Haematology and Bone Marrow Transplant Unit, IRCCS San Raffaele Scientific Institute, Milan, Italy.
Department of Haematology and EBMT Paris Study Office/CEREST-TC, Saint Antoine Hospital, Paris, France.
Bone Marrow Transplant. 2021 Aug;56(8):1842-1849. doi: 10.1038/s41409-021-01248-3. Epub 2021 Mar 5.
The use of haplo-HCT with posttransplant cyclophosphamide (PT-Cy) is a new standard in the treatment of hematological diseases. A paucity of data exists on risk factors for engraftment failure in haplo-HCT with PT-Cy. We analyzed 1939 adults with acute myeloid leukemia (AML) who received a first haplo-HCT from 2010 to 2019. Status at haplo-HCT was first complete remission (CR1) in 72.5% of patients, secondary AML was reported in 9.9%. Median follow-up was 24.4 months and median age at haplo-HCT was 51 years. Stem cell source was bone marrow (BM) in 42% and peripheral blood stem cell (PBSC) in 58%, and 64% of patients received a myeloablative conditioning (MAC) regimen. Cumulative incidence of primary graft failure (GF) was 6%; GF was reported in 110 patients and 54 died before day +30 with no sign of cell recovery. Overall, 33 patients underwent a second HCT in a median time of 45 days and 13 were alive at last follow-up, the 2-year overall survival (OS) after second HCT being 32.4%. In multivariate analysis, factors independently associated with the risk of nonengraftment were: secondary AML (HR 1.30, p = 0.003), use of RIC (HR 1.22, p < 0.001), and use of BM (HR 1.21, p < 0.001). At 2 years, leukemia-free survival (LFS) and OS for the entire population was 55.2% (95% CI: 52.6-57.6) and 60.9% (95% CI: 58.4-63.3), respectively. Incidence of GF after haplo-HCT with PT-Cy is lower than reported T-cell-depleted haplo-HCT. Optimization of conditioning regimen and graft source should be considered for reducing the risk of GF in haplo-HCT recipients using PT-Cy.
在血液系统疾病的治疗中，使用单倍体造血干细胞移植（haplo-HCT）联合移植后环磷酰胺（PT-Cy）已成为新的标准。然而，关于 haplo-HCT 联合 PT-Cy 中移植物失败的风险因素的数据还很有限。我们分析了 1939 例 2010 年至 2019 年期间接受首次 haplo-HCT 的急性髓系白血病（AML）成人患者。72.5%的患者在 haplo-HCT 时处于完全缓解（CR1）状态，9.9%的患者为继发性 AML。中位随访时间为 24.4 个月，haplo-HCT 时的中位年龄为 51 岁。干细胞来源为骨髓（BM）占 42%，外周血造血干细胞（PBSC）占 58%，64%的患者接受了清髓性预处理方案（MAC）。原发性移植物失败（GF）的累积发生率为 6%；110 例患者发生 GF，其中 54 例在+30 天前死亡且无细胞恢复迹象。总体而言，33 例患者在中位时间为 45 天内行第二次 HCT，13 例患者在最后一次随访时仍存活，第二次 HCT 后 2 年的总生存率（OS）为 32.4%。多变量分析显示，与非植入相关的独立危险因素包括：继发性 AML（HR 1.30，p=0.003）、使用RIC（HR 1.22，p<0.001）和使用 BM（HR 1.21，p<0.001）。在整个研究人群中，2 年时无白血病生存（LFS）和 OS 分别为 55.2%（95%CI：52.6-57.6）和 60.9%（95%CI：58.4-63.3）。haplo-HCT 联合 PT-Cy 后移植物失败的发生率低于报道的 T 细胞耗竭 haplo-HCT。对于使用 PT-Cy 的 haplo-HCT 受者，应考虑优化预处理方案和移植物来源，以降低移植物失败的风险。
