Onida Francesco, de Wreede Liesbeth C, van Biezen Anja, Eikema Diderik-Jan, Byrne Jenny L, Iori Anna P, Schots Rik, Jungova Alexandra, Schetelig Johannes, Finke Jürgen, Veelken Hendrik, Johansson Jan-Erik, Craddock Charles, Stelljes Matthias, Theobald Matthias, Holler Ernst, Schanz Urs, Schaap Nicolaas, Bittenbring Jörg, Olavarria Eduardo, Chalandon Yves, Kröger Nicolaus
Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, University of Milan, Milan, Italy.
Department of Medical Statistics & Bioinformatics, Leiden University Medical Centre, Leiden, The Netherlands.
Br J Haematol. 2017 Jun;177(5):759-765. doi: 10.1111/bjh.14619. Epub 2017 Mar 28.
Atypical chronic myeloid leukaemia (aCML) is an aggressive malignancy for which allogeneic haematopoietic stem cell transplantation (allo-HSCT) represents the only curative option. We describe transplant outcomes in 42 patients reported to the European Society for Blood and Marrow Transplantation (EBMT) registry who underwent allo-HSCT for aCML between 1997 and 2006. Median age was 46 years. Median time from diagnosis to transplant was 7 months. Disease status was first chronic phase in 69%. Donors were human leucocyte antigen (HLA)-identical siblings in 64% and matched unrelated (MUD) in 36%. A reduced intensity conditioning was employed in 24% of patients. T-cell depletion was applied in 87% and 26% of transplants from MUD and HLA-identical siblings, respectively. According to the EBMT risk-score, 45% of patients were 'low-risk', 31% 'intermediate-risk' and 24% 'high-risk'. Following allo-HSCT, 87% of patients achieved complete remission. At 5 years, relapse-free survival was 36% and non-relapse mortality (NRM) was 24%, while relapse occurred in 40%. Patient age and the EBMT score had an impact on overall survival. Relapse-free survival was higher in MUD than in HLA-identical sibling HSCT, with no difference in NRM. In conclusion, this study confirmed that allo-HSCT represents a valid strategy to achieve cure in a reasonable proportion of patients with aCML, with young patients with low EBMT risk score being the best candidates.
非典型慢性髓系白血病(aCML)是一种侵袭性恶性肿瘤,异基因造血干细胞移植(allo-HSCT)是其唯一的治愈选择。我们描述了42例于1997年至2006年间因aCML接受allo-HSCT并向欧洲血液与骨髓移植学会(EBMT)登记处报告的患者的移植结果。中位年龄为46岁。从诊断到移植的中位时间为7个月。69%的患者疾病状态为首次慢性期。64%的供者为人类白细胞抗原(HLA)相合同胞,36%为匹配无关供者(MUD)。24%的患者采用了减低强度预处理。分别在87%和26%来自MUD和HLA相合同胞的移植中应用了T细胞去除。根据EBMT风险评分,45%的患者为“低风险”,31%为“中风险”,24%为“高风险”。allo-HSCT后,87%的患者实现完全缓解。5年时,无复发生存率为36%,非复发死亡率(NRM)为24%,40%的患者出现复发。患者年龄和EBMT评分对总生存有影响。MUD移植的无复发生存率高于HLA相合同胞HSCT,NRM无差异。总之,本研究证实allo-HSCT是使相当一部分aCML患者获得治愈的有效策略,EBMT风险评分低的年轻患者是最佳候选者。