Role of Reduced-Intensity Conditioning Allogeneic Hematopoietic Stem-Cell Transplantation in Older Patients With De Novo Myelodysplastic Syndromes: An International Collaborative Decision Analysis
Role of Reduced-Intensity Conditioning Allogeneic Hematopoietic Stem-Cell Transplantation in Older Patients With De Novo Myelodysplastic Syndromes: An International Collaborative Decision Analysis
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DOI：
10.1200/jco.2012.46.8652
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发表时间：
2013-07-20
影响因子：
45.3
通讯作者：
Cutler, Corey
中科院分区：
文献类型：
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作者：
Koreth, John;Pidala, Joseph;Cutler, Corey
目的骨髓增生异常综合征(MDS)是一种克隆性造血系统疾病，多见于60岁以上的患者，用传统疗法无法治愈。低强度调理(RIC)异基因造血干细胞移植具有潜在的治愈作用，但存在额外的死亡风险。我们通过国际预后评分系统(IPSS)对老年MDS患者的RIC移植和非移植治疗进行了评估。患者和方法评估了一个马尔可夫决策模型，该模型对不同MDS和移植状态进行了生活质量效用估计。结果是预期寿命(LE)和质量调整后的预期寿命(QALE)。共有514名年龄在60-70岁的新发MDS患者接受了评估。排除慢性粒单核细胞白血病、孤立的5q综合征、无法分类和治疗相关的MDS。使用T细胞耗竭或人类白细胞抗原不相合或脐带血供者的移植也被排除在外。按IPSS风险分层的RIC移植(n=132)与非贫血低/中-1 IPSS患者(n=123)、贫血低/中-1 IPSS患者(n=94)和中-2/高IPSS患者(n=165)的最佳支持治疗进行了比较。QALE和敏感性分析并不支持跨可信的效用估计进行RIC移植。对于中-2/高IPSS MDS，RIC移植LE为36个月，非移植治疗为28个月。QALE和敏感性分析在可信的效用估计中有利于RIC移植。结论对于60-70岁的初发MDS患者，所青睐的治疗方法因IPSS风险而异。对于低/中-1 IPSS，首选非移植方法。对于中-2/高IPSS，RIC移植提供了总体和质量调整的生存益处。(C)美国临床肿瘤学会2013年
PurposeMyelodysplastic syndromes (MDS) are clonal hematopoietic disorders that are more common in patients aged >= 60 years and are incurable with conventional therapies. Reduced-intensity conditioning (RIC) allogeneic hematopoietic stem-cell transplantation is potentially curative but has additional mortality risk. We evaluated RIC transplantation versus nontransplantation therapies in older patients with MDS stratified by International Prognostic Scoring System (IPSS) risk.Patients and MethodsA Markov decision model with quality-of-life utility estimates for different MDS and transplantation states was assessed. Outcomes were life expectancy (LE) and quality-adjusted life expectancy (QALE). A total of 514 patients with de novo MDS aged 60 to 70 years were evaluated. Chronic myelomonocytic leukemia, isolated 5q-syndrome, unclassifiable, and therapy-related MDS were excluded. Transplantation using T-cell depletion or HLA-mismatched or umbilical cord donors was also excluded. RIC transplantation (n = 132) stratified by IPSS risk was compared with best supportive care for patients with nonanemic low/intermediate-1 IPSS (n = 123), hematopoietic growth factors for patients with anemic low/intermediate-1 IPSS (n = 94), and hypomethylating agents for patients with intermediate-2/high IPSS (n = 165).ResultsFor patients with low/intermediate-1 IPSS MDS, RIC transplantation LE was 38 months versus 77 months with nontransplantation approaches. QALE and sensitivity analysis did not favor RIC transplantation across plausible utility estimates. For intermediate-2/high IPSS MDS, RIC transplantation LE was 36 months versus 28 months for nontransplantation therapies. QALE and sensitivity analysis favored RIC transplantation across plausible utility estimates.ConclusionFor patients with de novo MDS aged 60 to 70 years, favored treatments vary with IPSS risk. For low/intermediate-1 IPSS, nontransplantation approaches are preferred. For intermediate-2/high IPSS, RIC transplantation offers overall and quality-adjusted survival benefit. (C) 2013 by American Society of Clinical Oncology