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老年人镰状细胞病的管理:当前和新兴疗法的考虑因素。

Management of Older Adults with Sickle Cell Disease: Considerations for Current and Emerging Therapies.

机构信息

Division of Hematology, Department of Medicine, Duke University School of Medicine, Durham, NC, USA.

Duke Claude D. Pepper Older Americans Independence Center, Durham, NC, USA.

出版信息

Drugs Aging. 2023 Apr;40(4):317-334. doi: 10.1007/s40266-023-01014-8. Epub 2023 Feb 28.

Abstract

People with sickle cell disease (SCD) are living longer than ever before, with the median survival increasing from age 14 years in 1973, beyond age 40 years in the 1990s, and as high as 61 years in recent cohorts from academic centers. Improvements in survival have been attributed to initiatives, such as newborn screening, penicillin prophylaxis, vaccination against encapsulated organisms, better detection and treatment of splenic sequestration, and improved transfusion support. There are an estimated 100,000 people living with SCD in the United States and millions of people with SCD globally. Given that the number of older adults with SCD will likely continue to increase as survival improves, better evidence on how to manage this population is needed. When managing older adults with SCD (defined herein as age ≥ 40 years), healthcare providers should consider the potential pitfalls of extrapolating evidence from existing studies on current and emerging therapies that have typically been conducted with participants at mean ages far below 40 years. Older adults with SCD have historically had little to no representation in clinical trials; therefore, more guidance is needed on how to use current and emerging therapies in this population. This article summarizes the available evidence for managing older adults with SCD and discusses potential challenges to using approved and emerging drugs in this population.

摘要

患有镰状细胞病 (SCD) 的人比以往任何时候都活得更长,中位生存期从 1973 年的 14 岁延长到 20 世纪 90 年代的 40 岁以上,最近来自学术中心的队列高达 61 岁。生存的改善归因于各种举措,例如新生儿筛查、青霉素预防、针对囊胞体的疫苗接种、更好地检测和治疗脾功能亢进以及改进输血支持。在美国,估计有 10 万人患有 SCD,在全球范围内,有数百万患有 SCD 的人。鉴于随着生存的改善,患有 SCD 的老年人的数量可能会继续增加,因此需要更好的证据来管理这一人群。在管理患有 SCD 的老年人(定义为年龄≥40 岁)时,医疗保健提供者应考虑从现有研究中推断现有和新兴疗法的证据的潜在陷阱,这些研究通常是在平均年龄远低于 40 岁的参与者中进行的。患有 SCD 的老年人在历史上几乎没有或没有参与临床试验;因此,需要更多关于如何在该人群中使用现有和新兴疗法的指导。本文总结了管理患有 SCD 的老年人的现有证据,并讨论了在该人群中使用已批准和新兴药物的潜在挑战。

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