Cost–effectiveness of treatments for high-risk myelodysplastic syndromes after failure of first-line hypomethylating agent therapy

Abstract

Purpose: To evaluate optimal salvage therapy in high-risk myelodysplastic syndromes patients who have failed a first-line hypomethylating agent (HMA) therapy, given that treatment choice is challenging. Methods: Using published literature and expert opinion, we developed a Markov model to evaluate the cost–effectiveness of current treatments for patients who failed first-line HMA therapy. The model predicted costs, life years, quality-adjusted life years and incremental cost-effectiveness ratios. Sensitivity analyses were conducted to assess the impact of uncertainty in model inputs. Results: Supportive care was the least expensive option ($65,704/patient) with the shortest survival (0.48 years). Low- and high-intensity chemotherapies and hematopoietic cell transplantation increased survival and costs with incremental cost-effectiveness ratios of $108,808, 306,103 and 318,163/life year, respectively. Switching HMA was more costly and less efficacious than another treatment option, namely low-intensity chemotherapy. Conclusions: Subsequent treatments in myelodysplastic syndrome patients who failed first-line HMA significantly increase costs, while only providing marginal clinical benefit and substantially increasing treatment-related morbidities. Additional treatment options would benefit resource allocation, clinical decision-making and patient outcomes.

Keywords:

• cost–effectiveness analysis
• economic modeling
• health care resource utilization
• hypomethylating agents
• myelodysplastic syndromes

Financial & competing interests disclosure

Onconova Therapuetics, Inc. funded the research described in the manuscript. TJ McKearn and ME Petrone are employees of Onconova Therapuetics, Inc. and at the time the analyses were conducted S Megaffin was also an employee – he is now President of Churchill Pharmaceuticals. TGK Bentley, JD Ortendahl and AM Anene are employees of Partnership for Health Analytic Research, LLC, which received payment from Onconova Therapeutics, Inc. to conduct the analyses described in this manuscript. CR Cogle and S Mukherjee have consulted with Partnership for Health Analytic Research, LLC. The authors have no other relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript apart from those disclosed. Peer reviewers on this manuscript were reimbursed for their time.