A retrospective study of treosulfan versus busulfan‐based conditioning in pediatric patients. (21st July 2022)

• Record Type: Journal Article
• Authors: Olivas‐Mazón, Raquel; Bueno, David; Sisinni, Luisa; Mozo, Yasmina; Casado‐Abad, Gema; Martínez, Antonio Pérez
• Is Part Of: European journal of haematology. Volume 109:Number 5(2022)
• Page Start: 474
• Page End: 482
• Publication Date: 2022-07-21
• Subjects: busulfan -- HSCT‐conditioning -- pediatric -- treosulfan; Hematology -- Periodicals; Blood -- Diseases -- Periodicals; Blood -- Periodicals; 616.15005
• DOI: 10.1111/ejh.13828
• Languages: English
• ISSNs: 0902-4441

Abstract

Abstract: Objectives: To compare the outcomes of treosulfan‐based vs busulfan‐based conditioning regimens in allogeneic hematopoietic stem cell transplantation (HSCT) in pediatric patients. Methods: Retrospective study of all consecutive patients (2012–2019) treated with allogenic HSCT and treosulfan‐ or busulfan‐based conditioning regimens at a single center. Results: A total of 101 HSCT were included: 66 HSCT with busulfan and 35 with treosulfan. In malignant diseases ( n = 62), busulfan‐based conditioning was more commonly employed than treosulfan: 82.3% vs 17.7%. However, the use of treosulfan for malignant diseases increased over time: 6.5% of HSCT in 2012–2015 vs 29% of HSCT in 2015–2019 ( p = .02). The cohort of treosulfan had more children under 1‐year of age than the busulfan cohort (31 vs 13%; p = .033). The percentage of patients who received serotherapy was 73 and 89% in the nonmalignant and malignant groups, respectively. The engraftment, time to neutrophil, and platelet engraftment were not significantly different between the busulfan and the treosulfan cohorts. Rate of grade II‐IV acute GvHD was significantly higher in the busulfan cohort than the treosulfan cohort (39% vs 15%; p = .016). No differences were observed in endothelial damage complications, chronic GvHD, relapse, overall survival, and transplant‐related mortality. Conclusions: Busulfan‐based conditioning regimens are used more frequently for children undergoing allogenic HSCT, but treosulfan‐based conditioning is gaining acceptance. Treosulfan‐based conditioning is associated with lower rates of acute GvHD, and no significant differences on overall survival were observed compared with busulfan.