OBJECTIVES: To study the responsiveness (sensitivity to change) of the Motor Function Measure (MFM) in detecting change in neuromuscular disease patients with the intent of using this measure in future clinical trials. DESIGN: Prospective cohort observational study. SETTING: Inpatient and outpatient facilities for follow-up and treatment of neuromuscular diseases. PARTICIPANTS: Patients (N=152) with various neuromuscular diseases aged 6 to 60 years. INTERVENTIONS: Not applicable. MAIN OUTCOME MEASURE(S): We used the MFM total score and its 3 subscores on 2 measurements grossly 1 year apart. The physicians and the patients (or proxy) were asked to provide their perceived change in functional status since the first MFM. These changes were expressed in 3 outcomes: deterioration, stability, or improvement. RESULTS: The overall 12-month-standardized mean change of the total score mean ± SD annual total score change was -2.4±5.5 points (P<.001), with patients with Duchenne muscular dystrophy (DMD) presenting the most significant change (-5.8±6.3, P<.001). The change in patients reporting deterioration (34%) was significantly larger than that of those reporting stability (47%) or improvement (10%) (-4.4±6.4 vs -2.0±5.6 and +0.9±4.4 points, respectively, P<.01). The 12-month-standardized total score changes were significantly greater in physician-rated deteriorated (49%) versus stable patients (51%), with mean differences in scores being -5.3±7.6 and -1.2±5.3, respectively (P<.001). CONCLUSIONS: The MFM showed a good responsiveness, especially in patients with DMD and agreements with patients' and physicians' perceived change. Confirming this responsiveness requires larger age groups of patients with DMD and other neuromuscular diseases as well as disease-specific interexamination delays.
OBJECTIVES: To study the responsiveness (sensitivity to change) of the Motor Function Measure (MFM) in detecting change in neuromuscular diseasepatients with the intent of using this measure in future clinical trials. DESIGN: Prospective cohort observational study. SETTING: Inpatient and outpatient facilities for follow-up and treatment of neuromuscular diseases. PARTICIPANTS: Patients (N=152) with various neuromuscular diseases aged 6 to 60 years. INTERVENTIONS: Not applicable. MAIN OUTCOME MEASURE(S): We used the MFM total score and its 3 subscores on 2 measurements grossly 1 year apart. The physicians and the patients (or proxy) were asked to provide their perceived change in functional status since the first MFM. These changes were expressed in 3 outcomes: deterioration, stability, or improvement. RESULTS: The overall 12-month-standardized mean change of the total score mean ± SD annual total score change was -2.4±5.5 points (P<.001), with patients with Duchenne muscular dystrophy (DMD) presenting the most significant change (-5.8±6.3, P<.001). The change in patients reporting deterioration (34%) was significantly larger than that of those reporting stability (47%) or improvement (10%) (-4.4±6.4 vs -2.0±5.6 and +0.9±4.4 points, respectively, P<.01). The 12-month-standardized total score changes were significantly greater in physician-rated deteriorated (49%) versus stable patients (51%), with mean differences in scores being -5.3±7.6 and -1.2±5.3, respectively (P<.001). CONCLUSIONS: The MFM showed a good responsiveness, especially in patients with DMD and agreements with patients' and physicians' perceived change. Confirming this responsiveness requires larger age groups of patients with DMD and other neuromuscular diseases as well as disease-specific interexamination delays.
Authors: Lilian A Y Fernandes; Fátima A Caromano; Silvana M B Assis; Michele E Hukuda; Mariana C Voos; Eduardo V Carvalho Journal: Braz J Phys Ther Date: 2014 Nov-Dec Impact factor: 3.377
Authors: Katherine G Meilleur; Minal S Jain; Linda S Hynan; Ching-Yi Shieh; Eunice Kim; Melissa Waite; Michelle McGuire; Courtney Fiorini; Allan M Glanzman; Marion Main; Kristy Rose; Tina Duong; Roxanna Bendixen; Melody M Linton; Irene C Arveson; Carmel Nichols; Kelly Yang; Kenneth H Fischbeck; Kathryn R Wagner; Kathryn North; Ami Mankodi; Christopher Grunseich; Elizabeth J Hartnett; Michaele Smith; Sandra Donkervoort; Alice Schindler; Angela Kokkinis; Meganne Leach; A Reghan Foley; James Collins; Francesco Muntoni; Anne Rutkowski; Carsten G Bönnemann Journal: Neuromuscul Disord Date: 2014-09-28 Impact factor: 4.296
Authors: Sandra Donkervoort; Payam Mohassel; Lucia Laugwitz; Maha S Zaki; Erik-Jan Kamsteeg; Reza Maroofian; Katherine R Chao; Corien C Verschuuren-Bemelmans; Veronka Horber; Annemarie J M Fock; Riley M McCarty; Minal S Jain; Victoria Biancavilla; Grace McMacken; Matthew Nalls; Nicol C Voermans; Hasnaa M Elbendary; Molly Snyder; Chunyu Cai; Tanya J Lehky; Valentina Stanley; Susan T Iannaccone; A Reghan Foley; Hanns Lochmüller; Joseph Gleeson; Henry Houlden; Tobias B Haack; Rita Horvath; Carsten G Bönnemann Journal: Am J Med Genet A Date: 2020-08-10 Impact factor: 2.578
Authors: Silvia Regina Pinheiro Malheiros; Talita Dias da Silva; Francis Meire Favero; Luiz Carlos de Abreu; Felipe Fregni; Denise Cardoso Ribeiro; Carlos Bandeira de Mello Monteiro Journal: Neuropsychiatr Dis Treat Date: 2015-12-30 Impact factor: 2.570