Local sclerotherapy with polydocanol (Aethoxysklerol®) for the treatment of Epistaxis in Rendu-Osler-Weber or Hereditary Hemorrhagic Telangiectasia (HHT): 15 years of experience

Abstract

Hereditary Haemorrhagic Telangiectasia or Rendu-Osler-Weber syndrome is a rare autosomal dominant vascular disease characterized by mucocutaneous and gastrointestinal telangiectases and localized arteriovenous malformations in lung, brain and liver. Epistaxis, due to rupture of telangiectases of the nasal mucosa, is the most frequent clinical manifestation, leading in many cases to severe impairment of the quality of life in the patients. Though several treatments have been used to reduce epistaxis, none have been completely effective, with the exception of polydocanol (Aethoxysklerol®) in submucosal or subpericondrial injections, which was first presented in 2000 with very good results. After fifteen years using polydocanol in submucosal injections on 45 patients and with nearly 300 injections, we have observed that in 95% of all cases, their nose bleeds improved with respect to frequency and quantity without any important side effects. There was just one case of septal perforation, another with increased septal perforation, and one patient who suffered from dizziness and blurred vision for a few minutes. In this paper the results obtained using this technique over a fifteen-year period will be presented and evaluated.

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

“…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, 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 …”

Complications from office sclerotherapy for epistaxis due to hereditary hemorrhagic telangiectasia (HHT or Osler‐Weber‐Rendu)

“…Stemming predominantly (90% of cases) from mutations in the transforming growth factor β (TGF‐β)/bone morphogenic protein (BMP) angiogenic regulation cascade, including the Endoglin ( ENG ) and ALK1/ACVRL1 loci, HHT showcases hemorrhagic complications through rupture of the characteristically thin‐walled, discontinuous vascular architecture, defined by inadequate elastin and smooth muscle fibers. Consequently, these architectural deficits lead to altered hemostasis due to defective vasoconstriction . Vascular malformations in HHT manifest via progressive elastin loss and consequent luminal dilatation of postcapillary venules, which is followed by arteriolar and capillary enlargement .…”

“…Furthermore, during this process, affected vessels also become longer and more tortuous, commonly meandering toward the nearest mucosal or cutaneous surface where they are at increased exposure to the external environment . The resultant ultra‐structural findings are tortuous and dilated superficial vessels (ie, telangiectasias) in the nasal, buccal, and gastrointestinal mucosa, as well as in the superficial skin vessels, and arteriovenous malformations in larger vessels in the liver, lungs, and brain …”

“…As the primary presenting manifestation of HHT, epistaxis represents an unpredictable, potentially life‐consuming complication that is nearly ubiquitous in these patients, affecting approximately 90% of the 1:5000 afflicted individuals before the age of 21 years . Typically, epistaxis is a daily nuisance in these patients and arises as a consequence of irritation of the fragile vessels within the nasal mucosa by minor trauma or through crusting from the drying effect of air conduction through the nasal cavity, which results in vascular damage and rupture .…”

“…Typically, epistaxis is a daily nuisance in these patients and arises as a consequence of irritation of the fragile vessels within the nasal mucosa by minor trauma or through crusting from the drying effect of air conduction through the nasal cavity, which results in vascular damage and rupture . Moreover, incidence, symptom severity, and the degree of systemic involvement of HHT vascular lesions typically increase with age, and severity may range from minor periodic bleeding to severe potentially life‐threatening hemorrhage or arteriovenous shunting . Although exsanguination and other life‐threatening events in HHT from epistaxis are extremely rare, epistaxis can impose drastically on the quality of life of HHT patients .…”