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

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

Research output: Contribution to journalArticlepeer-review

52 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 languageEnglish (US)
Pages (from-to)522-532
Number of pages11
JournalMuscle and Nerve
Volume51
Issue number4
DOIs
StatePublished - Apr 1 2015

Bibliographical note

Publisher Copyright:
© 2014 Wiley Periodicals, Inc..

Keywords

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

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