Skip to main page content
U.S. flag

An official website of the United States government

Dot gov

The .gov means it’s official.
Federal government websites often end in .gov or .mil. Before sharing sensitive information, make sure you’re on a federal government site.

Https

The site is secure.
The https:// ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely.

Access keys NCBI Homepage MyNCBI Homepage Main Content Main Navigation
. 2020 Jan;55(1):165-171.
doi: 10.1038/s41409-019-0651-9. Epub 2019 Sep 2.

Pediatric acute GVHD: clinical phenotype and response to upfront steroids

Margaret L MacMillan  1   2 , Shernan G Holtan  3   4 , Armin Rashidi  3   4 , Todd E DeFor  3   5 , Bruce R Blazar  3   6 , Daniel J Weisdorf  3   4
Affiliations

Pediatric acute GVHD: clinical phenotype and response to upfront steroids

Margaret L MacMillan et al. Bone Marrow Transplant. 2020 Jan.

Abstract

To better understand the clinical phenotype of acute graft-versus-host disease (GVHD) in children, we examined the GVHD clinical stage, grade, and response to prednisone 60 mg/m2/day PO in a diverse group of 370 pediatric patients with acute GVHD treated from 1990 to 2016 at a single institution. Overall response [complete response (CR) + partial response (PR)] at day 28 occurred in 65%, (CR 52%; PR 13%). Initial GVHD grade did not predict day 28 response. However, the Minnesota GVHD Risk Score predicted response with 68% standard risk (SR)-GVHD patients achieving CR/PR at day 28 versus 48% high risk (HR)-GVHD patients (p < 0.01). Multivariable analysis confirmed that response rates were lower in patients with HR-GVHD [odds ratio (OR), 0.4, p < 0.01] and in recipients of HLA mismatched URD (OR 0.4, p = 0.03). Transplant-related mortality (TRM) at 2 years was greater in HR-GVHD patients, recipients of HLA-partially matched or mismatched unrelated donor (URD) grafts, but not umbilical cord blood (UCB). These data highlight the importance of including children in novel acute GVHD treatment trials. Compared with initial GVHD grade, the Minnesota GVHD Risk Score better demarcates risk of steroid failure and TRM in children and could be used for risk stratification in pediatric acute GVHD studies.

PubMed Disclaimer

Conflict of interest statement

Conflict of interest The authors declare that they have no conflict of interest.

Figures

Fig. 1
Fig. 1
Day 28 response by Minnesota Risk group. CR = complete response. PR = partial response
Fig. 2
Fig. 2
Survival at 2 years after onset of steroid therapy by response to steroids at day 28
Fig. 3
Fig. 3
Cumulative incidence of TRM at 2 years after onset of steroid therapy by response to steroids at day 28
See this image and copyright information in PMC

References

    1. Carpenter PA, MacMillan ML. Management of acute graft-versu-shost disease in children. Pediatr Clin North Am. 2010;57:273–95. - PMC - PubMed
    1. Weisdorf D, Haake R, Blazar B, Miller W, McGlave P, Ramsay N, et al. Treatment of moderate/severe acute graft-versus-host disease after allogeneic bone marrow transplantation: an analysis of clinical risk features and outcome. Blood. 1990;75:1024–30. - PubMed
    1. MacMillan ML, Weisdorf DJ, Wagner JE, DeFor TE, Burns LJ, Ramsay NK, et al. Response of 443 patients to steroids as primary therapy for acute graft-versus-host disease: comparison of grading systems. Biol Blood Marrow Transplant. 2002;8:387–94. - PubMed
    1. Weisdorf D, Spellman S, Haagenson M, Horowitz M, Lee S, Anasetti C, et al. Classification of HLA-matching for retrospective analysis of unrelated donor transplantation: revised definitions to predict survival. Biol Blood Marrow Transplant. 2008;14:748–58. - PMC - PubMed
    1. Barker JN, Davies SM, DeFor T, Ramsay NK, Weisdorf DJ,Wagner JE. Survival after transplantation of unrelated donor umbilical cord blood is comparable to that of human leukocyte antigen-matched unrelated donor bone marrow: results of a matched-pair analysis. Blood. 2001;97:2957–61. - PubMed

Publication types