Outpatient ATG-free hematopoietic transplantation for aplastic anemia in limited-resource environments offers excellent results: Data from a single LATAM center

• Published in: Blood cells, molecules, & diseases Vol. 109; p. 102885
• Main Authors: Jaime-Pérez, José Carlos, González-Treviño, Mariana, Gómez-De León, Andrés, Campos-Bocardo, Miguel A., Barragán-Longoria, Renata V., Cantú-Rodríguez, Olga Graciela, Gutiérrez-Aguirre, César Homero, Gómez-Almaguer, David
• Format: Journal Article
• Language: English
• Published: United States Elsevier Inc 01-11-2024
• Subjects: Ambulatory transplant; Hematopoietic cell transplant; Outpatient HSCT; Peripheral blood HSCT; Severe aplastic anemia; T-cell replete graft; Outpatient HSCT; Severe aplastic anemia; Peripheral blood HSCT; Hematopoietic cell transplant; T-cell replete graft; Ambulatory transplant
• DOI: 10.1016/j.bcmd.2024.102885
• EISSN: 1096-0961
• ISSN: 1079-9796; 1096-0961

To document the results of outpatient hematopoietic stem cell transplantation (HSCT) from the peripheral blood (PB) of sibling donors without anti-thymocyte globulin (ATG) in the conditioning regimen.
Patients from a low-income population with severe AA who received a PB, unmanipulated sibling HLA-identical HSCT between 2000 and 2020 at a single institution were studied. Survival was the primary outcome.
Forty-one transplants were performed. Time between diagnosis and transplant was five months (1–104). Median age was 37 (range, 4–61) years; 25 (61 %) recipients were males and 32 (78 %) had treatment failure, 9 (22 %) have not received treatment. ATG was administered in 5 (12.2 %) cases; the graft source was PB in 38 (92.7 %) transplants. Twenty-six (63.4 %) transplants were carried out in the outpatient setting. Infections developed in 14 (34.1 %) patients. Primary graft failure (GF) occurred in 3 (7.3 %) patients. The 15-year OS was 81 %, EFS was 77.4 %. Patients with high pre-HSCT transfusion burden had lower OS (p = 0.035) and EFS (p = 0.026). Previous treatment failure and age were not associated with lower OS (p = 0.115, p = 0.069) or EFS (p = 0.088, p = 0.5, respectively).
HLA-identical T-cell replete outpatient HSCT from the PB of sibling donors for AA patients using ATG-free conditioning offers excellent long-term survival.
•Delayed AA diagnosis to hematopoietic transplant is associated with poorer results.•Transplant outcomes without ATG in the conditioning regimen are acceptable.•Unmanipulated peripheral blood can be used successfully in allografting AA patients.•Peripheral blood HSCT in severe AA is a valid option when BMT is not available.