Author:

Keywords:

Social Sciences, Science & Technology, Life Sciences & Biomedicine, Economics, Health Care Sciences & Services, Health Policy & Services, Pharmacology & Pharmacy, Business & Economics, PRIMARY IMMUNE THROMBOCYTOPENIA, PER-RESPONDER ANALYSIS, DOUBLE-BLIND, INTRAVENOUS IMMUNOGLOBULIN, PLATELET COUNTS, ELTROMBOPAG, ROMIPLOSTIM, RITUXIMAB, PURPURA, GUIDELINES, Adult, Humans, Thrombopoietin, Cost-Benefit Analysis, Thrombocytopenia, Hemorrhage, Quality-Adjusted Life Years, 11 Medical and Health Sciences, 14 Economics, 3214 Pharmacology and pharmaceutical sciences, 3801 Applied economics, 4203 Health services and systems

Abstract:

OBJECTIVES: Thrombopoietin (TPO) mimetics are a potential alternative to platelet transfusion to minimize blood loss in patients with thrombocytopenia. This systematic review aimed to evaluate the cost-effectiveness of TPO mimetics, compared with not using TPO mimetics, in adult patients with thrombocytopenia. METHODS: Eight databases and registries were searched for full economic evaluations (EEs) and randomized controlled trials (RCTs). Incremental cost-effectiveness ratios (ICERs) were synthesized as cost per quality-adjusted life year gained (QALY) or as cost per health outcome (e.g. bleeding event avoided). Included studies were critically appraised using the Philips reporting checklist. RESULTS: Eighteen evaluations from nine different countries were included, evaluating the cost-effectiveness of TPO mimetics compared with no TPO, watch-and-rescue therapy, the standard of care, rituximab, splenectomy or platelet transfusion. ICERs varied from a dominant strategy (i.e. cost-saving and more effective), to an incremental cost per QALY/health outcome of EUR 25,000-50,000, EUR 75,000-750,000 and EUR > 1 million, to a dominated strategy (cost-increasing and less effective). Few evaluations (n = 2, 10%) addressed the four principal types of uncertainty (methodological, structural, heterogeneity and parameter). Parameter uncertainty was most frequently reported (80%), followed by heterogeneity (45%), structural uncertainty (43%) and methodological uncertainty (28%). CONCLUSIONS: Cost-effectiveness of TPO mimetics in adult patients with thrombocytopenia ranged from a dominant strategy to a significant incremental cost per QALY/health outcome or a strategy that is clinically inferior and has increased costs. Future validation and tackling the uncertainty of these models with country-specific cost data and up-to-date efficacy and safety data are needed to increase the generalizability.