2020 Update to Spinal Muscular Atrophy Management in Saudi Arabia

Frontiers in Pediatrics

Fahad A. Bashiri¹  Fahad Alsohime²  Mohamad-Hani Temsah²  Yazed AlRuthia³  Khalid Hundallah⁴ 

[1] College of Medicine, King Saud University, Riyadh, Saudi Arabia;Division of Pediatric Neurology, Department of Pediatrics, King Saud University Medical City, Riyadh, Saudi Arabia;College of Medicine, King Saud University, Riyadh, Saudi Arabia;Pediatric Intensive Care Unit, Pediatric Department, King Saud University Medical City, Riyadh, Saudi Arabia;Department of Clinical Pharmacy, College of Pharmacy, King Saud University, Riyadh, Saudi Arabia;Pharmacoeconomics Research Unit, College of Pharmacy, King Saud University, Riyadh, Saudi Arabia;Division of Neurology, Department of Pediatrics, Prince Sultan Military Medical City, Riyadh, Saudi Arabia;

关键词: spinal muscular atrophy;    gene therapy;    nusinersen;    onasemnogene abeparvovec;    risdiplam;    Saudi Arabia;   

DOI  :  10.3389/fped.2021.684134

来源: Frontiers

【 摘 要 】

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.

【 授权许可】

CC BY