Division of Hematology, Department of Medicine, Karolinska Institute, Stockholm, Sweden.
Section of Hematology and Coagulation, Department of Medicine, Sahlgrenska University Hospital, Göteborg, Sweden.
Biol Blood Marrow Transplant. 2019 Sep;25(9):1770-1778. doi: 10.1016/j.bbmt.2019.05.038. Epub 2019 Jun 6.
Secondary AML (s-AML), including AML with an antecedent hematologic disorder (AHD-AML) and therapy-related AML (t-AML), constitutes a large proportion of patients with AML and is considered to confer a dismal prognosis. The role of allogeneic hematopoietic cell transplantation (HCT) in patients with s-AML and the extent to which HCT is performed in these patients has been little studied to date. We used the population-based Swedish AML Registry comprising 3337 intensively treated adult patients over a 17-year period to study the role of HCT within the group of patients with s-AML as well as compared with patients with de novo AML. HCT was performed in 576 patients (22%) with de novo AML, in 74 patients (17%) with AHD-AML, and in 57 patients (20%) with t-AML. At 5 years after diagnosis, there were no survivors among patients with previous myeloproliferative neoplasms who did not undergo HCT, and corresponding survival for patients with antecedent myelodysplastic syndromes and t-AML was and 2% and 4%, respectively. HCT was compared with chemotherapy consolidation in s-AML using 3 models: (1) a 200-day landmark analysis, in which HCT was favorable compared with conventional consolidation (P = .04, log-rank test); (2) a multivariable Cox regression with HCT as a time-dependent variable, in which the hazard ratio for mortality was 0.73 (95% confidence interval, 0.64 to 0.83) for HCT and favored HCT in all subgroups; and (3) a propensity score matching analysis, in which the 5-year overall survival (OS) and relapse-free survival in patients with s-AML in first complete remission (CR1) was 48% and 43%, respectively, for patients undergoing HCT versus 20% and 21%, respectively, for those receiving chemotherapy consolidation (P = .01 and .02, respectively, log-rank test). Our observational data suggest that HCT improves survival and offers the only realistic curative treatment option in patients with s-AML.
继发性急性髓系白血病（s-AML），包括伴有先前血液系统疾病的 AML（AHD-AML）和治疗相关的 AML（t-AML），在 AML 患者中占很大比例，被认为预后不良。异基因造血细胞移植（HCT）在 s-AML 患者中的作用以及迄今为止在这些患者中进行 HCT 的程度尚未得到充分研究。我们使用基于人群的瑞典 AML 登记处，该登记处包含 3337 名在 17 年内接受强化治疗的成年患者，以研究 HCT 在 s-AML 患者组中的作用，并与新发 AML 患者进行比较。HCT 用于 576 例（22%）新发 AML 患者、74 例（17%）AHD-AML 患者和 57 例（20%）t-AML 患者。在诊断后 5 年，未接受 HCT 的先前骨髓增生性肿瘤患者无存活者，先前骨髓增生异常综合征和 t-AML 患者的相应存活率分别为 2%和 4%。在 s-AML 中，我们使用 3 种模型将 HCT 与化疗巩固治疗进行了比较：（1）200 天的里程碑分析，其中 HCT 与常规巩固治疗相比具有优势（P=0.04，对数秩检验）；（2）多变量 Cox 回归，其中 HCT 作为时间依赖性变量，死亡风险比为 0.73（95%置信区间，0.64 至 0.83），HCT 在所有亚组中均有利；（3）倾向评分匹配分析，在首次完全缓解（CR1）的 s-AML 患者中，HCT 的 5 年总生存率（OS）和无复发生存率分别为 48%和 43%，而接受化疗巩固治疗的患者分别为 20%和 21%（P=0.01 和 0.02，对数秩检验）。我们的观察性数据表明，HCT 可改善生存，并为 s-AML 患者提供唯一现实的治愈治疗选择。
