关键词: Hematopoietic stem cell transplantation Myelodysplastic syndromes Prognosis Somatic mutations

Mesh : Humans Myelodysplastic Syndromes / therapy genetics mortality diagnosis Retrospective Studies Hematopoietic Stem Cell Transplantation / methods Male Female Middle Aged Prognosis Aged Adult Clinical Decision-Making Transplantation, Homologous High-Throughput Nucleotide Sequencing Young Adult

来  源:   DOI:10.1016/j.leukres.2024.107529

Abstract:
Allogeneic hematopoietic stem cell transplantation (HSCT) remains the only curative option for patients with Myelodysplastic syndromes (MDS). For many years, the selection of patients to allogeneic HSCT has been largely based on use of the International Prognostic Scoring System-Revised (IPSS-R). However, the recent broader application of next generation sequencing in clinical practice provided an abundance of molecular data and led to the introduction of molecular prognostic scores as IPSS-Molecular (IPSS-M). In this paper, we retrospectively analyzed the outcomes of 57 consecutive MDS patients treated with allogeneic HSCT in our center. Re-stratification from IPSS-R to IPSS-M occurred in almost half of patients. The application of IPSS-M to our cohort demonstrated a stronger prognostic separation compared to IPSS-R and improved the C-index. Very high-risk IPSS-M patients showed worse outcomes following HSCT compared to high-risk patients. This study provides data supporting the need of integrating molecular information in the transplant decision making of patients with MDS. This allows an earlier and better identification of patients to whom the transplant should be advised.
摘要:
异基因造血干细胞移植(HSCT)仍然是骨髓增生异常综合征(MDS)患者的唯一治疗选择。多年来,同种异体HSCT患者的选择主要基于国际预后评分系统修订(IPSS-R)的使用.然而,下一代测序在临床实践中的最新广泛应用提供了丰富的分子数据,并导致将分子预后评分作为IPSS-分子(IPSS-M)引入.在本文中,我们回顾性分析了在我们中心接受同种异体HSCT治疗的57例连续MDS患者的结局.几乎一半的患者发生了从IPSS-R到IPSS-M的重新分层。与IPSS-R相比,IPSS-M在我们队列中的应用显示出更强的预后分离,并改善了C指数。与高风险患者相比,非常高风险的IPSS-M患者在HSCT后的预后较差。这项研究提供了支持在MDS患者的移植决策中整合分子信息的需要的数据。这允许更早和更好地识别应建议移植的患者。
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