Hereditary hemorrhagic telangiectasia
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Benjamin Tillman, MD Vanderbilt University Nashville, TN, USA |
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2 regimens on this page
2 variants on this page
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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
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 (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
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 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 PubMed 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 PubMed NCT01408030