评估心理社会风险和弹性,以支持在状细胞疾病中准备基因治疗:一个共识声明
Steven J Hardy1,2, Lori E Crosby3,4, Jerlym S Porter5
1Center for Cancer and Blood Disorders, Children's National Hospital, Washington, DC.
JAMA network open
|August 21, 2024
概括
基因疗法为状细胞疾病 (SCD) 提供了新的希望. 患者准备性评估对于成功的基因疗法至关重要,专注于理解,动机和心理社会福祉,以获得最佳的结果.
科学领域:
- 血液学 血液学 血液学
- 遗传学 是一个遗传学.
- 公共卫生 公共卫生
背景情况:
- 状细胞疾病 (SCD) 是全球最常见的遗传性血液疾病.
- 基因疗法和基因组编辑代表了SCD治疗的重大进步.
- 历史利用需要仔细考虑SCD患者对基因疗法的准备.
研究的目的:
- 为解决在状细胞疾病中对基因疗法的患者准备资源的需求.
- 为基因治疗前的患者准备性评估制定共识性建议和实际资源.
主要方法:
- 召集了包括专家,患者和护理人员在内的患者准备和弹性工作组.
- 在两年内制定了共识建议.
- 创建了资源,以指导准备性评估的实施.
主要成果:
- 确定了基因治疗前患者准备性评估的五个关键目标.
- 建议侧重于提高教育成果,评估知识,动机和心理社会因素.
- 强调评估患者的理解,兴趣,动机以及心理社会风险和弹性的重要性.
结论:
- 患者的准备程度和心理社会因素对SCD的个体和集体基因疗法成功有重大影响.
- 医疗机构,行业,支付人,政策制定者和临床医生共同负责优化患者准备和获取.
- 开发的资源可以指导实施SCD的基因治疗前准备和弹性评估.
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