PUBLICATION: Journal of American Medical Association; JAMA. 2016;316(9):943-951. doi:10.1001/jama.2016.11724
We are very pleased to report the first multi-center clinical study on nosebleed treatment has been published and released in the Journal of American Medical Association: “Effect of Topical Intranasal Therapy on Epistaxis Frequency in Patients with Hereditary Hemorrhagic Telangiectasia a Randomized Clinical Trial” on September 6, 2016.
This is the first randomized double blind placebo controlled Phase II Clinical trial ever documented! This study has propelled other studies forward, such as the Avastin Epistaxis study at Stanford University and the Paznopanib trial with Glaxo Smith Kline Pharmaceutical Company.
120 patients received topical therapy of either Avastin 1% (4mg/d), Estriol 0.1% (0.4mg/d), Tranexamic acid 10% (40mg/d) or placebo (0.9% saline) for 12 weeks to assess the risks and benefits for treatment of moderate to severe nose bleeding. As you know all too well, interventional techniques such as laser and cautery have provided temporary help, but the need for better therapeutic approaches in managing nosebleeds is desperately needed.
The study showed treatment with topical therapy is safe, well tolerated and improves nosebleed severity in most patients. Though the study didn’t show any impact on nosebleed frequency, patients on Avastin showed a borderline reduction in nosebleed duration in comparison with the tranexamic acid and placebo over the 24 weeks.
As HHT patients do not routinely irrigate the nose prior to the administration of drug therapies (which might improve absorption of medication), we do not know if this procedure impacted the results. Additionally, it may be that a higher concentration of the Avastin (bevacizamab) could have produced greater improvement.
This study concluded simple treatment with nasal saline spray twice daily is beneficial to many patients. The addition of tranexamic acid, estriol or bevacizumab does not seem to offer significant advantage over plain saline. However, if you are currently using one of these medicated sprays, do not discontinue before speaking with your doctor.