Hereditary hemorrhagic telangiectasia

From HemOnc.org - A Hematology Oncology Wiki
Jump to: navigation, search
Section editor
Tillman Benjamin-2.jpg
Benjamin Tillman, MD
Vanderbilt University
Nashville, TN
2 regimens on this page
2 variants on this page


All lines of therapy

Placebo

back to top

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 Seems not superior (*)
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 Seems not superior
Whitehead et al. 2016 (NOSE) Randomized Phase II (C) 1. Bevacizumab nasal spray
2. Estriol nasal spray
3. Tranexamic acid
Seems not superior

Note: No active treatment. In TAHHT, the primary outcome of hemoglobin levels was not statistically significantly different, but the secondary outcome of epistaxis was inferior for this arm.

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 SD article PubMed
  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
  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
  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

Tamoxifen monotherapy

back to top

Regimen

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

Hormonotherapy

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

back to top

Regimen

Study Evidence Comparator Comparative Efficacy
Geisthoff et al. 2014 (TAHHT) Phase III (E) Placebo Seems not superior (*)
Gaillard et al. 2014 (ATERO) Phase III (E) Placebo Shorter mean duration of epistaxis

Note: 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 SD article PubMed
  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
  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