Latest revision as of 01:09, 4 May 2024

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Section editor
	Benjamin Tillman, MD Vanderbilt University Nashville, TN, USA LinkedIn

Are you looking for a regimen but can't find it here? For placebo or observational studies in this condition, please visit this page. If you still can't find it, please let us know so we can add it!

2 regimens on this page 2 variants on this page

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	Dates of enrollment	Evidence	Comparator	Comparative Efficacy
Yaniv et al. 2009	2005-02 to 2006-01	Randomized Phase 2 (E-esc)	Placebo	Superior frequency of epistaxis

Endocrine therapy

Tamoxifen (Nolvadex) 20 mg PO once per day

6-month course

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 contains dosing details in manuscript PubMed

Tranexamic acid monotherapy

Regimen

Study	Dates of enrollment	Evidence	Comparator	Comparative Efficacy
Geisthoff et al. 2014 (TAHHT)	2002-03-01 to 2002-05-31	Phase 3b (E-esc)	Placebo	Did not meet primary endpoint of hemoglobin levels¹
Gaillard et al. 2014 (ATERO)	2006-09 to 2009-12	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

Tranexamic acid (Cyklokapron) by the following study-specific criteria:
- TAHHT: 1000 mg PO three times per day
- ATERO: 1500 mg PO twice per day

3-month course

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 contains dosing details in manuscript 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 contains dosing details in manuscript 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

@@ Line 3: / Line 3: @@
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 </div>
-{{#lst:Section editor transclusions|heme}}
+{{#lst:Editorial board transclusions|heme}}
+''Are you looking for a regimen but can't find it here? For placebo or observational studies in this condition, please visit [[Hereditary hemorrhagic telangiectasia - null regimens|this page]]. If you still can't find it, please let us know so we can add it!''<br>
 {| class="wikitable" style="float:right; margin-right: 5px;"
 |-
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 {{TOC limit|limit=3}}
 =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. [https://doi.org/10.7326/m20-1443 Second International Guidelines for the Diagnosis and Management of Hereditary Hemorrhagic Telangiectasia]
+*'''2020:''' Faughnan et al. [https://doi.org/10.7326/m20-1443 Second International Guidelines for the Diagnosis and Management of Hereditary Hemorrhagic Telangiectasia] [https://www.ncbi.nlm.nih.gov/pubmed/32894695 PubMed]
-===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] [https://www.ncbi.nlm.nih.gov/pubmed/19553198 PubMed]
 =All lines of therapy=
-==Placebo==
-<div class="toccolours" style="background-color:#eeeeee">
-===Regimen===
-{| class="wikitable sortable" style="width: 100%; text-align:center;"
-!style="width: 25%"|Study
-!style="width: 25%"|[[Levels_of_Evidence#Evidence|Evidence]]
-!style="width: 25%"|Comparator
-!style="width: 25%"|[[Levels_of_Evidence#Comparative_efficacy|Comparative Efficacy]]
-|-
-|[https://doi.org/10.1002/lary.20065 Yaniv et al. 2009]
-| style="background-color:#1a9851" |Randomized Phase 2 (C)
-|[[#Tamoxifen_monotherapy|Tamoxifen]]
-| style="background-color:#d73027" |Inferior frequency of epistaxis
-|-
-|[https://doi.org/10.1016/j.thromres.2014.06.012 Geisthoff et al. 2014 (TAHHT)]
-| style="background-color:#1a9851" |Phase 3 (C)
-|[[#Tranexamic_acid_monotherapy|Tranexamic acid]]
-| style="background-color:#ffffbf" |Did not meet primary endpoint of hemoglobin levels<sup>1</sup>
-|-
-|[https://doi.org/10.1111/jth.12654 Gaillard et al. 2014 (ATERO)]
-| style="background-color:#1a9851" |Phase 3 (C)
-|[[#Tranexamic_acid_monotherapy|Tranexamic acid]]
-| style="background-color:#d73027" |Longer mean duration of epistaxis
-|-
-|[https://jamanetwork.com/journals/jama/fullarticle/2547754 Dupuis-Girod et al. 2016 (ALEGORI)]
-| style="background-color:#1a9851" |Randomized Phase 2 (C)
-|[[#Bevacizumab_nasal_spray_999|Bevacizumab nasal spray]]
-| style="background-color:#ffffbf" |Did not meet primary endpoint
-|-
-|[https://jamanetwork.com/journals/jama/fullarticle/2547755 Whitehead et al. 2016 (NOSE)]
-| style="background-color:#1a9851" |Randomized Phase 2 (C)
-|1. [[#Bevacizumab_nasal_spray_999|Bevacizumab nasal spray]]<br>2. [[#Estriol_nasal_spray_999|Estriol nasal spray]]<br> 3. [[#Tranexamic_acid_monotherapy|Tranexamic acid]]
-| style="background-color:#ffffbf" |Did not meet primary endpoint of epistaxis
-|-
-|}
-''<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.''
-</div></div>
-===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]
-# '''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] [https://clinicaltrials.gov/study/NCT01031992 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] [https://clinicaltrials.gov/study/NCT00355108 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. [https://jamanetwork.com/journals/jama/fullarticle/2547754 link to original article] [https://pubmed.ncbi.nlm.nih.gov/27599328/ PubMed] [https://clinicaltrials.gov/study/NCT02106520 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] [https://clinicaltrials.gov/study/NCT01408030 NCT01408030]
 ==Tamoxifen monotherapy {{#subobject:b1894c|Regimen=1}}==
 <div class="toccolours" style="background-color:#eeeeee">
 ===Regimen {{#subobject:9462b2|Variant=1}}===
 {| class="wikitable sortable" style="width: 100%; text-align:center;"
-!style="width: 25%"|Study
+!style="width: 20%"|Study
-!style="width: 25%"|[[Levels_of_Evidence#Evidence|Evidence]]
+!style="width: 20%"|Dates of enrollment
-!style="width: 25%"|Comparator
+!style="width: 20%"|[[Levels_of_Evidence#Evidence|Evidence]]
-!style="width: 25%"|[[Levels_of_Evidence#Comparative_efficacy|Comparative Efficacy]]
+!style="width: 20%"|Comparator
+!style="width: 20%"|[[Levels_of_Evidence#Comparative_efficacy|Comparative Efficacy]]
 |-
 |[https://doi.org/10.1002/lary.20065 Yaniv et al. 2009]
+|2005-02 to 2006-01
 | style="background-color:#1a9851" |Randomized Phase 2 (E-esc)
-|[[#Placebo|Placebo]]
+|[[Hereditary_hemorrhagic_telangiectasia_-_null_regimens#Placebo|Placebo]]
 | style="background-color:#1a9850" |Superior frequency of epistaxis
 |-
@@ Line 77: / Line 39: @@
 <div class="toccolours" style="background-color:#b3e2cd">
 ====Endocrine therapy====
-*[[Tamoxifen (Nolvadex)]]
+*[[Tamoxifen (Nolvadex)]] 20 mg PO once per day
+'''6-month course'''
 </div></div>
 ===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] '''contains dosing details in manuscript''' [https://pubmed.ncbi.nlm.nih.gov/19160429/ PubMed]
 ==Tranexamic acid monotherapy {{#subobject:ea894c|Regimen=1}}==
 <div class="toccolours" style="background-color:#eeeeee">
 ===Regimen {{#subobject:9134b2|Variant=1}}===
 {| class="wikitable sortable" style="width: 100%; text-align:center;"
-!style="width: 25%"|Study
+!style="width: 20%"|Study
-!style="width: 25%"|[[Levels_of_Evidence#Evidence|Evidence]]
+!style="width: 20%"|Dates of enrollment
-!style="width: 25%"|Comparator
+!style="width: 20%"|[[Levels_of_Evidence#Evidence|Evidence]]
-!style="width: 25%"|[[Levels_of_Evidence#Comparative_efficacy|Comparative Efficacy]]
+!style="width: 20%"|Comparator
+!style="width: 20%"|[[Levels_of_Evidence#Comparative_efficacy|Comparative Efficacy]]
 |-
 |[https://doi.org/10.1016/j.thromres.2014.06.012 Geisthoff et al. 2014 (TAHHT)]
-| style="background-color:#1a9851" |Phase 3 (E-esc)
+|2002-03-01 to 2002-05-31
-|[[#Placebo|Placebo]]
+| style="background-color:#1a9851" |Phase 3b (E-esc)
+|[[Hereditary_hemorrhagic_telangiectasia_-_null_regimens#Placebo|Placebo]]
 | style="background-color:#ffffbf" |Did not meet primary endpoint of hemoglobin levels<sup>1</sup>
 |-
 |[https://doi.org/10.1111/jth.12654 Gaillard et al. 2014 (ATERO)]
+|2006-09 to 2009-12
 | style="background-color:#1a9851" |Phase 3 (E-esc)
-|[[#Placebo|Placebo]]
+|[[Hereditary_hemorrhagic_telangiectasia_-_null_regimens#Placebo|Placebo]]
 | style="background-color:#1a9850" |Shorter mean duration of epistaxis (primary endpoint)
 |-
@@ Line 103: / Line 70: @@
 ''<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====
+====Supportive therapy====
-*[[Tranexamic acid (Cyklokapron)]]
+*[[Tranexamic acid (Cyklokapron)]] by the following study-specific criteria:
+**TAHHT: 1000 mg PO three times per day
+**ATERO: 1500 mg PO twice per day
+'''3-month course'''
 </div></div>
 ===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] [https://clinicaltrials.gov/study/NCT01031992 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] '''contains dosing details in manuscript''' [https://pubmed.ncbi.nlm.nih.gov/25005464/ PubMed] [https://clinicaltrials.gov/study/NCT01031992 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] [https://clinicaltrials.gov/study/NCT00355108 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] '''contains dosing details in manuscript''' [https://pubmed.ncbi.nlm.nih.gov/25040799/ PubMed] [https://clinicaltrials.gov/study/NCT00355108 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] [https://clinicaltrials.gov/study/NCT01408030 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://doi.org/10.1001/jama.2016.11724 link to original article] [https://pubmed.ncbi.nlm.nih.gov/27599329/ PubMed] [https://clinicaltrials.gov/study/NCT01408030 NCT01408030]
 [[Category:Hereditary hemorrhagic telangiectasia regimens]]
 [[Category:Disease-specific pages]]
 [[Category:Hemolytic process]]

Difference between revisions of "Hereditary hemorrhagic telangiectasia"

Latest revision as of 01:09, 4 May 2024

Contents

Guidelines

International Guidelines

All lines of therapy

Tamoxifen monotherapy

Regimen

Endocrine therapy

References

Tranexamic acid monotherapy

Regimen

Supportive therapy

References

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