Mendelson Avital, Liu Yunfeng, Bao Weili, Shi Patricia A
Laboratory of Stem Cell Biology and Engineering, New York Blood Center, New York, NY, USA.
Laboratory of Complement Biology, New York Blood Center, New York, NY, USA.
Blood Cells Mol Dis. 2024 Mar;105:102824. doi: 10.1016/j.bcmd.2024.102824. Epub 2024 Jan 19.
In preparation for hematopoietic stem cell mobilization and collection, current ex vivo gene therapy protocols for sickle cell disease require patients to undergo several months of chronic red cell transfusion. For health care equity, alternatives to red cell transfusion should be available. We examined whether treatment with GBT1118, the murine analog of voxelotor, could be a safe and feasible alternative to red cell transfusion. We found that 3 weeks of treatment with GBT1118 increased the percentage of bone marrow hematopoietic stem cells and upon plerixafor mobilization, the percentage of peripheral blood hematopoietic stem cells. Our data suggest that voxelotor should be further explored for its potential safety and utility as preparation for hematopoietic stem cell mobilization and collection.
在为造血干细胞动员和采集做准备时，目前用于镰状细胞病的体外基因治疗方案要求患者接受数月的慢性红细胞输血。为了实现医疗公平，应该有红细胞输血的替代方案。我们研究了伏索利特的鼠类类似物GBT1118治疗是否可以作为红细胞输血的一种安全可行的替代方案。我们发现，用GBT1118治疗3周可提高骨髓造血干细胞的百分比，并且在使用普乐沙福进行动员后，外周血造血干细胞的百分比也会提高。我们的数据表明，应进一步探索伏索利特在造血干细胞动员和采集准备方面的潜在安全性和实用性。
