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

Abstract

Background Our previous studies have demonstrated the tolerability and low side-effect profile of office-based sclerotherapy with sodium tetradecyl sulfate (STS) for treating recurrent epistaxis due to hereditary hemorrhagic telangiectasia (HHT). The objective of this study was to use a prospective randomized trial to determine the effectiveness of sclerotherapy with STS vs standard treatment. Methods This prospective randomized trial (conducted from November 1, 2011, through January 31, 2014) involved 17 patients with recurrent epistaxis due to HHT. We defined standard treatment as continuation of any treatment that the patient had previously undergone, such as moisturization, packing, and cautery. We used a crossover design, so study participants were randomized to either sclerotherapy or standard treatment during the first time period, and then to the other during the second period. The primary outcome measure was frequency and severity of epistaxis, as measured by the epistaxis severity score (ESS). The ESS is a 10-point scale, with higher scores corresponding to more bleeding. Results After controlling for treatment order, bleeding was substantially better controlled after sclerotherapy; the ESS after sclerotherapy was nearly one point lower than after standard treatment (−0.95, 1-sided p = 0.027). Treatment order, baseline ESS, the number of lesions, moisturization practices, and a history of previous blood transfusions did not significantly affect the results. Conclusion This trial demonstrated that sclerotherapy with STS (vs standard treatment) significantly reduced epistaxis due to HHT.

The minimal important difference of the epistaxis severity score in hereditary hemorrhagic telangiectasia

“…These outcome measurements are not standardized, reproducible, or validated in HHT patients. More recent prospective studies using the ESS as a primary outcome measurement have identified rose geranium oil, bevacizumab, and sclerotherapy as potentially successful therapies to treat HHT epistaxis . In order to continue investigating new treatment modalities with a high level of evidence, we believe that the ESS should be adopted as a primary outcome measurement of future randomized clinical trials.…”

“…More recent prospective studies using the ESS as a primary outcome measurement have identified rose geranium oil, bevacizumab, and sclerotherapy as potentially successful therapies to treat HHT epistaxis. 7,8,24 In order to continue investigating new treatment modalities with a high level of evidence, we believe that the ESS should be adopted as a primary outcome measurement of future randomized clinical trials. The MID of the ESS may be referenced in the design, critique, and interpretation of these trials.…”

Sclerotherapy and Topical Nasal Propranolol: An Effective and Safe Therapy for HHT‐Epistaxis

“…In another questionnaire including 220 HHT patients, almost half of them reported daily bleeding, and 76.5% of them had at least one weekly bleeding . The severity of epistaxis generally worsens with age and threatens patient's life, causing chronic anemia and frequently requiring blood transfusions, invasive procedures, and hospitalizations . These situations lead to a significant negative impact on the quality of life of HHT patients.…”

Epistaxis in hereditary hemorrhagic telangiectasia: an evidence based review of surgical management

“…Sclerotherapy with sodium tetradecyl sulfate (STS) was described for use in HHT in 2011 [ 31 ]. A follow-up prospective trial recently demonstrated the efficacy of sclerotherapy when compared to “standard therapy” (defined in this study as any treatment that the patient had previously undergone, such as packing or cautery) [ 32 ]. Sclerotherapy works via injection of STS into the vascular lesion, which theoretically leads to thickening of the vessel wall and subsequently less bleeding.…”

“…Further research into this treatment modality is warranted. Level of Evidence: Level 1b (1 RCT) [ 32 ] ; level 4 (1 case series) [ 31 ]…”