Garin Margaret C, Clark Leslie, Chumney Elinor C G, Simpson Kit N, Highland Kristin B
Department of Medicine, Hospital of the University of Pennsylvania, Philadelphia, Pennsylvania, USA.
Clin Drug Investig. 2009;29(10):635-46. doi: 10.2165/11317820-000000000-00000.
Clinicians must choose between an increasing number of medications for the treatment of pulmonary arterial hypertension (PAH) with different routes of administration, adverse effects, costs and efficacies. We constructed a decision analysis to help inform treatment choices in PAH.
We created a Markov-type model to evaluate 1-year treatment with bosentan, treprostinil, epoprostenol, inhaled iloprost, sildenafil, sitaxentan and ambrisentan. Transition probabilities were based on observed transitions between WHO functional classes, adjusted by relative risk of improvement in a 6-minute walk test. Utilities were based on reported values for each functional class, adjusted for burden of treatment administration. Costs were estimated from Medicare and Medicaid reimbursement data. Sensitivity analyses evaluated changes in efficacy, utilities and costs.
Treatment with sildenafil was less costly and resulted in a greater gain in quality-adjusted life-years (QALYs) compared with other treatments. Treatment with ambrisentan and bosentan resulted in the same gain in QALYs as sildenafil, but at a higher cost. Sensitivity analyses had minimal impact on these results.
Based on this model, sildenafil is a cost-effective treatment for PAH with a low price and a net increase in QALYs. The results from this analysis are a tool to help guide clinicians in deciding which PAH medications to use; however, the final decisions should be individualized because the effectiveness of therapy, resulting utilities and acceptable costs will differ with each patient.
临床医生在治疗肺动脉高压（PAH）时，面对越来越多具有不同给药途径、不良反应、成本和疗效的药物，必须做出选择。我们构建了一个决策分析模型，以辅助指导PAH的治疗选择。
我们创建了一个马尔可夫类型模型，来评估波生坦、曲前列尼尔、依前列醇、吸入用伊洛前列素、西地那非、西他生坦和安立生坦1年的治疗效果。转移概率基于世界卫生组织功能分级之间观察到的转移情况，并根据6分钟步行试验改善的相对风险进行调整。效用值基于各功能分级报告的值，并根据治疗给药负担进行调整。成本根据医疗保险和医疗补助报销数据估算。敏感性分析评估疗效、效用值和成本的变化。
与其他治疗方法相比，西地那非治疗成本更低，且质量调整生命年（QALY）增益更大。安立生坦和波生坦治疗的QALY增益与西地那非相同，但成本更高。敏感性分析对这些结果影响极小。
基于该模型，西地那非是一种治疗PAH性价比高的药物，价格低廉且QALY净增加。该分析结果是帮助指导临床医生决定使用哪种PAH药物的工具；然而，最终决策应因人而异，因为每个患者的治疗效果、由此产生的效用值和可接受成本会有所不同。
