Combination of Haploidentical Hematopoietic Stem Cell Transplantation with Umbilical Cord-Derived Mesenchymal Stem Cells in Patients with Severe Aplastic Anemia: A Retrospective Controlled Study

Abstract

Objective: We retrospectively compared the outcomes of patients with severe aplastic anemia (SAA) who received haploidentical hematopoietic stem cell transplantation (haplo-HSCT) combined or not combined with umbilical cord-derived mesenchymal stem cells (UC-MSCs). Materials and Methods: A total of 101 patients with SAA were enrolled in this study and treated with haplo-HSCT plus UC-MSC infusion (MSC group, n=47) or haplo-HSCT alone (non-MSC group, n=54). Results: The median time to neutrophil engraftment in the MSC and non-MSC group was 11 (range: 8-19) and 12 (range: 8-23) days, respectively (p=0.049), with a respective cumulative incidence (CI) of 97.82% and 97.96% (p=0.101). Compared to the non-MSC group, the MSC group had a lower CI of chronic graft-versus-host disease (GVHD) (8.60+-0.25% vs. 24.57+-0.48%, p=0.048), but similar rates of grades IIIV acute GVHD (23.40+-0.39% vs. 24.49+-0.39%, p=0.849), grades III-IV acute GVHD (8.51+-0.17% vs. 10.20+-0.19%, p=0.765), and moderatesevere chronic GVHD (2.38+-0.06% vs. 7.45+-0.18%, p=0.352) were observed. The estimated 5-year overall survival (OS) rates were 78.3+-6.1% and 70.1+-6.3% (p=0.292) while the estimated 5-year GVHD-free, failure-free survival (GFFS) rates were 76.6+-6.2% and 56.7+-6.9% (p=0.045) in the MSC and non-MSC groups, respectively. Conclusion: In multivariate analysis, graft failure was the only adverse predictor for OS. Meanwhile, graft failure, grades III-IV acute GVHD, and moderate-severe chronic GVHD could predict worse GFFS. Our results indicated that haplo-HSCT combined with UC-MSCs infusion was an effective and safe option for SAA patients.

Keywords

• severe aplastic anemia
• haploidentical
• hematopoietic stem cell transplantation
• mesenchymal stem cells