Finke J, Schmoor C, Bertz H, Marks R, Wäsch R, Zeiser R, Hackanson B
Department of Hematology/Oncology and Stem Cell Transplantation, University Medical Center, Freiburg, Germany.
Center for Clinical Trials and Biostatistics, University Medical Center, Freiburg, Germany.
Bone Marrow Transplant. 2016 Jun;51(6):771-7. doi: 10.1038/bmt.2015.338. Epub 2016 Jan 11.
The outcome of patients with therapy-related myelodysplasia (t-MDS) or t-AML is very poor. The only curative treatment option implements allogeneic hematopoietic cell transplantation (aHCT); however, long-term follow-up data beyond 5 years are scarce. Here we report on a cohort of 79 consecutive patients with a median age of 58 years (range (r): 20-76) at transplantation and a median follow-up of 7.5 years (r: 0.07-19.0). Only 19 (24.1%) patients were in CR before aHCT. Non-relapse mortality and relapse rates were 23% (95% confidence interval, 15-35%) and 42% (32-55%) at 5 years, and 32% (22-46%) and 44% (34-57%) at 10 years, respectively. Disease-free survival (DFS) and overall survival (OS) rates were 35% (24-46%) and 38% (27-49%) at 5 years, and 24% (14-36%) and 24% (13-36%) at 10 years, respectively. Although cytogenetic aberrations were associated with shorter DFS and higher relapse risk, persistent disease at the time of transplantation, an unrelated donor and patient age were not associated with shorter OS. In conclusion, long-term survival beyond 10 years of t-MDS/t-AML patients after aHCT is possible, even for refractory patients. Therefore, early donor search and rapid transplantation are warranted, also to decrease the risk of disease-related deterioration of patients' performance status.
治疗相关的骨髓增生异常综合征(t-MDS)或治疗相关的急性髓系白血病(t-AML)患者的预后非常差。唯一的治愈性治疗选择是进行异基因造血细胞移植(aHCT);然而,超过5年的长期随访数据很少。在此,我们报告了一组连续79例患者,移植时的中位年龄为58岁(范围:20 - 76岁),中位随访时间为7.5年(范围:0.07 - 19.0年)。只有19例(24.1%)患者在aHCT前处于完全缓解(CR)状态。5年时的非复发死亡率和复发率分别为23%(95%置信区间,15 - 35%)和42%(32 - 55%),10年时分别为32%(22 - 46%)和44%(34 - 57%)。5年时的无病生存率(DFS)和总生存率(OS)分别为35%(24 - 46%)和38%(27 - 49%),10年时分别为24%(14 - 36%)和24%(13 - 36%)。虽然细胞遗传学异常与较短的DFS和较高的复发风险相关,但移植时的持续性疾病、无关供体和患者年龄与较短的OS无关。总之,即使是难治性患者,t-MDS/t-AML患者在aHCT后存活超过10年也是有可能的。因此,有必要尽早寻找供体并快速进行移植,以降低患者因疾病相关导致身体状况恶化的风险。