Objectives/hypothesis: Objectives were to assess available information on hormonal therapy for bleeding in hereditary hemorrhagic telangiectasia (HHT), to determine whether there is a role for hormonal therapy as an initial therapeutic option, and to report the second known case of response in HHT to antihormonal therapy.
Study design: Literature review and case report.
Methods: The literature on hormonal and antihormonal therapy for HHT was reviewed. Medical records for the case reported in the present study were evaluated to confirm the diagnosis and assess responses to surgical and nonsurgical treatments.
Results: All reports of success using hormonal therapy for HHT-related bleeding were either retrospective or uncontrolled with the exception of two. Anecdotal evidence with high-dose estrogen appeared to show success, but serious side effects have discouraged use. One controlled trial found no benefit for intermediate dose, single-agent estrogen. The other controlled trial appeared to show benefit with low-dose estrogen-progesterone in HHT with gastrointestinal tract bleeding and was supported by an uncontrolled study showing efficacy in epistaxis. The case reported in the present study demonstrated long-term cessation of epistaxis with tamoxifen in a postmenopausal woman.
Conclusions: Systemic estrogen-progesterone at doses used for oral contraception may eliminate bleeding in symptomatic HHT and is a reasonable initial option in fertile women. There is no information on possible effects of lower-dose estrogen-progesterone used in postmenopausal women for hormone replacement therapy. Tamoxifen has dramatically eliminated HHT-related bleeding in two cases. It is well tolerated in postmenopausal women and should be considered for randomized clinical trials.