School of International Pharmaceutical Business, China Pharmaceutical University, Nanjing, China; Department of Pharmacy, The First Affiliated Hospital of Soochow University, Suzhou, China.
Department of Pharmacy, The First Affiliated Hospital of Soochow University, Suzhou, China.
Ann Palliat Med. 2022 Aug;11(8):2709-2719. doi: 10.21037/apm-22-880.
Chemotherapy-induced thrombocytopenia (CIT) is a common adverse reaction to chemotherapy that can lead to treatment delay, platelet transfusion, thereby increasing treatment costs, reducing chemotherapy effectiveness and affecting prognosis. Based on real-world data, this study analyzed the safety, efficacy, and economic of recombinant human thrombopoietin (rhTPO) and recombinant human interleukin-11 (rhIL-11) in the treatment of CIT in hematological tumors from the perspective of the health care system.
We retrospectively collected the data of hematological tumor patients treated with rhTPO and rhIL-11 due to thrombocytopenia caused by chemotherapy. The propensity score matching (PSM) method was used to balance the baseline information of the two groups and they were further stratified according to the degree of thrombocytopenia (grade I-II and grade III-IV). The platelet compliance rate at 2 weeks of treatment was used as the efficacy evaluation index, and the cost-effectiveness method was used to evaluate the economic value of the two drugs in the treatment of thrombocytopenia based on drug effectiveness. Univariate and probabilistic sensitivity analyses were performed.
A total of 1,571 patients met the inclusion and exclusion criteria, and 476 patients were included after 1:1 PSM. For patients with grade I-II thrombocytopenia, no significant difference in the platelet compliance rate was found between the two groups after 1 and 2 weeks of treatment. The platelet compliance rate in the rhTPO group was higher than that in the rhIL-11 group for patients with grade III-IV thrombocytopenia. Cost-effectiveness analysis (CEA) showed that the incremental cost-effectiveness ratio (ICER) for the rhTPO and rhIL-11 groups was 226,615.8. The ICER value was sensitive to the platelet compliance rate of the two groups, the cost of rhTPO, the cost of platelet transfusion in the rhTPO group. Probabilistic sensitivity analysis showed that when willingness to pay was less than approximately 220,000 yuan, rhIL-11 economy presented 100% better than that of rhTPO.
In CIT treatment for hematological tumors, rhTPO yielded a higher platelet compliance rate than rhIL-11 treatment, especially for patients with grade III-IV thrombocytopenia. However, whether rhTPO has economic advantages still requires further exploration.
化疗引起的血小板减少症（CIT）是化疗的常见不良反应，可导致治疗延迟、血小板输注，从而增加治疗成本、降低化疗效果并影响预后。本研究基于真实世界数据，从医疗保健系统的角度分析了重组人血小板生成素（rhTPO）和重组人白细胞介素-11（rhIL-11）治疗血液系统肿瘤 CIT 的安全性、疗效和经济性。
我们回顾性收集了因化疗引起血小板减少症而接受 rhTPO 和 rhIL-11 治疗的血液系统肿瘤患者的数据。采用倾向评分匹配（PSM）方法平衡两组的基线信息，并根据血小板减少程度（I 度-II 度和 III 度-IV 度）进一步分层。治疗 2 周时的血小板依从率作为疗效评价指标，基于药物疗效，采用成本效果分析法评价两种药物治疗血小板减少症的经济性。进行了单因素和概率敏感性分析。
共纳入 1571 例符合纳入排除标准的患者，1:1 PSM 后纳入 476 例患者。对于 I 度-II 度血小板减少症患者，治疗 1 周和 2 周后两组血小板依从率无显著差异。对于 III 度-IV 度血小板减少症患者，rhTPO 组的血小板依从率高于 rhIL-11 组。成本效果分析（CEA）显示，rhTPO 和 rhIL-11 组的增量成本效果比（ICER）为 226615.8。ICER 值对两组血小板依从率、rhTPO 成本、rhTPO 组血小板输注成本敏感。概率敏感性分析显示，当支付意愿低于约 220000 元时，rhIL-11 经济状况比 rhTPO 好 100%。
在血液系统肿瘤 CIT 治疗中，rhTPO 组的血小板依从率高于 rhIL-11 组，尤其是 III 度-IV 度血小板减少症患者。然而，rhTPO 是否具有经济优势仍需进一步探讨。
