European principles of inhibitor management in patients with haemophilia: implications of new treatment options
Jazyk angličtina Země Velká Británie, Anglie Médium electronic
Typ dokumentu dopisy
PubMed
32831110
PubMed Central
PMC7444030
DOI
10.1186/s13023-020-01511-8
PII: 10.1186/s13023-020-01511-8
Knihovny.cz E-zdroje
- Klíčová slova
- Bypassing agents, Emicizumab, Factor IX, Factor VIII, Guidelines, Haemophilia, Immune tolerance, Inhibitors,
- MeSH
- faktor IX MeSH
- faktor VIII MeSH
- hemofilie A * farmakoterapie MeSH
- lékařství * MeSH
- lidé MeSH
- poruchy pohlavních chromozomů * MeSH
- Check Tag
- lidé MeSH
- Publikační typ
- dopisy MeSH
- Názvy látek
- faktor IX MeSH
- faktor VIII MeSH
Canterbury Christ Church University Kent UK
Children's University Hospital and Masaryk University Brno Czech Republic
European Haemophilia Consortium Brussels Belgium
Zobrazit více v PubMed
PLF G, Hermans C, O’Mahony B, de KP BM, Batorova A, et al. European principles of inhibitor management in patients with haemophilia. Orphanet J Rare Dis. 2018;13(1):66. doi: 10.1186/s13023-018-0800-z. PubMed DOI PMC
Oldenburg J, Mahlangu JN, Kim B, Schmitt C, Callaghan MU, Young G, et al. Emicizumab prophylaxis in hemophilia a with inhibitors. N Engl J Med. 2017;377(9):809–818. doi: 10.1056/NEJMoa1703068. PubMed DOI
Young G, Liesner R, Chang T, Sidonio R, Oldenburg J, Jimenez-Yuste V, et al. A multicenter, open-label phase 3 study of emicizumab prophylaxis in children with hemophilia a with inhibitors. Blood. 2019;134(24):2127–2138. doi: 10.1182/blood.2019001869. PubMed DOI PMC
Ebbert PT, Xavier F, Seaman CD, Ragni MV. Emicizumab prophylaxis in patients with haemophilia a with and without inhibitors. Haemophilia. 2020;26(1):41–46. doi: 10.1111/hae.13877. PubMed DOI
Page D. Parent testimonial: a caregiver whose son with inhibitors has been receiving emicizumab. Transfus Apher Sci. 2019;58:5. doi: 10.1016/j.transci.2018.12.008. PubMed DOI
Gruppo RA, Malan D, Kapocsi J, Nemes L, Hay CRM, Boggio L, et al. Phase 1, single-dose escalating study of marzeptacog alfa (activated), a recombinant factor VIIa variant, in patients with severe hemophilia. J Thromb Haemost. 2018;16(10):1984–1993. doi: 10.1111/jth.14247. PubMed DOI
Butterfield JSS, Hege KM, Herzog RW, Kaczmarek R. A molecular revolution in the treatment of hemophilia. Mol Ther. 2019;28(4):997. doi: 10.1016/j.ymthe.2019.11.006. PubMed DOI PMC
The Hemophilia Inhibitor Prevention Trial. https://www.clinicaltrials.gov/ct2/show/NCT04303559. 22-7-2020.
A Study to Evaluate the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Subcutaneous Emicizumab in Participants From Birth to 12 Months of Age With Hemophilia A Without Inhibitors (HAVEN 7). https://www.clinicaltrials.gov/ct2/show/NCT04431726?term=emicizumab&draw=2&rank=6 . 22-7-0020.
Pierce GF, Hart DP, Kaczmarek R. Safety and efficacy of emicizumab and other novel agents in newborns and infants. Haemophilia. 2019;25(5):e334–e335. doi: 10.1111/hae.13822. PubMed DOI
Jenkins PV, Bowyer A, Burgess C, Gray E, Kitchen S. Murphy P et al. Haemophilia: Laboratory coagulation tests and emicizumab treatment A United Kingdom Haemophilia Centre Doctors' Organisation guideline; 2019. p. 151. PubMed
Susen S, Gruel Y, Godier A, Harroche A, Chambost H, Lasne D, et al. Management of bleeding and invasive procedures in haemophilia a patients with inhibitor treated with emicizumab (Hemlibra((R)) ): proposals from the French network on inherited bleeding disorders (MHEMO), the French reference Centre on Haemophilia, in collaboration with the French working group on perioperative Haemostasis (GIHP) Haemophilia. 2019;25(5):731–737. doi: 10.1111/hae.13817. PubMed DOI
Castaman G, Santoro C, Coppola A, Mancuso ME, Santoro RC, Bernardini S, et al. Emergency management in patients with haemophilia a and inhibitors on prophylaxis with emicizumab: AICE practical guidance in collaboration with SIBioC, SIMEU, SIMEUP, SIPMeL and SISET. Blood Transfus. 2019;18:1–8. PubMed PMC
Carcao M, Escuriola-Ettingshausen C, Santagostino E, Oldenburg J, Liesner R, Nolan B, et al. The changing face of immune tolerance induction in haemophilia a with the advent of emicizumab. Haemophilia. 2019;25(4):676–684. PubMed PMC
Batsuli G, Zimowski KL, Tickle K, Meeks SL, Sidonio RF., Jr Immune tolerance induction in paediatric patients with haemophilia a and inhibitors receiving emicizumab prophylaxis. Haemophilia. 2019;25(5):789–796. doi: 10.1111/hae.13819. PubMed DOI
The Hemophilia Inhibitor Eradication Trial. https://clinicaltrials.gov/ct2/show/NCT04303572.
A Study to Evaluate the Efficacy, Safety, and Pharmacokinetics of Prophylactic Emicizumab Versus no Prophylaxis in Hemophilia A Participants With Inhibitors (HAVEN 1). https://www.clinicaltrials.gov/ct2/show/NCT02622321.
Kajiwara M, Shima M, Yoshioka A. Two haemophilia patients with inhibitors who became ambulatory after physiotherapy under haemostatic cover with bypassing agents. Haemophilia. 2013;19(5):e301–e304. doi: 10.1111/hae.12179. PubMed DOI
