OBJECTIVES : Myelodysplastic syndromes (MDS) are clonal haematopoietic stem cell disorders characterised by ineffective haematopoiesis leading to blood cytopenias, with a high incidence of progression to acute myeloid leukaemia (AML). The objective of this study was to undertake a systematic literature review (SLR) of economic models to inform the design of a model for evaluating the cost-effectiveness of anaemia treatments in MDS.

METHODS : A SLR and a search of national reimbursement and technology appraisal (TA) organisations were conducted to identify economic evaluations of pharmacological interventions in the management of MDS. Electronic databases (MEDLINE, Embase, The Cochrane Library and EconLit) were searched from inception to July 2017. Data from eligible studies was extracted and assessed for methodological quality and applicability.

RESULTS : The SLR identified 19 studies and two TA reports describing 16 economic models. The majority (80%) utilised a Markov model structure with health states comprising: MDS, stratified by transfusion status (reduced dependence, dependent [+/- chelation therapy] or independent); progression to AML; and death. Models grouped differential MDS risk categories within a single MDS health state (low and intermediate risk MDS, n=5; intermediate and high risk, n=7; MDS del(5q), n=4). Cardiac, hepatic or diabetes related co-morbidities were also considered. Time-independent disease progression rates were typically derived from pivotal trials. The majority (n=8) employed a lifetime horizon. Health state costs comprised of drug, administration costs, testing (blood, liver, metabolic), transfusions, hospitalisation, physician visits, iron chelation and adverse events. Health state utilities varied considerably; mean (range) utilities for transfusion independent MDS, transfusion dependent MDS and AML were 0.88 (0.84 to 0.91), 0.56 (0.50 to 0.65) and 0.57 (0.45 to 0.67), respectively.

CONCLUSIONS : Given the structural consistency across published studies, a lifetime Markov model with MDS, AML and death health states and 28-day time cycle appears suitable for evaluating the cost-effectiveness of treatments for anaemia in MDS.