Abstract

Information on incidence, and factors associated with mortality is a prerequisite to improve outcome after hematopoietic stem cell transplantation (HSCT). Therefore, 55′668 deaths in 114′491 patients with HSCT (83.7% allogeneic) for leukemia were investigated in a landmark analysis for causes of death at day 30 (very early), day 100 (early), at 1 year (intermediate) and at 5 years (late). Mortality from all causes decreased from cohort 1 (1980–2001) to cohort 2 (2002–2015) in all post-transplant phases after autologous HSCT. After allogeneic HSCT, mortality from infections, GVHD, and toxicity decreased up to 1 year, increased at 5 years; deaths from relapse increased in all post-transplant phases. Infections of unknown origin were the main cause of infectious deaths. Lethal bacterial and fungal infections decreased from cohort 1 to cohort 2, not unknown or mixed infections. Infectious deaths were associated with patient-, disease-, donor type, stem cell source, center, and country- related factors. Their impact varied over the post-transplant phases. Transplant centres have successfully managed to reduce death after HSCT in the early and intermediate post-transplant phases, and have identified risk factors. Late post-transplant care could be improved by focus on groups at risk and better identification of infections of “unknown origin”.

Highlights

  • Besides the risk of relapse, hematopoietic stem cell transplantation (HSCT) remains associated with significant early and late treatment related mortality (TRM)

  • This retrospective, observational study included all patients with HSCT from all donor types and stem cell sources for acute lymphoblastic leukemia (ALL), acute myeloid leukemia (AML) or chronic myeloid leukemia (CML) between 1980 and 2015, reported by 588 centers from 51 countries to the EBMT database

  • In allogeneic HSCT, they were successful in reducing deaths from graft-vs.-host disease (GVHD), infections and other causes in the very early and early post-transplant time phases, despite an increase in the patient pre-transplant risk profile

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Summary

Introduction

Besides the risk of relapse, hematopoietic stem cell transplantation (HSCT) remains associated with significant early and late treatment related mortality (TRM). The number of transplants has substantially increased [2,3,4], indications for HSCT have broadened [5], and new technologies have been introduced [4, 6,7,8]. It is estimated today that more than 1.4 million transplants have been performed so far worldwide; about 70′000 patients are being treated annually with HSCT, half of them in Europe [3]

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