Trunk Andrew D, Patel Sagar S, Prchal Josef T, Sborov Douglas W, Zander Axel R, Lee Catherine J
Transplant and Cellular Therapy Program, Huntsman Cancer Institute, University of Utah, USA.
Leuk Res Rep. 2023 Aug 26;20:100388. doi: 10.1016/j.lrr.2023.100388. eCollection 2023.
Allogeneic stem cell transplant can have high morbidity and mortality in patients with myelofibrosis (MF) and multiple myeloma (MM). This phase 2 study used a novel myeloablative regimen of split-dose busulfan, fludarabine, and then post-transplant cyclophosphamide. Four patients with MF and 2 with MM were enrolled. At 1 year, non-relapse mortality was 33.3%, and overall survival was 50%. Incidence of acute and chronic GVHD was 33.3% and 16.7%, respectively. Those surviving beyond 1 year (MF = 1, MM = 2) had durable remissions with a median follow-up of 42 months. This small study demonstrates relative safety & favorable key outcomes using this novel approach.
异基因干细胞移植在骨髓纤维化（MF）和多发性骨髓瘤（MM）患者中可能具有较高的发病率和死亡率。这项2期研究使用了一种新型的清髓方案，即分剂量白消安、氟达拉滨，然后进行移植后环磷酰胺治疗。招募了4例MF患者和2例MM患者。1年时，非复发死亡率为33.3%，总生存率为50%。急性和慢性移植物抗宿主病（GVHD）的发生率分别为33.3%和16.7%。存活超过1年的患者（MF = 1例，MM = 2例）获得了持久缓解，中位随访时间为42个月。这项小型研究表明，使用这种新方法具有相对安全性和良好的关键结果。
