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Published on: March 17, 2020
Durable discontinuation of systemic therapy in patients affected by chronic graft-versus-host disease
George L Chen1, Lynn Onstad2, Paul J Martin2
1Department of Medicine, Roswell Park Comprehensive Cancer Center, Buffalo, NY, USA. George.chen@roswellpark.org.
Insights
Most patients with chronic graft-versus-host disease (GvHD) require long-term systemic therapy (ST). Durable discontinuation of ST, indicating GvHD resolution, was achieved by 32% of patients over 10 years.
Area of Science:
- Hematology
- Immunology
- Oncology
Background:
- Chronic graft-versus-host disease (GvHD) is a significant complication following allogeneic stem cell transplantation.
- Long-term systemic therapy (ST) is often necessary for managing chronic GvHD.
- Durable discontinuation of ST is a key indicator of treatment success and disease resolution.
Purpose of the Study:
- To identify factors associated with achieving durable discontinuation of systemic therapy (ST) in patients with chronic graft-versus-host disease (GvHD).
- To estimate the cumulative incidence of durable ST discontinuation over time.
- To inform management strategies for chronic GvHD based on factors influencing treatment cessation.
Main Methods:
- Analysis of data from 684 patients in two prospective cohorts from the Chronic GvHD Consortium.
- Definition of durable ST discontinuation as cessation of all ST for at least 12 months.
- Multivariate analysis to assess predictors of durable ST discontinuation, including conditioning regimen, GvHD manifestation, symptom score, and graft source.
Main Results:
- The estimated cumulative incidence of durable ST discontinuation at 10 years was 32% (95% CI: 28%-37%).
- Factors associated with lower likelihood of discontinuation included myeloablative conditioning, moderate/severe lower gastrointestinal GvHD, and higher Lee symptom overall score.
- Mild lower gastrointestinal GvHD and cord blood as graft source were associated with a higher likelihood of ST discontinuation.
Conclusions:
- A minority of patients achieve durable discontinuation of systemic therapy for chronic GvHD.
- Most patients require prolonged systemic therapy, highlighting the chronic nature of the disease.
- Understanding factors influencing treatment cessation is crucial for optimizing chronic GvHD management.
Abstract:
Successful treatment of chronic graft-versus-host disease (GvHD) often requires long-term systemic therapy (ST). Durable discontinuation of ST reflects the resolution of active chronic GvHD. We evaluated the factors associated with durable ST discontinuation, defined as cessation of all ST for ≥12 months, using data from two prospectively followed cohorts from the Chronic GvHD Consortium (n=684). Transplant sources were peripheral blood (89%), bone marrow (6.6%), and cord blood (4.4%) from HLA matched related (37.6%), HLA matched unrelated (45%), and other donor types (18%). Half of the patients received non-myeloablative conditioning. The median time from transplantation to chronic GvHD diagnosis was 7.7 months (range, 1.0-141.3) and the median time from chronic GvHD onset to enrollment into the cohorts was 0.9 months (range, 0.0-12.0). The cumulative incidence estimate of durable ST discontinuation was 32% (95% confidence interval: 28%-37%) at 10 years after enrollment into the cohort. Among patients who discontinued ST, the median time from chronic GvHD diagnosis to durable ST discontinuation was 3.6 years (range, 1.2-10.5). In multivariate analysis, patients who received myeloablative conditioning, had chronic GvHD manifested as moderate/severe lower gastrointestinal involvement, and had a higher (worse) Lee symptom overall score were less likely to attain durable ST discontinuation. In contrast, mild lower gastrointestinal involvement and cord blood (vs. peripheral blood) as the graft source were associated with a greater likelihood of ST discontinuation. Although a minority of patients can discontinue ST permanently, most patients require prolonged ST. Viewing chronic GvHD in this way has implications for management approaches.
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