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首页> 外文期刊>The Canadian Journal of Neurological Sciences: le Journal Canadien des Sciences Neurologiques >P.060 Time to treatment effect in Spinal Muscular Atrophy Type 1 (SMA1): an indirect comparison of treatments
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P.060 Time to treatment effect in Spinal Muscular Atrophy Type 1 (SMA1): an indirect comparison of treatments

机译：P.060脊髓肌萎缩型治疗效果1（SMA1）：治疗的间接比较

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Background: SMA1 is a rapidly progressing disease resulting in death/permanent ventilation by 2 years. This study compared clinical trial data evaluating the relationship between treatment timing, time to treatment effect, and clinical outcomes in SMA1 patients Methods: A post-hoc indirect treatment comparison was conducted to measure time-to-effect differences in AVXS-101 (CL-101, NCT02122952, cohort 2) vs nusinersen (ENDEAR, NCT02193074) or risdiplam (FIREFISH, NCT02913482) using CHOP-INTEND scores. Results: Compared with nusinersen, AVXS-101 more rapidly increased mean CHOP-INTEND score from baseline (9.8- and 14.9-point increase at 1- and 2-months post-AVXS-101 vs ≤5-point increase at 2-months post-nusinersen). Greater survival benefits and lower rates of permanent ventilatory support were also observed in AVXS-101- vs nusinersen-treated patients. Compared with risdiplam treatment, AVXS-101 improved median CHOP-INTEND scores (14.0-point increase at 2-months post-AVXS-101 vs 5.5-point increase at ~2-months post-risdiplam). Treatment differences were maintained through 8-months with additional improvements at all time-points. Conclusions: Although patients in these 3 cohorts are not entirely matched (e.g. age, disease severity), useful comparisons can still be made. Based on CHOP-INTEND scores, the treatment effect of AVXS-101 appears to be more rapid vs nusinersen or risdiplam. These findings suggest that timely restoration of SMN protein may be essential for maximizing outcomes in SMA1 patients.
机译：背景：SMA1是一种迅速进展的疾病，导致2年后的死亡/永久通风。本研究比较了临床试验数据，评估治疗时间之间的关系，治疗效果的时间，以及SMA1患者的临床结果：进行了HOC间接治疗比较，以测量AVXS-101的效应时间差异（CL- 101，NCT02122952，COSINERSEN（INDEAR，NCT02193074）或RISDIPLAM（FIREFISH，NCT02913482）使用Chop-Intend分数。结果：与NUSINERSEN相比，AVXS-101比AVXS-101后1-和2个月的9.8-和14.9点增加的速度增加，从基线开始增加（9.8-14.9点≤5点。 -nusinersen）。在AVXS-101-VS Nusinersen治疗的患者中也观察到更高的生存益处和更低的永久性通气支持率。与Risdimpiplam处理相比，AVXS-101改善了中位数斩波意图分数（2个月后14.0点增加-101 vs 5.5点在Risdiplam后的5.5点增加）。治疗差异在8个月内维持在所有时间点的其他改进。结论：虽然这3个群组中的患者不完全匹配（例如，年龄，疾病严重程度），但仍然可以进行有用的比较。基于Chop-Intend分数，AVXS-101的治疗效果似乎更快速VS NUSINSEN或RISDIPLAM。这些发现表明，SMN蛋白的及时恢复对于最大化SMA1患者的结果可能是必不可少的。

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