Improvement of survival after hematopoietic stem cell transplantation and trends at a pediatric transplantation center; a three-decade journey

Pediatr Transplant. 2024 Feb;28(1):e14691. doi: 10.1111/petr.14691.

Abstract

Background: Advances in stem cell transplantation have resulted in improved outcomes.

Methods: This is a retrospective study aimed to analyze changes in patient profile, transplantation, graft characteristics, and outcome among 241 pediatric patients who received stem cell transplantation in a single center between 1993 and 2019.

Results: In the 2010-2019, compared with the 1993-2009 period, a significantly higher 5-year overall survival (60% vs. 44%, p = .022) and an event-free survival (53% vs. 34%, p = .025) were observed. Cumulative incidence of deaths due to relapse or progression between the 1993-2009 and 2010-2019 periods were 33% and 26% respectively (p = .66). Cumulative incidence of non-relapse mortality was significantly higher during the 1993-2009 period compared with the 2010-2019 period for malignant diseases (57.7% vs. 28.3%, p = .007). The overall survival from acute graft-versus-host disease between 1993 and 2009 was 11% versus 46% between 2010 and 2019 (p = .0001). The overall survival from infection in both eras did not show any difference (p = .41).

Conclusions: Development in transplantation technology has led to a decrease in non-relapse mortality and better control of graft-versus-host disease. However, relapse and infection remained as major causes of death. Studies evaluating institutional trends in patients undergoing HSCT and analyzing their mortality profile, can improve the management of patients, leading to a reduction in transplant-related problems.

Keywords: event-free survival; hematopoietic stem cell transplantation; overall survival.

MeSH terms

  • Child
  • Graft vs Host Disease* / epidemiology
  • Graft vs Host Disease* / etiology
  • Hematopoietic Stem Cell Transplantation* / adverse effects
  • Humans
  • Recurrence
  • Retrospective Studies
  • Transplantation, Homologous / adverse effects