Department of Pharmacy, Beijing Friendship Hospital, Capital Medical University, Yong-an Road, Xi-Cheng District, Beijing, 100050, PR China.
School of Pharmacy, Capital Medical University, Beijing, PR China.
BMC Health Serv Res. 2023 Apr 12;23(1):361. doi: 10.1186/s12913-023-09365-z.
This study aims to evaluate the cost-effectiveness of immunosuppressive therapy for patients with progressive idiopathic membranous nephropathy (IMN) from the Chinese healthcare system perspective.
To estimate the cost-effectiveness of four regimens namely cyclophosphamide, cyclosporine, rituximab and tacrolimus-rituximab in treatment of IMN recommended by the updated Kidney Disease: Improving Global Outcomes (KDIGO) guideline 2021, a Markov model with five discrete states (active disease, remission, dialysis, kidney transplant and death) based on IMN patients aged 50 or above over a 30-years time horizon was constructed. Total costs were imputed from the Chinese healthcare system perspective, and health outcomes were converted into quality-adjusted life years (QALYs). The incremental cost-effectiveness ratio (ICER) was used to describe the results. The willingness-to-pay (WTP) threshold was set at $12,044 (China's 2021 Gross Domestic Product per capita). Sensitivity analyses were performed to test the uncertainties of the results.
Compared with cyclophosphamide, both cyclosporine (incremental cost $28,337.09, incremental QALY-1.63) and tacrolimus-rituximab (incremental cost $28,324.13, incremental QALY -0.46) were considered at strictly dominated for their negative values in QALYs, and the ICER value of rituximab was positive (incremental cost $9,162.19, incremental QALY 0.44). Since the ICER of rituximab exceeds the pre-determined threshold, cyclophosphamide was likely to be the best choice for the treatment of IMN within the acceptable threshold range. The results of the sensitivity analysis revealed that the model outcome was mostly affected by the probability of remission in rituximab. In a probabilistic sensitivity analysis, cyclophosphamide had 62.4% probability of being cost-effective compared with other regimens when the WTP was $12,044 per QALY. When WTP exceeded $18,300, rituximab was more cost-effective than cyclophosphamide.
Compared with cyclosporine, rituximab and tacrolimus-rituximab, our model results indicated that cyclophosphamide represented the most cost-effective regimen for patients with progressive IMN in China.
本研究旨在从中国医疗保健系统的角度评估免疫抑制疗法治疗进展性特发性膜性肾病（IMN）患者的成本效益。
为了评估环磷酰胺、环孢素、利妥昔单抗和他克莫司-利妥昔单抗四种方案在治疗 2021 年更新的肾脏病：改善全球预后（KDIGO）指南推荐的 IMN 患者中的成本效益，根据 50 岁或以上的 IMN 患者，构建了一个具有五个离散状态（活动疾病、缓解、透析、肾移植和死亡）的 30 年时间范围内的 Markov 模型。总费用从中国医疗保健系统的角度进行估算，健康结果转换为质量调整生命年（QALY）。增量成本效益比（ICER）用于描述结果。意愿支付（WTP）阈值设定为 12044 美元（中国 2021 年人均国内生产总值）。进行敏感性分析以检验结果的不确定性。
与环磷酰胺相比，环孢素（增量成本为 28337.09 美元，增量 QALY-1.63）和他克莫司-利妥昔单抗（增量成本为 28324.13 美元，增量 QALY-0.46）的 QALY 均为负值，被认为是严格占主导地位的，而利妥昔单抗的 ICER 值为正（增量成本为 9162.19 美元，增量 QALY 为 0.44）。由于利妥昔单抗的 ICER 超过了预定的阈值，因此在可接受的阈值范围内，环磷酰胺可能是治疗 IMN 的最佳选择。敏感性分析的结果表明，模型结果主要受利妥昔单抗缓解概率的影响。在概率敏感性分析中，当 WTP 为每个 QALY 12044 美元时，与其他方案相比，环磷酰胺有 62.4%的可能性具有成本效益。当 WTP 超过 18300 美元时，利妥昔单抗比环磷酰胺更具成本效益。
与环孢素、利妥昔单抗和他克莫司-利妥昔单抗相比，我们的模型结果表明，在中国，环磷酰胺是治疗进展性特发性膜性肾病患者最具成本效益的方案。
