Difference between revisions of "Inherited coagulopathy"
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'''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.''' | '''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.''' | ||
==ASH/ISTH/NHF/WFH== | ==ASH/ISTH/NHF/WFH== | ||
| − | *'''2021:''' James et al. [https://doi.org/10.1182/bloodadvances.2020003265 ASH ISTH NHF WFH 2021 guidelines on the diagnosis of von Willebrand disease] [https://www.ncbi.nlm.nih.gov/pubmed/33570651 PubMed] | + | *'''2021:''' James et al. [https://doi.org/10.1182/bloodadvances.2020003265 ASH ISTH NHF WFH 2021 guidelines on the diagnosis of von Willebrand disease] [https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7805340/ link to PMC article] [https://pubmed.ncbi.nlm.nih.gov/33570651 PubMed] |
| − | *'''2021:''' Connell et al. [https://doi.org/10.1182/bloodadvances.2020003264 ASH ISTH NHF WFH 2021 guidelines on the management of von Willebrand disease] [https://www.ncbi.nlm.nih.gov/pubmed/33570647 PubMed] | + | *'''2021:''' Connell et al. [https://doi.org/10.1182/bloodadvances.2020003264 ASH ISTH NHF WFH 2021 guidelines on the management of von Willebrand disease] [https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7805326/ link to PMC article] [https://pubmed.ncbi.nlm.nih.gov/33570647 PubMed] |
==BSH== | ==BSH== | ||
| − | *'''2020:''' Rayment et al. [https://doi.org/10.1111/bjh.16704 Guidelines on the use of prophylactic factor replacement for children and adults with Haemophilia A and B] [https:// | + | *'''2020:''' Rayment et al. [https://doi.org/10.1111/bjh.16704 Guidelines on the use of prophylactic factor replacement for children and adults with Haemophilia A and B] [https://pubmed.ncbi.nlm.nih.gov/32390158 PubMed] |
=Factor VIII deficiency (Hemophilia A), replacement products= | =Factor VIII deficiency (Hemophilia A), replacement products= | ||
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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://doi.org/10.1056/NEJMoa1703068 link to original article] [https://pubmed.ncbi.nlm.nih.gov/28691557/ PubMed] | # '''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://doi.org/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://doi.org/10.1182/blood.2019001869 link to original article] [https://pubmed.ncbi.nlm.nih.gov/31697801/ 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://doi.org/10.1182/blood.2019001869 link to original article] [https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6908828/ link to PMC article] [https://pubmed.ncbi.nlm.nih.gov/31697801/ PubMed] |
==Rituximab monotherapy {{#subobject:6d79c2|Regimen=1}}== | ==Rituximab monotherapy {{#subobject:6d79c2|Regimen=1}}== | ||
Latest revision as of 23:47, 6 July 2024
| Section editor | |
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Benjamin Tillman, MD Vanderbilt University Nashville, TN, USA |
- We have moved How I Treat articles to a dedicated page.
3 regimens on this page
3 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.
ASH/ISTH/NHF/WFH
- 2021: James et al. ASH ISTH NHF WFH 2021 guidelines on the diagnosis of von Willebrand disease link to PMC article PubMed
- 2021: Connell et al. ASH ISTH NHF WFH 2021 guidelines on the management of von Willebrand disease link to PMC article PubMed
BSH
- 2020: Rayment et al. Guidelines on the use of prophylactic factor replacement for children and adults with Haemophilia A and B PubMed
Factor VIII deficiency (Hemophilia A), replacement products
To be completed
Factor VIII deficiency (Hemophilia A), without inhibitors, all lines of therapy
Emicizumab monotherapy
Regimen
| Study | Dates of enrollment | Evidence | Comparator | Comparative Efficacy |
|---|---|---|---|---|
| Mahlangu et al. 2018 (HAVEN 3) | 2016-09 to NR | Phase 3 (E-esc) | No prophylaxis | Lower bleeding rate (primary endpoint) |
| Negrier et al. 2023 (HAVEN 6) | 2020-02-10 to 2021-08-31 | Non-randomized phase 3 |
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 NCT02847637
- HAVEN 6: Negrier C, Mahlangu J, Lehle M, Chowdary P, Catalani O, Bernardi RJ, Jimenez-Yuste V, Beckermann BM, Schmitt C, Ventriglia G, Windyga J, d'Oiron R, Moorehead P, Koparkar S, Teodoro V, Shapiro AD, Oldenburg J, Hermans C. Emicizumab in people with moderate or mild haemophilia A (HAVEN 6): a multicentre, open-label, single-arm, phase 3 study. Lancet Haematol. 2023 Mar;10(3):e168-e177. Epub 2023 Jan 27. link to original article PubMed NCT04158648
Factor VIII deficiency (Hemophilia A), with inhibitors, all lines of therapy
Emicizumab monotherapy
Regimen
| Study | Dates of enrollment | Evidence | Comparator | Comparative Efficacy |
|---|---|---|---|---|
| Oldenburg et al. 2017 (HAVEN 1) | 2015-11-17 to NR | Phase 3 (E-esc) | No prophylaxis | Lower bleeding rate (primary endpoint) |
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 link to PMC article PubMed
Rituximab monotherapy
Regimen
| Study | Dates of enrollment | Evidence |
|---|---|---|
| Leissinger et al. 2014 (RICH) | 2006-08 to 2010-11 | Phase 2 |
Immunosuppressive therapy
- Rituximab (Rituxan) 375 mg/m2 IV once on day 1
7-day cycle for 4 cycles (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 dosing details PubMed
- RICH: 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. Epub 2014 Jun 12. link to original article link to PMC article contains dosing details in manuscript PubMed NCT00331006
Factor IX deficiency (Hemophilia B), all lines of therapy
To be completed