State Key Laboratory of Experimental Hematology, National Clinical Research Center for Blood Diseases, Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Tianjin, China.
State Key Laboratory of Experimental Hematology, National Clinical Research Center for Blood Diseases, Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Tianjin, China.
Transplant Cell Ther. 2021 Aug;27(8):666.e1-666.e9. doi: 10.1016/j.jtct.2021.05.006. Epub 2021 May 18.
Marrow fibrosis (MF) is usually accompanied with primary myelodysplastic syndromes (MDS) and no consensus has been reached on the relationship between MF and prognosis. We retrospectively analyzed 239 MDS and MDS derived acute myeloid leukemia patients with known grade of MF who received allogeneic stem cell transplantation (allo-HSCT). Of these, it included 121 (50.6%) without fibrosis (MF-0), 81 (33.9%) with mild fibrosis (MF-1), 37 (15.5%) with moderate to severe fibrosis (MF-2/3). MF-2/3 was associated with more pronounced dysmegakaryopoiesis (P =.002), more frequent karyotype abnormality (P = .039) and increased leukemic transformation. Spliceosome and ras pathway mutation occurred more frequently in patients with MF-2/3. After allo-HSCT, neutrophil and platelet engraftment was significantly delayed in patients with MF-2/3 than those with MF-1 and MF-0 (P = .031, P = .05, respectively). The estimated 3-year overall survival (OS) rates and disease-free survival (DFS) rates were significantly lower in patients with MF-2/3 than in those with MF-0 or MF-1 (P = .018, P = .018, respectively). Notably, in the subgroup of patients with more than 10% bone marrow blasts, MF-2/3 was independently associated with shorter OS and DFS (P = .012, P = .012, respectively) and has improved outcomes for these patients who achieved complete remission (CR) before allo-HSCT. Overall, MF-2/3 as an additional risk factor have the inferior prognosis for MDS and MDS-AML patients with bone marrow blasts ≥10%. Using pretransplantation cytoreductive therapy to obtain CR for these patients may benefit from allo-HSCT.
骨髓纤维化（MF）通常伴有原发性骨髓增生异常综合征（MDS），MF 与预后之间的关系尚未达成共识。我们回顾性分析了 239 例已知 MF 程度的 MDS 和 MDS 相关急性髓系白血病患者，这些患者均接受了异基因造血干细胞移植（allo-HSCT）。其中，无纤维化（MF-0）121 例（50.6%），轻度纤维化（MF-1）81 例（33.9%），中重度纤维化（MF-2/3）37 例（15.5%）。MF-2/3 与明显的巨核细胞发育不良（P=.002）、更频繁的核型异常（P=.039）和白血病转化有关。剪接体和 ras 通路突变在 MF-2/3 患者中更常见。allo-HSCT 后，MF-2/3 患者的中性粒细胞和血小板植入明显延迟，与 MF-1 和 MF-0 患者相比（P=.031，P=.05）。MF-2/3 患者的 3 年总生存率（OS）和无病生存率（DFS）明显低于 MF-0 或 MF-1 患者（P=.018，P=.018）。值得注意的是，在骨髓原始细胞比例超过 10%的患者亚组中，MF-2/3 与较短的 OS 和 DFS 独立相关（P=.012，P=.012），并且对于在 allo-HSCT 前获得完全缓解（CR）的患者，其结局得到改善。总体而言，MF-2/3 作为附加危险因素，对骨髓原始细胞比例≥10%的 MDS 和 MDS-AML 患者预后较差。对这些患者进行移植前细胞减少治疗以获得 CR 可能有益于 allo-HSCT。
