酪氨酸激酶抑制剂时代完全缓解状态下Ph染色体阳性急性淋巴细胞白血病异基因造血干细胞移植预后分析

OBJECTIVE: To study the clinical results and prognostic factors for allo-HSCT of Philadelphia chromosome-positive (Ph(+)) acute lymphoblastic leukemia (ALL) in complete remission (CR) in the era of tyrosine kinase inhibitors (TKI). METHODS: We performed a retrospective analysis of the clinical characteristics of 116 patients with Ph(+)ALL who underwent allo-HSCT while in CR. RESULTS: The study population included 72 men and 44 women. The median patient age was 20 years (4–64 years). The patients received sibling-identical donor (n=21), haplo (n=77), and unrelated donor (n=18) HSCT. The overall survival (OS) rate at 5 years was 73.2% (95% CI 63.8%–80.5%). In particular, the 5-year OS can reach 87.5% when the time from diagnosis to transplant is <180 days. The 5-years DFS was 61.4% (95% CI 51.8%–69.7%), the 5-year molecular and morphology cumulative relapse incidence was 18.5% (95% CI 12.6%–27.3%), and the 5-year TRM was 19.9% (95% CI 13.8%–28.7%). A multivariate analysis showed that an age range of 15–39 years (HR=2.730, P=0.044), time from diagnosis to HSCT ≥ 180 days (HR=4.534, P=0.010), and Ⅲ–Ⅳgrade aGVHD (HR=7.558, P=0.000) were significantly associated with an inferior overall survival. Limited cGVHD subgroup had better OS (HR=0.300, P=0.034). Sex, WBC count at diagnosis, type of BCR-ABL fusion genes, somatic gene mutations, CR(1) or >CR(1), MRD negative or positive, conditioning regimen based on TBI or Bu, conditioning intensity, donor source, GVHD prophylactic proposal using cyclosporine or tacrolimus, presence/absence of CMV viremia, and presence/absence of EBV viremia were not significantly different in terms of the OS and DFS. CONCLUSION: Factors influencing the overall survival of Ph(+) ALL patients who underwent allo-HSCT in CR in the TKI era include age, time form diagnosis to HSCT, and aGVHD severity.