了解护理人员对脊髓肌肉缩疾病修饰疗法的经验:一种定性研究
Lena Xiao1,2, Sohee Kang1,2, Djurdja Djordjevic1,2
1Pediatrics, The Hospital for Sick Children, Toronto, Ontario, Canada.
Archives of disease in childhood
|July 7, 2023
概括
脊柱肌缩 (SMA) 儿童的照顾者面临的挑战,由于成本和监管障碍,他们无法获得新的疾病修饰疗法. 他们的经历凸显了严重的不平等和情感负担,影响了治疗机会和家庭福祉.
科学领域:
- 神经学 神经学
- 遗传学 是一个遗传学.
- 儿科 儿科 儿科
背景情况:
- 脊椎肌肉缩 (SMA) 是一种严重的神经肌肉疾病,影响运动功能和呼吸.
- 新兴的疾病修饰疗法 (DMT) 如nusinersen,onasemnogene abeparvovec和risdiplam正在改变SMA护理.
- 了解护理人员对这些新治疗方法的看法对于优化患者的治疗结果至关重要.
研究的目的:
- 探索SMA儿童接受DMT的护理人员的生活经验.
- 确定与获得和使用这些改变生活的疗法相关的挑战和促进者.
- 了解DMT对SMA家庭的情感和实际影响.
主要方法:
- 采用了定性研究设计,利用与家庭护理人员的半结构面试.
- 采访被录音,文字转录,并使用内容分析进行分析.
- 15名儿童的护理人员与SMA类型1,2和3的儿童参与.
主要成果:
- 护理人员报告说,在获得DMT方面存在严重的不平等现象,理由是监管部门的批准变化,高成本和基础设施不足.
- 关键主题包括决策中的挑战,以及与治疗有关的希望,恐惧和不确定性的经验.
- 护理人员经常采取广泛的努力,以确保治疗机会,强调公平和正义的问题.
结论:
- 引入DMT已经彻底改变了SMA护理人员的经验.
- 持续和公平地获得DMT仍然是一个主要问题,受到复杂的监管,财务和资格因素的影响.
- 护理人员的洞察力为其他罕见疾病治疗的医疗保健提供了宝贵的教训.
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