Zamanillo Irene, Poza Maria, Ayala Rosa, Rapado Inmaculada, Martinez-Lopez Joaquín, Cedena Maria Teresa
Hematology Department and Research Institute (imas12), University Hospital 12 Octubre, Madrid, Spain.
Front Oncol. 2023 May 18;13:1199023. doi: 10.3389/fonc.2023.1199023. eCollection 2023.
The IPSS-M is a recently published score for risk stratification in myelodysplastic syndromes (MDS), based on clinical and molecular data. We aimed to evaluate its relevance on treatment choice in a real-life setting.
We retrospectively collected clinical, cytogenetic and molecular data from 166 MDS patients. We calculated IPSS-R and IPSS-M scores and compared Overall Survival (OS) and Leukemia Free Survival (LFS). We also analyzed which patients would have been affected by the re-stratification in terms of clinical management.
We found that 86.1% of the patients had at least one genetic alteration. The most frequent mutated genes were (25.9%), (16.8%) and (14.4%). IPSS-M re-stratified 48.2% of the patients, of which 16.9% were downgraded and 31.3% were upgraded. IPSS-M improved outcome prediction, with a Harrell's c-index of 0.680 vs 0.626 for OS and 0.801 vs 0.757 for LFS. In 22.2% of the cohort, the reclassification of the IPSS-M could potentially affect clinical management; 17.4% of the patients would be eligible for treatment intensification and 4.8% for treatment reduction.
IPSS-M implementation in clinical practice could imply different treatment approaches in a significant number of patients. Our work validates IPSS-M in an external cohort and confirms its applicability in a real-life setting.
国际预后评分系统-分子版（IPSS-M）是最近发布的用于骨髓增生异常综合征（MDS）风险分层的评分系统，基于临床和分子数据。我们旨在评估其在实际临床环境中对治疗选择的相关性。
我们回顾性收集了166例MDS患者的临床、细胞遗传学和分子数据。我们计算了国际预后评分系统修订版（IPSS-R）和国际预后评分系统-分子版（IPSS-M）得分，并比较了总生存期（OS）和无白血病生存期（LFS）。我们还分析了哪些患者在临床管理方面会受到重新分层的影响。
我们发现86.1%的患者至少有一处基因改变。最常见的突变基因是（此处原文缺失具体基因名称）（25.9%）、（此处原文缺失具体基因名称）（16.8%）和（此处原文缺失具体基因名称）（14.4%）。IPSS-M对48.2%的患者进行了重新分层，其中16.9%的患者分层降低，31.3%的患者分层升高。IPSS-M改善了预后预测，总生存期的Harrell c指数为0.680，而之前为0.626；无白血病生存期的Harrell c指数为0.801，而之前为0.757。在22.2%的队列中，IPSS-M的重新分类可能会影响临床管理；17.4%的患者符合强化治疗标准，4.8%的患者符合减少治疗标准。
在临床实践中实施IPSS-M可能意味着在相当数量的患者中采用不同的治疗方法。我们的研究在外部队列中验证了IPSS-M，并证实了其在实际临床环境中的适用性。
