Omer Tarig Fathi Abdelazim, Abu-Baker Khalid Sharafeldin, Mohammed Kawari, Zahra Abdulla Isa Yusuf Hasan, Zainab Abdulmajeed Toorani
{"title":"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)。","authors":"Omer Tarig Fathi Abdelazim, Abu-Baker Khalid Sharafeldin, Mohammed Kawari, Zahra Abdulla Isa Yusuf Hasan, Zainab Abdulmajeed Toorani","doi":"10.1177/15473287251362882","DOIUrl":null,"url":null,"abstract":"<p><p>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.</p>","PeriodicalId":94214,"journal":{"name":"Stem cells and development","volume":" ","pages":"363-373"},"PeriodicalIF":2.0000,"publicationDate":"2025-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":"{\"title\":\"Advances in Sickle Cell Disease Treatment: A Comparative Review of Hematopoietic Stem Cell Transplantation and Gene Therapy (Casgevy and Lyfgenia).\",\"authors\":\"Omer Tarig Fathi Abdelazim, Abu-Baker Khalid Sharafeldin, Mohammed Kawari, Zahra Abdulla Isa Yusuf Hasan, Zainab Abdulmajeed Toorani\",\"doi\":\"10.1177/15473287251362882\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<p><p>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.</p>\",\"PeriodicalId\":94214,\"journal\":{\"name\":\"Stem cells and development\",\"volume\":\" \",\"pages\":\"363-373\"},\"PeriodicalIF\":2.0000,\"publicationDate\":\"2025-09-01\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"Stem cells and development\",\"FirstCategoryId\":\"1085\",\"ListUrlMain\":\"https://doi.org/10.1177/15473287251362882\",\"RegionNum\":0,\"RegionCategory\":null,\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"2025/7/31 0:00:00\",\"PubModel\":\"Epub\",\"JCR\":\"\",\"JCRName\":\"\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"Stem cells and development","FirstCategoryId":"1085","ListUrlMain":"https://doi.org/10.1177/15473287251362882","RegionNum":0,"RegionCategory":null,"ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"2025/7/31 0:00:00","PubModel":"Epub","JCR":"","JCRName":"","Score":null,"Total":0}
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.