Brammer J E, Saliba R M, Jorgensen J L, Ledesma C, Gaballa S, Poon M, Maziarz R T, Champlin R E, Hosing C, Kebriaei P
Department of Stem Cell Transplantation and Cellular Therapy, The University of Texas MD Anderson Cancer Center, Houston, TX, USA.
Department of Hematopathology, The University of Texas MD Anderson Cancer Center, Houston, TX, USA.
Bone Marrow Transplant. 2017 Jan;52(1):20-27. doi: 10.1038/bmt.2016.194. Epub 2016 Sep 12.
This study aims to provide a detailed analysis of allogeneic stem cell transplantation (allo-SCT) outcomes in a large T-cell acute lymphoblastic leukemia (T-ALL) cohort with a specific emphasis on the effects of pre-transplant minimal residual disease (MRD) and disease subtype, including the aggressive early-thymic precursor (ETP) subtype. Data from 102 allo-SCT patients with a diagnosis of T-ALL from three centers were retrospectively analyzed. Patients were grouped into four T-ALL subtypes: ETP, early, cortical and mature. At 3 years, overall survival (OS), PFS, non-relapse mortality and cumulative incidence (CI) progression were 35, 33, 11 and 55%, respectively. Patients transplanted in first complete remission (CR1) had a 3-year OS of 62% versus those transplanted in CR2 or greater (24%) (hazards ratio 1.6, P=0.2). Patients with MRD positivity at the time of transplant had significantly higher rates of progression compared with those with MRD negativity (76 vs 34%, hazards ratio 2.8, P=0.006). There was no difference in OS, PFS or cumulative incidence (CI) progression between disease subtypes, including ETP (n=16). ETP patients transplanted in CR1 (n=10) had OS of 47%, comparable to other disease subtypes, suggesting that allo-SCT can overcome the poor prognosis associated with ETP. MRD status at transplant was highly predictive of disease relapse, suggesting novel therapies are necessary to improve transplant outcomes.
本研究旨在对一大群T细胞急性淋巴细胞白血病(T-ALL)患者的异基因干细胞移植(allo-SCT)结果进行详细分析,特别关注移植前微小残留病(MRD)和疾病亚型的影响,包括侵袭性早期胸腺祖细胞(ETP)亚型。对来自三个中心的102例诊断为T-ALL的allo-SCT患者的数据进行了回顾性分析。患者被分为四种T-ALL亚型:ETP、早期、皮质型和成熟型。3年时,总生存率(OS)、无进展生存率(PFS)、非复发死亡率和累积发病率(CI)进展率分别为35%、33%、11%和55%。首次完全缓解(CR1)时接受移植的患者3年总生存率为62%,而在CR2或更高缓解状态下接受移植的患者为24%(风险比1.6,P=0.2)。移植时MRD阳性的患者与MRD阴性的患者相比,进展率显著更高(76%对34%,风险比2.8,P=0.006)。包括ETP(n=16)在内的疾病亚型之间,OS、PFS或累积发病率(CI)进展没有差异。在CR1时接受移植的ETP患者(n=10)的总生存率为47%,与其他疾病亚型相当,这表明allo-SCT可以克服与ETP相关的不良预后。移植时的MRD状态对疾病复发具有高度预测性,这表明需要新的治疗方法来改善移植结果。