Quality of Life in SMA Patients Under Treatment With Nusinersen
Background: Spinal Muscular Atrophy (SMA) is a severe neurodegenerative disease, characterized by progressive muscle weakness and atrophy. The approval of the antisense oligonucleotide (ASO) nusinersen now provides an effective pharmacological approach with the potential to slow down or stop disease...
| Main Authors: | , , , , , , , , |
|---|---|
| Format: | Article |
| Language: | English |
| Published: |
Frontiers Media S.A.
2021-03-01
|
| Series: | Frontiers in Neurology |
| Subjects: | |
| Online Access: | https://www.frontiersin.org/articles/10.3389/fneur.2021.626787/full |
| _version_ | 1818870319221309440 |
|---|---|
| author | Lucas Mix Benedikt Winter Claudia D. Wurster Sophia Platen Simon Witzel Zeljko Uzelac Heiko Graf Albert C. Ludolph Albert C. Ludolph Dorothée Lulé |
| author_facet | Lucas Mix Benedikt Winter Claudia D. Wurster Sophia Platen Simon Witzel Zeljko Uzelac Heiko Graf Albert C. Ludolph Albert C. Ludolph Dorothée Lulé |
| author_sort | Lucas Mix |
| collection | DOAJ |
| description | Background: Spinal Muscular Atrophy (SMA) is a severe neurodegenerative disease, characterized by progressive muscle weakness and atrophy. The approval of the antisense oligonucleotide (ASO) nusinersen now provides an effective pharmacological approach with the potential to slow down or stop disease progression with a potentially major impact on patients' well-being.Objective: This study evaluates quality of life (QoL) in pediatric and adult patients over the course of therapy with nusinersen.Methods: Twenty-six SMA patients treated with nusinersen were evaluated regarding global QoL (gQoL), health-related QoL (HRQoL) and depressiveness. Assessments were conducted three times over the first 6 months of treatment. Applied were different questionnaires: the Anamnestic Comparative Self-Assessment (ACSA) for gQoL, the Short Form-36 Health Survey (SF-36) for HRQoL in adult patients and the ALS Depression Inventory 12 Items (ADI-12) for depressiveness. The sample was matched with 22 healthy controls.Results: Despite severe physical restrictions, patients reported high levels of QoL and low levels of depressiveness at study entry. Early disease onset and low levels of physical functioning were associated with better gQoL and lower levels of depressiveness. A significant decrease of gQoL in patients was evident over the course of the study. Still, adult patients reported a significant increase in perceived health.Conclusions: Our study provides first insight that SMA patients experience a gQoL superior to healthy controls at start of therapy. This might indicate patients' high hopes and expectations toward treatment. gQoL returns to a level similar to that of healthy controls over the course of therapy. |
| first_indexed | 2024-12-19T12:05:08Z |
| format | Article |
| id | doaj.art-6039dca1d1cf457a98b054a201d41637 |
| institution | Directory Open Access Journal |
| issn | 1664-2295 |
| language | English |
| last_indexed | 2024-12-19T12:05:08Z |
| publishDate | 2021-03-01 |
| publisher | Frontiers Media S.A. |
| record_format | Article |
| series | Frontiers in Neurology |
| spelling | doaj.art-6039dca1d1cf457a98b054a201d416372022-12-21T20:22:22ZengFrontiers Media S.A.Frontiers in Neurology1664-22952021-03-011210.3389/fneur.2021.626787626787Quality of Life in SMA Patients Under Treatment With NusinersenLucas Mix0Benedikt Winter1Claudia D. Wurster2Sophia Platen3Simon Witzel4Zeljko Uzelac5Heiko Graf6Albert C. Ludolph7Albert C. Ludolph8Dorothée Lulé9Department of Neurology, Ulm University, Ulm, GermanyDepartment of Paediatrics, Ulm University, Ulm, GermanyDepartment of Neurology, Ulm University, Ulm, GermanyDepartment of Neurology, Ulm University, Ulm, GermanyDepartment of Neurology, Ulm University, Ulm, GermanyDepartment of Neurology, Ulm University, Ulm, GermanyDepartment of Psychiatry and Psychotherapy III, Ulm University, Ulm, GermanyDepartment of Neurology, Ulm University, Ulm, GermanyGerman Center for Neurodegenerative Diseases Ulm, Ulm, GermanyDepartment of Neurology, Ulm University, Ulm, GermanyBackground: Spinal Muscular Atrophy (SMA) is a severe neurodegenerative disease, characterized by progressive muscle weakness and atrophy. The approval of the antisense oligonucleotide (ASO) nusinersen now provides an effective pharmacological approach with the potential to slow down or stop disease progression with a potentially major impact on patients' well-being.Objective: This study evaluates quality of life (QoL) in pediatric and adult patients over the course of therapy with nusinersen.Methods: Twenty-six SMA patients treated with nusinersen were evaluated regarding global QoL (gQoL), health-related QoL (HRQoL) and depressiveness. Assessments were conducted three times over the first 6 months of treatment. Applied were different questionnaires: the Anamnestic Comparative Self-Assessment (ACSA) for gQoL, the Short Form-36 Health Survey (SF-36) for HRQoL in adult patients and the ALS Depression Inventory 12 Items (ADI-12) for depressiveness. The sample was matched with 22 healthy controls.Results: Despite severe physical restrictions, patients reported high levels of QoL and low levels of depressiveness at study entry. Early disease onset and low levels of physical functioning were associated with better gQoL and lower levels of depressiveness. A significant decrease of gQoL in patients was evident over the course of the study. Still, adult patients reported a significant increase in perceived health.Conclusions: Our study provides first insight that SMA patients experience a gQoL superior to healthy controls at start of therapy. This might indicate patients' high hopes and expectations toward treatment. gQoL returns to a level similar to that of healthy controls over the course of therapy.https://www.frontiersin.org/articles/10.3389/fneur.2021.626787/fullpatient reported outcomespinal muscular atrophyantisense oligonucleotidenusinersen (Spinraza)quality of lifedepressiveness |
| spellingShingle | Lucas Mix Benedikt Winter Claudia D. Wurster Sophia Platen Simon Witzel Zeljko Uzelac Heiko Graf Albert C. Ludolph Albert C. Ludolph Dorothée Lulé Quality of Life in SMA Patients Under Treatment With Nusinersen Frontiers in Neurology patient reported outcome spinal muscular atrophy antisense oligonucleotide nusinersen (Spinraza) quality of life depressiveness |
| title | Quality of Life in SMA Patients Under Treatment With Nusinersen |
| title_full | Quality of Life in SMA Patients Under Treatment With Nusinersen |
| title_fullStr | Quality of Life in SMA Patients Under Treatment With Nusinersen |
| title_full_unstemmed | Quality of Life in SMA Patients Under Treatment With Nusinersen |
| title_short | Quality of Life in SMA Patients Under Treatment With Nusinersen |
| title_sort | quality of life in sma patients under treatment with nusinersen |
| topic | patient reported outcome spinal muscular atrophy antisense oligonucleotide nusinersen (Spinraza) quality of life depressiveness |
| url | https://www.frontiersin.org/articles/10.3389/fneur.2021.626787/full |
| work_keys_str_mv | AT lucasmix qualityoflifeinsmapatientsundertreatmentwithnusinersen AT benediktwinter qualityoflifeinsmapatientsundertreatmentwithnusinersen AT claudiadwurster qualityoflifeinsmapatientsundertreatmentwithnusinersen AT sophiaplaten qualityoflifeinsmapatientsundertreatmentwithnusinersen AT simonwitzel qualityoflifeinsmapatientsundertreatmentwithnusinersen AT zeljkouzelac qualityoflifeinsmapatientsundertreatmentwithnusinersen AT heikograf qualityoflifeinsmapatientsundertreatmentwithnusinersen AT albertcludolph qualityoflifeinsmapatientsundertreatmentwithnusinersen AT albertcludolph qualityoflifeinsmapatientsundertreatmentwithnusinersen AT dorotheelule qualityoflifeinsmapatientsundertreatmentwithnusinersen |