دورية أكاديمية
Quality of life assessment in adult spinal muscular atrophy patients treated with nusinersen
| العنوان: | Quality of life assessment in adult spinal muscular atrophy patients treated with nusinersen |
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| المؤلفون: | Silvia Bonanno, Riccardo Zanin, Luca Bello, Irene Tramacere, Virginia Bozzoni, Luca Caumo, Manfredi Ferraro, Sara Bortolani, Gianni Sorarù, Mauro Silvestrini, Veria Vacchiano, Mara Turri, Raffaella Tanel, Rocco Liguori, Michela Coccia, Renato Emilio Mantegazza, Tiziana Mongini, Elena Pegoraro, Lorenzo Maggi |
| المساهمون: | Silvia Bonanno, Riccardo Zanin, Luca Bello, Irene Tramacere, Virginia Bozzoni, Luca Caumo, Manfredi Ferraro, Sara Bortolani, Gianni Sorarù, Mauro Silvestrini, Veria Vacchiano, Mara Turri, Raffaella Tanel, Rocco Liguori, Michela Coccia, Renato Emilio Mantegazza, Tiziana Mongini, Elena Pegoraro, Lorenzo Maggi |
| سنة النشر: | 2022 |
| المجموعة: | IRIS Università degli Studi di Bologna (CRIS - Current Research Information System) |
| مصطلحات موضوعية: | Adult, INQoL, Nusinersen, Quality of life, SMA |
| الوصف: | Objective: To retrospectively evaluate quality of life (QoL) in a large multicenter cohort of adult patients affected by spinal muscular atrophy (SMA) during nusinersen treatment. Methods: We included adult (≥ 18years) patients clinically and genetically defined as SMA2, SMA3 and SMA4, who started nusinersen treatment in adulthood. QoL was rated by the Individualized Neuromuscular Quality of Life (INQoL) questionnaire. Concurrent motor function evaluation included the Hammersmith Functional Motor Scale Expanded (HFMSE), the Revised Upper Limb Module (RULM), the 6min walking test (6MWT). Results: 189 completed questionnaires were collected during a 14months’ treatment period. 78 patients were included (7 SMA2 and 69 SMA3 and 2 SMA4) with mean disease duration at first nusinersen administration of 33.2years (± 12.5years). All the scores for each INQoL domain (weakness, fatigue, activities, independence, social relationship, emotions, body images) and the derived QoL total score, significantly improved during the observation period, except the muscle locking and pain items. Exploratory analyses suggested that emotions and social relationships were more relevant issues for females compared to males. Social relationships were affected also by a longer disease duration (> 30years). In SMA3 non-walker patients, activities ameliorate better compared to walkers. The HFMSE and RULM significantly improved from baseline; however, no associations with QoL total score and weakness, activities or independence were demonstrated. Conclusion: In our cohort, adult SMA patients showed a global improvement at the INQoL assessment over 14months of nusinersen treatment. QoL assessment is relevant to SMA multidisciplinary evaluation. |
| نوع الوثيقة: | article in journal/newspaper |
| وصف الملف: | STAMPA |
| اللغة: | English |
| العلاقة: | info:eu-repo/semantics/altIdentifier/pmid/34978620; info:eu-repo/semantics/altIdentifier/wos/WOS:000737691300001; volume:269; issue:6; firstpage:3264; lastpage:3275; numberofpages:12; journal:JOURNAL OF NEUROLOGY; http://hdl.handle.net/11585/897519Test; info:eu-repo/semantics/altIdentifier/scopus/2-s2.0-85122244123 |
| DOI: | 10.1007/s00415-021-10954-3 |
| الإتاحة: | https://doi.org/10.1007/s00415-021-10954-3Test http://hdl.handle.net/11585/897519Test |
| رقم الانضمام: | edsbas.A79F3F07 |
| قاعدة البيانات: | BASE |
| DOI: | 10.1007/s00415-021-10954-3 |
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