Guardiola P, Anderson J E, Bandini G, Cervantes F, Runde V, Arcese W, Bacigalupo A, Przepiorka D, O'Donnell M R, Polchi P, Buzyn A, Sutton L, Cazals-Hatem D, Sale G, de Witte T, Deeg H J, Gluckman E
Department of Hematology, Bone Marrow Transplant, Hôpital Saint-Louis, Paris, France.
Blood. 1999 May 1;93(9):2831-8.
Agnogenic myeloid metaplasia (AMM) is a chronic myeloproliferative disorder in which patients with poor prognostic features, receiving conventional treatments, have a median survival of less than 3 years. In this retrospective multicenter study, we analyze the results and try to define the indications for allogeneic stem cell transplantation in AMM. From January 1979 to November 1997, 55 patients with a median age of 42 years were transplanted from HLA-matched related (n = 49) or alternative (n = 6) donors for AMM. A multivariate analysis was conducted to identify factors associated with posttransplant outcome. The median posttransplant follow-up was 36 months (range, 6 to 223). The 5-year probability of survival was 47% +/- 8% for the overall group, and 54% +/- 8% for patients receiving an unmanipulated HLA-matched related transplant. The 1-year probability of transplant-related mortality was 27% +/- 6%. Hemoglobin level </=100 g/L and osteomyelosclerosis before transplant adversely affected the outcome. The probability of developing grade III-IV acute graft-versus-host disease (GVHD) was 33% +/- 8%. Sixteen of 45 patients developed extensive chronic GVHD. At last follow-up, 22 patients were in complete histohematologic remission. Treatment failure was observed in 13 cases. Age at transplant and karyotype were predictors of treatment failure. Allogeneic stem cell transplantation is an effective treatment leading to cure in a substantial number of patients with AMM. A better characterization of the variables affecting the posttransplant outcome should lead to a decreased transplant-related mortality and an improvement in these results.
原发性骨髓化生(AMM)是一种慢性骨髓增殖性疾病,预后特征较差的患者接受传统治疗后的中位生存期不到3年。在这项回顾性多中心研究中,我们分析了结果,并试图确定AMM患者异基因干细胞移植的指征。1979年1月至1997年11月,55例中位年龄为42岁的患者因AMM接受了来自人类白细胞抗原(HLA)匹配的亲属供者(n = 49)或替代供者(n = 6)的移植。进行多变量分析以确定与移植后结局相关的因素。移植后的中位随访时间为36个月(范围6至223个月)。整个研究组的5年生存率为47%±8%,接受未处理的HLA匹配亲属移植的患者为54%±8%。移植相关死亡率的1年概率为27%±6%。移植前血红蛋白水平≤100 g/L和骨髓硬化对结局有不利影响。发生III-IV级急性移植物抗宿主病(GVHD)的概率为33%±8%。45例患者中有16例发生广泛的慢性GVHD。在最后一次随访时,22例患者达到完全血液学缓解。观察到13例治疗失败。移植时的年龄和核型是治疗失败的预测因素。异基因干细胞移植是一种有效的治疗方法,可使大量AMM患者治愈。更好地描述影响移植后结局的变量应能降低移植相关死亡率并改善这些结果。