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Allogeneic blood or marrow transplantation with haploidentical donor and post-transplantation cyclophosphamide in patients with myelofibrosis: a multicenter study
Leukemia ( IF 11.4 ) Pub Date : 2021-10-18 , DOI: 10.1038/s41375-021-01449-1
Siddharth Kunte 1 , Lisa Rybicki 2 , Auro Viswabandya 3 , Roni Tamari 4 , Asad Bashey 5 , Alla Keyzner 6 , Madiha Iqbal 7 , Michael R Grunwald 8 , Bhagirathbhai Dholaria 9 , Hany Elmariah 10 , Michael Ozga 11 , Anurag Singh 12 , Sameem Abedin 13 , Amy E DeZern 14 , Richard J Jones 14 , Vikas Gupta 3 , Aaron T Gerds 1 , Tania Jain 14
Affiliation  

We report the results from a multicenter retrospective study of 69 adult patients who underwent haploidentical blood or marrow transplantation (haplo-BMT) with post-transplantation cyclophosphamide (PTCy) for chronic phase myelofibrosis. The median age at BMT was 63 years (range, 41–74). Conditioning regimens were reduced intensity in 54% and nonmyeloablative in 39%. Peripheral blood grafts were used in 86%. The median follow-up was 23.1 months (range, 1.6–75.7). At 3 years, the overall survival, relapse-free survival (RFS), and graft-versus-host-disease (GVHD)-free-RFS were 72% (95% CI 59–81), 44% (95% CI 29–59), and 30% (95% CI 17–43). Cumulative incidences of non-relapse mortality and relapse were 23% (95% CI 14–34) and 31% (95% CI 17–47) at 3 years. Spleen size ≥22 cm or prior splenectomy (HR 6.37, 95% CI 2.02–20.1, P = 0.002), and bone marrow grafts (HR 4.92, 95% CI 1.68–14.4, P = 0.004) were associated with increased incidence of relapse. Cumulative incidence of acute GVHD grade 3–4 was 10% at 3 months and extensive chronic GVHD was 8%. Neutrophil engraftment was reported in 94% patients, at a median of 20 days (range, 14–70). In conclusion, haplo-BMT with PTCy is feasible in patients with myelofibrosis. Splenomegaly ≥22 cm and bone marrow grafts were associated with a higher incidence of relapse in this study.



中文翻译:

骨髓纤维化患者的单倍相合供体和移植后环磷酰胺的同种异体血液或骨髓移植:一项多中心研究

我们报告了一项多中心回顾性研究的结果,该研究纳入了 69 名接受半相合血液或骨髓移植 (haplo-BMT) 和移植后环磷酰胺 (PTCy) 治疗慢性期骨髓纤维化的成年患者。BMT 的中位年龄为 63 岁(范围 41-74 岁)。54% 的调理方案强度降低,39% 的调理方案为非清髓性。86% 的患者使用了外周血移植物。中位随访时间为 23.1 个月(范围 1.6–75.7)。3 年时,总生存率、无复发生存率 (RFS) 和无移植物抗宿主病 (GVHD) 的 RFS 分别为 72% (95% CI 59–81)、44% (95% CI 29) –59) 和 30% (95% CI 17–43)。3 年时非复发死亡率和复发的累积发生率为 23%(95% CI 14-34)和 31%(95% CI 17-47)。脾脏大小≥22 cm 或之前进行过脾切除术(HR 6.37,95% CI 2.02–20.1,P = 0.002)和骨髓移植(HR 4.92,95% CI 1.68–14.4,P  = 0.004)与复发率增加相关。3 个月时急性 GVHD 3-4 级的累积发生率为 10%,广泛的慢性 GVHD 为 8%。94% 的患者报告了中性粒细胞植入,中位时间为 20 天(范围,14-70)。总之,具有 PTCy 的单倍体 BMT 在骨髓纤维化患者中是可行的。在这项研究中,脾肿大 ≥ 22 cm 和骨髓移植与较高的复发率相关。

更新日期:2021-10-18
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