Difference between revisions of "Hereditary hemorrhagic telangiectasia"
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=Guidelines= | =Guidelines= | ||
==International Guidelines== | ==International Guidelines== | ||
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===Older=== | ===Older=== | ||
*'''2009:''' Faughnan et al. [https://doi.org/10.1136/jmg.2009.069013 International guidelines for the diagnosis and management of hereditary haemorrhagic telangiectasia] | *'''2009:''' Faughnan et al. [https://doi.org/10.1136/jmg.2009.069013 International guidelines for the diagnosis and management of hereditary haemorrhagic telangiectasia] | ||
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=All lines of therapy= | =All lines of therapy= | ||
==Placebo== | ==Placebo== | ||
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===Regimen=== | ===Regimen=== | ||
{| class="wikitable sortable" style="width: 100%; text-align:center;" | {| class="wikitable sortable" style="width: 100%; text-align:center;" | ||
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''<sup>1</sup>In TAHHT, the primary outcome of hemoglobin levels was not statistically significantly different, but the secondary outcome of epistaxis was inferior for this arm.''<br> | ''<sup>1</sup>In TAHHT, the primary outcome of hemoglobin levels was not statistically significantly different, but the secondary outcome of epistaxis was inferior for this arm.''<br> | ||
''Note: No active treatment.'' | ''Note: No active treatment.'' | ||
| − | + | </div></div> | |
===References=== | ===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. [https://doi.org/10.1002/lary.20065 link to original article] [https://pubmed.ncbi.nlm.nih.gov/19160429 PubMed] | # 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. [https://doi.org/10.1002/lary.20065 link to original article] [https://pubmed.ncbi.nlm.nih.gov/19160429 PubMed] | ||
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# '''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. [https://jamanetwork.com/journals/jama/fullarticle/2547754 link to original article] [https://pubmed.ncbi.nlm.nih.gov/27599328 PubMed] NCT02106520 | # '''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. [https://jamanetwork.com/journals/jama/fullarticle/2547754 link to original article] [https://pubmed.ncbi.nlm.nih.gov/27599328 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. [https://jamanetwork.com/journals/jama/fullarticle/2547755 link to original article] [https://pubmed.ncbi.nlm.nih.gov/27599329 PubMed] NCT01408030 | # '''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. [https://jamanetwork.com/journals/jama/fullarticle/2547755 link to original article] [https://pubmed.ncbi.nlm.nih.gov/27599329 PubMed] NCT01408030 | ||
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==Tamoxifen monotherapy {{#subobject:b1894c|Regimen=1}}== | ==Tamoxifen monotherapy {{#subobject:b1894c|Regimen=1}}== | ||
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===Regimen {{#subobject:9462b2|Variant=1}}=== | ===Regimen {{#subobject:9462b2|Variant=1}}=== | ||
{| class="wikitable sortable" style="width: 100%; text-align:center;" | {| class="wikitable sortable" style="width: 100%; text-align:center;" | ||
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|- | |- | ||
|} | |} | ||
| + | <div class="toccolours" style="background-color:#b3e2cd"> | ||
====Endocrine therapy==== | ====Endocrine therapy==== | ||
*[[Tamoxifen (Nolvadex)]] | *[[Tamoxifen (Nolvadex)]] | ||
| − | + | </div></div> | |
===References=== | ===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. [https://doi.org/10.1002/lary.20065 link to original article] [https://pubmed.ncbi.nlm.nih.gov/19160429 PubMed] | # 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. [https://doi.org/10.1002/lary.20065 link to original article] [https://pubmed.ncbi.nlm.nih.gov/19160429 PubMed] | ||
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==Tranexamic acid monotherapy {{#subobject:ea894c|Regimen=1}}== | ==Tranexamic acid monotherapy {{#subobject:ea894c|Regimen=1}}== | ||
| − | + | <div class="toccolours" style="background-color:#eeeeee"> | |
===Regimen {{#subobject:9134b2|Variant=1}}=== | ===Regimen {{#subobject:9134b2|Variant=1}}=== | ||
{| class="wikitable sortable" style="width: 100%; text-align:center;" | {| class="wikitable sortable" style="width: 100%; text-align:center;" | ||
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| − | '<sup>1</sup>In TAHHT, the primary outcome of hemoglobin levels was not statistically significantly different, but the secondary outcome of epistaxis was superior for this arm.'' | + | ''<sup>1</sup>In TAHHT, the primary outcome of hemoglobin levels was not statistically significantly different, but the secondary outcome of epistaxis was superior for this arm.'' |
| + | <div class="toccolours" style="background-color:#b3e2cd"> | ||
====Hemostasis therapy==== | ====Hemostasis therapy==== | ||
*[[Tranexamic acid (Cyklokapron)]] | *[[Tranexamic acid (Cyklokapron)]] | ||
| − | + | </div></div> | |
===References=== | ===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. [https://doi.org/10.1016/j.thromres.2014.06.012 link to original article] [https://pubmed.ncbi.nlm.nih.gov/25005464 PubMed] NCT01031992 | # '''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. [https://doi.org/10.1016/j.thromres.2014.06.012 link to original article] [https://pubmed.ncbi.nlm.nih.gov/25005464 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. [https://doi.org/10.1111/jth.12654 link to original article] [https://pubmed.ncbi.nlm.nih.gov/25040799 PubMed] NCT00355108 | # '''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. [https://doi.org/10.1111/jth.12654 link to original article] [https://pubmed.ncbi.nlm.nih.gov/25040799 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. [https://jamanetwork.com/journals/jama/fullarticle/2547755 link to original article] [https://pubmed.ncbi.nlm.nih.gov/27599329 PubMed] NCT01408030 | # '''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. [https://jamanetwork.com/journals/jama/fullarticle/2547755 link to original article] [https://pubmed.ncbi.nlm.nih.gov/27599329 PubMed] NCT01408030 | ||
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[[Category:Hereditary hemorrhagic telangiectasia regimens]] | [[Category:Hereditary hemorrhagic telangiectasia regimens]] | ||
[[Category:Disease-specific pages]] | [[Category:Disease-specific pages]] | ||
[[Category:Hemolytic process]] | [[Category:Hemolytic process]] | ||
Revision as of 00:30, 1 March 2023
2 regimens on this page
2 variants on this page
|
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 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
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 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