Background: Although experts recommend presymptomatic coil embolotherapy for patients with hereditary hemorrhagic telangiectasia (HHT) who have pulmonary arteriovenous malformations (PAVMs), this approach has not been studied prospectively and is not applied universally. We used decision analysis to evaluate the optimal treatment strategy for HHT patients with asymptomatic PAVMs.
Methods: We developed a Markov model to evaluate the following three strategies: no embolotherapy; embolotherapy only in the event of a PAVM complication; and immediate embolotherapy. Our model incorporated PAVM complications, embolotherapy effectiveness and complications, and the possibility of PAVM growth or reperfusion of successfully embolized PAVMs. The base case was a 40-year-old man with HHT and an asymptomatic PAVM with a 3-mm feeding artery. We modeled the natural history of HHT and the clinical course of embolotherapy based on review of the medical literature. We incorporated quality-of-life weights derived from the direct assessment of patient preferences (n = 45) and a literature review.
Results: No embolotherapy, embolotherapy only in the event of a PAVM complication, and immediate embolotherapy were associated with expected survival times of 37.2, 37.6, and 39.0 years, respectively. After adjusting for quality of life, the corresponding estimates were 32.6, 34.1, and 37.2 quality-adjusted life-years. The outcome of the model was robust to changing model parameters within plausible ranges.
Conclusions: Patients with HHT and a PAVM with a feeding artery of >or= 3 mm have improved life expectancy and quality-adjusted survival with immediate embolotherapy. Embolotherapy should be the standard of care in such circumstances.