFM DMD Corticosteroids Group</aucorp><aucorp>MFM DMD Corticosteroids Group</aucorp><format>journal</format><genre>article</genre><ristype>JOUR</ristype><atitle>Corticosteroids in Duchenne muscular dystrophy: impact on the motor function measure sensitivity to change and implications for clinical trials</atitle><jtitle>Developmental medicine and child neurology</jtitle><addtitle>Dev Med Child Neurol</addtitle><date>2018-02</date><risdate>2018</risdate><volume>60</volume><issue>2</issue><spage>185</spage><epage>191</epage><pages>185-191</pages><issn>0012-1622</issn><issn>1469-8749</issn><eissn>1469-8749</eissn><abstract>Aim
To monitor the evolution of the motor function of ambulatory patients with Duchenne muscular dystrophy (DMD) treated by corticosteroids for 2 years in comparison with untreated patients.
Method
This observational, multicentre cohort study explores the evolution of the motor function measure (MFM) over a 24‐month period for 29 ambulant corticosteroids‐treated and 45 ambulant untreated patients with DMD.
Results
Significant differences were found between mean MFM scores in corticosteroids‐treated and untreated groups for domain 1 of the MFM (standing position and transfers; D1), domain 2 of the MFM (axial and proximal motor function; D2), and domain 3 of the MFM (distal motor function; D3). Subscores were between 0 months and 6 months, and 0 months and 24 months. For the D1 subscore specifically, there was a significant increase in the corticosteroids‐treated group (mean±standard deviation [SD] slope of change=12.6±15.5%/y), while a decrease was observed in the untreated group (−17.8±17.7%/y) between 0 months and 6 months (p<0.001). Sensitivity to change as assessed by standardized response means was high between 12 months and 24 months for D1 of both corticosteroids‐treated and untreated groups (1.0 and 1.2 respectively), and low for D2 and D3 of both treated and untreated groups.
Interpretation
Patients with DMD treated by corticosteroids present a different course of the disease as assessed by MFM, confirming the sensitivity to change of the MFM in this population.
What this paper adds
Corticosteroids have a quantitative impact on muscle strength 6 to 24 months after starting treatment.
Motor function measure is a valid outcome measure in Duchenne muscular dystrophy patients under corticosteroid treatment.
Resumen
Corticoides en distrofia muscular de Duchenne: su impacto en la sensibilidad al cambio de la medición de la función motora e implicancias para los ensayos clínicos
Objetivo
monitorear la evolución de la función motora en pacientes ambulatorios con distrofia muscular de Duchenne (DMD) tratada mediante el uso de corticoides a diferencia de pacientes no tratados.
Método
este estudio observacional, multi‐céntrico de cohorte, estudia la evolución de la medición de la función motora (MFM) durante un periodo de 24 meses en 29 pacientes ambulatorios con DMD tratados con corticoides y 45 pacientes con DMD ambulatorios no tratados.
Resultados
se reflejaron diferencias significativas en los promedios de MFM en grupos tratados y no tratados para el dominio 1 de MFM (bipedestación y transferencia, D1) y dominio 2 de MFM (función motora proximal y de eje, D2) y dominio 3 de MFM (función motora distal, D3). Las escalas de evaluación se realizaron entre los 0 y 6 meses, y entre los 0 y 24 meses. Para la escala especifica D1, hubo un incremento significativo en el grupo de niños tratado (promedio +/‐ desviación estándar de la pendiente del cambio = 12.6 +/‐ 15.5%/ años), mientras que en el grupo de niños no tratado se observo una disminución del (‐17.8 +/‐ 17.7%/y) entre 0 y 6 meses (p<0.001). La sensibilidad del cambio en MFM fue analizada mediante respuesta media estandarizada (SRM), sus resultados fueron: elevada entre 12 y 24 meses para D1 tanto para pacientes tratados como no tratados y baja para D2 y D3 en ambos grupos.
Interpretación
pacientes con DMD tratados con corticoides presentan un curso diferente de la enfermedad evaluado por MFM, confirmado la sensibilidad al cambio del MFM en esta población.
Resumo
Corticoesteróides em distrofia muscular de Duchenne: impacto na função motora, sensibilidade a mudanças e implicações para estudos clínicos
Objetivo
Monitorar a avaliação da função motora de pacientes com distrofia muscular de Duchenne (DMD) tratados com corticoesteróides por 2 anos em comparação com pacientes não tratados.
Método
Este estudo de coorte observacional multicêntrico explora a evolução da Medida da Função Motora (MFM) em um período de 24 meses para 20 pacientes com DMD ambuladores tratados com corticoesteróides e 45 pacientes com DMD ambuladores não tratados.
Resultados
Diferenças significativas foram encontradas entre os escores MFM médios nos grupos tratado e não tratado para o domínio 1 da MFM (posição em pé e transferências; D1), domínio 2 da MFM (função motora axial e proximal; D2), e domínio 3 da MFM (função motora distal; D3). Os sub‐escores estiveram entre 0 e 6 e 0 e 24 meses. Para o sub‐escore D1 especificamente, houve aumento significativo no grupo tratado com corticoesteróides (média ±desvio‐padrão [SD]; curva de mudança= 12,6 ±15,5%/y), enquanto houve redução no grupo não tratado (‐17,8±17,7%/y) entre 0 e 6 meses (p<0,001). A sensibilidade a mudanças avaliada pela media de resposta padronizada (MRP) foi alta entre 12 e 24 meses para D1 tanto para o grupo tratado quanto para o não tratado com corticoesteróides (1,0 e 1,2, respectivamente), e baixa para D2 e D2 para o grupo tratado e para o não tratado.
Interpretação
Pacientes com DMD tratados com corticoesteróides apresentam curso diferente da doença, como avaliado pela MFM, confirmando a sensibilidade a mudanças da MFM para esta população.
What this paper adds
Corticosteroids have a quantitative impact on muscle strength 6 to 24 months after starting treatment.
Motor function measure is a valid outcome measure in Duchenne muscular dystrophy patients under corticosteroid treatment.
This article is commented on by Domingos and Muntoni on page 117 of this issue.
This article's has been translated into Spanish and Portuguese.
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Video Podcast: https://www.youtube.com/watch?v=qNmTSgJiJco</abstract><cop>England</cop><pub>Wiley online library</pub><pmid>28990163</pmid><doi>10.1111/dmcn.13590</doi><tpages>7</tpages><orcidid>https://orcid.org/0000-0003-4995-0202</orcidid><oa>free_for_read</oa></addata></record> |
| fulltext | fulltext |
| identifier | ISSN: 0012-1622 |
| ispartof | Developmental medicine and child neurology, 2018-02, Vol.60 (2), p.185-191 |
| issn | 0012-1622 1469-8749 1469-8749 |
| language | eng |
| recordid | cdi_hal_primary_oai_HAL_hal_04477185v1 |
| source | MEDLINE; Wiley Free Content; Wiley Online Library All Journals |
| subjects | Adrenal Cortex Hormones - therapeutic use Child Cohort Studies Disability Evaluation Female Humans Life Sciences Male Movement Disorders - drug therapy Movement Disorders - etiology Muscle Strength - drug effects Muscular Dystrophy, Duchenne - complications Muscular Dystrophy, Duchenne - drug therapy Sensitivity and Specificity Severity of Illness Index Statistics, Nonparametric Time Factors |
| title | Corticosteroids in Duchenne muscular dystrophy: impact on the motor function measure sensitivity to change and implications for clinical trials |
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