التفاصيل البيبلوغرافية
| العنوان: |
Use of MFM-20 to monitor SMA types 1 and 2 patients treated with nusinersen. |
| المؤلفون: |
Le Goff, Laure, Seferian, Andreea, Phelep, Aurelie, Rippert, Pascal, Mathieu, Marie-Laure, Cances, Claude, de Lattre, Capucine, Durigneux, Julien, Gousse, Gaelle, Vincent-Genod, Dominique, Ribault, Shams, Gomez Garcia de la Banda, Marta, Quijano-Roy, Susana, Sarret, Catherine, Servais, Laurent, Vuillerot, Carole |
| المصدر: |
Neurological Sciences; Jan2023, Vol. 44 Issue 1, p329-337, 9p, 2 Charts, 3 Graphs |
| مصطلحات موضوعية: |
SPINAL muscular atrophy, TEST validity |
| مستخلص: |
Objective: To evaluate sensitivity to change and discriminant validity of the 20-item Motor Function Measure (MFM-20) in 2–7-year-old patients with spinal muscular atrophy types 1 (SMA1) or 2 (SMA2) treated with nusinersen. Methods: Children aged 2 to 7 years old with SMA1 or SMA2 treated with nusinersen were assessed at least three times using the MFM-20 over an average follow-up time of 17 months. Evolution of 4-month-standardized MFM-20 scores was calculated for each MFM-20 domain (D1 standing and transfers, D2 axial and proximal, D3 distal) and for the total score (TS). Results: Included in the study were 22 SMA1 subjects and 19 SMA2 subjects. Baseline MFM scores were significantly lower in patients with SMA1 than SMA2 (TS 29.5% vs. 48.3%, D1 4.5% vs. 10.6%, D2 43.6% vs. 72.6%, D3 51.2% vs. 75.0%). When considering the mean change during nusinersen treatment, standardized over a 4-month period, TS was improved for both SMA1 (+ 4.1%, SRM 1.5) and SMA2 (+ 2.8%, SRM 0.89) patients. For SMA1 patients, considerable changes were observed in D2 (+ 6.2%, SRM 0.89) and D3 (+ 6.0%, SRM 0.72), whereas the change in D1 was small (+ 0.5%, SRM 0.44). In SMA2 2 subjects, D3 was improved to a larger extent (+ 4.2%, SRM 0.53) than D1 (+ 1.8% SRM 0.63) or D2 (+ 3.2%, SRM 0.69). Conclusion: Our results validate use of MFM-20 to monitor function of young SMA1 and SMA2 subjects treated with nusinersen. Significant motor function improvements following treatment were observed in both SMA1 and SMA2 patients. [ABSTRACT FROM AUTHOR] |
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| قاعدة البيانات: |
Complementary Index |