Background The presence of mixed-lineage leukaemia rearrangement (MLL-r) in paediatric patients with acute myeloid leukaemia (AML) is a poor prognostic predictor. Whether allogeneic haematopoietic stem cell transplantation (allo-HSCT) is beneficial in such cases remains unclear. Methods We evaluated the outcomes and prognostic factors of allo-HSCT in 44 paediatric patients with MLL-r AML in the first complete remission (CR1) between 2014 and 2019 at our institution. Results For all the 44 patients, the 3-year overall survival (OS), event-free survival (EFS), and cumulative incidence of relapse (CIR) were 74.5%, 64.1%, and 29.1%, respectively. Among them, 37 (84.1%) patients received haploidentical (haplo)-HSCT, and the 3-year OS, EFS, and CIR were 73.0%, 65.6%, and 26.4%, respectively. The 100-day cumulative incidence of grade II–IV acute graft-versus-host disease (aGVHD) post-transplantation was 27.3%, and that of grade III–IV aGVHD was 15.9%. The overall 3-year cumulative incidence of chronic graft-versus-host disease (cGVHD) post-transplantation was 40.8%, and that of extensive cGVHD was 16.7%. Minimal residual disease (MRD)-positive (MRD +) status pre-HSCT was significantly associated with lower survival and higher risk of relapse. The 3-year OS, EFS, and CIR differed significantly between patients with MRD + pre-HSCT (n = 15; 48.5%, 34.3% and 59%) and those with MRD-pre-HSCT (n = 29; 89.7%, 81.4% and 11.7%). Pre-HSCT MRD + status was an independent risk factor in multivariate analysis. Conclusions Allo-HSCT (especially haplo-HSCT) can be a viable strategy in these patients, and pre-HSCT MRD status significantly affected the outcomes.
Background: The role of allogeneic hematopoietic stem cell transplantation (allo-HSCT) for children with high-risk (HR) T- cell acute lymphoblastic leukemia (T-ALL) in first complete remission (CR1) is still under critical discussion. Moreover, relapse is still the main factor affecting survival. This study explored the effect of allo-HSCT (especially haploidentical HSCT (haplo-HSCT) ) on improving survival and reducing relapse for children with HR T-ALL in CR1 and the prognostic factors of childhood T-ALL in order to identify who could benefit from HSCT.Methods: Seventy-four newly diagnosed pediatric T-ALL patients were included in this study and stratified into low-risk chemotherapy cohort (n=16), high-risk chemotherapy cohort (n=31) and high-risk transplant cohort (n=27). The characteristics, survival outcomes and prognostic factors of all patients were analyzed.Results: Patient prognosis in the high-risk chemotherapy cohort was significantly inferior to the low-risk chemotherapy cohort (5-year overall survival (OS): 51.2%±10% vs. 100%, P = 0.003; 5-year event-free survival (EFS): 48.4%±9.8% vs. 93.8%±6.1%, P = 0.01; 5-year cumulative incidence of relapse (CIR): 45.5%±0.8% vs. 6.3%±0.4%, P = 0.043). For high-risk patients, allo-HSCT could improve the 5-year EFS and CIR compared to chemotherapy (5-year EFS: 77.0%±8.3% vs. 48.4%±9.8%, P = 0.041; 5-year CIR: 11.9%±0.4% vs. 45.5%±0.8%, P = 0.011). 5-year OS in high-risk transplant cohort had a trend for better than that in high-risk chemotherapy cohort ( 77.0%±8.3% vs. 51.2%±10%, P = 0.084). Haplo-HSCT could reduce relapse and had a trend for improving long-term survival for HR patients when compared to the high-risk chemotherapy cohort (5-year OS: 80.0%±8.9% vs. 51.2%±10%, P = 0.093; 5-year EFS: 80.0%±8.9% vs. 48.4%±9.8%, P = 0.047; 5-year CIR: 13.9%±0.6% vs. 45.5%±0.8%, P = 0.022). Minimal residual disease (MRD) re-emergence was the independent risk factor associated with 5-year OS, EFS and CIR.Conclusions: allo-HSCT, especially haplo-HSCT, might effectively improve the survival outcomes for HR childhood T-ALL in CR1.
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