Center of Excellence in Rehabilitation Medicine, UMC Utrecht Brain Center and De Hoogstraat Rehabilitation, University Medical Center Utrecht, Utrecht University, Utrecht, the Netherlands.
Ecare4you, Amersfoort, the Netherlands.
Dev Med Child Neurol. 2021 Jul;63(7):816-823. doi: 10.1111/dmcn.14825. Epub 2021 Feb 6.
To gain insight into parents' perspectives about their decision-making process concerning nusinersen treatment for their child, including perceived needs and concerns, and to explore factors that influence this process.
This was an exploratory qualitative interview study among parents of children with spinal muscular atrophy types 1 to 3. Data were analysed using inductive thematic analysis.
Nineteen parents of 16 children representing 13 families participated. A wide variety of perspectives was reported ranging from a biomedical approach, which focused on battling the disease, to a holistic approach, which aimed for a good quality of life for their child. The most important factors that helped parents to decide were honest and neutral communication with their physician and access to available information.
It is important physicians understand that there are different perspectives influencing the decision-making process. Physicians should create an environment that allows parents to accept or reject treatment by communicating honestly and openly with them and by discussing both options extensively. Clear information about pros and cons, recent developments in research, and the experiences of other parents should be made available to enable parents to make an informed decision. What this paper adds Parents perceived different needs and concerns about nusinersen treatment, which emphasized individual differences. Parents' perspectives varied from battling the disease to preserving quality of life. Life expectancy, stopping deterioration, and improving quality of life were the perceived benefits of nusinersen treatment. Open communication about the pros and cons of treatment with clinicians facilitated decision-making. Clear and honest information facilitated the alignment of values and goals.
深入了解家长在决定是否为其脊髓性肌萎缩症 1 至 3 型患儿接受nusinersen 治疗时的决策过程，包括他们的需求和关注点，以及探讨影响这一过程的因素。
这是一项针对脊髓性肌萎缩症 1 至 3 型患儿家长的探索性定性访谈研究。采用归纳主题分析对数据进行分析。
13 个家庭中有 19 名患儿的家长参与，代表了 16 名患儿。报告了各种各样的观点，从关注疾病的生物医学方法到旨在为孩子提供良好生活质量的整体方法。帮助家长做出决定的最重要因素是与医生进行诚实和中立的沟通，以及获取可用信息。
了解影响决策过程的不同观点很重要。医生应该创造一个环境，通过与家长坦诚和开放地沟通，并广泛讨论两种选择，使家长能够接受或拒绝治疗。应该提供有关利弊、研究进展和其他家长经验的明确信息，以使家长能够做出明智的决定。
家长对 nusinersen 治疗有不同的需求和关注点，强调了个体差异。
家长的观点从与疾病作斗争到维持生活质量各有不同。
延长预期寿命、停止恶化和提高生活质量是 nusinersen 治疗的预期益处。
与临床医生就治疗的优缺点进行开放沟通有助于决策。
清晰、诚实的信息有助于价值观和目标的协调一致。
