Prophylaxis for severe haemophilia: Clinical challenges in the absence as well as in the presence of inhibitors

Haemophilia

Volumn 14, Issue SUPPL. 3, 2008, Pages 196-201

  Fischer, Kathelijn ^a,b   Valentino, L ^c   Ljung, R ^d   Blanchette, V ^e  

^a UNIVERSITY MEDICAL CENTER UTRECHT   (Netherlands)

^b UNIVERSITY MEDICAL CENTER UTRECHT   (Netherlands)

^c Rush Children's Hospital and Rush University Medical Center   (United States)

^d LUND UNIVERSITY HOSPITAL   (Sweden)

^e HOSPITAL FOR SICK CHILDREN   (Canada)

Author keywords

Arthropathy; Bypassing agents; Dose; Frequency; Inhibitors; Prophylaxis

Indexed keywords

ACTIVATED PROTHROMBIN COMPLEX; BLOOD CLOTTING FACTOR 8; BLOOD CLOTTING FACTOR CONCENTRATE; RECOMBINANT BLOOD CLOTTING FACTOR 7A;

ADULT; ARTHROPATHY; ARTICLE; BRAIN HEMORRHAGE; CHILD; CLINICAL TRIAL; DISEASE SEVERITY; DRUG DOSE INCREASE; DRUG DOSE REDUCTION; DRUG DOSE REGIMEN; HEMARTHROSIS; HEMOPHILIA; HEMOPHILIA A; HEMOPHILIA B; HUMAN; IMMUNOLOGICAL TOLERANCE; LIFESTYLE MODIFICATION; ONSET AGE; PATIENT ATTITUDE; PATIENT COMPLIANCE; PATIENT EDUCATION; PHYSICAL ACTIVITY; PRIORITY JOURNAL; PROPHYLAXIS; PSYCHOLOGICAL ASPECT; QUALITY OF LIFE; SECONDARY PREVENTION; SELF CARE;

ADOLESCENT; CHILD; COHORT STUDIES; DRUG ADMINISTRATION SCHEDULE; FACTOR IX; FACTOR VIII; HEMARTHROSIS; HEMOPHILIA A; HUMANS; MALE; PATIENT COMPLIANCE; QUALITY OF LIFE; RANDOMIZED CONTROLLED TRIALS AS TOPIC; TREATMENT OUTCOME;

EID: 44249113317     PISSN: 13518216     EISSN: 13652516     Source Type: Journal    
DOI: 10.1111/j.1365-2516.2008.01736.x     Document Type: Article

References (43)

1. Nilsson IM, Berntorp E, Lofqvist T, Pettersson H. Twenty-five years' experience of prophylactic treatment in severe haemophilia A and B. J Intern Med 1992; 232: 25-32.
2. Löfqvist T, Nilsson IM, Berntorp E, Pettersson H. Haemophilia prophylaxis in young patients - a long-term follow-up. J Intern Med 1997; 241: 395-400.
3. Ahlberg A. Haemophilia in Sweden VII. Incidence, treatment and prophylaxis of arthropathy and other musculo-skeletal manifestations of haemophilia A and B. Acta Orthop Scand 1965; 77(Suppl.): 5-99.
4. Berntorp E, Astermark J, Bjorkman S et al. Consensus perspectives on prophylactic therapy for haemophilia: Summary statement. Haemophilia 2003; 9(Suppl. 1): 1-4.
5. National Haemophilia Foundation. MASAC Recommendations Concerning Prophylaxis (Regular Administration of Clotting Factor Concentrate to Prevent Bleeding), 2007 (Chapter Advisory 197). Available at: http://www.haemophilia.org/NHFWeb/MainPgs/MainNHF.aspx?menuid= 57&contentid=1007 (last accessed on March 23rd 2008).
6. Aledort LM, Haschmeyer RH, Pettersson H. A longitudinal study of orthopaedic outcomes for severe factor-VIII-deficient haemophiliacs. The Orthopaedic Outcome Study Group. J Intern Med 1994; 236: 391-9.
7. Fischer K, Van der Bom JG, Mauser-Bunschoten EP et al. Changes in treatment strategies for severe haemophilia over the last 3 decades: effects on clotting factor consumption and arthropathy. Haemophilia 2001; 7: 446-52.
8. Steen Carlsson K, Hojgard S, Glomstein A et al. On-demand vs. prophylactic treatment for severe haemophilia in Norway and Sweden: differences in treatment characterstics and outcome. Haemophilia 2003; 9: 555-66.
9. Feldman BM, Pai M, Rivard GE et al. Tailored prophylaxis in severe haemophilia A: Interim results from the first 5 years of the Canadian Haemophilia Primary Prophylaxis Study. J Thromb Haemost 2006; 4: 1228-36.
10. Manco-Johnson MJ, Abshire TC, Shapiro AD et al. Prophylaxis versus episodic treatment to prevent joint disease in boys with severe haemophilia. N Engl J Med 2007; 357: 535-44.
11. Astermark J, Petrini P, Tengborn L et al. Primary prophylaxis in severe haemophilia should be started at an early age but can be individualized. Br J Haematol 1999; 105: 1109-13.
12. Fischer K, Van der Bom JG, Mauser-Bunschoten EP et al. Effects of postponing prophylactic treatment on long-term outcome in patients with severe haemophilia. Blood 2002; 99: 2337-41.
13. Kreuz W, Escuriola-Ettingshausen C, Funk M et al. When should prophylactic treatment in patients with haemophilia A and B start? - the German experience. Haemophilia 1998; 4: 413-7.
14. Van Dijk K, Fischer K, Van der Bom JG et al. Variability in clinical phenotype of severe haemophilia: The role of the first joint bleed. Haemophilia 2005; 11: 438-43.
15. Gouw SC, Van der Bom JG, Marijke van den BH. Treatment-related risk factors of inhibitor development in previously untreated patients with haemophilia A: The CANAL cohort study. Blood 2007; 109: 4648-54.
16. Ljung RC. Intracranial haemorrhage in haemophilia A and B. Haematology 2008; 140: 378-84.
17. Petrini P. What factors should influence the dosage and interval of prophylactic treatment in patients with severe haemophilia A and B? Haemophilia 2001; 7: 99-102.
18. Barlow JH, Stapley J, Ellard DR, Gilchrist M. Information and self-management needs of people living with bleeding disorders: A survey. Haemophilia 2007; 13: 264-70.
19. Kyngas HA, Kroll T, Duffy ME. Compliance in adolescents with chronic diseases: A review. J Adolesc Health 2000; 26: 379-88.
20. Kyngas H. Compliance of adolescents with chronic disease. J Clin Nurs 2000; 9: 549-56.
21. Fischer K, Collins P, Astermark J et al. Internal and external determinants of bleeding patterns on prophylaxis for severe haemophilia. Blood 2007; 110: 1145. (Abstract).
22. Hacker MR, Geraghty S, Manco-Johnson M. Barriers to compliance with prophylaxis therapy in haemophilia. Haemophilia 2001; 7: 392-6.
23. Lindvall K, Colstrup L, Wollter IM et al. Compliance with treatment and understanding of own disease in patients with severe and moderate haemophilia. Haemophilia 2006; 12: 47-51.
24. Fischer K, Van der Bom JG, Prejs R et al. Discontinuation of prophylactic therapy in severe haemophilia: Incidence and effects on outcome. Haemophilia 2001; 7: 544-50.
25. Van Dijk K, Fischer K, Van der Bom JG et al. Can long-term prophylaxis for severe haemophilia be stopped in adulthood? Results from Denmark and the Netherlands. Br J Haematol 2005; 130: 107-12.
26. White GC, Rosendaal FR, Aledort LM et al. 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.
27. Lusher JM. Inhibitor antibodies to factor VIII and factor IX: management. Semin Thromb Hemost 2008; 26: 179-88.
28. Ho AY, Height SE, Smith MP. Immune tolerance therapy for haemophilia. Drugs 2000; 60: 547-54.
29. Leissinger CA. Prevention of bleeds in haemophilia patients with inhibitors: Emerging data and clinical direction. Am J Hematol 2004; 77: 187-93.
30. Leissinger CA. Use of prothrombin complex concentrates and activated prothrombin complex concentrates as prophylactic therapy in haemophilia patients with inhibitors. Haemophilia 1999; 5(Suppl. 3): 25-32.
31. Soucie JM, Cianfrini C, Janco RL et al. Joint range-of-motion limitations among young males with haemophilia: Prevalence and risk factors. Blood 2004; 103: 2467-73.
32. Quintana-Molina M, Martinez-Bahamonde F, Gonzalez-Garcia E et al. Surgery in haemophilic patients with inhibitor: 20years of experience. Haemophilia 2004; 10(Suppl. 2): 30-40.
33. Scalone L, Montovani LG, Mannucci PM, Gringeri A. Quality of life is associated to the orthopaedic status in haemophilic patients with inhibitors. Haemophilia 2006; 12: 154-62.
34. Leissinger CA, Beeton DL, Ewing NP, Valentino LA. Prophylactic treatment with activated prothrombin complex concentrate (FEIBA) reduces the frequency of bleeding episodes in paediatric patients with haemophilia A and inhibitors. Haemophilia 2007; 13: 249-55.
35. Shapiro AD, Gilchrist GS, Hoots K, Cooper HA, Gastineau DA. Prospective randomised trial of two doses of rFVIIa (Novoseven) in haemophilia patients with inhibitors undergoing surgery. Thromb Haemost 1998; 80: 773-8.
36. Saxon BR, Shanks D, Jory CB, Williams V. Effective prophylaxis with daily recombinant factor VIIa (rFVIIa-Novoseven) in a child with high titre inhibitors and a target joint. Thromb Haemost 2001; 86: 1126-7.
37. Young G, McDaniel M, Nugent DJ. Prophylactic recombinant factor VIIa in haemophilia patients with inhibitors. Haemophilia 2005; 11: 203-7.
38. Konkle BA, Ebbessen E, Erhardtsen E et al. Randomized, prospective clinical trial of recombinant factor VIIa for secondary prohylaxis in haemophilia patients with inhibitors. J Thromb Haemost 2007; 5: 1904-13.
39. Haya S, Moret A, Cid AR, et al. Inhibitors in haemophilia A: Current management and open issues. haemophilia 2007; 13(Suppl. 5): 52-60.
40. Oldenburg J, Schwaab R, Brackmann HH. Induction of immune tolerance in haemophilia A inhibitor patients by the "Bonn Protocol": predictive parameter for therapy duration and outcome. Vox Sang 1999; 77 (Suppl. 1): 49-54.
41. Brackmann HH, Effenberger E, Hess L et al. INovoSeven in immune tolerance therapy. Blood Coagul Fibrinolysis 2000; 11(Suppl.1): S39-44.
42. Antunes SV, Vicari P, Cavalheiro S, Bordin JO. Intracranial haemorrhage among a population of haemophilic patients in Brazil. Haemophilia 2003; 9: 573-7.
43. Panicker J, Warrier I, Thormas R, Lusher JM. The overall effectiveness of prophylaxis in severe haemophilia. Haemophilia 2003; 9: 272-8.