Difference between revisions of "Inherited coagulopathy"
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===References=== | ===References=== | ||
− | # '''HAVEN 3:''' Mahlangu J, Oldenburg J, Paz-Priel I, Negrier C, Niggli M, Mancuso ME, Schmitt C, Jiménez-Yuste V, Kempton C, Dhalluin C, Callaghan MU, Bujan W, Shima M, Adamkewicz JI, Asikanius E, Levy GG, Kruse-Jarres R. Emicizumab prophylaxis in patients who have hemophilia A without inhibitors. N Engl J Med. 2018 Aug 30;379(9):811-822. [https://www.nejm.org/doi/full/10.1056/NEJMoa1803550 link to original article] [https:// | + | # '''HAVEN 3:''' Mahlangu J, Oldenburg J, Paz-Priel I, Negrier C, Niggli M, Mancuso ME, Schmitt C, Jiménez-Yuste V, Kempton C, Dhalluin C, Callaghan MU, Bujan W, Shima M, Adamkewicz JI, Asikanius E, Levy GG, Kruse-Jarres R. Emicizumab prophylaxis in patients who have hemophilia A without inhibitors. N Engl J Med. 2018 Aug 30;379(9):811-822. [https://www.nejm.org/doi/full/10.1056/NEJMoa1803550 link to original article] [https://pubmed.ncbi.nlm.nih.gov/30157389 PubMed] |
=Factor VIII deficiency (Hemophilia A), with inhibitors, all lines of therapy= | =Factor VIII deficiency (Hemophilia A), with inhibitors, all lines of therapy= | ||
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===References=== | ===References=== | ||
− | # '''HAVEN 1:''' Oldenburg J, Mahlangu JN, Kim B, Schmitt C, Callaghan MU, Young G, Santagostino E, Kruse-Jarres R, Negrier C, Kessler C, Valente N, Asikanius E, Levy GG, Windyga J, Shima M. Emicizumab prophylaxis in hemophilia A with inhibitors. N Engl J Med. 2017 Aug 31;377(9):809-818. Epub 2017 Jul 10. [https://www.nejm.org/doi/full/10.1056/NEJMoa1703068 link to original article] [https:// | + | # '''HAVEN 1:''' Oldenburg J, Mahlangu JN, Kim B, Schmitt C, Callaghan MU, Young G, Santagostino E, Kruse-Jarres R, Negrier C, Kessler C, Valente N, Asikanius E, Levy GG, Windyga J, Shima M. Emicizumab prophylaxis in hemophilia A with inhibitors. N Engl J Med. 2017 Aug 31;377(9):809-818. Epub 2017 Jul 10. [https://www.nejm.org/doi/full/10.1056/NEJMoa1703068 link to original article] [https://pubmed.ncbi.nlm.nih.gov/28691557 PubMed] |
− | # '''HAVEN 2:''' Young G, Liesner R, Chang T, Sidonio R, Oldenburg J, Jiménez-Yuste V, Mahlangu J, Kruse-Jarres R, Wang M, Uguen M, Doral MY, Wright LY, Schmitt C, Levy GG, Shima M, Mancuso ME. A multicenter, open-label phase 3 study of emicizumab prophylaxis in children with hemophilia A with inhibitors. Blood. 2019 Dec 12;134(24):2127-2138. [https://ashpublications.org/blood/article-lookup/doi/10.1182/blood.2019001869 link to original article] [https:// | + | # '''HAVEN 2:''' Young G, Liesner R, Chang T, Sidonio R, Oldenburg J, Jiménez-Yuste V, Mahlangu J, Kruse-Jarres R, Wang M, Uguen M, Doral MY, Wright LY, Schmitt C, Levy GG, Shima M, Mancuso ME. A multicenter, open-label phase 3 study of emicizumab prophylaxis in children with hemophilia A with inhibitors. Blood. 2019 Dec 12;134(24):2127-2138. [https://ashpublications.org/blood/article-lookup/doi/10.1182/blood.2019001869 link to original article] [https://pubmed.ncbi.nlm.nih.gov/31697801 PubMed] |
==Rituximab monotherapy {{#subobject:6d79c2|Regimen=1}}== | ==Rituximab monotherapy {{#subobject:6d79c2|Regimen=1}}== | ||
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===References=== | ===References=== | ||
− | # Kempton CL, Allen G, Hord J, Kruse-Jarres R, Pruthi RK, Walsh C, Young G, Soucie JM. Eradication of factor VIII inhibitors in patients with mild and moderate hemophilia A. Am J Hematol. 2012 Sep;87(9):933-6. [https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3645919/ link to PMC article] '''does not contain protocol''' [https:// | + | # Kempton CL, Allen G, Hord J, Kruse-Jarres R, Pruthi RK, Walsh C, Young G, Soucie JM. Eradication of factor VIII inhibitors in patients with mild and moderate hemophilia A. Am J Hematol. 2012 Sep;87(9):933-6. [https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3645919/ link to PMC article] '''does not contain protocol''' [https://pubmed.ncbi.nlm.nih.gov/22733686 PubMed] |
− | # Leissinger C, Josephson CD, Granger S, Konkle BA, Kruse-Jarres R, Ragni MV, Journeycake JM, Valentino L, Key NS, Gill JC, McCrae KR, Neufeld EJ, Manno C, Raffini L, Saxena K, Torres M, Marder V, Bennett CM, Assmann SF. Rituximab for treatment of inhibitors in haemophilia A: a phase II study. Thromb Haemost. 2014 Sep 2;112(3):445-58. [http://th.schattauer.de/en/contents/archive/issue/1975/manuscript/21293.html link to original article] [https:// | + | # Leissinger C, Josephson CD, Granger S, Konkle BA, Kruse-Jarres R, Ragni MV, Journeycake JM, Valentino L, Key NS, Gill JC, McCrae KR, Neufeld EJ, Manno C, Raffini L, Saxena K, Torres M, Marder V, Bennett CM, Assmann SF. Rituximab for treatment of inhibitors in haemophilia A: a phase II study. Thromb Haemost. 2014 Sep 2;112(3):445-58. [http://th.schattauer.de/en/contents/archive/issue/1975/manuscript/21293.html link to original article] [https://pubmed.ncbi.nlm.nih.gov/24919980 PubMed] |
=Factor IX deficiency (Hemophilia B), all lines of therapy= | =Factor IX deficiency (Hemophilia B), all lines of therapy= |
Revision as of 02:09, 25 February 2020
Section editor | |
---|---|
Benjamin Tillman, MD Vanderbilt University Nashville, TN |
3 regimens on this page
3 variants on this page
|
Guidelines
"How I treat"
Factor VIII deficiency (Hemophilia A), replacement products
To be completed
Factor VIII deficiency (Hemophilia A), without inhibitors, all lines of therapy
Emicizumab monotherapy
back to top |
Regimen
Study | Evidence | Comparator | Comparative Efficacy |
---|---|---|---|
Mahlangu et al. 2018 (HAVEN 3) | Phase III (E-esc) | No prophylaxis | Lower bleeding rate |
To be completed
Supportive therapy
References
- HAVEN 3: Mahlangu J, Oldenburg J, Paz-Priel I, Negrier C, Niggli M, Mancuso ME, Schmitt C, Jiménez-Yuste V, Kempton C, Dhalluin C, Callaghan MU, Bujan W, Shima M, Adamkewicz JI, Asikanius E, Levy GG, Kruse-Jarres R. Emicizumab prophylaxis in patients who have hemophilia A without inhibitors. N Engl J Med. 2018 Aug 30;379(9):811-822. link to original article PubMed
Factor VIII deficiency (Hemophilia A), with inhibitors, all lines of therapy
Emicizumab monotherapy
back to top |
Regimen
Study | Evidence | Comparator | Comparative Efficacy |
---|---|---|---|
Oldenburg et al. 2017 (HAVEN 1) | Phase III (E-esc) | No prophylaxis | Lower bleeding rate |
To be completed
Supportive therapy
References
- HAVEN 1: Oldenburg J, Mahlangu JN, Kim B, Schmitt C, Callaghan MU, Young G, Santagostino E, Kruse-Jarres R, Negrier C, Kessler C, Valente N, Asikanius E, Levy GG, Windyga J, Shima M. Emicizumab prophylaxis in hemophilia A with inhibitors. N Engl J Med. 2017 Aug 31;377(9):809-818. Epub 2017 Jul 10. link to original article PubMed
- HAVEN 2: Young G, Liesner R, Chang T, Sidonio R, Oldenburg J, Jiménez-Yuste V, Mahlangu J, Kruse-Jarres R, Wang M, Uguen M, Doral MY, Wright LY, Schmitt C, Levy GG, Shima M, Mancuso ME. A multicenter, open-label phase 3 study of emicizumab prophylaxis in children with hemophilia A with inhibitors. Blood. 2019 Dec 12;134(24):2127-2138. link to original article PubMed
Rituximab monotherapy
back to top |
Regimen
Study | Evidence |
---|---|
Leissinger et al. 2014 | Phase II |
Immunosuppressive therapy
- Rituximab (Rituxan) 375 mg/m2 IV once per day on days 1, 8, 15, 22
28-day course
References
- Kempton CL, Allen G, Hord J, Kruse-Jarres R, Pruthi RK, Walsh C, Young G, Soucie JM. Eradication of factor VIII inhibitors in patients with mild and moderate hemophilia A. Am J Hematol. 2012 Sep;87(9):933-6. link to PMC article does not contain protocol PubMed
- Leissinger C, Josephson CD, Granger S, Konkle BA, Kruse-Jarres R, Ragni MV, Journeycake JM, Valentino L, Key NS, Gill JC, McCrae KR, Neufeld EJ, Manno C, Raffini L, Saxena K, Torres M, Marder V, Bennett CM, Assmann SF. Rituximab for treatment of inhibitors in haemophilia A: a phase II study. Thromb Haemost. 2014 Sep 2;112(3):445-58. link to original article PubMed
Factor IX deficiency (Hemophilia B), all lines of therapy
To be completed