Risk factors associated with increased nonrelapse mortality and with poor overall survival in children with chronic graft-versus-host disease
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Updated: May 29, 2026

Bone Marrow Transplantation Platform to Investigate the Role of Dendritic Cells in Graft-versus-Host Disease
Published on: March 17, 2020
David A Jacobsohn1, Mukta Arora, John P Klein
1Division of Blood and Marrow Transplantation Center for Cancer and Blood Disorders, Children's National Medical Center, Washington, DC 20010, USA. dajacobs@cnmc.org
Insights
Identifying risk factors for chronic graft-versus-host disease (cGVHD) in pediatric transplant patients is crucial. Key factors for nonrelapse mortality and survival include HLA matching, graft type, and patient condition at diagnosis.
Area of Science:
- Hematology
- Immunology
- Pediatric Oncology
Background:
- Limited data exists on factors influencing nonrelapse mortality (NRM) and overall survival in pediatric patients with chronic graft-versus-host disease (cGVHD).
- Understanding these factors is vital for improving outcomes in this vulnerable population.
Purpose of the Study:
- To identify risk factors for NRM and survival in pediatric allograft recipients diagnosed with cGVHD.
- To provide data for risk stratification, clinical trial design, and patient counseling.
Main Methods:
- Multivariate analysis of data from 1117 pediatric subjects with leukemia or myelodysplastic syndrome.
- Analysis included patients transplanted from related donors, unrelated donors (URD), or unrelated cord blood (1995-2004).
- Identified risk factors for NRM and survival post-cGVHD diagnosis.
Main Results:
- Four variables linked to higher NRM: HLA partially matched/mismatched URD, peripheral blood stem cell graft, low Karnofsky/Lansky score (<80), and low platelet count (<100 × 10^9/L).
- Worse survival associated with: age >10 years, HLA partially matched/mismatched URD, advanced disease, low Karnofsky/Lansky score, and low platelet count.
- Cumulative incidence of discontinuing immunosuppression at 1, 3, and 5 years was 22%, 34%, and 37% respectively.
Conclusions:
- This study identifies key variables affecting outcomes in pediatric cGVHD patients.
- These findings can aid in risk stratification, clinical trial development, and family counseling.
- Further research can refine management strategies for pediatric cGVHD.
Abstract:
There is a paucity of information regarding the factors that affect nonrelapse mortality (NRM) and overall survival among children that develop chronic graft-versus-host disease (cGVHD). We performed multivariate analyses using data from the Center for International Blood and Marrow Transplant Research to identify risk factors for NRM and survival in 1117 pediatric subjects with leukemia or myelodysplastic syndrome, transplanted from related donors, unrelated donors (URD), or unrelated cord blood between 1995 and 2004. We identified 4 variables associated with higher NRM: HLA partially matched or mismatched URD, peripheral blood cell graft, Karnofsky/Lansky score < 80 at cGVHD diagnosis, and platelets < 100 × 10(9)/L at cGVHD diagnosis. Factors associated with significantly worse survival were: age > 10 years, transplantation from HLA partially matched or mismatched URD, advanced disease at transplantation, Karnofsky/Lansky < 80; and platelets < 100 × 10(9)/L. Cumulative incidence of discontinuation of systemic immune suppression at 1, 3, and 5 years after diagnosis of cGVHD were 22% (20%-25%), 34% (31%-37%), and 37% (34%-40%), respectively. This is the largest study elucidating variables affecting outcome after diagnosis of cGVHD in pediatric allograft recipients. These variables may be useful for risk stratification, development of future clinical trials, and family counseling in children with cGVHD.
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