PMID- 33166523 OWN - NLM STAT- MEDLINE DCOM- 20210419 LR - 20230725 IS - 1532-821X (Electronic) IS - 0003-9993 (Print) IS - 0003-9993 (Linking) VI - 102 IP - 4 DP - 2021 Apr TI - Responsiveness and Minimal Clinically Important Difference of the Motor Function Measure in Collagen VI-Related Dystrophies and Laminin Alpha2-Related Muscular Dystrophy. PG - 604-610 LID - S0003-9993(20)31214-4 [pii] LID - 10.1016/j.apmr.2020.10.116 [doi] AB - OBJECTIVES: To investigate the responsiveness of the motor function measure (MFM) and determine the minimal clinically important difference (MCID) in individuals with 2 common types of congenital muscular dystrophy (CMD). DESIGN: Observational, prospective, single center, cohort study. SETTING: National Institute of Neurological Disorders and Stroke (NINDS) of the National Institutes of Health (NIH). PARTICIPANTS: Individuals (N=44) with collagen VI-related dystrophies (COL6-RD, n=23) and 21 individuals laminin alpha2-related muscular dystrophy (LAMA2-RD, n=21) enrolled in a 4-year longitudinal natural history study. INTERVENTIONS: Not applicable. MAIN OUTCOME MEASURES: Responsiveness of the MFM-32 and the Rasch-scaled MFM-25 and the MCID of the MFM-32 determined from a patient-reported anchor with 2 different methods, within-patient and between-patient. RESULTS: The original MFM-32 and Rasch-scaled MFM-25 performed similarly overall in both the COL6-RD and LAMA2-RD populations, with all subscores (D1, standing and transfers; D2, axial and proximal; D3, distal) showing a significant decrease over time, except MFM D1 and D3 for LAMA2-RD. The MFM D1 subscore was the most sensitive to change for ambulant individuals, whereas the MFM D2 subscore was the most sensitive to change for nonambulant individuals. The MCID for the MFM-32 total score was calculated as 2.5 and 3.9 percentage points according to 2 different methods. CONCLUSIONS: The MFM showed strong responsiveness in individuals with LAMA2-RD and COL6-RD. Because a floor effect was identified more prominently with the Rasch-Scaled MFM-25, the use of the original MFM-32 as a quantitative variable with the assumption of scale linearity appears to be a good compromise. When designing clinical trials in congenital muscular dystrophies, the use of MCID for MFM should be considered to determine if a given intervention effects show not only a statistically significant change but also a clinically meaningful change. CI - Copyright (c) 2020 American Congress of Rehabilitation Medicine. All rights reserved. FAU - Le Goff, Laure AU - Le Goff L AD - Department of Pediatric Physical Medicine and Rehabilitation, Hospices Civils de Lyon, Bron, France. Electronic address: laure.le-goff@chu-lyon.fr. FAU - Meilleur, Katherine G AU - Meilleur KG AD - National Institute of Nursing Research, National Institutes of Health, Bethesda, MD; Neuromuscular and Neurogenetic Disorders of Childhood Section, National Institute of Neurological Disorders and Stroke, National Institutes of Health, Bethesda, MD. FAU - Norato, Gina AU - Norato G AD - Office of Biostatistics, National Institute of Neurological Disorders and Stroke, National Institutes of Health, Bethesda, MD. FAU - Rippert, Pascal AU - Rippert P AD - Public Health Center, Research and Clinical Epidemiology Department, Hospices Civils de Lyon, Lyon, France. FAU - Jain, Minal AU - Jain M AD - Mark O. Hatfield Clinical Research Center, National Institutes of Health, Bethesda, MD. FAU - Fink, Margaret AU - Fink M AD - Neuromuscular and Neurogenetic Disorders of Childhood Section, National Institute of Neurological Disorders and Stroke, National Institutes of Health, Bethesda, MD. FAU - Foley, A Reghan AU - Foley AR AD - Neuromuscular and Neurogenetic Disorders of Childhood Section, National Institute of Neurological Disorders and Stroke, National Institutes of Health, Bethesda, MD. FAU - Waite, Melissa AU - Waite M AD - Mark O. Hatfield Clinical Research Center, National Institutes of Health, Bethesda, MD. FAU - Donkervoort, Sandra AU - Donkervoort S AD - Neuromuscular and Neurogenetic Disorders of Childhood Section, National Institute of Neurological Disorders and Stroke, National Institutes of Health, Bethesda, MD. FAU - Bonnemann, Carsten G AU - Bonnemann CG AD - Neuromuscular and Neurogenetic Disorders of Childhood Section, National Institute of Neurological Disorders and Stroke, National Institutes of Health, Bethesda, MD. FAU - Vuillerot, Carole AU - Vuillerot C AD - Department of Pediatric Physical Medicine and Rehabilitation, Hospices Civils de Lyon, Bron, France; Neuromuscular and Neurogenetic Disorders of Childhood Section, National Institute of Neurological Disorders and Stroke, National Institutes of Health, Bethesda, MD; Neuromyogen Institute, CNRS UMR 5310-INSERM, University of Lyon, Lyon, France; University of Lyon 1, F-69100, Villeurbanne, France. LA - eng GR - Z99 CL999999/ImNIH/Intramural NIH HHS/United States PT - Journal Article PT - Observational Study PT - Research Support, N.I.H., Extramural DEP - 20201106 PL - United States TA - Arch Phys Med Rehabil JT - Archives of physical medicine and rehabilitation JID - 2985158R SB - IM MH - Adolescent MH - Adult MH - Child MH - Child, Preschool MH - Cohort Studies MH - *Disability Evaluation MH - Female MH - Humans MH - Longitudinal Studies MH - Male MH - *Minimal Clinically Important Difference MH - Motor Activity/*physiology MH - Muscular Dystrophies/*physiopathology/*rehabilitation MH - Prospective Studies MH - Young Adult PMC - PMC10363856 MID - NIHMS1865249 OTO - NOTNLM OT - Disability evaluation OT - Muscular dystrophies OT - Rehabilitation EDAT- 2020/11/10 06:00 MHDA- 2021/04/20 06:00 PMCR- 2023/07/24 CRDT- 2020/11/09 20:12 PHST- 2020/06/23 00:00 [received] PHST- 2020/09/16 00:00 [revised] PHST- 2020/10/01 00:00 [accepted] PHST- 2020/11/10 06:00 [pubmed] PHST- 2021/04/20 06:00 [medline] PHST- 2020/11/09 20:12 [entrez] PHST- 2023/07/24 00:00 [pmc-release] AID - S0003-9993(20)31214-4 [pii] AID - 10.1016/j.apmr.2020.10.116 [doi] PST - ppublish SO - Arch Phys Med Rehabil. 2021 Apr;102(4):604-610. doi: 10.1016/j.apmr.2020.10.116. Epub 2020 Nov 6.