Outcome reliability in non-Ambulatory Boys/Men with duchenne muscular dystrophy

MDA DMD Clinical Research Network

doi:10.1002/mus.24346

Outcome reliability in non-Ambulatory Boys/Men with duchenne muscular dystrophy

MDA DMD Clinical Research Network

Research output: Contribution to journal › Article › peer-review

62 Scopus citations

Abstract

Introduction: Therapeutic trials in Duchenne muscular dystrophy (DMD) often exclude non-ambulatory individuals. Here we establish optimal and reliable assessments in a multicenter trial. Methods: Non-ambulatory boys/men with DMD (N=91; 16.7±4.5 years of age) were assessed by trained clinical evaluators. Feasibility (percentage completing task) and reliability [intraclass correlation coefficients (ICCs) between morning and afternoon tests] were measured. Results: Forced vital capacity (FVC), assessed in all subjects, showed a mean of 47.8±22% predicted (ICC 0.98). Brooke Upper Extremity Functional Rating (Brooke) and Egen Klassifikation (EK) scales in 100% of subjects showed ICCs ranging from 0.93 to 0.99. Manual muscle testing, range of motion, 9-hole peg test, and Jebsen-Taylor Hand Function Test (JHFT) demonstrated varied feasibility (99% to 70%), with ICCs ranging from 0.99 to 0.64. We found beneficial effects of different forms of corticosteroids for the Brooke scale, percent predicted FVC, and hand and finger strength. Conclusions: Reliable assessment of non-ambulatory boys/men with DMD is possible. Clinical trials will have to consider corticosteroid use.

Original language	English
Pages (from-to)	522-532
Number of pages	11
Journal	Muscle and Nerve
Volume	51
Issue number	4
DOIs	https://doi.org/10.1002/mus.24346
State	Published - Apr 1 2015

Keywords

Corticosteroids
Duchenne muscular dystrophy
Non-ambulatory
Pulmonary function
Quality of life
Strength

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10.1002/mus.24346

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title = "Outcome reliability in non-Ambulatory Boys/Men with duchenne muscular dystrophy",

abstract = "Introduction: Therapeutic trials in Duchenne muscular dystrophy (DMD) often exclude non-ambulatory individuals. Here we establish optimal and reliable assessments in a multicenter trial. Methods: Non-ambulatory boys/men with DMD (N=91; 16.7±4.5 years of age) were assessed by trained clinical evaluators. Feasibility (percentage completing task) and reliability [intraclass correlation coefficients (ICCs) between morning and afternoon tests] were measured. Results: Forced vital capacity (FVC), assessed in all subjects, showed a mean of 47.8±22% predicted (ICC 0.98). Brooke Upper Extremity Functional Rating (Brooke) and Egen Klassifikation (EK) scales in 100% of subjects showed ICCs ranging from 0.93 to 0.99. Manual muscle testing, range of motion, 9-hole peg test, and Jebsen-Taylor Hand Function Test (JHFT) demonstrated varied feasibility (99% to 70%), with ICCs ranging from 0.99 to 0.64. We found beneficial effects of different forms of corticosteroids for the Brooke scale, percent predicted FVC, and hand and finger strength. Conclusions: Reliable assessment of non-ambulatory boys/men with DMD is possible. Clinical trials will have to consider corticosteroid use.",

keywords = "Corticosteroids, Duchenne muscular dystrophy, Non-ambulatory, Pulmonary function, Quality of life, Strength",

author = "{MDA DMD Clinical Research Network} and Connolly, {Anne M.} and Malkus, {Elizabeth C.} and Mendell, {Jerry R.} and Flanigan, {Kevin M.} and Miller, {J. Philip} and Schierbecker, {Jeanine R.} and Siener, {Catherine A.} and Golumbek, {Paul T.} and Zaidman, {Craig M.} and Mcdonald, {Craig M.} and Linda Johnson and Alina Nicorici and Karachunski, {Peter I.} and Day, {John W.} and Kelecic, {Jason M.} and Lowes, {Linda P.} and Alfano, {Lindsay N.} and Darras, {Basil T.} and Kang, {Peter B.} and Janet Quigley and Pasternak, {Amy E.} and Florence, {Julaine M.} and Pallavi Anand and Wulf, {Charlie O.} and Erica Goude and Dalton, {Joline C.} and Laurence Viollet-Callendret and Buser, {Karen K.} and Elizabeth Shriber and Rebecca Parad",

note = "Publisher Copyright: {\textcopyright} 2014 Wiley Periodicals, Inc..",

year = "2015",

month = apr,

day = "1",

doi = "10.1002/mus.24346",

language = "English",

volume = "51",

pages = "522--532",

journal = "Muscle and Nerve",

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T1 - Outcome reliability in non-Ambulatory Boys/Men with duchenne muscular dystrophy

AU - MDA DMD Clinical Research Network

AU - Connolly, Anne M.

AU - Malkus, Elizabeth C.

AU - Mendell, Jerry R.

AU - Flanigan, Kevin M.

AU - Miller, J. Philip

AU - Schierbecker, Jeanine R.

AU - Siener, Catherine A.

AU - Golumbek, Paul T.

AU - Zaidman, Craig M.

AU - Mcdonald, Craig M.

AU - Johnson, Linda

AU - Nicorici, Alina

AU - Karachunski, Peter I.

AU - Day, John W.

AU - Kelecic, Jason M.

AU - Lowes, Linda P.

AU - Alfano, Lindsay N.

AU - Darras, Basil T.

AU - Kang, Peter B.

AU - Quigley, Janet

AU - Pasternak, Amy E.

AU - Florence, Julaine M.

AU - Anand, Pallavi

AU - Wulf, Charlie O.

AU - Goude, Erica

AU - Dalton, Joline C.

AU - Viollet-Callendret, Laurence

AU - Buser, Karen K.

AU - Shriber, Elizabeth

AU - Parad, Rebecca

PY - 2015/4/1

Y1 - 2015/4/1

N2 - Introduction: Therapeutic trials in Duchenne muscular dystrophy (DMD) often exclude non-ambulatory individuals. Here we establish optimal and reliable assessments in a multicenter trial. Methods: Non-ambulatory boys/men with DMD (N=91; 16.7±4.5 years of age) were assessed by trained clinical evaluators. Feasibility (percentage completing task) and reliability [intraclass correlation coefficients (ICCs) between morning and afternoon tests] were measured. Results: Forced vital capacity (FVC), assessed in all subjects, showed a mean of 47.8±22% predicted (ICC 0.98). Brooke Upper Extremity Functional Rating (Brooke) and Egen Klassifikation (EK) scales in 100% of subjects showed ICCs ranging from 0.93 to 0.99. Manual muscle testing, range of motion, 9-hole peg test, and Jebsen-Taylor Hand Function Test (JHFT) demonstrated varied feasibility (99% to 70%), with ICCs ranging from 0.99 to 0.64. We found beneficial effects of different forms of corticosteroids for the Brooke scale, percent predicted FVC, and hand and finger strength. Conclusions: Reliable assessment of non-ambulatory boys/men with DMD is possible. Clinical trials will have to consider corticosteroid use.

AB - Introduction: Therapeutic trials in Duchenne muscular dystrophy (DMD) often exclude non-ambulatory individuals. Here we establish optimal and reliable assessments in a multicenter trial. Methods: Non-ambulatory boys/men with DMD (N=91; 16.7±4.5 years of age) were assessed by trained clinical evaluators. Feasibility (percentage completing task) and reliability [intraclass correlation coefficients (ICCs) between morning and afternoon tests] were measured. Results: Forced vital capacity (FVC), assessed in all subjects, showed a mean of 47.8±22% predicted (ICC 0.98). Brooke Upper Extremity Functional Rating (Brooke) and Egen Klassifikation (EK) scales in 100% of subjects showed ICCs ranging from 0.93 to 0.99. Manual muscle testing, range of motion, 9-hole peg test, and Jebsen-Taylor Hand Function Test (JHFT) demonstrated varied feasibility (99% to 70%), with ICCs ranging from 0.99 to 0.64. We found beneficial effects of different forms of corticosteroids for the Brooke scale, percent predicted FVC, and hand and finger strength. Conclusions: Reliable assessment of non-ambulatory boys/men with DMD is possible. Clinical trials will have to consider corticosteroid use.

KW - Corticosteroids

KW - Duchenne muscular dystrophy

KW - Non-ambulatory

KW - Pulmonary function

KW - Quality of life

KW - Strength

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U2 - 10.1002/mus.24346

DO - 10.1002/mus.24346

M3 - Article

C2 - 25056178

AN - SCOPUS:84925350947

SN - 0148-639X

VL - 51

SP - 522

EP - 532

JO - Muscle and Nerve

JF - Muscle and Nerve

IS - 4

ER -

Outcome reliability in non-Ambulatory Boys/Men with duchenne muscular dystrophy

Abstract

Keywords

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