Runde V, de Witte T, Arnold R, Gratwohl A, Hermans J, van Biezen A, Niederwieser D, Labopin M, Walter-Noel M P, Bacigalupo A, Jacobsen N, Ljungman P, Carreras E, Kolb H J, Aul C, Apperley J
University Hospital of Essen, Germany.
Bone Marrow Transplant. 1998 Feb;21(3):255-61. doi: 10.1038/sj.bmt.1701084.
Allogeneic bone marrow transplantation (BMT) offers a potential cure for younger patients with myelodysplastic syndromes (MDS) or secondary acute myeloid leukemia (sAML). More than 600 patients from 50 European centers have now been reported to the European Group for Blood and Marrow Transplantation (EBMT). We retrospectively analyzed 131 patients reported to the Chronic Leukemia Working Party of the EBMT who underwent BMT from HLA-identical siblings without prior remission induction chemotherapy. At the time of BMT 46 patients had refractory anemia (RA) or RA with ringed sideroblasts, 67 patients had more advanced MDS subtypes and 18 patients had progressed to sAML. The 5-year disease-free (DFS) and overall survival (OS) for the entire group of patients was 34 and 41%, respectively. Fifty patients died from transplant-related complications, most commonly graft-versus-host disease and/or infections. Relapse occurred in 28 patients between 1 and 33 months after BMT, resulting in an actuarial probability of relapse of 39% at 5 years. DFS and OS were dependent on pretransplant bone marrow blast counts. Patients with RA/RARS, RAEB, RAEB/T and sAML had a 5-year DFS of 52, 34, 19 and 26%, respectively. The 5-year OS for the respective patient groups was 57, 42, 24 and 28%. In a multivariate analysis, younger age, shorter disease duration, and absence of excess of blasts were associated with improved outcome. From these data we conclude that patients with myelodysplasia who have appropriate marrow donors, especially those aged less than 40 years and those with low medullary blast cell count should be treated with BMT as the primary treatment early in the course of their disease. Transplantation early after establishing the diagnosis of MDS may improve prognosis due to a lower treatment-related mortality and a lower relapse risk.
异基因骨髓移植（BMT）为年轻的骨髓增生异常综合征（MDS）或继发性急性髓系白血病（sAML）患者提供了治愈的可能。目前，欧洲血液与骨髓移植组（EBMT）已收到来自50个欧洲中心的600多名患者的报告。我们回顾性分析了EBMT慢性白血病工作组报告的131例患者，这些患者接受了来自 HLA 相同同胞的BMT，且未进行过缓解诱导化疗。在进行BMT时，46例患者患有难治性贫血（RA）或伴有环形铁粒幼细胞的RA，67例患者患有更晚期的MDS亚型，18例患者已进展为sAML。整个患者组的5年无病生存率（DFS）和总生存率（OS）分别为34%和41%。50例患者死于移植相关并发症，最常见的是移植物抗宿主病和/或感染。28例患者在BMT后1至33个月复发，5年复发的精算概率为39%。DFS和OS取决于移植前骨髓原始细胞计数。RA/RARS、RAEB、RAEB/T和sAML患者的5年DFS分别为52%、34%、19%和26%。各患者组的5年OS分别为57%、42%、24%和28%。多因素分析显示，年龄较小、病程较短以及原始细胞无增多与较好的预后相关。根据这些数据，我们得出结论，患有骨髓增生异常且有合适骨髓供体的患者，尤其是年龄小于40岁且骨髓原始细胞计数低的患者，应在疾病早期将BMT作为主要治疗方法。在确诊MDS后尽早进行移植可能会改善预后，因为与治疗相关的死亡率较低且复发风险较低。
