Meeting unmet needs in inhibitor patients

Haemophilia

Volumn 16, Issue SUPPL. 3, 2010, Pages 46-51

  Blanchette, V S ^a   Manco Johnson, M J ^b  

^a HOSPITAL FOR SICK CHILDREN   (Canada)

^b Mountain States Regional Hemophilia and Thrombosis Center   (United States)

Author keywords

Haemophilia; Haemophilia with inhibitors; Unmet needs

Indexed keywords

ACTIVATED PROTHROMBIN COMPLEX; AMINOCAPROIC ACID; BLOOD CLOTTING FACTOR 8; BLOOD CLOTTING FACTOR 9; PREDNISONE; RECOMBINANT BLOOD CLOTTING FACTOR 7A; RECOMBINANT BLOOD CLOTTING FACTOR 8; RITUXIMAB; TRANEXAMIC ACID;

ARTICLE; BLEEDING; DRUG CLEARANCE; DRUG EFFICACY; DRUG MEGADOSE; ENCEPHALOMYOPATHY; HEMARTHROSIS; HEMOPHILIA; HEMOPHILIA A; HEMOPHILIA B; HOMEOSTASIS; HUMAN; HYDROTHERAPY; IMMUNOLOGICAL TOLERANCE; NONHUMAN; PHYSIOTHERAPY; PLASMAPHERESIS; PREVALENCE; PRIORITY JOURNAL; PUBLICATION; QUALITY OF LIFE; RECURRENT DISEASE; SERUM SICKNESS; SINGLE DRUG DOSE; SYMPOSIUM; SYNOVECTOMY; SYNOVITIS; TREATMENT DURATION; TREATMENT INDICATION; TREATMENT RESPONSE;

BLOOD COAGULATION FACTOR INHIBITORS; BLOOD COAGULATION FACTORS; DISEASE MANAGEMENT; HEMOPHILIA A; HEMOPHILIA B; HEMORRHAGE; HUMANS; JOINT DISEASES; SYNOVITIS;

EID: 77953495146     PISSN: 13518216     EISSN: 13652516     Source Type: Journal    
DOI: 10.1111/j.1365-2516.2010.02260.x     Document Type: Article

References (28)

1. Wight J, Paisley S. The epidemiology of inhibitors in haemophilia A: a systematic review. Haemophilia 2003, 9:418-35.
2. Kessler CM. New perspectives in haemophilia treatment. Hematology Am Soc Hematol Educ Program 2005, 1:429-35.
3. Darby SC, Keeling DM, Spooner RJ. UK Haemophilia Centre Doctors' Organisation. The incidence of factor VIII and factor IX inhibitors in the hemophilia population of the UK and their effect on subsequent mortality, 1977-99. J Thromb Haemost 2004, 2:1047-54.
4. White GC, Rosendaal F, Aledort LM, Lusher JM, Rothschild C, Ingerslev J. Definitions in haemophilia. 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.
5. Hay CR, Baglin TP, Collins PW, Hill FG, Keeling DM. The diagnosis and management of factor VIII and IX inhibitors: a guideline from the UK Haemophilia Centre Doctors' Organization (UKHCDO). Br J Haematol 2000, 111:78-90.
6. Key NS, Aledort LM, Beardsley D. Home treatment of mild to moderate bleeding episodes using recombinant factor VIIa (Novoseven) in haemophiliacs with inhibitors. Thromb Haemost 1998, 80:912-8.
7. Kenet G, Lubetsky A, Luboshitz J, Martinowitz U. A new approach to treatment of bleeding episodes in young hemophilia patients: a single bolus megadose of recombinant activated factor VII (NovoSeven®). J Thromb Haemost 2003, 1:450-5.
8. Kavakli K, Makris M, Zulfikar B. Home treatment of haemarthroses using a single dose regimen of recombinant activated factor VII in patients with haemophilia and inhibitors. A multi-centre, randomised, double-blind, cross-over trial. Thromb Haemost 2006, 95:600-5.
9. Santagostino E, Mancuso ME, Rocino A, Mancuso G, Scaraggi F, Mannucci PM. A prospective randomized trial of high and standard dosages of recombinant factor VIIa for treatment of hemarthroses in hemophiliacs with inhibitors. J Thromb Haemost 2006, 4:367-71.
10. Young G, Shafer FE, Rojas P, Seremetis S. Single 270 μg kg-1-dose rFVIIa vs. standard 90 μg kg-1-dose rFVIIa and APCC for home treatment of joint bleeds in haemophilia patients with inhibitors: a randomized comparison. Haemophilia 2008, 14:287-94.
11. Villar A, Aronis S, Morfini M. Pharmacokinetics of activated recombinant coagulation factor VII (NovoSeven®) in children vs. adults with haemophilia A. Haemophilia 2004, 10:352-9.
12. DiMichele DM, Hoots WK, Pipe SW, Rivard GE, Santagostino E. International workshop on immune tolerance induction: consensus recommendations. Haemophilia 2007, 13(Suppl. 1):1-22.
13. Todd DJ, Helfgott SM. Serum sickness following treatment with rituximab. J Rheumatol 2007, 34:430-3.
14. Frick M, Maloney C, Tallman S, Samaras AT, McKoy JM, Bennett CL. Progressive multifocal leukoencephalopathy following rituximab treatment. Community Oncol 2009, 6:410-1.
15. Raffini L, Manno C. Modern management of haemophilic arthropathy. Br J Haematol 2007, 136:777-87.
16. Lafeber FPJG, Miossec P, Valentino LA. Physiopathology of haemophilic arthropathy. Haemophilia 2008, 14(Suppl. 4):3-9.
17. Lusher JM. Acute hemarthroses: the benefits of early versus late treatment with recombinant activated factor VII. Blood Coagul Fibrinolysis 2000, 11(Suppl. 1):S45-9.
18. McDonald A, Hoffman M, Hedner U, Roberts HR, Monroe DM. Restoring hemostatic thrombin generation at the time of cutaneous wounding does not normalize healing in hemophilia B. J Thromb Haemost 2007, 5:1577-83.
19. Nilsson IM, Berntorp E, Löfqvist T, Pettersson H. Twenty-five years' experience of prophylactic treatment in severe haemophilia A and haemophilia B. J Intern Med 1992, 232:25-32.
20. Fischer K, van der Bom JG, Mauser-Bunschoten EP. The effects of postponing prophylactic treatment on long-term outcome in patients with severe haemophilia. Blood 2002, 99:2337-41.
21. Manco-Johnson MJ, Abshire TC, Shapiro AD, Riske B, Hacker MR. Disease in boys with severe hemophilia. N Engl J Med 2007, 357:535-44.
22. Konkle BA, Ebbesen LS, Erhardtsen E. Randomized, prospective clinical trial of recombinant factor VIIa for secondary prophylaxis in hemophilia patients with inhibitors. J Thromb Haemost 2007, 5:1904-13.
23. Jiménez-Yuste V, Alvarez MT, Martín-Salces M. Prophylaxis in 10 patients with severe haemophilia A and inhibitor: different approaches for different clinical situations. Haemophilia 2009, 15:203-9.
24. Valentino LA, Carcao M, Mathew P. The application of bypassing-agent prophylaxis in haemophilia A patients with inhibitors: a meeting report. Haemophilia 2009, 15:959-65.
25. Manco-Johnson MJ, Nuss R, Lear J. 32P radiosynoviorthesis in children with hemophilia. J Pediatr Hematol Oncol 2002, 24:534-9.
26. Nuss R, Kilcoyne RF, Geraghty S. MRI findings in haemophilic joints treated with radiosynoviorthesis with development of an MRI scale of joint damage. Haemophilia 2000, 6:162-9.
27. Dunn AL, Manco-Johnson M, Busch MT, Balark KL, Abshire TC. Leukemia and P32 radionuclide synovectomy for hemophilic arthropathy. J Thromb Haemost 2005, 3:1541-2.
28. MASAC Document # 176. MASAC recommendations following radionuclide synovectomy. 2006, http://www.hemophilia.org/NHFWeb/MainPgs/MainNHF.aspx?menuid=57&contentid=692, Available at Accessed October 19, 2009