# Improving Outcomes Assessment in Chronic Graft versus Host Disease

> **NIH NIH R01** · FRED HUTCHINSON CANCER CENTER · 2024 · $681,867

## Abstract

PROJECT SUMMARY
Allogeneic hematopoietic cell transplantation (HCT) can cure many hematologic cancers and other life-
threatening hematologic diseases but 20-50% of survivors develop chronic graft-versus-host disease (cGVHD),
the leading cause of morbidity and mortality in transplant survivors. Chronic GVHD is an iatrogenic
complication that can affect multiple organs, leading to clinical manifestations similar to autoimmune diseases
such as systemic sclerosis, systemic lupus erythematosus, Sjogren’s syndrome, and lichen planus. Chronic
GVHD requires prolonged treatment with potent immunosuppressive agents and is associated with high
symptom burden and poor quality of life. The precise pathophysiology is unclear in humans. There are data
supporting involvement of T and B cells, macrophages, dendritic cells, fibroblasts, endothelial cells, cytokines,
chemokines and other proteins. However, information is derived from studies with few patients having
heterogeneous clinical manifestations. This renewal application will address key gaps in our understanding:
Can we identify biologically relevant cGVHD subgroups by studying large numbers of patients and using
sophisticated analytic techniques to identify “clusters” of similar patients? Is personalized medicine possible
based on knowledge of underlying pathophysiology and the likelihood of response? To address these
questions, we will use our extensive biorepository to test whether plasma proteins and peripheral blood cellular
populations cluster with clinical manifestations. While these studies are ongoing, a new cohort will be enrolled
to prospectively test the cluster findings and to investigate early biomarkers for treatment response.
Participants will be enrolled prior to starting a new systemic initial or second-line cGVHD treatment, then
followed at 1, 3 and 6 months later to assess clinical response. Successful completion of these aims will
advance our understanding of the biologic underpinnings of the different forms of human cGVHD and guide
therapeutic approaches, in order to decrease the morbidity and mortality of this common transplant
complication.

## Key facts

- **NIH application ID:** 10817093
- **Project number:** 5R01CA118953-15
- **Recipient organization:** FRED HUTCHINSON CANCER CENTER
- **Principal Investigator:** STEPHANIE J LEE
- **Activity code:** R01 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2024
- **Award amount:** $681,867
- **Award type:** 5
- **Project period:** 2007-07-18 → 2026-03-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10817093

## Citation

> US National Institutes of Health, RePORTER application 10817093, Improving Outcomes Assessment in Chronic Graft versus Host Disease (5R01CA118953-15). Retrieved via AI Analytics 2026-05-23 from https://api.ai-analytics.org/grant/nih/10817093. Licensed CC0.

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