Unique Reduced-Intensity Conditioning Haploidentical Peripheral Blood Stem Cell Transplantation Protocol for Patients
Jianli Xu1, Wenyan Miao1, Hailong Yuan1
1Hematologic Disease Center, First Affiliated Hospital of Xinjiang Medical University, Xinjiang Uygur Autonomous Region Research Institute of Hematology, Urumqi 830061, Xinjiang, China.
Transplantation and Cellular Therapy
|February 12, 2023
Summary
This study shows that reduced-intensity conditioning haploidentical hematopoietic stem cell transplantation using high-dose peripheral blood stem cells is effective for hematologic malignancies, with manageable graft-versus-host disease and good survival rates.
Area of Science:
- Hematology
- Oncology
- Immunology
Background:
- Reduced-intensity conditioning (RIC) haploidentical (haplo-) hematopoietic stem cell transplantation (HSCT) requires optimized graft sources for engraftment and immune reconstitution.
- Peripheral blood stem cells (PBSCs) offer advantages, but high-dose, non-T cell-depleted (non-TCD) PBSCs may increase graft-versus-host disease (GVHD) risk.
Purpose of the Study:
- To investigate the efficacy and safety of a novel RIC-haplo-HSCT protocol using high-dose, non-TCD in vitro PBSCs in intermediate to high-risk hematologic malignancies.
- To evaluate acute and chronic GVHD incidence, relapse rates, and survival outcomes.
Main Methods:
- Prospective, single-arm clinical study involving 47 patients (age 30-68) with hematologic malignancies.
- Conditioning regimen: fludarabine/Ara-C/busulfan (FAB).
- Graft source: high-dose non-TCD in vitro PBSCs. In vivo T-cell depletion with rabbit antithymocyte globulin and IL-2 receptor antagonist for GVHD prophylaxis.
Main Results:
- The 2-year cumulative incidence of grade II-IV acute GVHD was 30.3%, and grade III-IV was 10.2%.
- The 2-year cumulative incidence of chronic GVHD was 34.9% (localized 26.1%, extensive 8.7%).
- The 2-year cumulative incidence of relapse was 17.3%, overall survival was 71.2%, and disease-free survival was 66.2%.
Conclusions:
- The investigated RIC-haplo-PBSC transplantation protocol demonstrated effective treatment for hematologic malignancies with acceptable GVHD rates.
- The protocol shows promise, but further validation through larger prospective multicenter trials and experimental studies is warranted.
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