Department of Hematology and Oncology, Nagoya University Graduate School of Medicine, Nagoya, Japan.
Department of Hematology, Clinical Immunology and Infectious Diseases, Ehime University Graduate School of Medicine, Toon, Japan.
Biol Blood Marrow Transplant. 2019 Aug;25(8):1536-1543. doi: 10.1016/j.bbmt.2019.02.019. Epub 2019 Mar 1.
The choice of alternative donor is a major issue in allogeneic hematopoietic stem cell transplantation (HSCT) for patients with primary myelofibrosis (PMF) without an HLA-matched related donor. We conducted this retrospective study using the Japanese national registry data for 224 PMF patients to compare the outcomes of first allogeneic HSCT from HLA-matched related donor bone marrow (Rtd-BM), HLA-matched related donor peripheral blood stem cells (Rtd-PB), HLA-matched unrelated donor bone marrow (UR-BM), unrelated umbilical cord blood (UR-UCB), and other hematopoietic stem cell grafts. Nonrelapse mortality (NRM) rates at 1 year after Rtd-BM, Rtd-PB, UR-BM, UR-UCB, and other transplantations were 16%, 36%, 30%, 41%, and 48%, respectively. Multivariate analysis identified UR-UCB transplantation, other transplantation, frequent RBC transfusion before transplantation, and frequent platelet (PLT) transfusion before transplantation as predictive of higher NRM. Relapse rates at 1 year after Rtd-BM, Rtd-PB, UR-BM, UR-UCB, and other transplantation were 14%, 17%, 11%, 14%, and 15%, respectively. No specific factor was associated with the incidence of relapse. Overall survival (OS) at 1 and 4 years after Rtd-BM, Rtd-PB, UR-BM, UR-UCB, and other transplantation were 81% and 71%, 58% and 52%, 61% and 46%, 48% and 27%, and 48% and 41%, respectively. Multivariate analysis identified older patient age, frequent RBC transfusion before transplantation, and frequent PLT transfusion before transplantation as predictive of lower OS. In conclusion, UR-UCB transplantation, as well as UR-BM transplantation, can be selected for PMF patients without an HLA-identical related donor. However, careful management is required for patients after UR-UCB transplantation because of the high NRM. Further studies including more patients after HLA-haploidentical related donor and HLA-mismatched unrelated donor transplantation would provide more valuable information for patients with PMF when making decisions regarding the choice of alternative donor.
对于没有 HLA 匹配相关供体的原发性骨髓纤维化 (PMF) 患者，选择替代供体是异基因造血干细胞移植 (HSCT) 的一个主要问题。我们使用日本全国登记处的数据对 224 名 PMF 患者进行了这项回顾性研究，比较了来自 HLA 匹配的相关供体骨髓 (Rtd-BM)、HLA 匹配的相关供体外周血干细胞 (Rtd-PB)、HLA 匹配的无关供体骨髓 (UR-BM)、无关脐带血 (UR-UCB) 和其他造血干细胞移植物的首次异基因 HSCT 的结果。Rtd-BM、Rtd-PB、UR-BM、UR-UCB 和其他移植后 1 年的非复发死亡率 (NRM) 分别为 16%、36%、30%、41%和 48%。多变量分析确定 UR-UCB 移植、其他移植、移植前频繁输血红细胞 (RBC) 和移植前频繁输血血小板 (PLT) 是 NRM 较高的预测因素。Rtd-BM、Rtd-PB、UR-BM、UR-UCB 和其他移植后 1 年的复发率分别为 14%、17%、11%、14%和 15%。没有特定的因素与复发的发生有关。Rtd-BM、Rtd-PB、UR-BM、UR-UCB 和其他移植后 1 年和 4 年的总生存率 (OS) 分别为 81%和 71%、58%和 52%、61%和 46%、48%和 27%以及 48%和 41%。多变量分析确定年龄较大、移植前频繁输血 RBC 和移植前频繁输血 PLT 是 OS 较低的预测因素。总之，对于没有 HLA 完全匹配相关供体的 PMF 患者，可以选择 UR-UCB 移植和 UR-BM 移植。然而，由于 NRM 较高，需要对接受 UR-UCB 移植的患者进行谨慎管理。进一步的研究包括更多 HLA 半相合相关供体和 HLA mismatched 无关供体移植后的患者，将为 PMF 患者在选择替代供体时提供更有价值的信息。
