The benefits of prophylactic treatment with APCC in patients with haemophilia and high-titre inhibitors: A retrospective case series

Haemophilia

Volumn 15, Issue 3, 2009, Pages 733-742

  Valentino, Leonard A ^a  

^a RUSH UNIVERSITY MEDICAL CENTER   (United States)

Author keywords

APCC; Arthropathy; Haemophilia; High titre; Inhibitor; Prophylaxis

Indexed keywords

ACTIVATED PROTHROMBIN COMPLEX; AMINOCAPROIC ACID; MONOCLONAL ANTIBODY; RECOMBINANT BLOOD CLOTTING FACTOR 7A;

ADOLESCENT; ADULT; ANTIBODY TITER; ARTHROPATHY; ARTICLE; BLEEDING; CASE STUDY; CHILD; CLINICAL ARTICLE; COST BENEFIT ANALYSIS; DISEASE CONTROL; DRUG COST; DRUG EFFICACY; DRUG MEGADOSE; DRUG SAFETY; HEMARTHROSIS; HEMOPHILIA A; HEMOPHILIA B; HUMAN; MALE; OUTCOME ASSESSMENT; PATIENT IDENTIFICATION; PRIORITY JOURNAL; QUALITY OF LIFE; RETROSPECTIVE STUDY;

ARTHROPATHY, NEUROGENIC; BLOOD COAGULATION FACTOR INHIBITORS; BLOOD COAGULATION FACTORS; CHILD; CHILD, PRESCHOOL; COST-BENEFIT ANALYSIS; DOSE-RESPONSE RELATIONSHIP, DRUG; HEMARTHROSIS; HEMOPHILIA A; HUMANS; MALE; RETROSPECTIVE STUDIES; TREATMENT OUTCOME; YOUNG ADULT;

EID: 65449159707     PISSN: 13518216     EISSN: 13652516     Source Type: Journal    
DOI: 10.1111/j.1365-2516.2009.01980.x     Document Type: Article

References (40)

1. Manco-Johnson MJ, Abshire TC, Shapiro AD et al. Prophylaxis versus episodic treatment to prevent joint disease in boys with severe hemophilia. N Engl J Med 2007; 357: 535-44.
2. Yee TT, Beeton K, Griffioen A et al. Experience of prophylaxis treatment in children with severe haemophilia. Haemophilia 2002; 8: 76-82.
3. 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.
4. van den Berg HM, Fischer K, Mauser-Bunschoten EP et al. Long-term outcome of individualized prophylactic treatment of children with severe haemophilia. Br J Haematol 2001; 112: 561-5.
5. Hoots WK, Rodriguez N, Boggio L, Valentino LA. Pathogenesis of haemophilic synovitis: Clinical aspects. Haemophilia 2007; 13(Suppl 3): 4-9.
6. Soucie JM, Cianfrini C, Janco RL et al. Joint range-of-motion limitations among young males with hemophilia: Prevalence and risk factors. Blood 2004; 103: 2467-73.
7. Shapiro AD, Donfield SM, Lynn HS et al. Defining the impact of hemophilia: The Academic Achievement in Children with Hemophilia Study. Pediatrics 2001; 108: E105.
8. Gringeri A, von Mackensen S, Auerswald G et al. Health status and health-related quality of life of children with haemophilia from six West European countries. Haemophilia 2004; 10(Suppl 1): 26-33.
9. 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.
10. Berntorp E, Astermark J, Bjorkman S et al. Consensus perspectives on prophylactic therapy for haemophilia: Summary statement. Haemophilia 2003; 9(Suppl 1): 1-4.
11. MASAC Recommendation Concerning Prophylaxis (Regular Administration of Clotting Factor to Prevent Bleeding). New York, NY: Medical and Scientific Advisory Council of the United States National Hemophilia Foundation, 2006. Document #170.
12. Guidelines for the Management of Hemophilia. Montreal, QC: World Federation of Hemophilia, 2005.
13. Leissinger CA. Prevention of bleeds in hemophilia patients with inhibitors: Emerging data and clinical direction. Am J Hematol 2004; 77: 187-93.
14. Leissinger CA, Becton 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.
15. DiMichele D. Inhibitors: Resolving diagnostic and therapeutic dilemmas. Haemophilia 2002; 8: 280-7.
16. Ewenstein B, Giangrande P, Morfini M et al. Evaluation of FEIBA for prophylaxis in patients with inhibitors. Haemophilia 2004; 10(Suppl 3): 113-6.
17. Schino M, Centra A, Piseddu G, Sbrighi P. Management of bleeding with bypassing agents. Haemophilia 2000; 6: 294[Abstract 237].
18. Kreuz W, Escuriola-Ettinghausen C, Martinez IC, Mentzer D, Figura S, Klarmann D. Efficacy and safety of factor VIII inhibitor bypass activity (FEIBA) for long-term prophylaxis in patients with high-responding inhibitors. Blood 2000; 96: 265a[Abstract 1140].
19. Ewing N. Anamnestic responses in hemophilia patients with inhibitors on continuous prophylaxis with Factor Eight Inhibitor Bypassing Activity, Vapor Heated (FEIBA VH). J Thromb Haemost 2005; 3(Suppl 1): Abstract P2036.
20. Cheng S, Chen Y, Chiang J. FEIBA prophylaxis in hemophilia A patient with inhibitors decreases bleeding episodes, improves joint function and enhance quality of life. Haemophilia 2006; 12(Suppl 2): Abstract 14 PO 371.
21. Lambert T, Fressinaud E, Goudemand J, Rafowicz A. Can long-term prophylaxis with APCC improve the bleeding rate and quality of life of frequently bleeding haemophiliacs with inhibitor? Haemophilia 2006; 12(Suppl 2): Abstract 14 PO 399.
22. Ewing NP, Pullens L, De Guzman C. Anamnesis in patients with hemophilia and inhibitors who receive activated prothrombin complex concentrates for prophylaxis. J Thromb Haemost 2007; 5(Suppl 1): Abstract P-T-158.
23. Konkle BA, Ebbesen LS, Erhardtsen E et al. Randomized, prospective clinical trial of recombinant factor VIIa for secondary prophylaxis in hemophilia patients with inhibitors. J Thromb Haemost 2007; 5: 1904-13.
24. Valentino LA. Efficacy and safety of FEIBA prophylaxis in a patient with hemophilia B and high-titer inhibitor. J Thromb Haemost 2005; 3(Suppl 1): Abstract P1437.
25. Valentino LA. FEIBA VH prophylaxis in hemophilia A patients with inhibitors results in 95% reduction in bleeding episodes. Haemophilia 2004; 10(Suppl 3): 115[Abstract 22 PO 18].
26. Gilbert MS. Prophylaxis: Musculoskeletal evaluation. Semin Hematol 1993; 30(3 Suppl 2): 3-6.
27. De Kleijn P, Heijnen L, Van Meeteren NL. Clinimetric instruments to assess functional health status in patients with haemophilia: A literature review. Haemophilia 2002; 8: 419-27.
28. Hutchinson TA, Boyd NF, Feinstein AR, Gonda A, Hollomby D, Rowat B. Scientific problems in clinical scales, as demonstrated in the Karnofsky index of performance status. J Chronic Dis 1979; 32: 661-6.
29. Mor V, Laliberte L, Morris JN, Wiemann M. The Karnofsky Performance Status Scale. An examination of its reliability and validity in a research setting. Cancer 1984; 53: 2002-7.
30. Fischer K, van der Bom JG, Mauser-Bunschoten EP et al. The effects of postponing prophylactic treatment on long-term outcome in patients with severe hemophilia. Blood 2002; 99: 2337-41.
31. Kreuz W, Escuriola-Ettingshausen C, Funk M, Schmidt H, Kornhuber B. When should prophylactic treatment in patients with haemophilia A and B start? The German experience. Haemophilia 1998; 4: 413-7.
32. Valentino LA. Secondary prophylaxis therapy: What are the benefits, limitations and unknowns? Haemophilia 2004; 10: 147-57.
33. Monahan PE, Aledort LM. Factors affecting choice of hemostatic agent for the hemophilia patient with an inhibitor antibody. Am J Hematol 2004; 77: 346-50.
34. Hoots WK, Ebbesen LS, Konkle BA et al. Secondary prophylaxis with recombinant activated factor VII improves health-related quality of life of haemophilia patients with inhibitors. Haemophilia 2008; 14: 466-75.
35. Brackmann HH, Eickhoff HJ, Oldenburg J, Hammerstein U. Long-term therapy and on-demand treatment of children and adolescents with severe haemophilia A: 12 years of experience. Haemostasis 1992; 22: 251-8.
36. Manco-Johnson MJ, Nuss R, Geraghty S, Funk S, Kilcoyne R. Results of secondary prophylaxis in children with severe hemophilia. Am J Hematol 1994; 47: 113-7.
37. Liesner RJ, Khair K, Hann IM. The impact of prophylactic treatment on children with severe haemophilia. Br J Haematol 1996; 92: 973-8.
38. Hilgartner MW, Makipernaa A, Dimichele DM. Long-term FEIBA prophylaxis does not prevent progression of existing joint disease. Haemophilia 2003; 9: 261-8.
39. Gringeri A. Prospective controlled studies on prophylaxis: An Italian approach. Haemophilia 2003; 9(Suppl 1): 38-42; discussion 43.
40. Hilliard P, Funk S, Zourikian N et al. Hemophilia joint health score reliability study. Haemophilia 2006; 12: 518-25.