Safety and Feasibility Study of Reduced-Intensity Cyclophosphamide, Pentostatin, and Anti-Thymocyte Globulin Before Haploidentical Stem Cell Transplant for Severe Refractory or Recurrent Aplastic Anemia

What this Trial Is About

Researchers are evaluating a reduced-intensity treatment regimen including cyclophosphamide, pentostatin, and anti-thymocyte globulin before a CD4 T-cell depleted haploidentical hematopoietic cell transplant haploHCT for patients with severe aplastic anemia that is refractory or recurrent. This phase I trial aims to assess the safety and feasibility of this approach, including the ability to prepare the required cell product and reduce risks such as graft versus host disease. Participants receive cyclophosphamide both orally and intravenously, pentostatin intravenously, and anti-thymocyte globulin intravenously before undergoing the CD4 T-cell depleted haploHCT. The study includes procedures like bone marrow aspirate and biopsy, along with blood sample collection at screening and follow-up. The regimen is designed to prepare the patients body for the transplant by making room for healthy cells and modulating the immune response. During the study, participants will be monitored for adverse events, blood cell recovery, infections, graft versus host disease, survival rates, and donor cell engraftment over periods extending up to three years. Assessments include lab tests, bone marrow evaluations, and questionnaires. Researchers will also study immune cell profiles and bone marrow environment changes for up to two years after transplant to better understand treatment effects and long-term outcomes.

A Reduced-Intensity Conditioning Regimen (Cyclophosphamide, Pentostatin, Anti-thymocyte Globulin) Followed by Haploidentical Hematopoietic Stem Cell Transplant for the Treatment of Patients With Refractory or Recurrent Severe Aplastic Anemia