摘要
目的调查接受疾病修正治疗的5q脊髓性肌萎缩(SMA)患儿的康复现状和需求。方法2020年2月至2025年2月,选择山东大学附属儿童医院就诊的5q型SMA患儿,接受诺西那生钠或利司扑兰治疗超过3个月,采用电子二维码发放调查问卷。调查内容包括患儿基本信息、康复现状和康复需求。结果共收到电子问卷165份,有效问卷152份,其中男性78例,女性74例,平均(6.99±5.19)岁。在康复现状方面,105例(69.08%)会定期到专业机构康复,且规律关节拉伸的比例(106例,69.74%)与之接近;每周康复时长以≥7 h(71例,46.71%)和<1 h(32例,21.05%)占比较高,呈现“集中或不足”的分布;仅53例(34.87%)进行规律有氧运动,且仅34例(22.37%)能独自完成康复训练,136例(89.47%)需家人督导协助。21例(13.82%)对康复效果不满意。康复困难主要集中在经济问题(62.50%)和缺乏专业康复资源(59.87%)等方面。康复需求以专业康复培训指导(48.03%)和经济帮助(42.76%)为主。结论接受疾病修正治疗的5q SMA患儿仍存在广泛的康复需求,经济问题、照看者时间不足和SMA专业康复师和康复机构的缺乏是患儿康复面临的主要问题。经济帮助和专业的康复培训指导是SMA患者家长的迫切需求。
Objective To investigate the situation and related needs of rehabilitation for children with 5q spinal muscular atrophy(SMA)treated with disease-modifying treatment.Methods From February,2020 to February,2025,the children with 5q SMA were included,who visited Children's Hospital Affiliated to Shandong University and received Nusinersen or Risdiplam for more than three months.QR code was used to distribute electronic survey questionnaires.The basic information,rehabilitation status and rehabilitation needs were investigated.Results A total of 165 electronic questionnaires were collected with 152 valid ones.There were 78 males and 74 females with an average age of(6.99±5.19)years.Regarding the current rehabilitation status,105 cases(69.08%)received regular rehabilitation at professional institutions,with a similar proportion(106 cases,69.74%)performing regular joint stretching;the weekly rehabilitation duration was predominantly distributed as≥7 hours(71 cas es,46.71%)and<1 hour(32 cases,21.05%),showing a"concentrated or insufficient"pattern;only 53 cases(34.87%)engaged in regular aerobic exercise,and merely 34 cases(22.37%)could complete rehabilitation training independently,while 136 cases(89.47%)required supervision and assistance from family members.A total of 21 cases(13.82%)were dissatisfied with the rehabilitation outcomes.The main rehabilitation challenges included economic issues(62.50%)and lack of professional rehabilitation resources(59.87%).The primary rehabilitation needs were professional rehabilitation training and guidance(48.03%)and financial support(42.76%).Conclusion There is an extensive range of rehabilitation needs in children with 5q SMA undergoing disease-modifying treatment.The main problems included economic issues,insufficient time of the caregivers and the lack of professional rehabilitation therapists and institutions.Economic assistance and professional rehabilitation guidance are urgent needs for parents of SMA children.
作者
郭晓华
王杰
黄艳
马凯
GUO Xiaohua;WANG Jie;HUANG Yan;MA Kai(Department of Rehabilitation,Ji'nan Children's Hospital(Children's Hospital Affiliated to Shandong University),Ji'nan,Shandong 250022,China;Department of Neurology,Ji'nan Children's Hospital(Children's Hospital Affiliated to Shandong University),Ji'nan,Shandong 250022,China)
出处
《中国康复理论与实践》
北大核心
2025年第12期1450-1455,共6页
Chinese Journal of Rehabilitation Theory and Practice
基金
山东大学卫生健康委员会科技发展计划项目(No.2024304030)。
关键词
脊髓性肌萎缩
康复需求
问卷调查
疾病修正治疗
spinal muscular atrophy
rehabilitation needs
questionnaire
disease-modifying treatment
