Abstract：
OBJECTIVE: In 2016, nusinersen became the first disease-modifying medication approved by the U.S. Food and Drug Administration (FDA) for spinal muscular atrophy (SMA). With the later availability of risdiplam in 2020, individuals now have the option of switching from nusinersen to risdiplam. Limited published data exist to inform this decision. This study aims to evaluate the perceptions and experiences of adult participants and parents of minor participants who previously received nusinersen and switched to risdiplam for the treatment of SMA.
METHODS: Institutional Review Board (IRB) approval was obtained from the Wake Forest IRB prior to the initiation of this study. A cross-sectional, observational study, with qualitative and quantitative data gathered via questionnaire and medical record review, was performed. Inclusion criteria included (1) prior diagnosis of SMA, (2) previous treatment with nusinersen, and (3) change to treatment with risdiplam. No participants were excluded based on age.
RESULTS: Fourteen participants-eight adults and six children-were enrolled in the study. Respondents noted improvements in physical function with each medication. Overall, respondents reported worse satisfaction with the method of delivery of the intrathecally delivered nusinersen compared to the orally-delivered risdiplam, but no respondent reported negative overall satisfaction with either medication. A majority (78.6%) of respondents reported that switching from nusinersen to risdiplam was the correct decision.
CONCLUSIONS: These results suggest that most patients are satisfied when switching from nusinersen to risdiplam, with the method of delivery being a primary factor.
METHODS: Institutional Review Board (IRB) approval was obtained from the Wake Forest IRB prior to the initiation of this study. A cross-sectional, observational study, with qualitative and quantitative data gathered via questionnaire and medical record review, was performed. Inclusion criteria included (1) prior diagnosis of SMA, (2) previous treatment with nusinersen, and (3) change to treatment with risdiplam. No participants were excluded based on age.
RESULTS: Fourteen participants-eight adults and six children-were enrolled in the study. Respondents noted improvements in physical function with each medication. Overall, respondents reported worse satisfaction with the method of delivery of the intrathecally delivered nusinersen compared to the orally-delivered risdiplam, but no respondent reported negative overall satisfaction with either medication. A majority (78.6%) of respondents reported that switching from nusinersen to risdiplam was the correct decision.
CONCLUSIONS: These results suggest that most patients are satisfied when switching from nusinersen to risdiplam, with the method of delivery being a primary factor.
摘要：
目的：2016年，nusinersen成为美国食品和药物管理局（FDA）批准的第一种用于治疗脊髓性肌萎缩症（SMA）的疾病缓解药物。随着risdiplam在2020年的后期上市，个人现在可以选择从nusinersen切换到risdiplam。存在有限的公布数据来告知这一决定。这项研究旨在评估成年参与者和未成年参与者的父母的看法和经验，这些参与者先前接受了nusinersen并改用risdiplam治疗SMA。
方法：在本研究开始之前，机构审查委员会（IRB）获得了威克森林IRB的批准。横截面，观察性研究，通过问卷调查和病历审查收集的定性和定量数据，已执行。纳入标准包括(1)SMA的先前诊断，（2）以前用Nusinersen治疗，(3)改用利司普坦治疗。没有参与者根据年龄被排除在外。
结果：14名参与者-8名成人和6名儿童-被纳入研究。受访者注意到每种药物的身体功能改善。总的来说,与口服利司普坦相比，受访者对鞘内递送nusinersen的递送方法满意度较差，但没有受访者报告对这两种药物的总体满意度为阴性。大多数（78.6％）的受访者报告说，从nusinersen切换到risdiplam是正确的决定。
结论：这些结果表明，大多数患者在从nusinersen切换到利司普时是满意的，交付方式是主要因素。
方法：在本研究开始之前，机构审查委员会（IRB）获得了威克森林IRB的批准。横截面，观察性研究，通过问卷调查和病历审查收集的定性和定量数据，已执行。纳入标准包括(1)SMA的先前诊断，（2）以前用Nusinersen治疗，(3)改用利司普坦治疗。没有参与者根据年龄被排除在外。
结果：14名参与者-8名成人和6名儿童-被纳入研究。受访者注意到每种药物的身体功能改善。总的来说,与口服利司普坦相比，受访者对鞘内递送nusinersen的递送方法满意度较差，但没有受访者报告对这两种药物的总体满意度为阴性。大多数（78.6％）的受访者报告说，从nusinersen切换到risdiplam是正确的决定。
结论：这些结果表明，大多数患者在从nusinersen切换到利司普时是满意的，交付方式是主要因素。
