Prospective randomized trial of sclerotherapy vs standard treatment for epistaxis due to hereditary hemorrhagic telangiectasia.

Recurrent epistaxis is often the presenting symptom in patients with hereditary hemorrhagic telangiectasia (HHT); in fact, 90% of patients with HHT experience recurrent epistaxis.1 HHT has an overall frequency of 1:5000. It affects all ethnicities, and males and females equally.2 An autosomal dominant disease with variable penetrance and expression, it acts on genes that produce elastic fibers of blood vessels. Affected individuals exhibit vascular malformations, primarily in the nose, gastrointestinal (GI) tract, brain, liver, and lung. Lesions demonstrate markedly dilated, convoluted venules with abnormal smooth muscles, often directly connected to abnormally dilated arterioles.3 In the nasal epithelium, thin-walled telangiectasias are exposed to dryness and nasal crusting, leading to frequent damage to these vessels. When damage causes bleeding, the lack of effective elastic fibers prevents constriction, and difficult-to-control hemorrhage ensues. The severity of epistaxis usually worsens with advancing age; major life-threatening bleeding can result in frequent transfusions, invasive procedures, and hospitalizations.4 A stepwise approach to treatment is recommended, based on the severity of symptoms.5,6 The Epistaxis Severity Score (ESS) is a standardized scoring system assessing epistaxis severity and frequency, and is an accurate, reproducible, validated objective measure of epistaxis severity.7 The ESS is measured on a 10-point scale, with higher scores reflecting more severe and frequent bleeding. Current standard treatment for mild recurrent bleeding relies on careful and diligent moisturization of the nasal passages. Many patients require iron supplementation to treat anemia. Various topical and oral regimens are also used, such as hormones, antifibrinolytics, and antiangiogenic agents.8 Recurrent or severe epistaxis may necessitate blood transfusions and referral to an otolaryngologist. For moderately severe recurrent epistaxis, otolaryngologists use various forms of laser photocoagulation (CO2, argon, neodymium-doped yttrium aluminum garnet [Nd-YAG], flashlamp-pulsed dye, and potassium titanyl phosphate [KTP]), electrical cautery, or surgical microdebridement. More aggressive surgical options include septodermoplasty and complete nasal closure (Young’s procedure). Arterial embolization is used for emergent severe bleeding.8 Yet none of these modalities provide complete relief from bleeding and can be associated with significant side effects, such as nasal crusting, foul odor, and septal perforation. Their limitations have motivated the search for alternative treatment options. One alternative is sclerotherapy, by injecting vascular lesions with an agent that causes thickening of the vessel wall, obstruction of blood flow, clot formation, and collapse of the lesion. An established treatment modality for vascular malformations in the skin, GI tract, and genitourinary tract, sclerotherapy has been adopted for lesions in various sites in the head and neck,9–13 but not for HHT. Case reports and one large series in the literature have described sclerotherapy for epistaxis due to HHT, but have not been followed by prospective studies.14–17 In our previously published retrospective analysis of our pilot study, we described the results of sclerotherapy with sodium tetradecyl sulfate (STS) in 7 patients. All 7 described the procedure as tolerable, and all reported a reduction in the severity and frequency of bleeding. All 7 were willing to undergo sclerotherapy with STS again.8 In our subsequent analysis of outcomes in 36 patients undergoing repeated sclerotherapy, we found no reports of adverse sequelae such as septal perforation, increased bleeding, or foul smell; some patients described minimal transient procedural symptoms, but no serious complications.18 The purpose of the current prospective randomized trial was to determine the effectiveness of sclerotherapy with STS, vs standard treatment, for recurrent epistaxis due to HHT. We hypothesized a priori that the severity and frequency of epistaxis would be reduced with sclerotherapy compared to standard treatment.