Yae Yukako, Yuge Kotaro, Maeda Toshiyuki, Ichinose Fumio, Matsuo Muneaki, Kobayashi Osamu, Okanari Kazuo, Baba Yusei, Yonee Chihiro, Maruyama Shinsuke, Shibata Minoru, Fujii Tatsuya, Chinen Madoka, Yamashita Yushiro
Department of Pediatrics and Child Health, Kurume University School of Medicine, Kurume, Fukuoka, Japan.
Department of Pediatrics, Faculty of Medicine, Saga University, Saga, Japan.
Front Neurol. 2022 Sep 30;13:918255. doi: 10.3389/fneur.2022.918255. eCollection 2022.
This study evaluated the feasibility of a matching-pair test using eye-tracking technology to assess nusinersen effectiveness in patients with advanced spinal muscular atrophy (SMA) type I.
This prospective, observational study enrolled patients with 5q-SMA type I who had lost gross motor function. Three different levels of matching-pair tests were conducted using the eye-gaze system (My Tobii; TobiiDynavox Inc.) at baseline, and after 9 and 24 weeks of nusinersen treatment. The primary endpoint was the change from baseline in matching-pair test scores and response times (i.e., the time to answer matching-pair test) at 24 weeks from baseline. Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP-INTEND), Pediatric Quality of Life inventory for patients with Neuromuscular Disease (PedsQL-NM) and Numerical Rating Scale (NRS) scores were also assessed as secondary endpoints. Analysis of ocular fixation was performed as an additional analysis. This study was registered at https://www.umin.ac.jp/ctr/ (UMIN000033935).
Seven patients (one male, six female) aged 5-21 years (median 11 years) were enrolled; all patients were bedridden and six patients were ventilated. All seven patients were able to conduct level 1 matching-pair tests at each assessment; five patients were also able to conduct levels 2 and 3. Two patients (those with the highest CHOP-INTEND scores) were able to complete all tests correctly within 60 s. There was a non-significant trend toward improvement in CHOP-INTEND, PedsQL-NM, and NRS scores over the 6-month period. There were no significant differences in the number of actions, errors, correct answers, or response times between baseline and Week 9 or 24 at any level. However, the result of an additional analysis suggests that detection of eye movement would be useful to evaluate for advanced SMA.
Eye-tracking systems are possibly feasible for the assessment of treatment efficacy in patients with advanced SMA type I.
本研究评估了使用眼动追踪技术进行配对测试以评估诺西那生对晚期I型脊髓性肌萎缩症（SMA）患者有效性的可行性。
本前瞻性观察性研究纳入了丧失粗大运动功能的5q-SMA I型患者。在基线时以及诺西那生治疗9周和24周后，使用眼动注视系统（My Tobii；TobiiDynavox公司）进行了三种不同水平的配对测试。主要终点是从基线开始24周时配对测试分数和反应时间（即回答配对测试的时间）相对于基线的变化。费城儿童医院神经肌肉疾病婴儿测试（CHOP-INTEND）、神经肌肉疾病患者的儿童生活质量量表（PedsQL-NM）和数字评分量表（NRS）分数也作为次要终点进行评估。进行了眼注视分析作为额外分析。本研究已在https://www.umin.ac.jp/ctr/（UMIN000033935）注册。
纳入了7名年龄在5至21岁（中位数11岁）的患者（1名男性，6名女性）；所有患者均卧床不起，6名患者需要通气。所有7名患者在每次评估时都能够进行1级配对测试；5名患者还能够进行2级和3级测试。两名患者（CHOP-INTEND评分最高的患者）能够在60秒内正确完成所有测试。在6个月期间，CHOP-INTEND、PedsQL-NM和NRS分数有改善的趋势，但不显著。在任何水平上，基线与第9周或第24周之间的动作数量、错误数量、正确答案数量或反应时间均无显著差异。然而，额外分析的结果表明，眼动检测对于评估晚期SMA可能有用。
眼动追踪系统可能适用于评估晚期I型SMA患者的治疗效果。
