Cost-Effectiveness of Thrombopoietin Mimetics in Patients with Thrombocytopenia: A Systematic Review

Hans Van Remoortel (), Hans Scheers, Bert Avau, Jørgen Georgsen, Susan Nahirniak, Nadine Shehata, Simon J. Stanworth, Emmy De Buck, Veerle Compernolle and Philippe Vandekerckhove
Additional contact information
Hans Van Remoortel: Belgian Red Cross
Hans Scheers: Belgian Red Cross
Bert Avau: Belgian Red Cross
Jørgen Georgsen: Odense University Hospital
Susan Nahirniak: University of Alberta
Nadine Shehata: University of Toronto, Mount Sinai Hospital
Simon J. Stanworth: NHS Blood and Transplant
Emmy De Buck: Belgian Red Cross
Veerle Compernolle: Belgian Red Cross
Philippe Vandekerckhove: Leuven Institute for Healthcare Policy, KU Leuven

PharmacoEconomics, 2023, vol. 41, issue 8, No 4, 869-911

Abstract: 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.

Date: 2023
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DOI: 10.1007/s40273-023-01271-w

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