Centre for Evidence-Based Practice, Belgian Red Cross, Mechelen, Belgium.
Department of Public Health and Primary Care, Leuven Institute for Healthcare Policy, KU Leuven, Leuven, Belgium.
Pharmacoeconomics. 2023 Aug;41(8):869-911. doi: 10.1007/s40273-023-01271-w. Epub 2023 May 5.
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.
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.
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%).
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.
血小板生成素 (TPO) 模拟物是减少血小板减少症患者出血的潜在替代血小板输注方法。本系统评价旨在评估 TPO 模拟物与不使用 TPO 模拟物相比，在血小板减少症成年患者中的成本效益。
检索了 8 个数据库和登记处，以获取全经济评价 (EE) 和随机对照试验 (RCT)。合成增量成本效益比 (ICER) 为每获得一个质量调整生命年的成本 (QALY) 或每获得一个健康结果的成本 (如避免出血事件)。使用 Philips 报告清单对纳入的研究进行了严格评价。
纳入了来自 9 个不同国家的 18 项评估，评估了 TPO 模拟物与不使用 TPO、观察和救援治疗、标准治疗、利妥昔单抗、脾切除术或血小板输注相比的成本效益。ICER 从主导策略（即节省成本和更有效）到每 QALY/健康结果增量成本为 25,000-50,000 欧元、75,000-750,000 欧元和 > 100 万欧元不等，再到主导策略（成本增加和效果降低）。少数评估（n = 2，10%）解决了四种主要类型的不确定性（方法学、结构性、异质性和参数）。参数不确定性报告最多（80%），其次是异质性（45%）、结构性不确定性（43%）和方法学不确定性（28%）。
TPO 模拟物在血小板减少症成年患者中的成本效益从主导策略到每 QALY/健康结果的显著增量成本或临床效果较差且成本增加的策略不等。需要使用具有国家特定成本数据和最新疗效和安全性数据的未来验证和解决这些模型的不确定性，以提高普遍性。
