UNASSIGNED: We enrolled patients with SMA who were treated with nusinersen under the National Health Insurance coverage, with complete motor score records available and a minimum treatment duration of 6 months. To evaluate the motor function of patients, the Hammersmith Infant Neurological Examination-2 (HINE-2) was used for type 1 and the Expanded Hammersmith Functional Motor Scale (HFMSE) was used for types 2 and 3 patients. A significant improvement was defined as a HINE-2 score gain ≥5 for patients with type 1 and an HFMSE score ≥ 3 for patients with types 2 and 3 SMA. Effects of treatment timing were assessed. Patients with type 2 were further categorized based on baseline motor scores for outcome analysis. We also analyzed a second dataset from five tertiary hospitals with the information on parents/patients-reported impressions of improvement.
UNASSIGNED: The study comprised 137 patients, with 21, 103, and 13 patients representing type 1, 2, and 3 SMA, respectively. At the 3-year follow-up, the analysis encompassed 7 patients with type 1, 12 patients with type 2, and none with type 3. Nearly half of all enrolled patients across SMA types (42.8, 59.2 and 46.2%, respectively) reached the 2-year follow-up for analysis. Patients with type 1 SMA exhibited gradual motor function improvement over 1-, 2-, and 3-year follow-ups (16, 9, and 7 patients, respectively). Patients with type 2 SMA demonstrated improvement over 1-, 2-, and 3-year follow-ups (96, 61 and 12 patients, respectively). Early treatment from symptom onset resulted in better outcomes for patients with type 1 and 2 SMA. In the second dataset, 90.7% of 108 patients reported subjective improvement at the 1-year follow-up.
UNASSIGNED: Nusinersen treatment for types 1-3 SMA is safe and effective in long-term follow-up. Early treatment initiation was a significant factor affecting long-term motor outcome.
■我们在国家健康保险范围内纳入了接受nusinersen治疗的SMA患者,提供完整的运动评分记录,最少治疗时间为6个月。评价患者的运动功能,Hammersmith婴儿神经学检查-2(HINE-2)用于1型患者,扩展Hammersmith功能运动量表(HFMSE)用于2型和3型患者。显著改善定义为1型患者的HINE-2评分增加≥5,2型和3型SMA患者的HFMSE评分≥3。评估治疗时机的影响。根据基线运动评分对2型患者进行进一步分类以进行结果分析。我们还分析了来自五家三级医院的第二个数据集,其中包含有关父母/患者报告的改善印象的信息。
■该研究包括137名患者,有21、103和13名患者代表1、2和3型SMA,分别。在3年的随访中,该分析包括7例1型患者,12例2型患者,无1例3型患者.几乎一半的SMA类型的所有登记患者(42.8、59.2和46.2%,分别)达到了2年的随访分析。1型SMA患者的运动功能逐渐改善超过1,2-,和3年随访(16、9和7例患者,分别)。2型SMA患者表现出超过1-的改善,2-,和3年随访(96、61和12例患者,分别)。对于1型和2型SMA患者,症状发作后的早期治疗可带来更好的预后。在第二个数据集中,108例患者中有90.7%在1年随访时报告主观改善。
■Nusinersen治疗1-3型SMA在长期随访中是安全有效的。早期开始治疗是影响长期运动结果的重要因素。