Outcomes Measure Reliability in Non Ambulatory Boys and Men with Duchenne Muscular Dystrophy (DMD): Results from the Muscular Dystrophy Association DMD Clinical Research Network (P04.085)

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Abstract

Objective: To establish optimal and reliable assessments in non-ambulatory boys and men with DMD. Background Therapeutic trials in DMD most often exclude non-ambulatory individuals and consensus has not been reached regarding which measures are most reliable and feasible. Design/Methods: Clinical evaluators(CEs) from the five MDA-DMD CRN sites were trained in all assessments. Ninety-three non-ambulatory boys/men with DMD who were not on continuous ventilation (mean age 16.8±3.6 years) were assessed twice(AM/PM) on one day with a battery of assessments. Reliability using intra-class correlation coefficients(ICCs) was determined by comparing AM and PM measures. Feasibility was determined by the percentage that could perform the task. Results: Forced Vital Capacity(FVC)(100% of subjects) showed mean FVC 47±23% predicted with an ICC of 0.97. Manual muscle testing was performed for shoulder abduction (88% of subjects; ICC=0.95), elbow flexion (96.8% of subjects; ICC=0.98), wrist flexion (70% of subjects; ICC=0.92) and wrist extension (96% of subjects; ICC=0.89). Hand Held myometrywas performed for supine elbow flexion (71% of subjects; ICC=0.93), supine elbow extension (70% of subjects; ICC=0.94), grip (97% of subject; 0.92), pinch grip(95% of subjects; ICC=0.84), and key grip (99% of subjects; ICC=0.93). Upper extremity active range of motion(ROM) ICCs ranged from 0.89-0.99 and passive ROM ranged from 0.71 to 0.97. Nine-hole peg was performed in 77% of subjects with ICC ranging from 0.96-0.97 for dominant and non-dominant hands. Brooke upper extremity scale was performed on 100% of subjects with an ICC=0.99. Egen Klassifikation(EK) scale was performed in 100% of subjects with ICCs on individual questions ranging from 0.93 to 0.99. Jebsen-Taylor Hand Function test was performed in 84% of subjects with ICCs of individual tasks ranging from 0.64 to 0.98. Conclusions: This study demonstrates excellent reliability across most measures with well-trained CEs from five centers. Ongoing studies will test sensitivity to change across time. Supported by: Muscular Dystrophy Association. Disclosure: Dr. Florence has received personal compensation for activities with GlaxoSmithKline, Inc./Prosena and DART Therapeutics as a consultant. Dr. Connolly has received personal compensation for activities with Halo Therapeutics. Dr. Connolly has received research support from PTC Therapeutics. Dr. Miller has nothing to disclose. Dr. Malkus has nothing to disclose. Dr. Schierbecker has nothing to disclose. Dr. Siener has nothing to disclose. Dr. Wulf has nothing to disclose. Dr. Anand has nothing to disclose. Dr. McDonald has received personal compensation for activities with AVI BioPharma, and PTC Therapeutics. Dr. Goude has nothing to disclose. Dr. Johnson has nothing to disclose. Dr. Nicorici has nothing to disclose. Dr. Day has nothing to disclose. Dr. Karachunski has nothing to disclose. Dr. Dalton has nothing to disclose. Dr. Kelecic has nothing to disclose. Dr. Paulson has nothing to disclose. Dr. Naughton has nothing to disclose. Dr. Lowes has nothing to disclose. Dr. Alfano has nothing to disclose. Dr. Viollet has nothing to disclose. Dr. Flanigan has received personal compensaiton for activities with AVI Therapeutics, Prosensa Therapeutics, and PTC, Inc. Dr. Flanigan has received research support from PTC Therapeutics. Dr. Mendell has received research support from AVI BioPharmaceuticals Inc. Dr. Darras has received personal compensation for activities with UpToDate, Inc., Isis Pharmaceuticals, Inc, and Athena Diagnostics as a consultant.Dr. Darras has received research support from PTC Therapeutics, Inc. Dr. Quigley has nothing to disclose. Dr. Pasternak has nothing to disclose. Dr. Shriber has nothing to disclose. Dr. Parad has nothing to disclose. Dr. Cwik has nothing to disclose.

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