Comparison of outcomes after umbilical cord blood and unmanipulated haploidentical hematopoietic stem cell transplantation in children with high‐risk acute lymphoblastic leukemia

Abstract

Allogeneic hematopoietic stem cell transplantation (HSCT) is an effective therapy for children with high-risk acute lymphoblastic leukemia (ALL). Human leukocyte antigen (HLA)-haploidentical HSCT (haplo-HSCT) or umbilical cord blood transplantation (UCBT) are both important alternative sources of stem cells for those without an HLA-identical sibling donor or unrelated matched donor. We aimed to compare the therapeutic effects of single UCBT and unmanipulated haplo-HSCT in high-risk ALL children (n = 129). Hematopoietic recovery was significantly faster in haplo-HSCT recipients than in UCBT recipients. The 2-year cumulative incidences of relapse in the haplo-HSCT and UCBT groups were 16.1% and 24.1%, respectively (p = 0.169). The 2-year cumulative incidences of non-relapse mortality in the haplo-HSCT and UCBT groups were 12.8% and 18.8%, respectively (p = 0.277). The 2-year probabilities of overall survival in the haplo-HSCT and UCBT groups were 82.0% and 69.6%, respectively (p = 0.071), and the 2-year probability of disease-free survival in the haplo-HSCT group was higher than in the UCBT group (71.0% vs. 57.2%, p = 0.040). However, several variables (such as leukocyte count and cytogenetics at diagnosis) were different between the groups, and a possible center effect should also be considered. In addition, only mild and moderate chronic graft-versus-host disease (GVHD) was associated with significantly improved survival compared to those without chronic GVHD in multivariate analysis. Thus, our results show that both unmanipulated haplo-HSCT and UCBT are valid for high-risk ALL children lacking a HLA matched donor, and both strategies expand the donor pool for children in need.

Better outcomes of modified myeloablative conditioning without antithymocyte globulin versus myeloablative conditioning in cord blood transplantation for hematological malignancies: A retrospective (development) and a prospective (validation) study

“…Despite the possible GVL effect, cGVHD was reported to have an adverse effect on NRM41 and was the main cause of poor quality of life after transplantation 42. However, low probability of cGVHD has always been one of the advantages of CB,43 and many studies reported that the relapse rate in CBT was similar to or lower than in other kinds of HCT,44, 45, 46 which reflected the clinical separation of cGVHD and the GVL effect in CB. Furthermore, our previous study showed that long‐term survivors with CBT had less cGVHD and higher quality of life compared to HCT in HLA‐identical sibling's donors 47.…”

Haploidentical transplantation is associated with better overall survival when compared to single cord blood transplantation: an EBMT-Eurocord study of acute leukemia patients conditioned with thiotepa, busulfan, and fludarabine

“…Contemporary transplantation practice involving the use of double cord blood units in case that there are not enough stem cells in a single cord, flexible conditioning regimens, effective graft-versus-host disease (GvHD) prophylaxis platforms with non-T cell depleted (NTD) Haplo, and improved management of post-transplant complications, have brought improvement in outcomes of alternative donor transplantations [3, 7, 12]. Several studies have reported that results with UCBT and Haplo are comparable with those of transplants from HLA identical or matched unrelated donors [13–22]. …”

Superior survival of unmanipulated haploidentical haematopoietic stem cell transplantation compared with intensive chemotherapy as post‐remission treatment for children with very high‐risk philadelphia chromosome negative B‐cell acute lymphoblastic leukaemia in first complete remission

“…In view of the fact that haploidentical HSCT (haplo-HSCT) can provide all patients with a potential donor, it has become an important alternative choice for many VHR ALL children when undergoing transplantation in recent years. In our previous study, we demonstrated the safety and efficacy of haplo-HSCT in the treatment of paediatric VHR ALL (Mo et al, 2016). However, as far as we know, no study has compared the efficacy of intensive chemotherapy and haplo-HSCT as post-remission therapy in paediatric VHR Ph-negative B-ALL, and the role of haplo-HSCT for post-remission therapy in these patients is not well established.…”