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

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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.

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Authors and Affiliations

  1. Centre for Evidence-Based Practice, Belgian Red Cross, Mechelen, Belgium

    Hans Van Remoortel, Hans Scheers, Bert Avau & Emmy De Buck

  2. Department of Public Health and Primary Care, Leuven Institute for Healthcare Policy, KU Leuven, Leuven, Belgium

    Hans Van Remoortel, Hans Scheers, Emmy De Buck & Philippe Vandekerckhove

  3. Department of Clinical Immunology, South Danish Transfusion Service and Tissue Center, Odense University Hospital, Odense, Denmark

    Jørgen Georgsen

  4. Faculty of Medicine, University of Alberta, Edmonton, Canada

    Susan Nahirniak

  5. Transfusion and Transplantation Medicine, Alberta Precision Laboratories, Alberta, Canada

    Susan Nahirniak

  6. Departments of Medicine, Laboratory Medicine and Pathobiology, Institute of Health Policy Management and Evaluation, University of Toronto, Mount Sinai Hospital, Toronto, Canada

    Nadine Shehata

  7. Transfusion Medicine, NHS Blood and Transplant, Oxford, UK

    Simon J. Stanworth

  8. Radcliffe Department of Medicine, University of Oxford, NIHR Oxford Biomedical Research Centre, Oxford, UK

    Simon J. Stanworth

  9. Department of Haematology, Oxford University Hospitals NHS Foundation Trust, Oxford, UK

    Simon J. Stanworth

  10. Blood Services, Belgian Red Cross, Mechelen, Belgium

    Veerle Compernolle

  11. Faculty of Medicine and Health Sciences, Ghent University, Ghent, Belgium

    Veerle Compernolle

  12. Belgian Red Cross, Mechelen, Belgium

    Philippe Vandekerckhove

  13. Division of Epidemiology and Biostatistics, Department of Global Health, Faculty of Medicine and Health Sciences, Stellenbosch University, Stellenbosch, South Africa

    Philippe Vandekerckhove

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  1. Hans Van Remoortel
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Correspondence to Hans Van Remoortel.

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Funding

This work is supported in part by a grant from the European Blood Alliance (grant 2021-01) and in part by structural support from the Foundation for Scientific Research of the Belgian Red Cross. The sponsors had no part in the design of this protocol. The contents of this document do not necessarily reflect the views and policies of the European Blood Alliance, nor does the mentioning of trade names or commercial products constitute endorsement or recommendation of use.

Conflict of interest

Relevant financial conflicts of interest directly related to this review: All authors declared not having any relevant direct financial conflict of interest. Relevant financial conflicts of interest not directly related to this review: HV, HS, BA, EDB, VC and PV are employees of Belgian Red Cross-Flanders, which is responsible for supplying adequate quantities of safe blood products to hospitals in Flanders and Brussels on a continuous basis and is funded by the Ministry of Social Affairs. Belgian Red Cross-Flanders received a grant from the European Blood Alliance to conduct this review. SS is an employee of NHS Blood and Transplant, which is the blood service supplier for England and manufactures platelets, and Chair the International Collaboration for Transfusion Medicine Guidelines (ICTMG). JG and SN declared not having any other financial conflicts of interest. NS received personal fees from Canadian Blood Services for the International Collaboration for Transfusion Medicine Guidelines (ICTMG) (platelet guideline). SS received grant awards for multiple trials of platelets and tranexamic acid. Relevant intellectual conflicts of interest. HV, HS, BA, JG, EDB, VC and PV declared not having any intellectual conflict of interest. NS, SN and SS declared to be involved in the ICTMG platelet guideline (SS is the chair, SN is the co-chair).

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All (secondary) data generated or analysed during this study are included in this published article and its supplementary information files.

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Author contributions

HVR: conceptualization, investigation, formal analysis, writing—original draft, visualization, supervision, project administration, funding acquisition. HS: investigation, formal analysis, writing—review and editing, visualization. BA: conceptualization, investigation, writing—review and editing, supervision, project administration, funding acquisition. JG: validation, writing—review and editing. SN: validation, writing—review and editing. NS: validation, writing—review and editing. SS: validation, writing—review and editing. EDB: conceptualization, writing—review and editing, supervision. VC: conceptualization, writing—review and editing, supervision. PV: conceptualization, resources, writing—review and editing, supervision.

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Van Remoortel, H., Scheers, H., Avau, B. et al. Cost-Effectiveness of Thrombopoietin Mimetics in Patients with Thrombocytopenia: A Systematic Review. PharmacoEconomics 41, 869–911 (2023). https://doi.org/10.1007/s40273-023-01271-w

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