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    Sei qui:

    1. Patologie
    2. Emofilia - Bibliografia
    Emofilia

    Introduzione

    Epidemiologia

    Presentazione clinica e diagnosi

    Gestione clinica

    Regimi di trattamento

    Bibliografia

    Bibliografia
    1. Mannucci PM, Tuddenham EG. The hemophilias--from royal genes to gene therapy. N Engl J Med 2001; 344:1773-9
    2. White GC 2nd, Rosendaal F, Aledort LM, Lusher JM, Rothschild C, Ingerslev J; Factor VIII and Factor IX Subcommittee. Definitions in hemophilia. Recommendation of the scientific subcommittee on factor VIII and factor IX of the scientific and standardization committee of the International Society on Thrombosis and Haemostasis. Thromb Haemost 2001; 85:560
    3. Kasper CK, Lin JC. Prevalence of sporadic and familial haemophilia. Haemophilia 2007; 13:90-2
    4. Srivastava A, Santagostino E, Dougall A, et al. WFH Guidelines for the Management of Hemophilia 3rd edition. Haemophilia 2020; 26 Suppl 6: 1-158 
    5. 5. World Federation of Hemophilia. Report on the Annual Global Survey 2017. October 2018. Available at http://www1.wfh.org/publications/files/pdf-1714.pdf. Accessed September 8, 2020 
    6. Abbonizio F, Hassan HJ, Riccioni R, et al. New data from the Italian National Register of Congenital Coagulopathies, 2016 Annual Survey. Blood Transfus 2020; 18: 58-66 
    7. Rocino A, Coppola A, Franchini M, et al. Principles of treatment and update of recommendations for the management of haemophilia and congenital bleeding disorders in Italy. Blood Transfus 2014; 12: 575-98 
    8. Mannucci PM, Franchini M, Present and future challanges in the treatment of haemophilia: a clinician perspective, Blood Transfus. 2013 Sep; 11 Suppl 4:s77-81 
    9. Mannucci et al, Recessively inherited coagulation disorders, Blood. 2004 Sep 1;104(5):1243-52 
    10. Onwuzurike et al, Types of bleeding seen during the first 30 months of life in children with severe haemophilia A and B, Haemophilia. 1996 Jul; 2(3):137- 40 
    11. Chambost et al, What factors influence the age at diagnosis of hemophilia? Results of the French hemophilia cohort, J Pediatr. 2002 Oct;141(4):548-52 
    12. Margaglione et al, The Italian AICE-Genetics hemophilia A database: results and correlation with clinical phenotype, Haematologica. 2008 May; 93(5):722- 8. doi:10.3324/haematol.12427. Epub 2008 Apr 2 
    13. Srivastava et al. WFH Guidelines for the Management of Hemophilia, 3rd edition. Haemophilia. 2020 Aug;26 Suppl 6:1-158 
    14. Aledort LM, A longitudinal study of orthopaedic outcomes for severe factor-VIII- deficient haemophiliacs. The Orthopaedic Outcome Study Group, J Intern Med. 1994 Oct; 236(4):391-9 
    15. Astermark J, Primary prophylaxis in severe haemophilia should be started at an early age but can be individualized, Br J Haematol. 1999 Jun; 105(4):1109-13 
    16. Manco-Johnson et al, Prophylaxis versus episodic treatment to prevent joint disease in boys with severe hemophilia, N Engl J Med. 2007 Aug 9; 357(6):535-44 
    17. Steven W. Pipe New therapies for hemophilia (Hematology Am Soc Hematol Educ Program. 2016 Dec 2; 2016(1): 650–656) 
    18. Peyvandi et al. Future of coagulation factor replacement therapy. J Thromb Haemost. 2013; Suppl 1:84-98 
    19. Warren BB et al. Young adult outcomes of childhood prophylaxis for severe hemophilia A: results of the Joint Outcome Continuation Study. Blood Adv. 2020 Jun 9;4(11):2451-2459 
    20. Mahlangu J, Powell JS, Ragni MV, et al Phase 3 study of recombinant factor VIII Fc fusion protein in severe hemophilia A. Blood 123:317–325, 2014 
    21. Konkle BA, Stasyshyn O, Chowdary P, et al Pegylated, full-length, recombinant factor VIII for prophylactic and on-demand treatmentof severe hemophilia A. Blood 126:1078–1085, 2015 
    22. Nuto A, Yoshihashi K, Takeda M, et al Anti-factor IXa/X bispecific antibody (ACE910): Hemostatic potency against ongoing bleeds in ahemophilia A model and the possibility of routine supplementation. J Thromb Haemost 12:206–213, 2014 
    23. Sehgal A, Barros S, Ivanciu L, et al An RNAi therapeutic targeting antithrombin to rebalance the coagulation system and promotehemostasis in hemophilia. Nat Med 21:492–497, 2015 
    24. Chowdary P, Lethagen S, Friedrich U, et al Safety and pharmacokinetics of anti-TFPI antibody (concizumab) in healthy volunteers andpatients with hemophilia: A randomized first human dose trial. J Thromb Haemost 13:743–754, 2015 
    25. George LA: Hemophilia gene therapy comes of age. Blood Adv 1:2591–2599, 2017. 
    26. Franchini M, Mannucci PM. Hemophilia A in the third millennium. Blood Reviews 2013;
    27. 179-84 27. Ljung R, Auerswald G, Benson G, et al. Inhibitors in haemophilia A and B: Management of bleeds, inhibitor eradication and strategies for difficult-to-treat patients. Eur J Haematol 2019; 102: 111-22
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