Datasheet1_Parents' dilemma: A therapeutic decision for children with spinal muscular atrophy (SMA) type 1.pdf

BackgroundSMA type 1 is a severe neurodegenerative disorder that, in the absence of curative treatment, leads to death before 1 year of age without ventilatory support. Three innovative therapies are available to increase life expectancy.Purpose(i) To increase knowledge about parents' experiences with their decision to have opted for an innovative therapy; (ii) to assess the middle-term psychological consequences in the parents' lives.MethodsWe used an in-depth interview; a self-administrated questionnaire and self-report scales (BDI-II, STAI-Y, PSI-SF, SOC-13, PBA, DAS 16 and FICD). We compared parents hesitant before the decision to parents who were not-hesitant and the group of parents whose child was treated with gene therapy (GT) to parents whose child received another innovative therapy.Main resultsWe included n = 18 parents of 13 children. Parent's mean age was 34.7 (±5.2), child's average age was 44.3 months (±38.0). Retrospectively, most parents felt involved by doctors in decision-making on treatment, they felt their point of view was considered and were satisfied with the effects of the treatment. The group of parents “non-hesitant” was more depressed (p

背景：BackgroundSMA类型1是一种严重的神经退行性疾病，在缺乏治愈性治疗的情况下，会导致病人在1岁之前因缺乏呼吸支持而死亡。目前有三种创新疗法可供选择，以延长患者的生存期。目的：(i) 提升对父母选择创新疗法体验的认知；(ii) 评估对父母生活产生的中期心理影响。方法：本研究采用深入访谈、自行填写的问卷及自我报告量表（BDI-II、STAI-Y、PSI-SF、SOC-13、PBA、DAS 16和FICD）进行。我们将犹豫是否选择治疗方案的家长与毫不犹豫的家长以及接受基因治疗（GT）的家长群体与接受其他创新疗法的家长群体进行了比较。主要结果：纳入了n=18名13名儿童的家长。家长的平均年龄为34.7岁（±5.2），孩子的平均年龄为44.3个月（±38.0）。回顾性分析显示，大多数家长认为医生在治疗决策中给予了他们参与，他们认为自己的观点得到了考虑，并对治疗效果表示满意。‘毫不犹豫’的家长群体中抑郁症的发病率更高（p