Difference between revisions of "Hereditary hemorrhagic telangiectasia"
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− | + | </div> | |
− | + | {{#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> | |
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{| class="wikitable" style="float:right; margin-right: 5px;" | {| class="wikitable" style="float:right; margin-right: 5px;" | ||
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− | |<div style="background-color: #fee0d1; border: 1px solid #808000; padding: 5px; {{border-radius|16px}}" align="right"><font size="4"><b>{{#ask: [[-Has subobject::{{FULLPAGENAME}}]] | + | |<div style="background-color: #fee0d1; border: 1px solid #808000; padding: 5px; {{border-radius|16px}}" align="right"><font size="4"><b>{{#ask: [[-Has subobject::{{FULLPAGENAME}}]] |?Regimen |limit=10000|format=sum}} [[Tutorial#Regimens|regimens]] on this page</b></font></div> |
− | <div style="background-color: #deebf6; border: 1px solid #808000; padding: 5px; {{border-radius|16px}}"><font size="4"><b>{{#ask: [[-Has subobject::{{FULLPAGENAME}}]] | + | <div style="background-color: #deebf6; border: 1px solid #808000; padding: 5px; {{border-radius|16px}}"><font size="4"><b>{{#ask: [[-Has subobject::{{FULLPAGENAME}}]] |?Variant |limit=10000|format=sum}} [[Tutorial#Variants|variants]] on this page</b></font></div> |
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{{TOC limit|limit=3}} | {{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] [https://www.ncbi.nlm.nih.gov/pubmed/32894695 PubMed] | ||
+ | |||
+ | *'''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= | =All lines of therapy= | ||
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− | ==Tamoxifen | + | ==Tamoxifen monotherapy {{#subobject:b1894c|Regimen=1}}== |
− | + | <div class="toccolours" style="background-color:#eeeeee"> | |
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===Regimen {{#subobject:9462b2|Variant=1}}=== | ===Regimen {{#subobject:9462b2|Variant=1}}=== | ||
− | {| class="wikitable" style="width: 100%; text-align:center;" | + | {| class="wikitable sortable" style="width: 100%; text-align:center;" |
− | !style="width: | + | !style="width: 20%"|Study |
− | !style="width: | + | !style="width: 20%"|Dates of enrollment |
− | !style="width: | + | !style="width: 20%"|[[Levels_of_Evidence#Evidence|Evidence]] |
− | !style="width: | + | !style="width: 20%"|Comparator |
+ | !style="width: 20%"|[[Levels_of_Evidence#Comparative_efficacy|Comparative Efficacy]] | ||
|- | |- | ||
− | |[https:// | + | |[https://doi.org/10.1002/lary.20065 Yaniv et al. 2009] |
− | | style="background-color:#1a9851" |Randomized Phase | + | |2005-02 to 2006-01 |
− | |[[#Placebo|Placebo]] | + | | style="background-color:#1a9851" |Randomized Phase 2 (E-esc) |
+ | |[[Hereditary_hemorrhagic_telangiectasia_-_null_regimens#Placebo|Placebo]] | ||
| style="background-color:#1a9850" |Superior frequency of epistaxis | | style="background-color:#1a9850" |Superior frequency of epistaxis | ||
|- | |- | ||
|} | |} | ||
− | ==== | + | <div class="toccolours" style="background-color:#b3e2cd"> |
− | *[[Tamoxifen (Nolvadex)]] | + | ====Endocrine therapy==== |
− | + | *[[Tamoxifen (Nolvadex)]] 20 mg PO once per day | |
+ | '''6-month course''' | ||
+ | </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:// | + | # 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}}== | ==Tranexamic acid monotherapy {{#subobject:ea894c|Regimen=1}}== | ||
− | + | <div class="toccolours" style="background-color:#eeeeee"> | |
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===Regimen {{#subobject:9134b2|Variant=1}}=== | ===Regimen {{#subobject:9134b2|Variant=1}}=== | ||
− | {| class="wikitable" style="width: 100%; text-align:center;" | + | {| class="wikitable sortable" style="width: 100%; text-align:center;" |
− | !style="width: | + | !style="width: 20%"|Study |
− | !style="width: | + | !style="width: 20%"|Dates of enrollment |
− | !style="width: | + | !style="width: 20%"|[[Levels_of_Evidence#Evidence|Evidence]] |
− | !style="width: | + | !style="width: 20%"|Comparator |
+ | !style="width: 20%"|[[Levels_of_Evidence#Comparative_efficacy|Comparative Efficacy]] | ||
|- | |- | ||
− | |[https:// | + | |[https://doi.org/10.1016/j.thromres.2014.06.012 Geisthoff et al. 2014 (TAHHT)] |
− | | style="background-color:#1a9851" |Phase | + | |2002-03-01 to 2002-05-31 |
− | |[[#Placebo|Placebo]] | + | | style="background-color:#1a9851" |Phase 3b (E-esc) |
− | | style="background-color:#ffffbf" | | + | |[[Hereditary_hemorrhagic_telangiectasia_-_null_regimens#Placebo|Placebo]] |
+ | | style="background-color:#ffffbf" |Did not meet primary endpoint of hemoglobin levels<sup>1</sup> | ||
|- | |- | ||
− | |[https:// | + | |[https://doi.org/10.1111/jth.12654 Gaillard et al. 2014 (ATERO)] |
− | | style="background-color:#1a9851" |Phase | + | |2006-09 to 2009-12 |
− | |[[#Placebo|Placebo]] | + | | style="background-color:#1a9851" |Phase 3 (E-esc) |
− | | style="background-color:#1a9850" |Shorter mean duration of epistaxis | + | |[[Hereditary_hemorrhagic_telangiectasia_-_null_regimens#Placebo|Placebo]] |
+ | | style="background-color:#1a9850" |Shorter mean duration of epistaxis (primary endpoint) | ||
|- | |- | ||
|} | |} | ||
− | '' | + | ''<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"> |
− | *[[Tranexamic acid (Cyklokapron)]] | + | ====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''' | ||
+ | </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:// | + | # '''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:// | + | # '''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:// | + | # '''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] |
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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]] |
Latest revision as of 01:09, 4 May 2024
Section editor | |
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Benjamin Tillman, MD Vanderbilt University Nashville, TN, USA |
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 |
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