异基因造血干细胞移植治疗 BCR-ABL1 阳性 AML 的相对良好结局：来自欧洲血液和骨髓移植学会（EBMT）急性白血病工作组的一项调查。
Relatively favorable outcome after allogeneic stem cell transplantation for BCR-ABL1-positive AML: A survey from the acute leukemia working party of the European Society for blood and marrow transplantation (EBMT).
机构信息
Skåne University Hospital, Department of Hematology, Oncology and Radiation Physics, Stem Cell Centre, Lund University, Lund, Sweden.
Hôpital Saint Antoine, ALWP office, Service d'Hématologie et de Thérapie cellulaire, Paris, France.
出版信息
Am J Hematol. 2018 Jan;93(1):31-39. doi: 10.1002/ajh.24928. Epub 2017 Oct 31.
The aim of the study was to assess the role of allogeneic stem cell transplantation (SCT) in patients diagnosed with BCR-ABL1-positive acute myeloid leukemia (AML). Fifty-seven patients (median age, 48 years, range: 19-67) with BCR-ABL1 positive AML undergoing SCT were identified. The majority of the patients (70%) received a TKI before the transplant. At SCT 48 patients were in CR (45 in CR1), while 9 patients were transplanted in a more advanced stage of the disease. MRD was negative (BCR-ABL1/ABL < 10 ) at time of SCT in 36.1% (14/40). After SCT, 16 (61.5%) out of 26 patients with MRD positive at transplantation reached MRD negativity. After a median follow-up of 6.3 years (0.7-14.2), NRM, RI, LFS, OS, and GRFS at 5 years were 18.1%, 37%, 44.2%, 53.8%, and 32.1%, respectively. The cumulative incidence of acute GvHD grade II-IV was 16.4%, incidence of chronic GvHD 24.9%, and of extensive cGvHD 21.4%, respectively. In patients who received SCT in CR1, 5-yr NRM, RI, LFS, OS, and GRFS were 15.9%, 36.4%, 46.5%, 59.4%, and 34.9%, respectively. Univariate analysis showed that age (<50 vs. ≥50 years) was associated with RI (5-yr: 22.7 vs. 50%), LFS (5-yr: 61.9 vs. 31.8%), and GRFS (5-yr: 52.4 vs. 18.2%), whereas MRD-negative status before SCT was associated with an improved GRFS (38.9 vs. 16.7%). We conclude that the outcome of patients <50 years of age with BCR-ABL1-positive AML receiving allogeneic SCT in CR is relatively favorable, possibly reflecting the beneficial effect of the use of TKI.
研究目的在于评估异基因造血干细胞移植（SCT）在诊断为 BCR-ABL1 阳性急性髓系白血病（AML）患者中的作用。共鉴定出 57 例接受 SCT 的 BCR-ABL1 阳性 AML 患者（中位年龄 48 岁，范围：19-67 岁）。大多数患者（70%）在移植前接受了 TKI 治疗。在 SCT 时，48 例患者处于完全缓解（CR）状态（45 例处于 CR1），而 9 例患者在疾病更晚期接受移植。36.1%（14/40）的患者在 SCT 时 MRD 阴性（BCR-ABL1/ABL < 10）。移植后，26 例 MRD 阳性患者中有 16 例（61.5%）达到 MRD 阴性。中位随访 6.3 年后（0.7-14.2），无复发生存率（NRM）、复发率（RI）、无白血病生存率（LFS）、总生存率（OS）和无疾病生存率（GRFS）在 5 年时分别为 18.1%、37%、44.2%、53.8%和 32.1%。急性移植物抗宿主病（GVHD）Ⅱ-Ⅳ级的累积发生率为 16.4%，慢性 GVHD 的发生率为 24.9%，广泛慢性 GVHD 的发生率为 21.4%。在 CR1 中接受 SCT 的患者中，5 年时 NRM、RI、LFS、OS 和 GRFS 分别为 15.9%、36.4%、46.5%、59.4%和 34.9%。单因素分析显示，年龄（<50 岁与 ≥50 岁）与 RI（5 年：22.7%与 50%）、LFS（5 年：61.9%与 31.8%）和 GRFS（5 年：52.4%与 18.2%）相关，而 SCT 前 MRD 阴性与 GRFS 改善相关（38.9%与 16.7%）。我们得出结论，年龄 <50 岁的 BCR-ABL1 阳性 AML 患者接受 CR 状态下的异基因 SCT 的结局相对较好，这可能反映了 TKI 使用的有益效果。
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