Aksaralikitsunti Manassanan, Sanmaneechai Oranee
Division of Neurology, Department of Pediatrics, Faculty of Medicine Siriraj Hospital, Mahidol University, Bangkok, Thailand.
Division of Neurology, Department of Pediatrics, Faculty of Medicine Siriraj Hospital, Mahidol University, Bangkok, Thailand; Siriraj Center for Neuromuscular Diseases, Faculty of Medicine Siriraj Hospital, Mahidol University, Bangkok, Thailand.
Pediatr Neonatol. 2022 May;63(3):291-297. doi: 10.1016/j.pedneo.2022.01.002. Epub 2022 Mar 14.
Spinal muscular atrophy (SMA) is a chronic genetic disease that causes varying degrees of disability. There is currently no data specific to the health-related quality of life (HRQoL) of Thai children and adolescents with SMA. Accordingly, this study aimed to evaluate the HRQoL of Thai pediatric SMA, and to identify factors significantly associated with HRQoL and other aspects of quality of life. The findings of this study will help to improve overall care in this vulnerable pediatric population.
This prospective cross-sectional descriptive study was conducted at the Division of Neurology, Department of Pediatrics, Faculty of Medicine Siriraj Hospital, Mahidol University, Bangkok, Thailand during 2015-2018. HRQoL was measured using the Thai language version of the Pediatric Quality of Life Inventory™ 4.0 Generic Core Scale (PedsQL™). The PedsQL™ was administered to SMA children aged 2-18 years and their parents. Disease severity was evaluated by pediatric neurologists, and patients were classified according to their SMA subtype.
Forty-two Thai pediatric SMA (mean age: 9.8 ± 5.0 years, 25 males) and their families were recruited. The mean PedsQL™ total score was 57.3 ± 13.6 by child self-report, and 54.3 ± 14.8 by parent proxy-report. The PedsQL™ scores of Thai SMA children were found to be significantly lower than those reported in healthy Thai children. The mean PedsQL™ total score among healthy Thai children was 78.7 ± 9.3 by child self-report, and 79.0 ± 12.8 by parent proxy-report. Factors independently associated with lower HRQoL were non-ambulation, household income less than 18,500 Thai baht/month, mechanical ventilation, and inability to attend school.
HRQoL score was found to be an accurate reflection of patient and parent perception of SMA, which suggests the value of this tool for prioritizing interventions to improve the quality of life of Thai SMA. The results of this study also provide a baseline measurement of quality of life among Thai SMA.
脊髓性肌萎缩症（SMA）是一种导致不同程度残疾的慢性遗传疾病。目前尚无关于泰国SMA儿童和青少年健康相关生活质量（HRQoL）的具体数据。因此，本研究旨在评估泰国小儿SMA的HRQoL，并确定与HRQoL及生活质量其他方面显著相关的因素。本研究结果将有助于改善这一脆弱儿科人群的整体护理。
本前瞻性横断面描述性研究于2015年至2018年在泰国曼谷玛希隆大学诗里拉吉医院医学院儿科学系神经科进行。使用泰语版儿童生活质量量表™ 4.0通用核心量表（PedsQL™）测量HRQoL。PedsQL™应用于2至18岁的SMA儿童及其父母。由儿科神经科医生评估疾病严重程度，并根据SMA亚型对患者进行分类。
招募了42名泰国小儿SMA患者（平均年龄：9.8 ± 5.0岁，25名男性）及其家庭。儿童自我报告的PedsQL™总分平均为57.3 ± 13.6，家长代理报告的平均分为54.3 ± 14.8。发现泰国SMA儿童的PedsQL™得分显著低于健康泰国儿童报告的得分。健康泰国儿童的儿童自我报告的PedsQL™总分平均为78.7 ± 9.3，家长代理报告的平均分为79.0 ± 12.8。与较低HRQoL独立相关的因素包括不能行走、家庭月收入低于18,500泰铢、机械通气以及无法上学。
发现HRQoL评分准确反映了患者和家长对SMA的认知，这表明该工具对于优先开展改善泰国SMA患者生活质量的干预措施具有价值。本研究结果还提供了泰国SMA患者生活质量的基线测量值。
