Hereditary hemorrhagic telangiectasia

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Tillman Benjamin-2.jpg
Benjamin Tillman, MD
Vanderbilt University
Nashville, TN
2 regimens on this page
2 variants on this page


Guidelines

International Guidelines

Older

All lines of therapy

Placebo

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Regimen

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

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Regimen

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

Endocrine therapy

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

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Regimen

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

  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