骨髓增生异常/骨髓增殖性重叠肿瘤的异基因造血干细胞移植结局
Outcomes of Allogeneic Hematopoietic Stem Cell Transplantation for Myelodysplastic/Myeloproliferative Overlap Neoplasms.
作者信息
Desai Nihar, Rodriguez-Rodriguez Sergio, Chen Carol, Moya Tommy Alfaro, Al-Shaibani Eshrak, Novitzky-Basso Igor, Pasic Ivan, Michelis Fotios V, Viswabandya Auro, Kim Dennis, Kumar Rajat, Mattsson Jonas, Law Arjun Datt
机构信息
Division of Medical Oncology and Hematology, Hans Messner Allogeneic Blood and Marrow Transplant Program, Princess Margaret Cancer Centre, Toronto, Ontario, Canada.
Department of Medicine, University of Toronto, Toronto, Ontario, Canada.
出版信息
Eur J Haematol. 2025 Aug;115(2):142-152. doi: 10.1111/ejh.14430. Epub 2025 May 7.
Myelodysplastic/myeloproliferative overlap neoplasms (MDS/MPN) are rare hematological malignancies. We analyzed the outcomes of 75 patients undergoing allogeneic hematopoietic stem cell transplantation (HSCT) for MDS/MPN. Graft-versus-host disease (GvHD) prophylaxis included post-transplantation cyclophosphamide (PTCy) in 71% of patients, with 44 (59%) receiving a combination of anti-thymocyte globulin (ATG) and PTCy. The median follow-up was 44.4 months. Primary graft failure occurred in three patients (4%). The incidence of grade III-IV acute GvHD at day 100 was 13% (95% CI: 6-22). At 2 years, the incidence of moderate-severe chronic GvHD, non-relapse mortality (NRM), relapse, GvHD-free/relapse-free survival (GRFS), and overall survival (OS) was 31.7% (95% CI 20.7-43.2), 37.9% (26-49), 17.4% (95% CI: 10-27), 24.8% (95% CI: 15-36), and 51.6% (95% CI: 39-63), respectively. PTCy-based GvHD prophylaxis seemed to be associated with improved OS (HR: 0.5, 95% CI: 0.3-0.9, p = 0.03), NRM (HR: 0.4, 95% CI: 0.2-0.9, p = 0.03), and GRFS (HR: 0.5, 95% CI: 0.3-0.8, 0.009). On multivariable analysis, the use of the PTCy-containing regimen seemed to be associated with improved NRM (HR: 0.41; 95% CI: 0.2-0.8; p = 0.03), GRFS (HR: 0.47; 95% CI: 0.3-0.8; p = 0.009), and OS (HR: 0.49; 95% CI: 0.2-0.9; p = 0.03) without an increased risk of relapse.
骨髓增生异常/骨髓增殖性重叠肿瘤（MDS/MPN）是罕见的血液系统恶性肿瘤。我们分析了75例接受异基因造血干细胞移植（HSCT）治疗MDS/MPN患者的预后情况。移植物抗宿主病（GvHD）预防措施包括71%的患者使用移植后环磷酰胺（PTCy），其中44例（59%）接受了抗胸腺细胞球蛋白（ATG）与PTCy的联合治疗。中位随访时间为44.4个月。3例患者（4%）发生原发性移植失败。100天时III-IV级急性GvHD的发生率为13%（95%CI：6-22）。2年时，中重度慢性GvHD、非复发死亡率（NRM）、复发率、无GvHD/无复发生存率（GRFS）和总生存率（OS）分别为31.7%（95%CI 20.7-43.2）、37.9%（26-49）、17.4%（95%CI：10-27）、24.8%（95%CI：15-36）和51.6%（95%CI：39-63）。基于PTCy的GvHD预防似乎与OS改善（HR：0.5，95%CI：0.3-0.9，p = 0.03）、NRM改善（HR：0.4，95%CI：0.2-0.9，p = 0.03）和GRFS改善（HR：0.5，95%CI：0.3-0.8，p = 0.009）相关。多变量分析显示，使用含PTCy方案似乎与NRM改善（HR：0.41；95%CI：0.2-0.8；p = 0.03）、GRFS改善（HR：0.47；95%CI：0.3-0.8；p = 0.009）和OS改善（HR：0.49；95%CI：0.2-0.9；p = 0.03）相关，且复发风险未增加。
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