Steroid-refractory acute graft-versus-host disease graded III-IV in pediatric patients. A mono-institutional experience with a long-term follow-up

Pediatr Transplant. 2020 Nov;24(7):e13806. doi: 10.1111/petr.13806. Epub 2020 Aug 26.

Abstract

aGvHD remains a major obstacle to successful HSCT. We report our experience on steroid-refractory aGvHD III and IV from 1989 to 2017. Ninety patients with aGvHD III or IV were stratified according to the HSCT year: 1989-1998, 1999-2007, and 2008-2017 and to aGvHD extension (GvHD III vs IV) and finally the probability of OS, RI, and TRM was calculated accordingly. aGvHD III patients had a substantial improvement over time: day 100 OS raised from 64% (95% CI 39-89) in the first cohort to 100% in the latest (P = .022), and it was mainly due to a reduction of TRM (it was 28% [95% CI 12-65] in the first cohort to 0% in the latest (P = .01). The aGvHD IV patients did not present a significant improvement. Day 100 OS was 42% (95% CI 16-68) in the first group and 54% (95% CI 25-83) in the year 2008-2017 (P = NS), and the day-100 TRM was very similar (it was 57% [95% CI 36-90] in the first cohort and 45% [95% CI 23-89] in the latest (P = NS). We report significant improvements in OS and TRM in patients diagnosed with grade III aGvHD. Patients with the most severe aGvHD appear to have no or fewer benefits on long-term outcomes.

Keywords: children; severe acute graft-versus-host disease; survival.

MeSH terms

  • Acute Disease
  • Adolescent
  • Child
  • Child, Preschool
  • Drug Resistance*
  • Female
  • Follow-Up Studies
  • Glucocorticoids / pharmacology*
  • Graft vs Host Disease / diagnosis*
  • Graft vs Host Disease / drug therapy
  • Graft vs Host Disease / mortality
  • Hematologic Neoplasms / therapy*
  • Hematopoietic Stem Cell Transplantation / adverse effects*
  • Humans
  • Immunosuppressive Agents / therapeutic use*
  • Infant
  • Italy / epidemiology
  • Male
  • Prognosis
  • Retrospective Studies
  • Severity of Illness Index
  • Survival Rate / trends
  • Time Factors
  • Transplantation, Homologous

Substances

  • Glucocorticoids
  • Immunosuppressive Agents