诺西那生钠治疗2型和3型儿童脊髓性肌萎缩症的临床分析OA

Objective To evaluate the efficacy and safety of nusinersen in children with spinal muscular atrophy(SMA)types 2 and 3.Methods Prospective data were collected on motor function,compound muscle action potential(CMAP),and spinal Cobb angle from 29 children with SMA type 2 or 3 treated with nusinersen at the Children's Medical Center of Xiangya Hospital,Central South University,from January 2022 to November 2025.Pre-and post-treatment changes were analyzed.Results Of the 29 children,19(66%)had a follow-up duration of≥26 months.Compared with baseline,after 26 months of treatment,clinically meaningful improvement was observed in 53%(10/19)on the Hammersmith Functional Motor Scale-Expanded(HFMSE),78%(14/18)on the Revised Upper Limb Module(RULM),and 89%(16/18)on the Motor Function Measure 20-item/32-item(MFM-20/32).CMAP amplitudes increased from baseline in the bilateral median,ulnar,and radial nerves(P<0.05).The Cobb angle increased by a median of 6.2 degrees at 26 months of treatment(P<0.05).Compared with the non-rehabilitation group,the rehabilitation group showed greater improvement in MFM-20/32 scores and slower scoliosis progression(P<0.05).Across the 29 children,309 intrathecal injections were performed;the incidence of procedure-related adverse reactions was 15.2%(47/309),and no serious adverse events were reported.Conclusions Nusinersen is well tolerated and safe in children with SMA types 2 and 3.The proportions achieving clinically meaningful responses on motor-function scales and CMAP improvements increase over time,and rehabilitation training may enhance efficacy.