Gu Minsu, Kong Hyun-Ho
Department of Rehabilitation Medicine, Chungbuk National University Hospital, Cheongju 28644, Korea.
Children (Basel). 2021 Nov 11;8(11):1039. doi: 10.3390/children8111039.
Although nusinersen has been demonstrated to improve motor function in patients with spinal muscular atrophy (SMA), no studies have investigated its effect on fine manual dexterity. The present study aimed to investigate the ability of nusinersen to improve fine manual dexterity in patients with SMA type 2. A total of five patients with SMA type 2 were included. The Hammersmith Functional Motor Scale (expanded version) (HFMSE) and Purdue Pegboard (PP) tests were used to evaluate gross motor function and fine manual dexterity, respectively, until 18 months after nusinersen administration. HFMSE scores improved by 3-10 points (+13-53%) in all patients following nusinersen administration. PP scores also improved in all patients, from 4 to 9 points (+80-225%) in the preferred hand and from 3 to 7 points (+60-500%) in the non-preferred hand. These results suggest that nusinersen treatment improved both gross motor function and fine manual dexterity in children with SMA type 2. Addition of the PP test may aid in evaluating the fine manual dexterity essential for activities of daily living in these patients.
尽管已证明诺西那生可改善脊髓性肌萎缩症（SMA）患者的运动功能，但尚无研究调查其对精细手部灵活性的影响。本研究旨在调查诺西那生改善2型SMA患者精细手部灵活性的能力。共纳入5例2型SMA患者。使用哈默史密斯功能运动量表（扩展版）（HFMSE）和普渡钉板试验（PP）分别评估总体运动功能和精细手部灵活性，直至诺西那生给药后18个月。所有患者在接受诺西那生治疗后，HFMSE评分提高了3 - 10分（+13 - 53%）。所有患者的PP评分也有所提高，优势手从4分提高到9分（+80 - 225%），非优势手从3分提高到7分（+60 - 500%）。这些结果表明，诺西那生治疗改善了2型SMA儿童的总体运动功能和精细手部灵活性。增加PP试验可能有助于评估这些患者日常生活活动所需的精细手部灵活性。
