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沙特阿拉伯脊髓性肌萎缩症管理的2020年更新

2020 Update to Spinal Muscular Atrophy Management in Saudi Arabia.

作者信息

Bashiri Fahad A, Temsah Mohamad-Hani, Hundallah Khalid, Alsohime Fahad, AlRuthia Yazed

机构信息

College of Medicine, King Saud University, Riyadh, Saudi Arabia.

Division of Pediatric Neurology, Department of Pediatrics, King Saud University Medical City, Riyadh, Saudi Arabia.

出版信息

Front Pediatr. 2021 May 31;9:684134. doi: 10.3389/fped.2021.684134. eCollection 2021.

Abstract

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.

摘要

新型治疗策略在治疗脊髓性肌萎缩症(SMA)方面已显示出一些前景。然而,这些新策略的疗效和接受度仍有待探索。我们旨在调查沙特阿拉伯各地医生对SMA管理策略的意见和看法。这是一项横断面调查,通过在沙特儿科神经病学协会年会上向照顾SMA患者的医生发放自行填写的结构化问卷进行。总共纳入了72名不同神经亚专业的临床医生。48.6%的医生为其患者开具了诺西那生钠,其中39%的患者开始使用诺西那生钠治疗。不过,8.3%的医生为1 - 3名患者开具了onasemnogene abeparvovec,但他们的患者均未开始接受该治疗。64.3%的医生表示,在他们所在的医疗机构中,SMA唯一可用的治疗方法是支持性护理。约69.4%的医生表示对SMA基因治疗有中度到高度的了解,79.2%的医生会推荐基因治疗。48.6%的医生确认他们会在6个月大时开具基因治疗,78.3%的医生会为I型SMA患者开具基因治疗。沙特阿拉伯的儿科神经科医生对SMA的新型创新疗法持接受态度。然而,高昂的治疗获取成本、严格的法规、后勤问题和预算限制阻碍了这些疗法的采用和实施。

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