Cutler Corey S, Lee Stephanie J, Greenberg Peter, Deeg H Joachim, Pérez Waleska S, Anasetti Claudio, Bolwell Brian J, Cairo Mitchell S, Gale Robert Peter, Klein John P, Lazarus Hillard M, Liesveld Jane L, McCarthy Philip L, Milone Gustavo A, Rizzo J Douglas, Schultz Kirk R, Trigg Michael E, Keating Armand, Weisdorf Daniel J, Antin Joseph H, Horowitz Mary M
International Bone Marrow Transplant Registry, Medical College of Wisconsin, 8701 Watertown Plank Rd, PO Box 26509, Milwaukee, WI 53226, USA.
Blood. 2004 Jul 15;104(2):579-85. doi: 10.1182/blood-2004-01-0338. Epub 2004 Mar 23.
Bone marrow transplantation (BMT) can cure myelodysplastic syndrome (MDS), although transplantation carries significant risks of morbidity and mortality. Because the optimal timing of HLA-matched BMT for MDS is unknown, we constructed a Markov model to examine 3 transplantation strategies for newly diagnosed MDS: transplantation at diagnosis, transplantation at leukemic progression, and transplantation at an interval from diagnosis but prior to leukemic progression. Analyses using individual patient risk-assessment data from transplantation and nontransplantation registries were performed for all 4 International Prognostic Scoring System (IPSS) risk groups with adjustments for quality of life (QoL). For low and intermediate-1 IPSS groups, delayed transplantation maximized overall survival. Transplantation prior to leukemic transformation was associated with a greater number of life years than transplantation at the time of leukemic progression. In a cohort of patients under the age of 40 years, an even more marked survival advantage for delayed transplantation was noted. For intermediate-2 and high IPSS groups, transplantation at diagnosis maximized overall survival. No changes in the optimal transplantation strategies were noted when QoL adjustments were incorporated. For low- and intermediate-1-risk MDS, delayed BMT is associated with maximal life expectancy, whereas immediate transplantation for intermediate-2- and high-risk disease is associated with maximal life expectancy.
骨髓移植（BMT）可以治愈骨髓增生异常综合征（MDS），尽管移植存在显著的发病和死亡风险。由于MDS进行人类白细胞抗原（HLA）匹配的BMT的最佳时机尚不清楚，我们构建了一个马尔可夫模型，以研究新诊断的MDS的三种移植策略：诊断时移植、白血病进展时移植以及在诊断后的某个间隔但在白血病进展之前移植。使用来自移植和非移植登记处的个体患者风险评估数据，对所有4个国际预后评分系统（IPSS）风险组进行了分析，并对生活质量（QoL）进行了调整。对于低危和中危-1的IPSS组，延迟移植可使总生存期最大化。白血病转化前移植比白血病进展时移植的生命年数更多。在40岁以下的患者队列中，延迟移植的生存优势更为明显。对于中危-2和高危IPSS组，诊断时移植可使总生存期最大化。纳入QoL调整后，最佳移植策略没有变化。对于低危和中危-1风险的MDS，延迟BMT与最大预期寿命相关，而中危-2和高危疾病立即移植与最大预期寿命相关。
