镰状细胞病的治疗进展:造血干细胞移植和基因治疗的比较综述(Casgevy和Lyfgenia)。

IF 2
Stem cells and development Pub Date : 2025-09-01 Epub Date: 2025-07-31 DOI:10.1177/15473287251362882
Omer Tarig Fathi Abdelazim, Abu-Baker Khalid Sharafeldin, Mohammed Kawari, Zahra Abdulla Isa Yusuf Hasan, Zainab Abdulmajeed Toorani
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引用次数: 0

摘要

镰状细胞病(SCD)影响了巴林约2.1%的人口,是一种普遍存在的遗传性疾病,需要有效的治疗和长期管理。本综述重点介绍了两种创新基因疗法(Casgevy和Lyfgenia),并将其与造血干细胞移植(HSCT)的疗效和安全性进行了比较,HSCT是目前唯一可用于治疗SCD的选择。虽然HSCT在合适的供体中有90%的成功率,但其局限性包括供体稀缺和毒性。Casgevy和Lyfgenia等基因疗法在减少SCD并发症方面显示出有希望的疗效,同时绕过了这些限制。在巴林王国,巴林肿瘤中心于2023年12月批准了Casgevy,并于2025年2月中旬完成了第一例患者治疗,使巴林成为早期采用Casgevy的国家。这一里程碑标志着SCD和基因治疗历史上的关键时刻,因此值得探索围绕其实施的考虑。尽管这些疗法似乎为不适合进行HSCT的患者带来了希望,但它们的长期结果仍未得到评估——需要进一步的研究和长期随访来确认它们的安全性和持久性。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Advances in Sickle Cell Disease Treatment: A Comparative Review of Hematopoietic Stem Cell Transplantation and Gene Therapy (Casgevy and Lyfgenia).

Sickle cell disease (SCD), affecting approximately 2.1% of Bahrain's population, is a prevalent inherited disorder that necessitates effective treatments and long-term management. This review highlights two innovative gene therapies (Casgevy and Lyfgenia) and compares their efficacy and safety with hematopoietic stem cell transplantation (HSCT)-the only curative option currently available for SCD. While HSCT offers a 90% success rate with suitable donors, its limitations include donor scarcity and toxicity. Gene therapies like Casgevy and Lyfgenia show promising efficacy in reducing SCD complications while bypassing such limitations. In the Kingdom of Bahrain, the Bahrain Oncology Center approved Casgevy in December 2023 and completed its first patient treatment in mid-February 2025, making Bahrain an early adopter. This milestone marks a crucial moment in the history of both SCD and gene therapies and thus warrants exploring the considerations revolving around their implementation. Although these therapies seem to offer hope for patients ineligible for HSCT, their long-term outcomes remain unassessed-further studies with extended follow-up are needed to confirm their safety and durability.

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