Conditioning Regimen of 5-Day Decitabine Administration for Allogeneic Stem Cell Transplantation in Patients with Myelodysplastic Syndrome and Myeloproliferative Neoplasms
Conditioning Regimen of 5-Day Decitabine Administration for Allogeneic Stem Cell Transplantation in Patients with Myelodysplastic Syndrome and Myeloproliferative Neoplasms
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DOI：
10.1016/j.bbmt.2019.09.001
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发表时间：
2020-02-01
影响因子：
4.3
通讯作者：
Han, Ming-Zhe
中科院分区：
文献类型：
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作者：
Cao, Yi-Geng;He, Yi;Han, Ming-Zhe
异基因造血干细胞移植（allo-HSCT）是骨髓增生异常综合征（MDS）和骨髓增生异常/骨髓增生性肿瘤（MDS/MPN）患者的潜在治愈性治疗。然而，HSCT后复发仍然是治疗失败的主要原因。在这里，我们评估了一种新的预处理方案，包括地西他滨（Dec），白消安（Bu），环磷酰胺（Cy），氟达拉滨（Flu），阿糖胞苷（Ara-c）的MDS和MDS/MPN患者的allo-HSCT的疗效。共入组48例患者，包括44例MDS和4例慢性粒单核细胞白血病（CMML）。患者在第-9天至第-5天接受Dec 20 mg/m2/天，联合Bu/Cy/Flu/Ara-c改良的准备方案。中位随访时间为522天（范围：15 - 1313天），总生存率（OS）为86%，复发率为12%，非复发死亡率为12%。严重急性（III-IV级）移植物抗宿主病（GVHD）的发生率为23%，慢性GVHD的发生率为15%。2年时，高危和极高危MDS患者的OS分别为74%和86%。在具有低风险基因突变的患者中，如TP 53和ASXL 1（88%），以及具有>= 3个基因突变的患者（79%），生存率是有希望的。免疫监测研究的结果表明，适当的自然杀伤细胞对这些有利的临床结果作出了重要贡献。总体而言，这种新方案与MDS和MDS/MPN的allo-HSCT受者的低复发率、低GVHD发生率和严重程度以及令人满意的生存率相关。(C)2019年美国移植和细胞治疗学会。爱思唯尔公司出版
Allogeneic hematopoietic stem cell transplantation (allo-HSCT) is a potentially curative treatment for patients with myelodysplastic syndromes (MDS) and myelodysplastic/myeloproliferative neoplasms (MDS/MPN). However, post-HSCT relapse remains a major cause of treatment failure. Here we assessed the efficacy of a new conditioning regimen comprising decitabine (Dec), busulfan (Bu), cyclophosphamide (Cy), fludarabine (Flu), and cytarabirie (Ara-c) for allo-HSCT in patients with MDS and MDS/MPN. A total of 48 patients were enrolled, including 44 with MDS and 4 with chronic myelomonocytic leukemia (CMML). Patients received Dec 20 mg/m(2)/day on days -9 to -5, combined with a Bu/Cy/Flu/Ara-c-modified preparative regimen. At a median follow-up of 522 days (range, 15 to 1313 days), the overall survival (OS) was 86%, relapse incidence was 12%, and nonrelapse mortality was 12%. The incidence of severe acute (grade III-IV) graft-versus-host disease (GVHD) was 23% and that of chronic GVHD was 15%. At 2 years, OS was 74% and 86%, respectively for high-risk and very-high-risk patients with MDS. Survival was promising in patients with poor-risk gene mutations, such as TP53 and ASXL1 (88%), and in those with >= 3 gene mutations (79%). Results of immunomonitoring studies revealed that proper natural killer cells made essential contributions to these favorable clinical outcomes. Overall, this new regimen was associated with a low relapse rate, low incidence and severity of GVHD, and satisfactory survival in allo-HSCT recipients with MDS and MDS/MPN. (C) 2019 American Society for Transplantation and Cellular Therapy. Published by Elsevier Inc.