Status of rehabilitation for children with 5q spinal muscular atrophy in disease-modifying treatment:a cross-sec-tional survey
GUO Xiaohua
WANG Jie
HUANG Yan
MA Kai
Abstract：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 Hos-pital 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 reha-bilitation 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%)re-ceived regular rehabilitation at professional institutions,with a similar proportion(106 cases,69.74%)perform-ing 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 train-ing 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 profes-sional rehabilitation therapists and institutions.Economic assistance and professional rehabilitation guidance are urgent needs for parents of SMA children.
Keywords：spinal muscular atrophyrehabilitation needsquestionnairedisease-modifying treatment
Publication Date：2025-12-25
Online Publishing Date：2026-01-06(First online date of this platform, not the publication date of the document)
Pages：6( 1450-1455 )