Anti-thymocyte globulin combined with post-transplantation cyclophosphamide reduce graft-versus-host disease in hematopoietic stem cell transplantation for pediatric leukemia

Abstract

This retrospective analysis evaluated the use of anti-thymocyte globulin (ATG) with or without post-transplantation cyclophosphamide (PTCy) for graft-versus-host disease (GvHD) prophylaxis in children with acute leukemia undergoing hematopoietic stem cell transplantation (HSCT). The study included 57 children, with 35 in the ATG-PTCy group and 22 in the ATG group. While overall incidence of acute and chronic GvHD did not differ significantly between groups, the ATG-PTCy group had lower rates of grade II-IV acute GvHD (p = 0.013) and moderate-to-severe chronic GvHD (p = 0.001) compared to the ATG group. Importantly, ATG-PTCy significantly improved GvHD/relapse-free survival (GRFS) compared to ATG (65.71% vs. 36.63%; p = 0.003). There were no differences in engraftment, infection rates, immune reconstitution, overall survival, leukemia-free survival, relapse rate, or non-relapse mortality between the two groups. Combining ATG with PTCy may reduce moderate-to-severe GvHD and improve GRFS in children undergoing HSCT for acute leukemia.

Keywords:

• Acute leukemia
• child
• graft-versus-host disease
• anti-thymocyte globulin
• post-transplantation cyclophosphamide

Acknowledgments

We thank the team of the Hematology Department of the Capital Institute of Pediatrics for their collaboration. We would like to extend our sincere gratitude to Professor Xiaohui Li for her invaluable guidance to this research and to the authors involved. Her expertise and insights have been instrumental in shaping our work and we are deeply appreciative of her contributions.

Authors’ contributions

HMZ carried out the studies, participated in collecting data and statistical analysis, and drafted the manuscript. LR participated in the study design and revised the manuscript. LJH, HT, ZZX, FSQ, and XLT participated in patient management and the acquisition and interpretation of data. All authors read and approved the final manuscript.

Disclosure statement

The authors report there are no competing interests to declare

Ethics approval

This work has been carried out in accordance with the Declaration of Helsinki (2000) of the World Medical Association. This study was approved by the Ethics Committee of the Capital Institute of Pediatrics [SHERLLM2023037]. This article is a retrospective study. Therefore, the Ethics Committee waived the requirement to obtain distinct written informed consent from the patients.

Data availability statement

All data generated or analyzed during this study are included in this published article and its supplementary information file.

Additional information

Funding

The author(s) reported there is no funding associated with the work featured in this article.