Impact of Conditioning Regimen and Graft-versus-Host Disease Prophylaxis on The Outcome of Haploidentical Peripheral Blood Stem Cell Transplantation for High-Risk Severe Aplastic Anemia in Children and Young Adults: A Report from the Pediatric Severe Aplastic Anemia Consortium of India

• DOI: 10.1016/j.jtct.2022.12.010
• Publication Date: 2022-12-23T11:00:39Z
• Authors: Gaurav Kharya; Sarita R. Jaiswal; Sunil Bhat; Revathi Raj; Satya P. Yadav; Vikas Dua; Santanu Sen; Atish Bakane; Shobha Badiger; Ramya Uppuluri; Neha Rastogi; Mansi Sachdev; Bharti Sharma; Ashraf Saifullah; Suparno Chakrabarti
• Keywords: Aplastic anemia; ThioTEPA; Melphalan; Regimen; Total body irradiation

Allogenic hematopoietic cell transplantation (HCT) is the best curative approach for patients with severe aplastic anemia (SAA). The outcomes of HCT from haploidentical family donors (HFDs) have improved, making it a feasible option lacking an HLA-identical donor. However, data on HFD-HCT younger SAA sparse. In this multicenter retrospective study, we evaluated 79 undergoing SAA. All were heavily pretransfused, median time to was >12 months, and 67% had failed previous therapies. Conditioning based fludarabine (Flu)-cyclophosphamide (Cy)-antithymocyte globulin (ATG)/total body irradiation (TBI) or without thiotepa/melphalan (TT/Mel). Post-transplantation Cy (PTCy) calcineurin inhibitors (CNIs)/sirolimus used as graft-versus-host disease (GVHD) prophylaxis abatacept. rate primary graft failure (PGF) 16.43% overall, lower in conditioned TT/Mel. incidences acute chronic GVHD 26.4% 18.9%, respectively. At follow-up 48 overall survival (OS) event-free (EFS) 61.6% 58.1%, Both OS EFS better TT/Mel recipients abatacept prophylaxis. On multivariate analysis, use found favorably impact outcome variables, including EFS. Our study suggests that PTCy-based reasonable young high-risk SAA, whom optimization conditioning might further improve outcomes.