Hereditary hemorrhagic telangiectasia
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| Section editor | |
|---|---|
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Benjamin Tillman, MD Vanderbilt University Nashville, TN |
2 regimens on this page
2 variants on this page
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Guidelines
International Guidelines
- 2020: Faughnan et al. Second International Guidelines for the Diagnosis and Management of Hereditary Hemorrhagic Telangiectasia
Older
- 2009: Faughnan et al. International guidelines for the diagnosis and management of hereditary haemorrhagic telangiectasia
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
- 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
- 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 Clinical Trial Registry
- 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 Clinical Trial Registry
- 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 Clinical Trial Registry
- 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 Clinical Trial Registry
Tamoxifen monotherapy
Regimen
| Study | Evidence | Comparator | Comparative Efficacy |
|---|---|---|---|
| Yaniv et al. 2009 | Randomized Phase 2 (E-esc) | Placebo | Superior frequency of epistaxis |
Endocrine therapy
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 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 |
1In TAHHT, the primary outcome of hemoglobin levels was not statistically significantly different, but the secondary outcome of epistaxis was superior for this arm.
Hemostasis therapy
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 PubMed Clinical Trial Registry
- 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 Clinical Trial Registry
- 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 Clinical Trial Registry