Clinical neurology and neurosurgery · 2026
Feasibility and usefulness of personalised patient-reported outcome measures in the therapeutic follow-up of adult spinal muscular atrophy patients.
СтатьяМногоцентровое исследованиеНазвание и аннотация: Перевод в очереди
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Cintas Pascal, Pouplin Samuel, Debergé Louise, Laforêt Pascal, Solé Guilhem, Fabry Vincent, Acket Blandine, Letelier Alfredo, Pigeon Corentin, Petit Noémie, Nicolas Guillaume, Masurier Morgane, Caire Jean-Michel
Аннотация
BACKGROUND: Spinal muscular atrophy (SMA) is a rare neuromuscular disorder characterized by progressive muscle weakness and atrophy. In adult patients, treatment goals are to stabilize the disease or gain motor skills. However, functional scales commonly used to assess treatment response, such as the 32-item Motor Function Scale (MFM32) are less sensitive in severely affected adult patients due to floor effect. The study's objective was to evaluate personalised patient reported outcome (PRO), centred on patient expectations, that combines two patient-centred tools widely used in occupational therapy: the Canadian Occupational Performance Measure (COPM) and Global Attainment Scale (GAS).
METHODS: A multicenter, retrospective cohort study of patients treated for SMA was conducted. Collected data included patients' characteristics, treatments received, and outcome evaluations with MFM32, COPM and GAS.
RESULTS: Thirty-two patients with type 1, 2, or 3 SMA were included. At treatment initiation, mean MFM32 total score was 37.1 ± 24.3. COPM identified patient's expectations were primarily centred on the domain of mobility and self-care. After a mean treatment duration of 14.1 ± 4.3 months, a clinically significant improvement was reported for five (15.6%) patients with MFM32. Eleven (34.4%) patients showed improvement on performance and 10 (31.2%) on satisfaction score in their most significant patient-identified problem areas in daily function as assessed by the COPM. Twenty (62.5%) patients achieved at least one of their therapeutic goals measured with the GAS. A positive correlation was found between change in satisfaction score and GAS (r = 0.521, p = 0.003).
CONCLUSION: The combined COPM/GAS approach is a personalised fit-for-purpose instrument able to detect improvements, meaningfully significant to the patient, which are not always captured by usual functional scales.
METHODS: A multicenter, retrospective cohort study of patients treated for SMA was conducted. Collected data included patients' characteristics, treatments received, and outcome evaluations with MFM32, COPM and GAS.
RESULTS: Thirty-two patients with type 1, 2, or 3 SMA were included. At treatment initiation, mean MFM32 total score was 37.1 ± 24.3. COPM identified patient's expectations were primarily centred on the domain of mobility and self-care. After a mean treatment duration of 14.1 ± 4.3 months, a clinically significant improvement was reported for five (15.6%) patients with MFM32. Eleven (34.4%) patients showed improvement on performance and 10 (31.2%) on satisfaction score in their most significant patient-identified problem areas in daily function as assessed by the COPM. Twenty (62.5%) patients achieved at least one of their therapeutic goals measured with the GAS. A positive correlation was found between change in satisfaction score and GAS (r = 0.521, p = 0.003).
CONCLUSION: The combined COPM/GAS approach is a personalised fit-for-purpose instrument able to detect improvements, meaningfully significant to the patient, which are not always captured by usual functional scales.