Umbilical cord blood transplantation can overcome the poor prognosis of KMT2A-MLLT3 acute myeloid leukemia and can lead to good GVHD-free/relapse-free survival

Abstract

This is a retrospective study comparing the effectiveness of umbilical cord blood transplantation (UCBT) and chemotherapy for patients in the first complete remission period for acute myeloid leukemia with KMT2A-MLLT3 rearrangements. A total of 22 patients were included, all of whom achieved first complete remission (CR1) through 1–2 rounds of induction chemotherapy, excluding patients with an early relapse. Twelve patients were treated with UCBT, and 10 patients were treated with chemotherapy after 2 to 4 courses of consolidation therapy. The 3-year overall survival (OS) of the UCBT group was 71.3% (95% CI, 34.4–89.8%), and that of the chemotherapy group was 10% (95% CI, 5.89–37.3%). The OS of the UCBT group was significantly higher than that of the chemotherapy group (P = 0.003). The disease-free survival (DFS) of the UCBT group was 60.8% (95% CI, 25.0–83.6%), which was significantly higher than the 10% (95% CI, 5.72–35.8%) of the chemotherapy group (P = 0.003). The relapse rate of the UCBT group was 23.6% (95% CI, 0–46.8%), and that of the chemotherapy group was 85.4% (95% CI, 35.8–98.4%), which was significantly higher than that of the UCBT group (P < 0.001). The non-relapse mortality (NRM) rate in the UCBT group was 19.8% (95% CI, 0–41.3%), and that in the chemotherapy group was 0.0%. The NRM rate in the UCBT group was higher than that in the chemotherapy group, but there was no significant difference between the two groups (P = 0.272). Two patients in the UCBT group relapsed, two died of acute and chronic GVHD, and one patient developed chronic GVHD 140 days after UCBT and is still alive, so the GVHD-free/relapse-free survival (GRFS) was 50% (95% CI, 17.2–76.1%). AML patients with KMT2A-MLLT3 rearrangements who receive chemotherapy as their consolidation therapy after CR1 have a very poor prognosis. UCBT can overcome the poor prognosis and significantly improve survival, and the GRFS for these patients is very good. We suggest that UCBT is a better choice than chemotherapy for KMT2A-MLLT3 patients.

Integration of Transcriptomic Features to Improve Prognosis Prediction of Pediatric Acute Myeloid Leukemia With KMT2A Rearrangement

“…HSCT is one of the major consolidation therapies for pediatric AML with adverse factors. Most current evidence supported that HSCT could improve the outcomes of pediatric KMT2A ‐r AML 25 , 26 . Nonetheless, our findings indicated that only individuals categorized as high‐ and intermediate risk according to our prognostic system could potentially benefit from HSCT in CR1.…”

“…Most current evidence supported that HSCT could improve the outcomes of pediatric KMT2A -r AML. 25,26 Nonetheless, our findings indicated that only individuals categorized as high- and intermediate risk according to our prognostic system could potentially benefit from HSCT in CR1. Given the elevated therapy-related mortality associated with HSCT and its impact on long-term quality of life, the candidates and timepoint to receive HSCT should be carefully considered.…”

Current status and prospects of hematopoietic stem cell transplantation in China

“…As for other stem cell sources in allo-HSCT, umbilical cord blood transplantation in China also provides more choice for patients. [ 94 , 95 ] Moreover, based on the mechanism of HSCT, microtransplantation may bring hope for older patients with AML who are unfit for HSCT. [ 96 , 97 ]…”

“…In addition, the proportion of Chinese guidelines/consensus to international guidelines/consensus is increasing to 59.6% and 43.8% about transplantation norms and complication management, respectively. As for other stem cell sources in allo-HSCT, umbilical cord blood transplantation in China also provides more choice for patients [94,95] . Moreover, based on the mechanism of HSCT, microtransplantation may bring hope for older patients with AML who are unfit for HSCT [96,97] …”

“…As for other stem cell sources in allo-HSCT, umbilical cord blood transplantation in China also provides more choice for patients. [94,95] Moreover, based on the mechanism of HSCT, microtransplantation may bring hope for older patients with AML who are unfit for HSCT. [96,97] The transplantation systems of different medical centers have their own characteristics, and all have made contributions to improving the transplantation technology, aiming to make HSCT much simpler and controllable with regard to manufacturing and more stable in terms of efficacy.…”