Curry Mary, Peterson Ilse, Belter Lisa, Sarr Fatou, Whitmire Sarah, Schroth Mary, Jarecki Jill
Cure SMA, 925 Busse Road, Elk Grove Village, IL, 60007, USA.
Faegre Drinker Biddle and Reath LLP, 1500 K Street NW, Suite 1100, Washington, DC, 20005, USA.
Neurol Ther. 2023 Oct;12(5):1631-1647. doi: 10.1007/s40120-023-00516-2. Epub 2023 Jun 22.
As part of efforts to reduce diagnostic delays and enhance clinical trials, Cure SMA evaluated the effects of COVID-19 on SMA care and clinical trial conduct.
Spinal muscular atrophy (SMA) is an autosomal recessive neuromuscular disease characterized by progressive, potentially debilitating muscle weakness and atrophy. Uninterrupted access to early diagnosis, disease-modifying treatment, and care for SMA is vital to avoiding irreversible motor neuron death and achieving optimal patient outcomes.
Two surveys were conducted: a provider survey and a community survey. The Provider Impact Survey, distributed from November 24, 2020, through March 8, 2021, assessed COVID-19's effects on referrals for evaluation of suspected SMA, cancellations and delays of SMA-related care, and clinical trials. The Community Impact Survey was fielded in three waves between April 7, 2020 and July 19, 2021, in tandem with Cure SMA COVID-19 support programs.
A total of 48 completed provider surveys (22 from care sites, 26 from care-and-trial sites) reflected decreases in referrals for suspected SMA, increases in appointment cancellations and delays, and patient reluctance to attend in-person visits due to COVID-19. One-third of care-and-trial sites reported trial recruitment delays, and one-quarter reported pausing trial enrollment. Results of the Community Impact Survey, completed by 2047 individuals, showed similar disruptions, with 55% reporting changes or limitations in accessing essential SMA-related services.
This research evaluates the pandemic's interruption of SMA care and research. These insights can help mitigate and increase preparedness for future disruptive events. Expanded use of virtual tools including telehealth and remote monitoring may enhance continuity and access. However, additional research is required to evaluate their effectiveness. While this research was specific to SMA, its findings may have relevance for other patient communities.
作为减少诊断延迟和加强临床试验工作的一部分，脊髓性肌萎缩症治疗协会（Cure SMA）评估了新冠病毒病对脊髓性肌萎缩症（SMA）护理及临床试验开展的影响。
脊髓性肌萎缩症是一种常染色体隐性神经肌肉疾病，其特征为进行性、可能导致身体衰弱的肌肉无力和萎缩。不间断地获得早期诊断、疾病修正治疗以及SMA护理对于避免不可逆的运动神经元死亡和实现最佳患者治疗效果至关重要。
开展了两项调查：一项是医疗服务提供者调查，另一项是社区调查。“医疗服务提供者影响调查”于2020年11月24日至2021年3月8日进行，评估了新冠病毒病对疑似SMA评估转诊、SMA相关护理的取消和延迟以及临床试验的影响。“社区影响调查”于2020年4月7日至2021年7月19日分三个阶段进行，与Cure SMA新冠病毒病支持项目同步开展。
总共48份已完成的医疗服务提供者调查问卷（22份来自护理机构，26份来自护理及试验机构）反映出疑似SMA转诊减少、预约取消和延迟增加，以及患者因新冠病毒病而不愿进行面对面就诊。三分之一的护理及试验机构报告试验招募延迟，四分之一的机构报告暂停试验入组。由2047人完成的“社区影响调查”结果显示了类似的干扰情况，55%的受访者报告在获取基本的SMA相关服务方面有变化或受限。
本研究评估了疫情对SMA护理和研究的干扰。这些见解有助于减轻未来干扰性事件的影响并提高应对准备。扩大使用包括远程医疗和远程监测在内的虚拟工具可能会增强连续性和可及性。然而，需要更多研究来评估其有效性。虽然本研究特定于SMA，但其发现可能与其他患者群体相关。
