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Steroid Refractory Chronic Graft-Versus-Host Disease: Cost-Effectiveness Analysis
Fevzi F Yalniz1, Mohammad H Murad2, Stephanie J Lee3
1Division of Hematology, Department of Medicine, Mayo Clinic, Rochester, Minnesota.
Summary
Methotrexate is the most cost-effective treatment for chronic graft-versus-host disease (cGVHD), offering significant savings compared to other agents. This analysis highlights the economic disparities among treatments for cGVHD.
Area of Science:
- Hematology
- Oncology
- Pharmacoeconomics
Background:
- Chronic graft-versus-host disease (cGVHD) presents a growing clinical challenge with escalating treatment costs.
- Multiple lines of drug therapy are often required, increasing the economic burden on healthcare systems.
- Steroid-refractory cGVHD necessitates evaluation of alternative treatment strategies.
Purpose of the Study:
- To conduct a meta-analysis assessing the comparative effectiveness of various treatments for cGVHD.
- To perform a cost-effectiveness analysis of frequently used agents in steroid-refractory cGVHD.
- To identify the most economically viable treatment options for different organ manifestations of cGVHD.
Main Methods:
- Systematic literature search for studies on tacrolimus, sirolimus, rituximab, ruxolitinib, hydroxychloroquine, imatinib, bortezomib, ibrutinib, extracorporeal photopheresis, pomalidomide, and methotrexate.
- Inclusion criteria: median follow-up ≥ 6 months, ≥ 5 patients.
- Meta-analysis of overall response (ORR), complete response (CR), and partial response (PR); calculation of cost per CR and cost per CR+PR based on 6-month direct treatment costs.
Main Results:
- Forty-one studies with 1047 patients were included. CR rates varied from 7% (rituximab) to 30% (methotrexate); ORR ranged from 30% (tacrolimus) to 85% (ruxolitinib).
- Cost per CR ranged from US$680 (methotrexate) to US$1,187,657 (ruxolitinib). Cost per ORR ranged from US$453 (methotrexate) to US$242,236 (ibrutinib).
- Methotrexate was the most cost-effective for all organ systems. Pomalidomide and imatinib were among the least cost-effective for specific manifestations.
Conclusions:
- Significant cost-effectiveness differences exist among available cGVHD treatments.
- Methotrexate emerges as the most cost-effective strategy across all organ systems.
- Economic considerations are crucial for sequencing treatments in cGVHD management, given potential treatment cycling.
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