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...

Full description

Bibliographic Details
Main Authors: Fahad A. Bashiri, Mohamad-Hani Temsah, Khalid Hundallah, Fahad Alsohime, Yazed AlRuthia
Format: Article
Language:English
Published: Frontiers Media S.A. 2021-05-01
Series:Frontiers in Pediatrics
Subjects:
Online Access:https://www.frontiersin.org/articles/10.3389/fped.2021.684134/full
id doaj-ae47506a607a4297bd2aef5a626cd262
record_format Article
spelling 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
work_keys_str_mv AT fahadabashiri 2020updatetospinalmuscularatrophymanagementinsaudiarabia
AT fahadabashiri 2020updatetospinalmuscularatrophymanagementinsaudiarabia
AT mohamadhanitemsah 2020updatetospinalmuscularatrophymanagementinsaudiarabia
AT mohamadhanitemsah 2020updatetospinalmuscularatrophymanagementinsaudiarabia
AT khalidhundallah 2020updatetospinalmuscularatrophymanagementinsaudiarabia
AT fahadalsohime 2020updatetospinalmuscularatrophymanagementinsaudiarabia
AT fahadalsohime 2020updatetospinalmuscularatrophymanagementinsaudiarabia
AT yazedalruthia 2020updatetospinalmuscularatrophymanagementinsaudiarabia
AT yazedalruthia 2020updatetospinalmuscularatrophymanagementinsaudiarabia
_version_ 1721419519937740800