title: Cost-effectiveness of treatments for high-risk myelodysplastic syndromes after failure of first-line hypomethylating agent therapy.
abstract: OBJECTIVE
  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.
authors.affiliation: a Division of Hematology and Oncology, Department of Medicine, College of Medicine , University of Florida , Gainesville , FL , USA.
authors.forename: Christopher R
authors.initials: CR
authors.lastname: Cogle
authors.affiliation: b Partnership for Health Analytic Research, LLC , Beverly Drive , Beverly Hills , CA 90212 , USA.
authors.forename: Jesse D
authors.initials: JD
authors.lastname: Ortendahl
authors.affiliation: b Partnership for Health Analytic Research, LLC , Beverly Drive , Beverly Hills , CA 90212 , USA.
authors.forename: Tanya Gk
authors.initials: TG
authors.lastname: Bentley
authors.affiliation: b Partnership for Health Analytic Research, LLC , Beverly Drive , Beverly Hills , CA 90212 , USA.
authors.forename: Ayanna M
authors.initials: AM
authors.lastname: Anene
authors.affiliation: c Onconova Therapeutics, Inc , Newtown , PA , USA.|d Churchill Pharmaceuticals, LLC , King of Prussia , PA , USA.
authors.forename: Scott
authors.initials: S
authors.lastname: Megaffin
authors.affiliation: c Onconova Therapeutics, Inc , Newtown , PA , USA.
authors.forename: Thomas J
authors.initials: TJ
authors.lastname: McKearn
authors.affiliation: c Onconova Therapeutics, Inc , Newtown , PA , USA.
authors.forename: Michael E
authors.initials: ME
authors.lastname: Petrone
authors.affiliation: e Department of Hematology and Medical Oncology , Cleveland Clinic Taussig Cancer Institute , Cleveland , OH , USA.
authors.forename: Sudipto
authors.initials: S
authors.lastname: Mukherjee
mesh_terms: D000964:Antimetabolites, Antineoplastic / Q000191:economics / Q000627:therapeutic use*; D000066491:Clinical Decision-Making; D003362:Cost-Benefit Analysis; D006801:Humans; D008390:Markov Chains; D009190:Myelodysplastic Syndromes / Q000188:drug therapy* / Q000191:economics; D019057:Quality-Adjusted Life Years; D040841:Resource Allocation; D016879:Salvage Therapy / Q000191:economics / Q000379:methods*; D013534:Survival; D035501:Uncertainty
chemical_list: D000964:Antimetabolites, Antineoplastic
keywords: cost–effectiveness analysis; economic modeling; health care resource utilization; hypomethylating agents; myelodysplastic syndromes