Highland Kristin B, Strange Charlie, Mazur Joe, Simpson Kit N
Division of Pulmonary, Critical Care, Allergy, and Clinical Immunology, Medical University of South Carolina, 96 Jonathan Lucas Street, Suite 812 CSB, Charleston, SC 29425, USA.
Chest. 2003 Dec;124(6):2087-92. doi: 10.1378/chest.124.6.2087.
New therapies for pulmonary arterial hypertension (PAH) improve functional status, quality of life (QOL), and survival. Clinicians must chose between very different therapies without the availability of comparison studies. We constructed a "virtual" clinical trial to help inform these treatment choices.
We compare key outcomes related to survival, costs, and QOL using a Markov-type decision model to estimate the expected outcomes and costs for PAH patients treated for 1 year with bosentan and treprostinil compared to patients treated with epoprostenol, as well as patients treated with bosentan compared to those treated with treprostinil. The allowed transitions in the model were between World Health Organization functional class I to IV and death. Transition probabilities were based on observed transitions for bosentan. Treatment effect was estimated using 6-min walk data for treprostinil and epoprostenol. Utilities were calculated from estimated EuroQol health states. Cost was estimated from average wholesale price and Medicare reimbursement data. The effects of changing values of input variables on the key outcomes were calculated.
Treatment with bosentan compared to treatment with either epoprostenol or treprostinil was less costly and resulted in a greater gain in quality-adjusted life years (QALYs). Conversely, treprostinil was significantly more expensive than epoprostenol, without an appreciable gain in QALYs. These findings were not substantially affected by the reasonable adjustments of transition probabilities, utility values, or tachyphylaxis to epoprostenol.
Treatment with bosentan is more cost-effective than treatment with either treprostinil or epoprostenol. In addition, a net improvement in quality-adjusted survival may be expected.
肺动脉高压（PAH）的新疗法可改善功能状态、生活质量（QOL）并提高生存率。临床医生在缺乏对比研究的情况下，必须在截然不同的疗法之间做出选择。我们构建了一项“虚拟”临床试验，以辅助做出这些治疗选择。
我们使用马尔可夫型决策模型比较与生存、成本和生活质量相关的关键结局，以估计接受波生坦和曲前列尼尔治疗1年的PAH患者与接受依前列醇治疗的患者，以及接受波生坦治疗的患者与接受曲前列尼尔治疗的患者的预期结局和成本。模型中允许的转变是在世界卫生组织功能分级I至IV级与死亡之间。转变概率基于波生坦的观察到的转变情况。使用曲前列尼尔和依前列醇的6分钟步行数据估计治疗效果。根据估计的欧洲五维度健康量表健康状态计算效用值。成本根据平均批发价格和医疗保险报销数据进行估计。计算了输入变量值的变化对关键结局的影响。
与使用依前列醇或曲前列尼尔治疗相比，使用波生坦治疗成本更低，且在质量调整生命年（QALY）方面有更大的增益。相反，曲前列尼尔比依前列醇贵得多，而在QALY方面没有明显增益。这些发现并未因对转变概率、效用值或对依前列醇的快速耐受进行合理调整而受到实质性影响。
与使用曲前列尼尔或依前列醇治疗相比，使用波生坦治疗更具成本效益。此外，预计在质量调整生存率方面会有净改善。
