Hereditary Hemorrhagic Telangiectasias (HHT) is characterized by arteriovenous malformations (AVM), an autosomal dominant inheritance pattern, and age-related penetrance. It is diagnosed clinically with 3 or more Curacao criteria: telangiectasias, epistaxis, family history, visceral lesions. Pulmonary hypertension (PH) occurs in 15% of patients with HHT, most frequently due to a high output state associated with hepatic AVM. In addition, a small, but significant, portion of patients with HHT develop precapillary PH, which portends poorer outcomes than for patients with HHT alone. To date, only small case series of treatment of severe precapillary PH in patients with HHT exist. Among these series, there has been no experience with prostanoid therapy as a treatment for HHT-associated PH, possibly because of the theoretical increased risk of bleeding in these patients due to the anti-platelet effect of these drugs. The purpose of this report is to emphasize the potential role and safety of prostanoid therapy as treatment for severe precapillary PH in patients with HHT.
We retrospectively evaluated four patients with HHT and severe precapillary PH, treated with prostanoid therapy. Diagnostic hemodynamics were compared pre- and post-treatment using a paired t-test. Mean follow-up was 95 months.
All subjects demonstrated clinically significant improvement following treatment with prostanoid therapy (epoprostenol). Cardiac index improved from 2.2±0.8 L/min/m2 at baseline to 3.6±0.5L/min/m2 during therapy. Pulmonary vascular resistance decreased from 16±6 WU to 7±1 WU. Mean pulmonary arterial pressure decreased from 68±6 mmHg to 53±9 mmHg. Moreover, treatment with epoprostenol was safe; there were no clinically important bleeding episodes associated with therapy in these patients.
This is the first series to examine prostanoid therapy as treatment of severe HHT associated precapillary PH. All patients treated with this regimen demonstrated hemodynamic improvement and there were no unexpected safety issues. Further studies are warranted to evaluate this treatment regimen for HHT-associated PH.