Clinical Research: Pediatric
Long-Term Morbidity and Mortality in Children with Chronic Graft-versus-Host Disease Classified by National Institutes of Health Consensus Criteria after Allogeneic Hematopoietic Stem Cell Transplantation

https://doi.org/10.1016/j.bbmt.2015.07.025Get rights and content
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Highlights

  • The National Institutes of Health global score predicts the risk of nonrelapse mortality and disease-free survival in children with chronic graft-versus-host disease

  • Children with severe chronic graft-versus-host disease had significantly higher nonrelapse mortality and lower disease-free survival

  • Probability of continuing chronic graft-versus-host disease 8 years after onset of severe chronic graft-versus-host disease was 36%

  • Among survivors of more than 5 years, 22% had a performance score below 70%

  • Efforts to lower the risk of severe chronic graft-versus-host disease are necessary in pediatric hematopoietic stem cell transplantation

Abstract

We report the long-term morbidity and mortality of 105 pediatric patients who developed chronic graft-versus-host disease (cGVHD) after allogeneic hematopoietic stem cell transplantation (HSCT). According to the consensus criteria of the National Institutes of Health, the global severity of cGVHD was mild in 26 patients (25%), moderate in 30 patients (29%), and severe in 49 patients (47%). Patients with severe cGVHD had a significantly lower cumulative incidence of cGVHD remission and higher probability of continuing cGVHD at 8 years from cGVHD diagnosis compared with those with mild or moderate cGVHD. The 10-year cumulative incidence of nonrelapse mortality in severe cGVHD patients was significantly higher and the probability of disease-free survival was significantly lower than those among patients with mild and moderate cGVHD. Of the 59 patients who survived for more than 5 years, 20 (34%) (4 with moderate and 16 with severe cGVHD) had persistent functional impairment caused by cGVHD with a Karnofsky/Lansky performance score of 90% in 3 patients, 80% in 4 patients, and below 70% in 13 patients at the time of relapse, death, or last follow-up. Better therapeutic strategies are needed to lower the incidence of severe cGVHD, considering the longer life expectancy of pediatric HSCT survivors.

Key Words

Chronic graft-versus-host disease
Children
Nonrelapse mortality
Functional impairment
Performance status
National Institutes of Health consensus criteria

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Financial disclosure: See Acknowledgments on page 1979.