Difference between revisions of "Hereditary hemorrhagic telangiectasia"
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m (Text replacement - "style="background-color:#1a9851" |Phase III" to "style="background-color:#1a9851" |Phase 3") |
Warner-admin (talk | contribs) m (Text replacement - "|Randomized Phase II" to "|Randomized Phase 2") |
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|[https://onlinelibrary.wiley.com/doi/full/10.1002/lary.20065 Yaniv et al. 2009] | |[https://onlinelibrary.wiley.com/doi/full/10.1002/lary.20065 Yaniv et al. 2009] | ||
| − | | style="background-color:#1a9851" |Randomized Phase | + | | style="background-color:#1a9851" |Randomized Phase 2 (C) |
|[[#Tamoxifen_monotherapy|Tamoxifen]] | |[[#Tamoxifen_monotherapy|Tamoxifen]] | ||
| style="background-color:#d73027" |Inferior frequency of epistaxis | | style="background-color:#d73027" |Inferior frequency of epistaxis | ||
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|[https://jamanetwork.com/journals/jama/fullarticle/2547754 Dupuis-Girod et al. 2016 (ALEGORI)] | |[https://jamanetwork.com/journals/jama/fullarticle/2547754 Dupuis-Girod et al. 2016 (ALEGORI)] | ||
| − | | style="background-color:#1a9851" |Randomized Phase | + | | style="background-color:#1a9851" |Randomized Phase 2 (C) |
|Bevacizumab nasal spray | |Bevacizumab nasal spray | ||
| style="background-color:#ffffbf" |Did not meet primary endpoint | | style="background-color:#ffffbf" |Did not meet primary endpoint | ||
|- | |- | ||
|[https://jamanetwork.com/journals/jama/fullarticle/2547755 Whitehead et al. 2016 (NOSE)] | |[https://jamanetwork.com/journals/jama/fullarticle/2547755 Whitehead et al. 2016 (NOSE)] | ||
| − | | style="background-color:#1a9851" |Randomized Phase | + | | style="background-color:#1a9851" |Randomized Phase 2 (C) |
|1. Bevacizumab nasal spray<br> 2. Estriol nasal spray<br> 3. [[#Tranexamic_acid_monotherapy|Tranexamic acid]] | |1. Bevacizumab nasal spray<br> 2. Estriol nasal spray<br> 3. [[#Tranexamic_acid_monotherapy|Tranexamic acid]] | ||
| style="background-color:#ffffbf" |Did not meet primary endpoint of epistaxis | | style="background-color:#ffffbf" |Did not meet primary endpoint of epistaxis | ||
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|[https://onlinelibrary.wiley.com/doi/full/10.1002/lary.20065 Yaniv et al. 2009] | |[https://onlinelibrary.wiley.com/doi/full/10.1002/lary.20065 Yaniv et al. 2009] | ||
| − | | style="background-color:#1a9851" |Randomized Phase | + | | style="background-color:#1a9851" |Randomized Phase 2 (E-esc) |
|[[#Placebo|Placebo]] | |[[#Placebo|Placebo]] | ||
| style="background-color:#1a9850" |Superior frequency of epistaxis | | style="background-color:#1a9850" |Superior frequency of epistaxis | ||
Revision as of 17:25, 12 February 2022
| Section editor | |
|---|---|
 |
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
| back to top |
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 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
- 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
- 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
| back to top |
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
| back to top |
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 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