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Published on: October 31, 2012
Chronic graft-versus-host disease: a prospective cohort study
Mukta Arora1, Linda J Burns, Stella M Davies
1Department of Medicine, Blood and Marrow Transplant Program, University of Minnesota, Minneapolis, Minnesota, USA.
Insights
Chronic graft-versus-host disease (CGVHD) significantly impacts bone marrow transplant survival. This study identified key mortality predictors and found that most patients require prolonged immunosuppression, highlighting the need for better treatments and infection prevention strategies.
Area of Science:
- Hematology
- Transplantation Immunology
- Oncology
Background:
- Chronic graft-versus-host disease (CGVHD) is a significant complication after allogeneic bone marrow transplantation (BMT), leading to substantial morbidity and mortality.
- Understanding the natural history and predictors of CGVHD is crucial for improving patient outcomes.
Purpose of the Study:
- To longitudinally characterize the natural history of CGVHD in 159 patients.
- To identify reliable predictors of treatment response and long-term mortality in CGVHD patients.
- To assess the prevalence of active CGVHD and the feasibility of discontinuing immunosuppressive therapy.
Main Methods:
- Longitudinal study of 159 patients with CGVHD.
- Analysis of treatment response rates at 6 months, 1 year, and 2 years.
- Multivariate analysis to identify predictors of mortality, including age, disease onset, platelet count, and gastrointestinal involvement.
- Assessment of infection incidence and survival rates over a median follow-up of 8.4 years.
Main Results:
- Treatment response rates were 61% at 6 months, 53% at 1 year, and 50% at 2 years.
- High incidence of infections observed, decreasing over time.
- Overall survival rate was 40% at a median follow-up of 8.4 years; 1-year and 10-year survival rates were 63% and 39%, respectively.
- Predictors of increased mortality included age >20 years, progressive CGVHD onset, platelet count <100,000/µL, and GI involvement.
- No response or partial response at 6 months predicted higher mortality.
- Prevalence of active CGVHD was 33% at 2 years, with only 13% successfully discontinuing therapy.
- Among patients with clinical resolution, only 18% were off immunosuppressants by 2 years, and 89% by 4 years.
Conclusions:
- Despite initial treatment responses, most CGVHD patients experience prolonged active disease requiring extended immunosuppression.
- Improved infection prevention is necessary due to the high incidence of infections.
- Identification of high-risk groups may allow for more intensive regimens, and novel treatment strategies are needed to improve survival and reduce toxicity.
Abstract:
Chronic graft-versus-host disease (CGVHD) is a major cause of morbidity and mortality following allogeneic bone marrow transplantation (BMT). We studied 159 patients with CGVHD longitudinally to characterize the natural history of CGVHD and identify reliable predictors of response and long-term mortality. Rates of response to treatment were 61%, 53%, and 50% at 6 months, 1 year, and 2 years, respectively. A high incidence of infections (7 of 1,000 patient-days at 0 to 6 months, 2.5 of 1,000 patient-days at 6 months to 1 year, and 0.6 of 1,000 patient-days at 1 to 2 years) was observed. After a median follow-up of 8.4 years, an overall survival rate of 40% was observed. The overall survival rate was 63% (95% confidence interval [CI], 56%-71%) at 1 year, 51% (95% CI, 43%-59%) at 2 years, and 39% (95% CI, 31%-47%) at 10 years. In multivariate analysis, age older than 20 years (RR = 1.5; 95% CI, 0.9%-2.5%; P =.09), progressive onset of CGVHD (RR = 1.6; 95% CI, 1.0%-2.4%; P =.04), platelet count of <100,000/ microL (RR = 2.1; 95% CI, 1.3%-3.4%; P =.001), and GI involvement (RR = 1.5; 95% CI, 1.0%-2.4%; P =.05) were associated with increased mortality. Among patients surviving more than 6 months, no response (RR = 4.5; 95% CI, 1.9%-10.5%; P =.0006) and partial response (RR = 2.5; 95% CI, 1.1%-6.1%; P =.04) to treatment at 6 months also were significant predictors of mortality. The prevalence of active CGVHD was 33% at 2 years. However, the cumulative incidence of successful discontinuation of therapy was only 13% at 2 years. Among patients with clinical resolution of CGVHD, only 18% were off immunosuppressive therapy by 2 years, and 89% by 4 years. Despite high initial response rates, a large majority of patients had active disease requiring prolonged immunosuppression. This requires improved infection prevention for a longer time. Recognition of a high-risk group should facilitate assignment of more intensified regimens. Better treatment regimens need to be identified to improve survival and limit toxicity of prolonged immunosuppression.
