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Abstract：
BACKGROUND: The management of Spinal Muscular Atrophy (SMA) requires a multidisciplinary treatment approach, wherein rehabilitation constitutes an integral element. In this study, we examined the effects of rehabilitation among Chinese SMA patients and assessed the real-world efficacy of rehabilitation interventions.
METHODS: We conducted a cross-sectional online survey on SMA patients from June 9, 2023, to June 30, 2023, through the Meier Advocacy & Support Center using data from the Center\'s database and electronic questionnaires. The rehabilitation situation of the participants over the past 14 months were investigated. Logistic binary regression was used to analyze the relationship between Pediatric Quality of Life Inventory(PedsQL™) scores and rehabilitation.
RESULTS: A total of 186 questionnaires were finally analyzed. Only 29 patients did not rehabilitated in the past 14 months. A significant correlation between age and type of rehabilitation, as well as between age and duration of rehabilitation. Patients receiving no rehabilitation or solely home-based rehabilitation exhibited a higher median age of 8.4 compared to those undergoing standard rehabilitation or a combination of standard and home-based rehabilitation, with a median age of 4.9 (z-score = -4.49, p-value < 0.001). In addition, long-term rehabilitation (OR = 0.314, 95%CI = 0.106-0.927, p = 0.04) were negatively correlated with lower PedsQL™ Neuromuscular Module scores, and PedsQL scores in the long-term rehabilitation group were higher than those in the short-term and no-rehabilitation groups (54.2 ± 15.1 vs. 45.9 ± 14.4 and 42.3 ± 14.3, p = 0.01), with the most significant difference observed in the physical function section (59.0 ± 15.8 vs. 46.8 ± 15.2 and 45.6 ± 15.9, p < 0.01). Mobility and exercise (OR = 0.26, 95%CI = 0.08-0.81, p = 0.02), as well as assistive technology (OR = 0.28, 95%CI = 0.10-0.82, p = 0.02), were independently associated with a lower score in a negative direction.
CONCLUSIONS: The study found that long-term rehabilitation was linked to higher PedsQL scores in SMA patients, highlighting the need for standardized rehabilitation programs to enhance function and quality of life.
METHODS: We conducted a cross-sectional online survey on SMA patients from June 9, 2023, to June 30, 2023, through the Meier Advocacy & Support Center using data from the Center\'s database and electronic questionnaires. The rehabilitation situation of the participants over the past 14 months were investigated. Logistic binary regression was used to analyze the relationship between Pediatric Quality of Life Inventory(PedsQL™) scores and rehabilitation.
RESULTS: A total of 186 questionnaires were finally analyzed. Only 29 patients did not rehabilitated in the past 14 months. A significant correlation between age and type of rehabilitation, as well as between age and duration of rehabilitation. Patients receiving no rehabilitation or solely home-based rehabilitation exhibited a higher median age of 8.4 compared to those undergoing standard rehabilitation or a combination of standard and home-based rehabilitation, with a median age of 4.9 (z-score = -4.49, p-value < 0.001). In addition, long-term rehabilitation (OR = 0.314, 95%CI = 0.106-0.927, p = 0.04) were negatively correlated with lower PedsQL™ Neuromuscular Module scores, and PedsQL scores in the long-term rehabilitation group were higher than those in the short-term and no-rehabilitation groups (54.2 ± 15.1 vs. 45.9 ± 14.4 and 42.3 ± 14.3, p = 0.01), with the most significant difference observed in the physical function section (59.0 ± 15.8 vs. 46.8 ± 15.2 and 45.6 ± 15.9, p < 0.01). Mobility and exercise (OR = 0.26, 95%CI = 0.08-0.81, p = 0.02), as well as assistive technology (OR = 0.28, 95%CI = 0.10-0.82, p = 0.02), were independently associated with a lower score in a negative direction.
CONCLUSIONS: The study found that long-term rehabilitation was linked to higher PedsQL scores in SMA patients, highlighting the need for standardized rehabilitation programs to enhance function and quality of life.
摘要：
背景：脊髓性肌萎缩症（SMA）的治疗需要多学科的治疗方法，其中康复构成一个组成部分。在这项研究中,我们研究了中国SMA患者的康复效果,并评估了康复干预的真实世界疗效.
方法：我们从2023年6月9日至2023年6月30日，通过Meier倡导和支持中心，使用该中心数据库和电子问卷中的数据，对SMA患者进行了横断面在线调查。调查了参与者过去14个月的康复情况。采用Logistic二元回归分析儿科生活质量量表(PedsQL™)评分与康复的关系。
结果：最终分析了186份问卷。在过去的14个月中，只有29名患者没有康复。年龄和康复类型之间存在显着相关性，以及年龄和康复时间之间。与接受标准康复或标准康复和家庭康复相结合的患者相比，未接受康复或仅接受家庭康复的患者的中位年龄为8.4岁。中位年龄为4.9岁（z评分=-4.49，p值<0.001）。此外,长期康复（OR=0.314，95CI=0.106-0.927，p=0.04）与较低的PedsQL™神经肌肉模块评分呈负相关，长期康复组的PedsQL评分高于短期和非康复组（54.2±15.1vs.45.9±14.4和42.3±14.3，p=0.01），在身体功能部分观察到最显著的差异(59.0±15.8vs.46.8±15.2和45.6±15.9，p<0.01）。流动性和运动（OR=0.26，95CI=0.08-0.81，p=0.02），以及辅助技术（OR=0.28，95CI=0.10-0.82，p=0.02），与负方向的较低分数独立相关。
结论：研究发现，长期康复与SMA患者较高的PedsQL评分有关，强调需要标准化的康复计划，以提高功能和生活质量。
方法：我们从2023年6月9日至2023年6月30日，通过Meier倡导和支持中心，使用该中心数据库和电子问卷中的数据，对SMA患者进行了横断面在线调查。调查了参与者过去14个月的康复情况。采用Logistic二元回归分析儿科生活质量量表(PedsQL™)评分与康复的关系。
结果：最终分析了186份问卷。在过去的14个月中，只有29名患者没有康复。年龄和康复类型之间存在显着相关性，以及年龄和康复时间之间。与接受标准康复或标准康复和家庭康复相结合的患者相比，未接受康复或仅接受家庭康复的患者的中位年龄为8.4岁。中位年龄为4.9岁（z评分=-4.49，p值<0.001）。此外,长期康复（OR=0.314，95CI=0.106-0.927，p=0.04）与较低的PedsQL™神经肌肉模块评分呈负相关，长期康复组的PedsQL评分高于短期和非康复组（54.2±15.1vs.45.9±14.4和42.3±14.3，p=0.01），在身体功能部分观察到最显著的差异(59.0±15.8vs.46.8±15.2和45.6±15.9，p<0.01）。流动性和运动（OR=0.26，95CI=0.08-0.81，p=0.02），以及辅助技术（OR=0.28，95CI=0.10-0.82，p=0.02），与负方向的较低分数独立相关。
结论：研究发现，长期康复与SMA患者较高的PedsQL评分有关，强调需要标准化的康复计划，以提高功能和生活质量。
