在最佳实践中更新脊柱肌肉缩:治疗考虑的建议
Mary K Schroth1, Jennifer Deans1, Diana X Bharucha Goebel1
1Cure SMA (MKS); Clinical Care Education (JD), Cure SMA; Neurology and Pediatrics (DBG), Children's National, and National Institute of Neurological Diseases and Stroke, National Institutes of Health; Division of Neurology (WBB), Department of Pediatrics, Vanderbilt University; Department of Neurology (BTD), Boston Children's Hospital, Harvard Medical School; Department of Neurology (BHE), The Ohio State University Wexner Medical Center; Child Neurology (MVF), Indiana University; Division of Neuropediatrics (AK), Development and Rehabilitation, Department of Pediatrics, Inselspital, Bern University Hospital; Pediatric Neuromuscular (JK), Helen DeVos Children's Hospital; Neurology (CMP), Children's Hospital of the King's Daughters; Department of Pediatrics (AV), Division of Neurology, University of Arkansas for Medical Sciences, Arkansas Children's Hospital; and Department of Anesthesiology (RJG), Critical Care and Pain Medicine, Boston Children's Hospital, Harvard Medical School.
脊柱肌缩 (SMA) 治疗需要共享决策. 治疗选择的关键因素包括患者的观点,安全性,年龄和临床状况,以获得最佳的结果.
科学领域:
- 神经学 神经学
- 遗传学 遗传学 是一个
- 药理学 药理学是指药理学的学科.
背景情况:
- 脊髓肌肉缩 (SMA) 是一种影响运动神经元的遗传疾病.
- 对于SMA,有三种增强SMN的治疗方法.
- 现实世界的数据和临床经验对于治疗审查至关重要.
研究的目的:
- 审查SMN增强性治疗对SMA的使用.
- 确定有助于决定SMA治疗的因素.
- 为临床医生和患者提供建议.
主要方法:
- 对SMA增强SMN治疗方法的系统文献综述.
- 经过修改的Delphi技术与专家医疗保健提供者.
- 社区工作组对患者和护理人员的投入.
主要成果:
- 患者/护理人员的观点,安全性和副作用是治疗的关键考虑因素.
- 对于新诊断的SMA患者来说,年龄和SMN2副本数量很重要.
- 临床状态,并发症和生活质量影响治疗决策.
结论:
- 提供者,患者和护理人员之间的共同决策至关重要.
- 有效的护理协调和多学科护理对于SMA治疗的成功至关重要.
- 关于当前治疗方法的信息共享解决了SMA护理中的障碍.
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