MOLECULAR INTERNATIONAL PROGNOSTIC SCORING SYSTEM FOR MYELODYSPLASTIC SYNDROMES: A MONOCENTRIC EXPERIENCE

IF 0.7 Q4 HEMATOLOGY Leukemia Research Reports Pub Date : 2024-01-01 DOI:10.1016/j.lrr.2024.100431
E. Diral, G. Bergonzi, S. Mastaglio, C. Tresoldi, P. Ronchi, M. Ponzoni, M. Cristante, D. Clerici, L. Vago, M. Bernardi, F. Ciceri
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Abstract

Introduction

Allogeneic HSCT (aHSCT) is the only curative treatment, reserved for IPSS-R higher risk (HR, > 3.5) MDS. Molecular data have been integrated within the recently validated IPSS-Mol score system, in order to better predict clinical outcome. However, IPSS-Mol is not still used to guide clinical decisions. We aim to investigate IPSS-Mol significance in a cohort of MDS patients transplanted at our center.

Methods

We retrospectively analyzed a cohort of 74 MDS patients undergoing aHSCT between 2010-2022 at our center according to IPSS-R risk score. All patients received treosulfan-based conditioning regimens and PBSC as stem cell source from matched related/unrelated or haploidentical donors. NGS testing for somatic myeloid mutations was performed retrospectively on cryopreserved marrow cells at diagnosis and MDS risk score was then re-calculated according to IPSS-Mol.

Results

27 patients (36%) were lower risk (LR) at diagnosis and underwent aHSCT for disease progression. All the other patients were HR (IPSS-R > 3.5) and received aHSCT as upfront or consolidation treatment. At least one oncogenic mutation was found in 86.5% of cases by NGS testing. With IPSS-Mol 10 LR patients (37%) were re-stratified as HR (of note, 6/12 patients with intermediate IPSS-R ≤ 3.5), while 7 HR patients (15%) were re-stratified as LR.

Conclusions

aHSCT remains the only curative strategy in HR MDS. NGS testing and application of IPSS-Mol allow to better prognosticate MDS, mostly in patients with LR MDS and specifically in intermediate risk (≤3.5) group. This could help in guiding treatment and specifically optimizing the use of aHSCT in MDS.

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骨髓增生异常综合征分子国际预后评分系统:单中心经验
导言同种异体造血干细胞移植(aHSCT)是唯一可治愈的治疗方法,适用于 IPSS-R 高风险(HR, > 3.5)MDS。为了更好地预测临床结果,分子数据已被整合到最近通过验证的 IPSS-Mol 评分系统中。然而,IPSS-Mol 仍未被用于指导临床决策。我们旨在研究 IPSS-Mol 在本中心接受移植的一组 MDS 患者中的意义。所有患者均接受了以曲硫烷为基础的调理方案,并以匹配的亲缘/非亲缘或单倍体供者的PBSC作为干细胞来源。对诊断时冷冻保存的骨髓细胞进行体细胞骨髓突变的NGS检测,然后根据IPSS-Mol重新计算MDS风险评分。其他患者均为HR(IPSS-R >3.5),接受了aHSCT作为前期或巩固治疗。86.5%的病例通过NGS检测发现了至少一种致癌突变。通过IPSS-Mol,10例LR患者(37%)被重新分层为HR(值得注意的是,6/12例患者的中间IPSS-R≤3.5),而7例HR患者(15%)被重新分层为LR。NGS检测和IPSS-Mol的应用可以更好地预测MDS的预后,主要针对LR MDS患者,尤其是中危(≤3.5)组患者。这有助于指导治疗,特别是优化 MDS 患者的 aHSCT 的使用。
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来源期刊
Leukemia Research Reports
Leukemia Research Reports Medicine-Oncology
CiteScore
1.70
自引率
0.00%
发文量
70
审稿时长
23 weeks
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