Hematology Department, Hospital Clínico Universitario, INCLIVA, University of Valencia, Valencia, Spain.
Hemotherapy and Hemostasis Department, Hospital Clinic, Barcelona, Spain.
Biol Blood Marrow Transplant. 2020 Dec;26(12):2237-2244. doi: 10.1016/j.bbmt.2020.07.022. Epub 2020 Jul 24.
Accurate prognostic tools are crucial to assess the risk/benefit ratio of allogeneic hematopoietic cell transplantation (allo-HCT) in patients with myelofibrosis (MF). We aimed to evaluate the performance of the Myelofibrosis Transplant Scoring System (MTSS) and identify risk factors for survival in a multicenter series of 197 patients with MF undergoing allo-HCT. After a median follow-up of 3.1 years, 47% of patients had died, and the estimated 5-year survival rate was 51%. Projected 5-year risk of nonrelapse mortality and relapse incidence was 30% and 20%, respectively. Factors independently associated with increased mortality were a hematopoietic cell transplantation-specific comorbidity index (HCT-CI) ≥3 and receiving a graft from an HLA-mismatched unrelated donor or cord blood, whereas post-transplant cyclophosphamide (PT-Cy) was associated with improved survival. Donor type was the only parameter included in the MTSS model with independent prognostic value for survival. According to the MTSS, 3-year survival was 62%, 66%, 37%, and 17% for low-, intermediate-, high-, and very high-risk groups, respectively. By pooling together the low- and intermediate-risk groups, as well as the high- and very high-risk groups, we pinpointed 2 categories: standard risk and high risk (25% of the series). Three-year survival was 62% in standard-risk and 25% in high-risk categories (P < .001). We derived a risk score based on the 3 independent risk factors for survival in our series (donor type, HCT-CI, and PT-Cy). The corresponding 5-year survival for the low-, intermediate-, and high-risk categories was 79%, 55%, and 32%, respectively (P < .001). In conclusion, the MTSS model failed to clearly delineate 4 prognostic groups in our series but may still be useful to identify a subset of patients with poor outcome. We provide a simple prognostic scoring system for risk/benefit considerations before transplantation in patients with MF.
准确的预后工具对于评估骨髓纤维化（MF）患者异基因造血细胞移植（allo-HCT）的风险/获益比至关重要。我们旨在评估骨髓纤维化移植评分系统（MTSS）在 197 例接受 allo-HCT 的 MF 患者多中心系列中的表现，并确定生存的危险因素。中位随访 3.1 年后，47%的患者死亡，估计 5 年生存率为 51%。预计 5 年非复发死亡率和复发率分别为 30%和 20%。与死亡率增加相关的因素是造血细胞移植特定合并症指数（HCT-CI）≥3 以及接受 HLA 不合无关供体或脐带血移植，而移植后环磷酰胺（PT-Cy）与生存改善相关。供体类型是唯一包含在 MTSS 模型中并对生存具有独立预后价值的参数。根据 MTSS，低、中、高和极高风险组的 3 年生存率分别为 62%、66%、37%和 17%。将低危和中危组以及高危和极高危组合并在一起，我们确定了 2 个类别：标准风险和高风险（占系列的 25%）。标准风险组和高风险组的 3 年生存率分别为 62%和 25%（P<.001）。我们根据本系列中对生存有影响的 3 个独立危险因素（供体类型、HCT-CI 和 PT-Cy）得出一个风险评分。低、中、高危组的 5 年生存率分别为 79%、55%和 32%（P<.001）。总之，MTSS 模型未能在我们的系列中清楚地区分 4 个预后组，但可能仍然有助于识别预后不良的患者亚组。我们为 MF 患者移植前的风险/获益考虑提供了一个简单的预后评分系统。
