Hereditary hemorrhagic telangiectasia - null regimens

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The purpose of this page is to provide references to "null therapy" such as placebo and observation. These references provide further insight into the historical development of the treatment landscape. See the main HHT page for regimens that include active treatment.
=All lines of therapy=
==Placebo==
===Regimen===
Study 	Dates of enrollment 	Evidence 	Comparator 	Comparative Efficacy
Geisthoff et al. 2014 (TAHHT) 	2002-03-01 to 2002-05-31	Phase 3 (C) 	Tranexamic acid 	Did not meet primary endpoint of hemoglobin levels1
Yaniv et al. 2009 	2005-02 to 2006-01 	Randomized Phase 2 (C)	Tamoxifen 	Higher frequency of epistaxis
Gaillard et al. 2014 (ATERO) 	2006-09 to 2009-12 	Phase 3 (C) 	Tranexamic acid 	Longer mean duration of epistaxis
Whitehead et al. 2016 (NOSE) 	2011-08 to 2014-03 	Randomized Phase 2 (C)	1. Bevacizumab nasal spray2. Estriol nasal spray 3. Tranexamic acid	Did not meet primary endpoint of epistaxis
Dupuis-Girod et al. 2016 (ALEGORI)	2014-04 to 2015-01 	Randomized Phase 2 (C)	Bevacizumab nasal spray 	Did not meet primary endpoint
Al-Samkari et al. 2024 (PATH-HHT) 	2019-11-05 to 2023-06-27	Randomized (C) 	Pomalidomide 	Worse Epistaxis Severity Score at 24 weeks (primary endpoint)
1In TAHHT, the primary outcome of hemoglobin levels was not statistically significantly different, but the secondary outcome of epistaxis was inferior for this arm.
Note: No active treatment.
===References===
# Yaniv E, Preis M, Hadar T, Shvero J, Haddad M. Antiestrogen therapy for hereditary hemorrhagic telangiectasia: a double-blind placebo-controlled clinical trial. Laryngoscope. 2009 Feb;119(2):284-8. link to original article EuroPMC
# TAHHT: Geisthoff UW, Seyfert UT, Kübler M, Bieg B, Plinkert PK, König J. Treatment of epistaxis in hereditary hemorrhagic telangiectasia with tranexamic acid - a double-blind placebo-controlled cross-over phase IIIB study. Thromb Res. 2014 Sep;134(3):565-71. Epub 2014 Jun 16. link to original article EuroPMC NCT01031992
# ATERO: Gaillard S, Dupuis-Girod S, Boutitie F, Rivière S, Morinière S, Hatron PY, Manfredi G, Kaminsky P, Capitaine AL, Roy P, Gueyffier F, Plauchu H; ATERO Study Group. Tranexamic acid for epistaxis in hereditary hemorrhagic telangiectasia patients: a European cross-over controlled trial in a rare disease. J Thromb Haemost. 2014 Sep;12(9):1494-502. Epub 2014 Jul 29. link to original article EuroPMC NCT00355108
# ALEGORI: Dupuis-Girod S, Ambrun A, Decullier E, Fargeton AE, Roux A, Bréant V, Colombet B, Rivière S, Cartier C, Lacombe P, Chinet T, Blivet S, Blondel JH, Gilbert-Dussardier B, Dufour X, Michel J, Harle JR, Dessi P, Faure F. Effect of Bevacizumab Nasal Spray on Epistaxis Duration in Hereditary Hemorrhagic Telangectasia: A Randomized Clinical Trial. JAMA. 2016 Sep 6;316(9):934-42. link to original article EuroPMC NCT02106520
# NOSE: Whitehead KJ, Sautter NB, McWilliams JP, Chakinala MM, Merlo CA, Johnson MH, James M, Everett EM, Clancy MS, Faughnan ME, Oh SP, Olitsky SE, Pyeritz RE, Gossage JR. Effect of Topical Intranasal Therapy on Epistaxis Frequency in Patients With Hereditary Hemorrhagic Telangiectasia: A Randomized Clinical Trial. JAMA. 2016 Sep 6;316(9):943-51. link to original article EuroPMC NCT01408030
#PATH-HHT: Al-Samkari H, Kasthuri RS, Iyer VN, Pishko AM, Decker JE, Weiss CR, Whitehead KJ, Conrad MB, Zumberg MS, Zhou JY, Parambil J, Marsh D, Clancy M, Bradley L, Wisniewski L, Carper BA, Thomas SM, McCrae KR. Pomalidomide for Epistaxis in Hereditary Hemorrhagic Telangiectasia. N Engl J Med. 2024 Sep 19;391(11):1015-1027. link to original article link to PMC article EuroPMC NCT03910244
Category:Hereditary hemorrhagic telangiectasia regimens
Category:Disease-specific pages
Category:Hemolytic process
Category:Null regimens