The cost-effectiveness of thrombopoietin mimetics varies substantially in patients with thrombocytopenia


Thrombopoietin (TPO) mimetics are potential alternatives to platelet transfusion for reducing blood loss in patients with thrombocytopenia (low platelet count). Based on an identified evidence gap in a previously developed scoping review on existing systematic reviews in the field of platelet transfusion, we conducted a systematic review to identify full economic evaluations and randomized controlled trials assessing the cost-effectiveness of TPO mimetics (e.g. romiplostim, avatrombopag, eltrombopag, lusutrombopag) compared to platelet transfusions or other first-line (corticosteroids or intravenous immunoglobulins) or second-line treatment modalities (immunosuppressants, splenectomy, rituximab, danazol).

Eight databases and registries were searched, resulting in 20 full economic evaluations and 1 randomized controlled trial, which showed that the cost-effectiveness of TPO mimetics, in terms of the incremental cost per quality-adjusted life year (QALY), ranged from a dominant strategy (i.e. cost-saving and clinically superior), to a significant incremental cost per QALY, or a dominated strategy (i.e. increased costs and clinically inferior), see Figure 1. 

Future validation and tackling the uncertainty of these models with country-specific cost data and up-to-date efficacy, safety, and utility data are needed to increase the generalizability of the current cost-effectiveness estimates and to use these data in international evidence-based guideline initiatives. 

This systematic review has been published in Pharmacoeconomics and can be found here.