目的:在利司普坦获得批准后,对于患有脊髓性肌萎缩症(SMA)的儿童,疾病改善治疗(DMT)的可能性更大.参与JEWELFISH研究的SMA非治疗初治受试者,设计用于评估利司普坦的安全性和耐受性,在接受risdiplam之前,需要经历一个冲洗期。本研究旨在调查接受nusinersen治疗90天内患者服用利司普坦的安全性。
方法:收集了接受nusinersen治疗的SMA患者的数据,然后在最后一次服用nusinersen的90天内接受了利司普坦,包括人口特征,关于用nusinersen和risdiplam治疗的信息,不良事件,在90天的随访期内进行实验室评估,以中位数(范围)表示。
结果:共报告了15名SMA患儿,包括8名男性和7名女性。先前接受的nusinersen治疗的剂量中位数为8(6-17)剂,首次接受利司普坦治疗的中位年龄为4.3(1.9-11.2)岁.具体来说,8名儿童在最近的nusinersen治疗后30天或更短的时间内接受了利司普坦,2在nusinersen后31-60天,和5在nusinersen后61-89天。发热的不良事件,肺炎,4例患者出现呕吐和皮疹.
结论:我们的研究显示,在90天的洗脱期内,接受nusinersen治疗后接受利司普兰治疗的患者的安全性数据良好。这补充了DMT时代的JEWELFISH研究,为临床医生提供额外的指导,但是需要其他中心的额外数据。
OBJECTIVE: Following the approval of risdiplam, there are more possibilities for disease-modifying therapy (DMT) in children with spinal muscular atrophy (SMA). Non-treatment-naïve subjects with SMA involved in the JEWELFISH study, designed to evaluate the safety and tolerability of risdiplam, were required to undergo a washout period before receiving risdiplam. This study aims to investigate the safety of administering risdiplam in patients within 90 days of receiving treatment with nusinersen.
METHODS: Data were collected on SMA patients who had undergone treatment with nusinersen, and who then received risdiplam within 90 days of their last dose of nusinersen, including demographic characteristics, information on treatment with nusinersen and risdiplam, adverse events, and laboratory assessments in a follow-up period of 90 days, presented as median (range).
RESULTS: A total of 15 children with SMA were reported, including 8 males and 7 females. The median number of doses of previous nusinersen treatment received was 8 (6-17) doses, and the median age at first risdiplam treatment was 4.3 (1.9-11.2) years. Specifically, 8 children received risdiplam 30 days or less after their most recent nusinersen treatment, 2 at 31-60 days after nusinersen, and 5 at 61-89 days post-nusinersen. Adverse events of pyrexia, pneumonia, vomiting and rash were reported in 4 patients.
CONCLUSIONS: Our study showed good safety data on patients who received risdiplam following nusinersen within the washout period of 90 days. This supplements the JEWELFISH study in the era of DMT, providing additional guidance for clinicians, but additional data from other centers is needed.