Addressing the slow uptake of gene therapy for sickle cell disease: a lack of interest, a high cost, or a more complex issue?

IF 2.8 4区医学 Q2 HEMATOLOGY

Expert Review of Hematology Pub Date : 2026-10-01 Epub Date: 2026-07-31 DOI:10.1080/17474086.2026.2711730

Lakshmanan Krishnamurti, Aishwarya Aiyer, Karthik Rayapureddi

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引用次数: 0

Abstract

Introduction: The approval of Gene Therapy (GT) for sickle cell disease (SCD) marks a major milestone in hematology, offering curative potential for SCD without the need for a allogeneic transplantation. Yet early real-world uptake has been slow. This delay in integration is likely due to complex intertwined barriers that require further elucidation.

Areas covered: We conducted a structured review of the literature, searching PubMed/MEDLINE, Embase, and ClinicalTrials.gov through April 2026 for evidence on the economic, institutional, clinician-level, and patient-level barriers limiting adoption of GT for SCD, as well as global disparities in access.

Expert opinion: Slow uptake of GT reflects the expected challenge of introducing a complex, resource-intensive intervention into a fragmented care system for an underserved population with a chronic debilitating illness. Addressing these barriers requires coordinated efforts from payers, institutions, clinicians, and patient communities. Without deliberate structural reform, GT risks widening existing health disparities rather than alleviating them.

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解决镰状细胞病基因治疗的缓慢接受：缺乏兴趣，高成本，还是一个更复杂的问题？

（GT）治疗镰状细胞病（SCD）的批准标志着血液学的一个重要里程碑，它提供了治疗潜力，而不需要匹配的兄弟姐妹供体。然而，现实世界对这种技术的早期吸收很慢。这种整合的延迟可能是由于复杂的交织障碍，需要进一步阐明。覆盖领域：我们对文献进行了结构化的回顾，检索PubMed/MEDLINE、Embase和ClinicalTrials.gov，直到2026年4月，寻找经济、制度、临床水平和患者水平上限制采用GT治疗SCD的障碍的证据，以及全球在获取方面的差异。专家意见：GT的缓慢采用反映了在分散的护理系统中引入复杂的资源密集型干预措施的预期挑战。解决这些障碍需要支付方、机构、临床医生和患者社区的协调努力。如果没有深思熟虑的结构性改革，GT就有可能扩大而不是缓解现有的健康差距。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

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来源期刊

Expert Review of Hematology HEMATOLOGY-

CiteScore

4.70

自引率

3.60%

发文量

审稿时长

6-12 weeks

期刊介绍： Advanced molecular research techniques have transformed hematology in recent years. With improved understanding of hematologic diseases, we now have the opportunity to research and evaluate new biological therapies, new drugs and drug combinations, new treatment schedules and novel approaches including stem cell transplantation. We can also expect proteomics, molecular genetics and biomarker research to facilitate new diagnostic approaches and the identification of appropriate therapies. Further advances in our knowledge regarding the formation and function of blood cells and blood-forming tissues should ensue, and it will be a major challenge for hematologists to adopt these new paradigms and develop integrated strategies to define the best possible patient care. Expert Review of Hematology (1747-4086) puts these advances in context and explores how they will translate directly into clinical practice.