预测骨髓纤维化患者同种异体造血细胞移植后的生存:骨髓纤维化移植评分系统(MTSS)的性能和新预后模型的发展

Juan-Carlos Hernández-Boluda , Arturo Pereira , Alberto Alvarez-Larran , Ana-Africa Martín , Ana Benzaquen , Lourdes Aguirre , Elvira Mora , Pedro González , Jorge Mora , Nieves Dorado , Antonia Sampol , Valentín García-Gutiérrez , Oriana López-Godino , María-Laura Fox , Juan Luis Reguera , Manuel Pérez-Encinas , María-Jesús Pascual , Blanca Xicoy , Rocío Parody , Leslie González-Pinedo , José-Luis Piñana
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引用次数: 12

摘要

准确的预后工具对于评估骨髓纤维化(MF)患者异体造血细胞移植(alloc - hct)的风险/收益比至关重要。我们的目的是评估骨髓纤维化移植评分系统(MTSS)的性能,并在197名接受同种异体hct治疗的MF患者的多中心系列研究中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%。与死亡率增加独立相关的因素是造血细胞移植特异性合并症指数(HCT-CI)≥3和接受hla不匹配的非亲属供体或脐带血移植,而移植后环磷酰胺(PT-Cy)与生存率提高相关。供体类型是MTSS模型中唯一具有独立生存预后价值的参数。根据MTSS,低、中、高、高危组的3年生存率分别为62%、66%、37%和17%。通过将低、中危组以及高、高危组汇总在一起,我们确定了两类:标准危组和高危组(占该系列的25%)。标准风险组的3年生存率为62%,高风险组为25% (P <措施)。我们根据三个独立的生存风险因素(供体类型、HCT-CI和PT-Cy)得出了一个风险评分。低、中、高风险组相应的5年生存率分别为79%、55%和32% (P <措施)。总之,在我们的研究中,MTSS模型未能清晰地描述4个预后组,但可能仍然有助于识别预后不良的患者亚群。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。
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Predicting Survival after Allogeneic Hematopoietic Cell Transplantation in Myelofibrosis: Performance of the Myelofibrosis Transplant Scoring System (MTSS) and Development of a New Prognostic Model

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.

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来源期刊
CiteScore
6.60
自引率
0.00%
发文量
1061
审稿时长
3-6 weeks
期刊介绍: Biology of Blood and Marrow Transplantation publishes original research reports, reviews, editorials, commentaries, letters to the editor, and hypotheses and is the official publication of the American Society for Transplantation and Cellular Therapy. The journal focuses on current technology and knowledge in the interdisciplinary field of hematopoetic stem cell transplantation.
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