Improvement of overall survival after allogeneic hematopoietic stem cell transplantation for children and adolescents: a three-decade experience of a single institution

Abstract

International audienceAllogeneic stem cell transplantation (allo-SCT) has become an essential component of the treatment for a variety of diseases in pediatric patients. During the past decades, advances in the transplant technology, availability of hematopoietic stem cells and supportive care not only have resulted in improved outcomes, but also have expanded the transplant options. However, these features have been studied mainly in adult populations. This investigation analyzed changes in patient profile, transplantation, graft characteristics and outcome among 250 children and adolescent patients who received allo-SCT in a single center between 1983 and 2010. In the 2000–2010, compared with the 1983–1999 period, a significantly higher 5-year overall survival (64% versus 52%, P=0.03) was observed together with a significant decrease of non-relapse mortality (27% versus 9%, P=0.0002). The progression-free survival was comparable between the two periods (49% versus 57%; P=0.17). The 5-year cumulative incidence of relapse was 24% between 1983 and 1999, and 34% between 2000 and 2010 (P=0.08). Major advances in supportive care practice have been made over the past decade, resulting in a significant survival benefit for the pediatric population undergoing allo-SCT. However, post-transplant relapse remains the leading cause of failure of this therapeutic approach, and preventing relapse represents a major challenge toda

Reduction in Mortality after Umbilical Cord Blood Transplantation in Children Over a 20-Year Period (1995-2014)

“…Gooley et al [12] reported that mortality among children and adults after allogeneic stem cell transplantation at the Fred Hutchinson Cancer Research Center fell 52% from 1993 to 1997 to 2003 to 2007. Similarly, 5-year overall survival among children who underwent allogeneic HSCT at a single institution improved from 52% to 64% between 1983 to 1999 and 2000 to 2010 [13]. Finally, using data reported to the Center for International Blood and Marrow Transplant Research, Horan et al [14] found that the 2-year overall survival of children after unrelated donor bone marrow transplantation improved from 35% in 1987 to 1995 to 58% in 2003 to 2006.…”

“…Finally, using data reported to the Center for International Blood and Marrow Transplant Research, Horan et al [14] found that the 2-year overall survival of children after unrelated donor bone marrow transplantation improved from 35% in 1987 to 1995 to 58% in 2003 to 2006. These studies consisted mostly of adults, were limited to patients with hematologic malignancies, included relatively few UCBT recipients, or did not include data from the past 10 years [12][13][14][15][16]. In this study we present detailed data on causes of death in the largest single-center cohort of pediatric UCBT recipients reported to date.…”

Long-Term Effects of Haematopoietic Stem Cell Transplantation after Pediatric Cancer: A Qualitative Analysis of Life Experiences and Adaptation Strategies

“…From a medical perspective, different authors showed over the last 25 years an increase in HSCT complexity but also major advances in supportive care and a refinement of donor selection (Mateos et al, 2013; Brissot et al, 2016; Svenberg et al, 2016). All these developments have probably impacted on the physical and psychosocial consequences following HSCT after a pediatric cancer.…”

Alternative-Donor Hematopoietic Stem Cell Transplantation with Post-Transplantation Cyclophosphamide for Nonmalignant Disorders

“…Advances in supportive care have greatly improved the success of allo-HSCT for this patient population [1, 2]. However, the use of HSCT is limited by the lack of human leukocyte antigen (HLA)-matched donors; only 50% of children in need of an allo-HSCT have an HLA-matched donor, and this number is less than 20% in some minority populations [3].…”