Hereditary hemorrhagic telangiectasia

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Last updated on 2024-12-02:3 regimens on this page
3 variants on this page
=Guidelines=
Given the rapid change in evidence in many areas of hematology/oncology, readers are encouraged to consider any guideline published 5+ years ago to be for historical purposes, only.
==International Guidelines==
*2020: Faughnan et al. Second International Guidelines for the Diagnosis and Management of Hereditary Hemorrhagic Telangiectasia EuroPMC
*2009: Faughnan et al. International guidelines for the diagnosis and management of hereditary haemorrhagic telangiectasia EuroPMC
=All lines of therapy=
==Pomalidomide monotherapy==
===Regimen===
Study 	Dates of enrollment 	Evidence 	Comparator	Comparative Efficacy
Al-Samkari et al. 2024 (PATH-HHT)	2019-11-05 to 2023-06-27	Randomized (E-esc)	Placebo 	Better Epistaxis Severity Score at 24 weeks (primary endpoint)
====Targeted therapy====
*Pomalidomide (Pomalyst) 4 mg PO once per day on days 1 to 28
28-day cycle for 6 cycles
===References===
#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 dosing details in manuscript have been reviewed by our editors EuroPMC NCT03910244
==Tamoxifen monotherapy==
===Regimen===
Study 	Dates of enrollment	Evidence 	Comparator	Comparative Efficacy
Yaniv et al. 2009	2005-02 to 2006-01 	Randomized Phase 2 (E-esc)	Placebo 	Lower frequency of epistaxis
====Endocrine therapy====
*Tamoxifen (Nolvadex) 20 mg PO once per day
6-month course
===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 dosing details in manuscript have been reviewed by our editors EuroPMC
==Tranexamic acid monotherapy==
===Regimen===
Study 	Dates of enrollment 	Evidence 	Comparator	Comparative Efficacy
Geisthoff et al. 2014 (TAHHT)	2002-03-01 to 2002-05-31	Phase 3b (E-esc)	Placebo 	Did not meet primary endpoint of hemoglobin levels1
Gaillard et al. 2014 (ATERO) 	2006-09 to 2009-12 	Phase 3 (E-esc) 	Placebo 	Shorter mean duration of epistaxis (primary endpoint)
1In TAHHT, the primary outcome of hemoglobin levels was not statistically significantly different, but the secondary outcome of epistaxis was better for this arm.
====Supportive therapy====
*Tranexamic acid (Cyklokapron) by the following study-specific criteria:
**TAHHT: 1000 mg PO three times per day
**ATERO: 1500 mg PO twice per day
3-month course
===References===
# 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 dosing details in manuscript have been reviewed by our editors 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 dosing details in manuscript have been reviewed by our editors EuroPMC NCT00355108
# 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
Category:Hereditary hemorrhagic telangiectasia regimens
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Category:Hemolytic process