Quality of life in patients with spinal muscular atrophy in Brazil: patient self-assessment and carer perception
ABSTRACT Objectives: The aim of this study was to assess the perception of quality of life of patients with spinal muscular atrophy (SMA) and investigate whether there is a correlation between patients’ perception and that of their carers. Methods: Cross-sectional analytical observational study. I...
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Sociedade de Pediatria de São Paulo
2025-01-01
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| Series: | Revista Paulista de Pediatria |
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| author | Maíra Coelho Marise Bueno Zonta Salmo Raskin Silvia Valderramas |
| author_facet | Maíra Coelho Marise Bueno Zonta Salmo Raskin Silvia Valderramas |
| author_sort | Maíra Coelho |
| collection | DOAJ |
| description | ABSTRACT Objectives: The aim of this study was to assess the perception of quality of life of patients with spinal muscular atrophy (SMA) and investigate whether there is a correlation between patients’ perception and that of their carers. Methods: Cross-sectional analytical observational study. In the first part, socioeconomic, demographic, clinical, and treatment information were collected from patients diagnosed with SMA, regardless of type, sex, or age. SMA type 1 does not sit; SMA type 2 sits; SMA type 3 walks; and SMA type 4 begins in adulthood. In the second part of the study, patients aged between 2 and 25 years and their caregivers responded to the Pediatric Quality of Life Inventory 4.0 questionnaire. Results: Of the 235 families recruited, 167 were eligible to respond to the questionnaire, 115 caregivers and 49 patients were included. The results point to a different perception of quality of life between patients and caregivers. Patients with SMA type 2 perceive more impaired physical and emotional capacity compared to SMA type 3 and 1, respectively. As for caregivers, the perception of quality of life in relation to physical and social capacity and the total score are worse for patients with SMA type 1 compared to other types. Perceptions correlate with emotional capacity in SMA type 2 and the total aspect in SMA type 3. Conclusions: Patients with SMA type 2 had a worse perception of their quality of life than other patients. Perception differed between patients and their caregivers, with the former having a better perception than the latter. |
| format | Article |
| id | doaj-art-08de37d652fe4e588b336d9d2ae3d6be |
| institution | Kabale University |
| issn | 1984-0462 |
| language | English |
| publishDate | 2025-01-01 |
| publisher | Sociedade de Pediatria de São Paulo |
| record_format | Article |
| series | Revista Paulista de Pediatria |
| spelling | doaj-art-08de37d652fe4e588b336d9d2ae3d6be2025-01-21T07:40:52ZengSociedade de Pediatria de São PauloRevista Paulista de Pediatria1984-04622025-01-014310.1590/1984-0462/2025/43/2024073Quality of life in patients with spinal muscular atrophy in Brazil: patient self-assessment and carer perceptionMaíra Coelhohttps://orcid.org/0000-0001-8256-0084Marise Bueno Zontahttps://orcid.org/0000-0003-1652-7723Salmo Raskinhttps://orcid.org/0000-0002-7191-0592Silvia Valderramashttps://orcid.org/0000-0001-7295-2439ABSTRACT Objectives: The aim of this study was to assess the perception of quality of life of patients with spinal muscular atrophy (SMA) and investigate whether there is a correlation between patients’ perception and that of their carers. Methods: Cross-sectional analytical observational study. In the first part, socioeconomic, demographic, clinical, and treatment information were collected from patients diagnosed with SMA, regardless of type, sex, or age. SMA type 1 does not sit; SMA type 2 sits; SMA type 3 walks; and SMA type 4 begins in adulthood. In the second part of the study, patients aged between 2 and 25 years and their caregivers responded to the Pediatric Quality of Life Inventory 4.0 questionnaire. Results: Of the 235 families recruited, 167 were eligible to respond to the questionnaire, 115 caregivers and 49 patients were included. The results point to a different perception of quality of life between patients and caregivers. Patients with SMA type 2 perceive more impaired physical and emotional capacity compared to SMA type 3 and 1, respectively. As for caregivers, the perception of quality of life in relation to physical and social capacity and the total score are worse for patients with SMA type 1 compared to other types. Perceptions correlate with emotional capacity in SMA type 2 and the total aspect in SMA type 3. Conclusions: Patients with SMA type 2 had a worse perception of their quality of life than other patients. Perception differed between patients and their caregivers, with the former having a better perception than the latter.http://www.scielo.br/scielo.php?script=sci_arttext&pid=S0103-05822025000100422&lng=en&tlng=enChild neurologyFamily impactMuscular atrophy spinalPatient-reported outcomes |
| spellingShingle | Maíra Coelho Marise Bueno Zonta Salmo Raskin Silvia Valderramas Quality of life in patients with spinal muscular atrophy in Brazil: patient self-assessment and carer perception Revista Paulista de Pediatria Child neurology Family impact Muscular atrophy spinal Patient-reported outcomes |
| title | Quality of life in patients with spinal muscular atrophy in Brazil: patient self-assessment and carer perception |
| title_full | Quality of life in patients with spinal muscular atrophy in Brazil: patient self-assessment and carer perception |
| title_fullStr | Quality of life in patients with spinal muscular atrophy in Brazil: patient self-assessment and carer perception |
| title_full_unstemmed | Quality of life in patients with spinal muscular atrophy in Brazil: patient self-assessment and carer perception |
| title_short | Quality of life in patients with spinal muscular atrophy in Brazil: patient self-assessment and carer perception |
| title_sort | quality of life in patients with spinal muscular atrophy in brazil patient self assessment and carer perception |
| topic | Child neurology Family impact Muscular atrophy spinal Patient-reported outcomes |
| url | http://www.scielo.br/scielo.php?script=sci_arttext&pid=S0103-05822025000100422&lng=en&tlng=en |
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