Hereditary hemorrhagic telangiectasia

Section editor
	Benjamin Tillman, MD Vanderbilt University Nashville, TN, USA LinkedIn

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2 regimens on this page 2 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 PubMed

• 2009: Faughnan et al. International guidelines for the diagnosis and management of hereditary haemorrhagic telangiectasia PubMed

All lines of therapy

Placebo

Regimen

Study	Evidence	Comparator	Comparative Efficacy
Yaniv et al. 2009	Randomized Phase 2 (C)	Tamoxifen	Inferior frequency of epistaxis
Geisthoff et al. 2014 (TAHHT)	Phase 3 (C)	Tranexamic acid	Did not meet primary endpoint of hemoglobin levels1
Gaillard et al. 2014 (ATERO)	Phase 3 (C)	Tranexamic acid	Longer mean duration of epistaxis
Dupuis-Girod et al. 2016 (ALEGORI)	Randomized Phase 2 (C)	Bevacizumab nasal spray	Did not meet primary endpoint
Whitehead et al. 2016 (NOSE)	Randomized Phase 2 (C)	1. Bevacizumab nasal spray 2. Estriol nasal spray 3. Tranexamic acid	Did not meet primary endpoint of epistaxis

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

1. 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 PubMed
2. 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 PubMed NCT01031992
3. 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 PubMed NCT00355108
4. 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 PubMed NCT02106520
5. 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 PubMed NCT01408030

Tamoxifen monotherapy

Regimen

Study	Evidence	Comparator	Comparative Efficacy
Yaniv et al. 2009	Randomized Phase 2 (E-esc)	Placebo	Superior frequency of epistaxis

Endocrine therapy

• Tamoxifen (Nolvadex)

References

1. 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 PubMed

Tranexamic acid monotherapy

Regimen

Study	Evidence	Comparator	Comparative Efficacy
Geisthoff et al. 2014 (TAHHT)	Phase 3 (E-esc)	Placebo	Did not meet primary endpoint of hemoglobin levels1
Gaillard et al. 2014 (ATERO)	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 superior for this arm.

Supportive therapy

• Tranexamic acid (Cyklokapron)

References

1. 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 PubMed NCT01031992
2. 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 PubMed NCT00355108
3. 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 PubMed NCT01408030