2020 Update to Spinal Muscular Atrophy Management in Saudi Arabia
Novel therapeutic strategies have shown some promise in treating spinal muscular atrophy (SMA). However, the outcomes and acceptance of these new strategies are yet to be explored. We aimed to investigate physicians' opinions and perceptions toward management strategies of SMA across Saudi Arab...
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doaj-ae47506a607a4297bd2aef5a626cd2622021-05-31T04:22:54ZengFrontiers Media S.A.Frontiers in Pediatrics2296-23602021-05-01910.3389/fped.2021.6841346841342020 Update to Spinal Muscular Atrophy Management in Saudi ArabiaFahad A. Bashiri0Fahad A. Bashiri1Mohamad-Hani Temsah2Mohamad-Hani Temsah3Khalid Hundallah4Fahad Alsohime5Fahad Alsohime6Yazed AlRuthia7Yazed AlRuthia8College of Medicine, King Saud University, Riyadh, Saudi ArabiaDivision of Pediatric Neurology, Department of Pediatrics, King Saud University Medical City, Riyadh, Saudi ArabiaCollege of Medicine, King Saud University, Riyadh, Saudi ArabiaPediatric Intensive Care Unit, Pediatric Department, King Saud University Medical City, Riyadh, Saudi ArabiaDivision of Neurology, Department of Pediatrics, Prince Sultan Military Medical City, Riyadh, Saudi ArabiaCollege of Medicine, King Saud University, Riyadh, Saudi ArabiaPediatric Intensive Care Unit, Pediatric Department, King Saud University Medical City, Riyadh, Saudi ArabiaDepartment of Clinical Pharmacy, College of Pharmacy, King Saud University, Riyadh, Saudi ArabiaPharmacoeconomics Research Unit, College of Pharmacy, King Saud University, Riyadh, Saudi ArabiaNovel therapeutic strategies have shown some promise in treating spinal muscular atrophy (SMA). However, the outcomes and acceptance of these new strategies are yet to be explored. We aimed to investigate physicians' opinions and perceptions toward management strategies of SMA across Saudi Arabia. This is a cross-sectional survey using a self-administered, structured questionnaire sent to physicians who care for SMA patients during the Saudi Pediatric Neurology Society annual conference. A total of 72 clinicians of different neurological subspecialties were included. 48.6% prescribed nusinersen to their patients, with 39% of them having patients started on nusinersen. Though, 8.3% prescribed onasemnogene abeparvovec for 1–3 patients, while none of their patients started on the treatment. 64.3% stated that the only treatment available for SMA in their settings is supportive care. Around 69.4% described having a moderate to high knowledge on SMA gene therapy, and 79.2% would recommend it. 48.6% confirmed they would prescribe gene therapy at the age of 6 months, and 78.3% would prescribe it for type-I SMA. Pediatric neurologists are receptive to novel and innovative therapies for SMA in Saudi Arabia. However, the high treatment acquisition cost, strict regulations, logistical issues, and budget constraints delay their adoption and implementation.https://www.frontiersin.org/articles/10.3389/fped.2021.684134/fullspinal muscular atrophygene therapynusinersenonasemnogene abeparvovecrisdiplamSaudi Arabia |
collection |
DOAJ |
language |
English |
format |
Article |
sources |
DOAJ |
author |
Fahad A. Bashiri Fahad A. Bashiri Mohamad-Hani Temsah Mohamad-Hani Temsah Khalid Hundallah Fahad Alsohime Fahad Alsohime Yazed AlRuthia Yazed AlRuthia |
spellingShingle |
Fahad A. Bashiri Fahad A. Bashiri Mohamad-Hani Temsah Mohamad-Hani Temsah Khalid Hundallah Fahad Alsohime Fahad Alsohime Yazed AlRuthia Yazed AlRuthia 2020 Update to Spinal Muscular Atrophy Management in Saudi Arabia Frontiers in Pediatrics spinal muscular atrophy gene therapy nusinersen onasemnogene abeparvovec risdiplam Saudi Arabia |
author_facet |
Fahad A. Bashiri Fahad A. Bashiri Mohamad-Hani Temsah Mohamad-Hani Temsah Khalid Hundallah Fahad Alsohime Fahad Alsohime Yazed AlRuthia Yazed AlRuthia |
author_sort |
Fahad A. Bashiri |
title |
2020 Update to Spinal Muscular Atrophy Management in Saudi Arabia |
title_short |
2020 Update to Spinal Muscular Atrophy Management in Saudi Arabia |
title_full |
2020 Update to Spinal Muscular Atrophy Management in Saudi Arabia |
title_fullStr |
2020 Update to Spinal Muscular Atrophy Management in Saudi Arabia |
title_full_unstemmed |
2020 Update to Spinal Muscular Atrophy Management in Saudi Arabia |
title_sort |
2020 update to spinal muscular atrophy management in saudi arabia |
publisher |
Frontiers Media S.A. |
series |
Frontiers in Pediatrics |
issn |
2296-2360 |
publishDate |
2021-05-01 |
description |
Novel therapeutic strategies have shown some promise in treating spinal muscular atrophy (SMA). However, the outcomes and acceptance of these new strategies are yet to be explored. We aimed to investigate physicians' opinions and perceptions toward management strategies of SMA across Saudi Arabia. This is a cross-sectional survey using a self-administered, structured questionnaire sent to physicians who care for SMA patients during the Saudi Pediatric Neurology Society annual conference. A total of 72 clinicians of different neurological subspecialties were included. 48.6% prescribed nusinersen to their patients, with 39% of them having patients started on nusinersen. Though, 8.3% prescribed onasemnogene abeparvovec for 1–3 patients, while none of their patients started on the treatment. 64.3% stated that the only treatment available for SMA in their settings is supportive care. Around 69.4% described having a moderate to high knowledge on SMA gene therapy, and 79.2% would recommend it. 48.6% confirmed they would prescribe gene therapy at the age of 6 months, and 78.3% would prescribe it for type-I SMA. Pediatric neurologists are receptive to novel and innovative therapies for SMA in Saudi Arabia. However, the high treatment acquisition cost, strict regulations, logistical issues, and budget constraints delay their adoption and implementation. |
topic |
spinal muscular atrophy gene therapy nusinersen onasemnogene abeparvovec risdiplam Saudi Arabia |
url |
https://www.frontiersin.org/articles/10.3389/fped.2021.684134/full |
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