一项范围审查,探索遗传血红蛋白病的治疗定义和语言
Marilyn S Baffoe-Bonnie1,2,3, K Jameson Floyd1, Alicia A Livinski4
1Health Disparities Unit, Social and Behavioral Research Branch, National Human Genome Research Institute, Bethesda, MD.
Genetics in medicine open
|March 22, 2024
概括
对于状细胞病和β血病治疗的"治愈"的定义仍然不清楚,尽管该术语被广泛使用. 这篇评论强调了需要精确的语言,以细胞为基础的治疗血红蛋白病变.
科学领域:
- 血液学 血液学 血液学
- 细胞和基因疗法细胞和基因疗法
- 医学伦理 医学伦理
背景情况:
- 状细胞病 (SCD) 和β血病是潜在的治愈性基于细胞的疗法的首要目标.
- 骨髓移植,干细胞移植和基因疗法是这些血红蛋白病的当前研究和治疗方式.
- "治愈"一词经常用于这些疗法,但缺乏治愈的明确定义.
研究的目的:
- 进行范围审查,探讨在SCD和β血病的基于细胞疗法的背景下"治愈"的定义和使用情况.
- 确定这些血红蛋白病变的治疗成功和并发症如何描述.
- 分析当前术语的科学,伦理和社会影响.
主要方法:
- 对五个数据库进行了全面的搜索.
- 两位评论员在标题/摘要和全文层面对文章进行了选.
- 纳入标准侧重于SCD或β血病的基于细胞的疗法 (BMT,SCT,基因疗法),报告临床/心理社会结果或利益相关者的观点的原始数据.
主要成果:
- "治愈"一词被广泛使用,但很少被定义,对其含义没有共识.
- 治疗的未定义的委词是常见的.
- 在如何描述治疗成功和并发症方面存在显著差异.
结论:
- 对于SCD和β血症的基于细胞的疗法中治疗意图和治疗结果的准确和明确术语非常需要.
- 这些发现强调了集中生活经验和将非专业知识与科学理解相结合的重要性.
- 伦理和社会影响需要重新评估如何传达治疗目标和结果.
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