The inhibitors – a challenge for the management of patients with hereditary haemophilia A
References
- 1. TABRINZIA-TABRIZI S., GHOLAMPOUR M., MANSOURITORGHABEH H.
A close insight to factor VIII inhibitor in the congenital hemophilia A . Expert Rev Hematol. 2016;9 :903-913. - 2. RODRIGUEZ-MERCHÁN E.C.
Orthopedic surgery is possible in hemophilic patients with inhibitors . Am J Orthop (Belle Mead NJ). 2012;41 :570-574. - 3. FINN J.D., OZELO M.C., SABATINO D.E., FRANCK H.W., MERRICKS E.P., CRUDELE J.M.,
et al .Eradication of neutralizing antibodies to factor VIII in canine hemophilia A after liver gene therapy . Blood. 2010;116 :5842-5848. - 4. LAI J., HOUGH C., TARRANT J., LILLICRAP D.
Biological considerations of plasma-derived and recombinant factor VIII immunogenicity . Blood. 2017;129 :3147-3154. - 5. OWAIDAH T., MOMEN A.A., ALZAHRANI H., ALMUSA A., ALKASIM F., TARAWAH A.,
et al. The prevalence of factor VIII and IX inhibitors among Saudi patients with hemophilia: Results from the Saudi national hemophilia screening program . Medicine (Baltimore). 2017;96 :e5456. - 6. BENSON G., AUERSWALD G., ELEZOVIĆ I., LAMBERT T., LJUNG R., MORFINI M.,
et al. Immune tolerance induction in patients with severe hemophilia with inhibitors: expert panel views and recommendations for clinical practice . Eur J Haematol. 2012;88 :371-379. - 7. ROCINO A., FRANCHINI M., COPPOLA A.
Treatment and Prevention of Bleeds in Haemophilia Patients with Inhibitors to Factor VIII/IX . J Clin Med. 2017;6 ;pii:E46. - 8. ECKHARDT C.L., LOOMANS J.I., VAN VELZEN A.S., PETERS M., MAUSER-BUNSHOTEN E.P., SCHWAAB R.,
et al. Inhibitor development and mortality in non-severe hemophilia A . J Thromb Haemost. 2015;13 :1217-1225. - 9. FRANCHINI M., LIPPI G.
Prevention of inhibitor development in hemophilia A in 2016. A glimpse into the future? Thromb Res. 2016;148 :96-100. - 10. VAN DEN BERG H.M., HASHEMI S.M., FISCHER K., PETRINI P., LJUNG R., RAFOWICZ A.,
et al. Increased inhibitor incidence in severe haemophilia A since 1990 attributable to more low titre inhibitors . Thromb Haemost. 2016;115 :729-737. - 11. VAN DEN BERG H.M., LJUNG R.; PEDNET STUDY GROUP.
Can a “center effect” explain the higher frequency of inhibitors for a second-generation recombinant factor VIII product? Blood. 2015;126 :2164-2165. - 12. FISCHER K., IORIO A., LASSILA R., PEYVANDI F., CALIZZANI G., GATT A.,
et al. Inhibitor development in non-severe haemophilia across Europe . Thromb Haemost. 2015;114 :670-675. - 13. KARAMAN K, AKBAYRAM S, GARIPARDIÇ M, ÖNER AF.
Diagnostic evaluation of our patients with hemophilia A: 17-year experience . Turk Pediatri Ars. 2015;50 :96-101. - 14. BLATNY J., KOMRSKA V., BLAZEK B., PENKA M., OVESNA P.; CZECH NATIONAL HAEMOPHILIA PROGRAMME.
Inhibitors incidence rate in Czech previously untreated patients with haemophilia A has not increased since introduction of recombinant factor VIII treatment in 2003 . Blood Coagul Fibrinolysis. 2015;26 :673-678. - 15. HALIMEH S., BIDLINGMAIER C., HELLER C., GUTSCHE S., HOLZHAUER S., KENET G.,
et al. Risk factors for high-titer inhibitor development in children with hemophilia A: results of a cohort study . Biomed Res Int. 2013;2013 :901975. - 16. TUNSTALL O., ASTERMARK J.
Strategies for reducing inhibitor formation in severe haemophilia . Eur J Haematol. 2015;94 Suppl 77:45-50. - 17. ÁLVAREZ T., SOTO I., ASTERMARK J.
Non-genetic risk factors and their influence on the management of patients in the clinic . Eur J Haematol. 2015;94 Suppl 77:2-6. - 18. CARACO M., RE W., EWENSTEIN B.
The role of previously untreated patient studies in understanding the development of FVIII inhibitors . Haemophilia. 2016;22 :22-31. - 19. TER AVEST P.C., FISCHER K., MANCUSO M.E., SANTAGOSTINO E., YUSTE V.J., VAN DEN BERG H.M.,
et al. Risk stratification for inhibitor development at first treatment for severe hemophilia A: a tool for clinical practice . J Thromb Haemost. 2008;6 :2048-2054. - 20. MARCUCCI M., MANCUSO M.E., SANTAGOSTINO E., KENET G., ELALFY M., HOLZHAUER S.,
et al. Type and intensity of FVIII exposure on inhibitor development in PUPs with haemophilia A . A patient-level meta-analysis. Thromb Haemost. 2015;113 :958-967. - 21. KURNIK K., BIDLINGMAIER C., ENGL W., CHEHADEH H., REIPERT B., AUERSWALD G.
New early prophylaxis regimen that avoids immunological danger signals can reduce FVIII inhibitor development . Haemophilia. 2010;16 :256-262. - 22. JAMES E.A., VAN HAREN S.D., ETTINGER R.A., FIJNVANDRAAT K., LIBERMAN J.A., KWOK W.W.,
et al. T-cell responses in two unrelated hemophilia A inhibitor subjects include an epitope at the factor VIII R593C missense site . J Thromb Haemost. 2011;9 :689-699. - 23. YADA K., NOGAMI K., TAKEYAMA M., OGIWARA K., WAKABAYASHI H., SHIMA M.
Mild hemophilia A patient with novel Pro1809Leu mutation develops an anti-C2 antibody inhibiting allogeneic but not autologous factor VIII activity . J Thromb Haemost. 2015;13 :1843-1853. - 24. SHIMA M., LILLICRAP D., KRUSE-JARRES R.
Alternative therapies for the management of inhibitors . Haemophilia. 2016;22 Suppl 5:36-41. - 25. LAI J.D., MOOREHEAD P.C., SPONAGLE K., STEINITZ K.N., REIPERT B.M., HOUGH C,
et al. Concurrent influenza vaccination reduces anti-FVIII antibody responses in murine hemophilia A . Blood. 2016;127 :3439-3449. - 26. LÖVGREN K.M., SØNDERGAARD H., SKOV S., WIINBERG B.
Joint bleeds increase the inhibitor response to human factor VIII in a rat model of severe haemophilia A . Haemophilia 2016;22 :772-779. - 27. FISCHER K., LASSILA R., PEYVANDI F., CALIZZANI G., GATT A., LAMBERT T.,
et al. Inhibitor development in haemophilia according to concentrate. Four-year results from the European HAemophilia Safety Surveillance (EUHASS) project . Thromb Haemost. 2015;113 :968-975. - 28. MANCUSO M.E., MANNUCCI P.M., ROCINO A., GARAGIOLA I., TAGLIAFERRI A., SANTAGOSTINO E.
Source and purity of factor VIII products as risk factors for inhibitor development in patients with hemophilia A . J Thromb Haemost. 2012;10 :781-790. - 29. FEISTRITZER C., SCHMIDT S.
Reversal of direct oral anticoagulants in hemophilia treatment: ASH meeting 2015 . Memo. 2016;9 :131-135. - 30. MATHEW P., DINTER H., CHURCH N., HUMPHRIES T.J., KULKARNI R.
Inhibitors in haemophilia A: a perspective on clotting factor products as a potential contributing factor . Haemophilia. 2016;22 :334-341. - 31. CALVEZ T., CHAMBOST H., CLAEYSSENS-DONADEL S., D’OIRON R., GOULET V., GUILLET B.,
et al. Recombinant factor VIII products and inhibitor development in previously untreated boys with severe hemophilia A . Blood. 2014;124 :3398-3408. - 32. COLLINS P.W., PALMER B.P., CHALMERS E.A., HART D.P., LIESNER R., RANGARAJAN S.,
et al. Factor VIII brand and the incidence of factor VIII inhibitors in previously untreated UK children with severe hemophilia A, 2000-2011 . Blood. 2014;124 :3389-3397. - 33. AZNAR J.A., MORET A., IBÁÑEZ F., VILA C., CABRERA N., MESA E.,
et al. Inhibitor development after switching of FVIII concentrate in multitransfused patients with severe haemophilia A . Haemophilia. 2014;20 :624-629. - 34. KOCHER S., ASMELASH G., MAKKI V., MÜLLER S., KREKELER S., ALESCI S.,
et al. Inhibitor development after changing FVIII/IX products in patients with haemophilia . Hamostaseologie. 2012;32 Suppl 1:S39-42. - 35. PARRA LOPEZ R., NEMES L., JIMENEZ-YUSTE V., RUSEN L., CID A.R., CHARNIGO R.J.,
et al. Prospective surveillance study of haemophilia A patients switching from moroctocog alfa or other factor VIII products to moroctocog alfa albumin-free cell culture (AF-CC) in usual care settings . Thromb Haemost. 2015;114 :676-684. - 36. MANNUCCI P.M., MANCUSO M.E., FRANCHINI M.
Tailoring hemostatic therapies to lower inhibitor development in previously untreated patients with severe hemophilia A . J Thromb Haemost. 2016;14 :1330-1336. - 37. KAHLE J., ORLOWSKI A., STICHEL D., HEALEY J.F., PARKER E.T., JAKEMIN M.,
et al. Frequency and epitope specificity of anti-factor VIII C1 domain antibodies in acquired and congenital hemophilia A . Blood. 2017; pii:blood-2016-11-751347. - 38. MATINO D., GARGARO M., SANTAGOSTINO E., DI MINNO M.N., CASTAMAN G., MORFINI M.,
et al. IDO1 suppresses inhibitor development in hemophilia A treated with factor VIII . J Clin Invest. 2015;125 :3766-3781. - 39. OLDENBURG J., LACROIX-DESMAZES S., LILLICRAP D.
Alloantibodies to therapeutic factor VIII in hemophilia A: the role of von Willebrand factor in regulating factor VIII immunogenicity . Haematologica. 2015;100 :149-156. - 40. MILLER L., WEISSMÜLLER S., RINGLER E., CRAUWELS P., van ZANDBERGEN G., SEITZ R.,
et al. Danger signal-dependent activation of human dendritic cells by plasma-derived factor VIII products . Thromb Haemost. 2015;114 :268-276. - 41. JACQUEMIN M., SAINT-REMY J.M.
T cell response to FVIII . Cell Immunol. 2016;301 :8-11. - 42. WANG X., SU J., SHERMAN A., ROGERS G.L., LIAO G., HOFFMAN B.E.,
et al. Plant-based oral tolerance to hemophilia therapy employs a complex immune regulatory response including LAP+CD4+ T cells . Blood. 2015;125 :2418-2427. - 43. ALLACHER P., BAUMGARTNER C.K., PORDES A.G., AHMAD R.U., SCHWARZ H.P., REIPERT B.M.
Stimulation and inhibition of FVIII-specific memory B-cell responses by CpG-B (ODN 1826), a ligand for Toll-like receptor 9 . Blood. 2011;117 :259-267. - 44. WERWITZKE S., VOLLACK N., VON HORNUNG M., KALIPPKE K., KUTZSCHBACH J., TRUMMER A.,
et al. Deletion or inhibition of Fc gamma receptor 2B (CD32) prevents FVIII-specific activation of memory B cells in vitro . Thromb Haemost. 2015;114 :1127-1135. - 45. BATOROVA A., JANKOVICOVA D., MORONGOVA A., BUBANSKA E., PRIGANCOVA T., HORAKOVA J.,
et al. Inhibitors in Severe Hemophilia A: 25-Year Experience in Slovakia . Semin Thromb Hemost. 2016;42 :550-562. - 46. MANNUCCI P.M., GARAGIOLA I.
Factor VIII products in haemophilia A: one size fits all? Thromb Haemost 2015;113 :911-914. - 47. DASGUPTA S., REPESSE Y., BAYRY J., NAVARRETE A.M., WOOTLA B., DELIGNAT S.,
et al. VWF protects FVIII from endocytosis by dendritic cells and subsequent presentation to immune effectors . Blood. 2007;109 :610–612. - 48. QADURA M., WATERS B., BURNETT E., CHEGENI R., BRADSHAW S., HOUGH C.,
et al. Recombinant and plasma-derived factor VIII products induce distinct splenic cytokine microenvironments in haemophilia A mice . Blood. 2009;114 :871-880. - 49. GHIO M., CONTINI P., OTTONELLO L., ARDUINO N., GRINGERI A., INDIVERI F.,
et al. Effect of clotting factors concentrates on lymphocyte and neutrophil function in vitro . Thromb Haemost. 2003;89 :365–373. - 50. PEYVANDI F., MANNUCCI P.M., GARAGIOLA I., EL-BESHLAWY A., ELALFY M., RAMANAN V.,
et al. A Randomized Trial of Factor VIII and Neutralizing Antibodies in Hemophilia A . N Engl J Med. 2016;374 :2054-2064. - 51. MANNUCCI P.M., MANCUSO M.E., SANTAGOSTINO E.
How we choose factor VIII to treat haemophilia . Blood. 2012;119 :4108-4114. - 52. PEYVANDI F., MANNUCCI P.M., PALLA R., ROSENDAAL F.R.
SIPPET: methodology, analysis and generalizability . Haemophilia. 2017;23 :353-361. - 53. AFONJA O., KOZAK R., PETRARO P., MICHAELS L.A., MATHEW P., LEMM G.,
et al. Baby hamster kidney cell-derived recombinant factor VIII: a quarter century of learning and clinical experience . Expert Rev Hematol. 2016;9 :1151-1164. - 54. HARTMANN J., CROTEAU S.E.
2017 Clinical trials update: Innovations in hemophilia therapy . Am J Hematol. 2016;91 :1252-1260. - 55. YOON J., SCHMIDT A., ZHANG A.H., KÖNIGS C., KIM Y.C., SCOTT D.W.
FVIII-specific human chimeric antigen receptor T-regulatory cells suppress T- and B-cell responses to FVIII . Blood. 2017;129 :238-245.
Language: English
Page range: 143 - 152
Submitted on: Mar 3, 2018
Published on: Aug 29, 2018
Published by: N.G. Lupu Internal Medicine Foundation
In partnership with: Paradigm Publishing Services
Publication frequency: 4 times per year
Keywords:
Related subjects:
© 2018 Romeo-Gabriel Mihăilă, published by N.G. Lupu Internal Medicine Foundation
This work is licensed under the Creative Commons Attribution-NonCommercial-NoDerivatives 4.0 License.