North American prophylaxis studies for persons with severe haemophilia: Background, rationale and design

Haemophilia

Volumn 9, Issue SUPPL. 1, 2003, Pages 44-49

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

^a UNIVERSITY OF COLORADO   (United States)

^b HOSPITAL FOR SICK CHILDREN   (Canada)

Author keywords

Clinical trials; Haemophilia; Prevention of joint damage; Prophylaxis

Indexed keywords

BLOOD CLOTTING FACTOR 8 CONCENTRATE; BLOOD CLOTTING FACTOR CONCENTRATE;

ARTHROPATHY; ARTICLE; BLEEDING; CLINICAL PROTOCOL; CLINICAL TRIAL; CONTROLLED CLINICAL TRIAL; CONTROLLED STUDY; DISEASE COURSE; DISEASE SEVERITY; DRUG DOSE REGIMEN; DRUG EFFICACY; EVIDENCE BASED MEDICINE; HEALTH CARE COST; HEMARTHROSIS; HEMOPHILIA; HUMAN; INDIVIDUALIZATION; MALE; METHODOLOGY; MORBIDITY; NORTH AMERICA; NUCLEAR MAGNETIC RESONANCE IMAGING; PRESCHOOL CHILD; PRIORITY JOURNAL; RANDOMIZED CONTROLLED TRIAL; RATING SCALE; SENSITIVITY ANALYSIS; SUBSTITUTION THERAPY; VALIDATION PROCESS;

EID: 0038102270     PISSN: 13518216     EISSN: None     Source Type: Journal    
DOI: 10.1046/j.1365-2516.9.s1.15.x     Document Type: Article

References (33)

1. DiMichele D, Neufeld EJ. Hemophilia: a new approach to an old disease. Hematol/Oncol Clin North Am 1998; 12: 1315-44.
2. Hilgartner MW. Current treatment of hemophilic arthropathy. Curr Opin Pediatr 2002; 14: 46-9.
3. Bohn RL, Avorn J, Glynn RJ, Choodnovskiy I, Haschemeyer R, Aledort LM. Prophylactic use of factor VIII: an economic evaluation. Thromb Haemost 1998; 79: 932-7.
4. Standards and Criteria, US National Hemophilia Foundation webpage, www.hemophilia.org.
5. Gilbert MS. Musculoskeletal complications of hemophilia: the joint. Haemophilia 2000; 6 (Suppl. 1): 34-7.
6. Rodriquez-Merchan EC. The destructive capabilities of the synovium in the haemophilic joint. Haemophilia 1998; 4: 506-10.
7. Morris CJ, Blake DR, Wainwright AC, Steven MM. Relationship between iron deposits and tissue damage in the synovium: an ultrastructural study. Ann Rheum Dis 1986; 45: 21-6.
8. Speer DP. Early pathogenesis of hemophilic arthropathy. Evolution of the subchondral cyst. Clin Orthop 1984; 185: 250-65.
9. Arnold WD, Hilgartner MW. Hemophilic arthropathy. Current concepts of pathogenesis and management. J Bone Joint Surg Am 1977; 59: 287-305.
10. Aronstam A, Wassef M, Hamad Z, McLellan DS. The effect of bleeding patterns on the response of haemophilic haemarthroses to different doses of factor VIII. Clin Laboratory Haematol 1981; 3: 107-12.
11. Nuss R, Kilcoyne RF, Geraghty S, Wiedel J, Manco-Johnson M. Utility of magnetic resonance imaging for management of hemophilic arthropathy in children. J Pediatr 1993; 123: 388-92.
12. Nuss R, Kilcoyne RF, Geraghty S, et al. MRI findings in hemophilic joints treated with radiosynoviorthesis with development of an MRI scale of joint damage. Haemophilia 2000; 6: 162-9.
13. Aledort LM, Haschmeyer RH, Pettersson H. A longitudinal study of orthopaedic outcomes for severe factor VIII-deficient haemophiliacs. The Orthopaedic Outcome Study Group. J Int Med 1994; 236: 391-9.
14. 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.
15. Teigland JC, Tjonnfjord GE, Evensen SA, Charania B. Synovectomy for haemophilic arthropathy: 6-21 years of follow-up in 16 patients. J Intern Med 1994; 235: 239-43.
16. Wiedel JD. Arthroscopic synovectomy of the knee in hemophilia: 10-to-15 year followup. Clin Orthop 1996; 328: 46-53.
17. Ekert H, Moorehead M, Williamson G. Home therapy for haemophilia. A follow-up study. Med J Aust 1981; 2: 21-3.
18. Brettler DB, Forsberg AD, O'Connell FD, Cederbaum AI, Chaitman AK, Levine PH. A long-term study of hemophilic arthropathy of the knee joint on a program of factor VIII replacement given at time of each hemarthrosis. Am J Hematol 1985; 18: 13-8.
19. Johnson JB. The management of hemophilia with lyophile human plasma intravenously injected. J Am Med Assoc 1942; 118: 799-802.
20. Ahlberg A. Haemophilia in Sweden. VII. Incidence, treatment and prophylaxis of arthropathy and other musculoskeletal manifestations of haemophilia A and B. Acta Orthop Scand Supplement 1965; 77: 3-132.
21. Robinson PM, Tittley P, Smiley RK. Prophylactic therapy in classical hemophilia: a preliminary report. Can Med Ass J 1967; 97: 559.
22. Shanbrom E, Thelin GM. Experimental prophylaxis of severe hemophilia with a factor VIII concentrate. JAMA 1969; 208: 1853-6.
23. Van Creveld S. Prophylaxis of joint hemorrhages in hemophilia. Acta Haematol 1969; 41: 206-14.
24. Hirschman RJ, Itscoitz SB, Shulman NR. Prophylactic treatment of factor VIII deficiency. Blood 1970; 35: 189-94.
25. 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.
26. 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.
27. Petrini P. What factors influence the dosage and interval of prophylactic treatment in patients with severe haemophilia A and B? Haemophilia 2001; 7: 99-102.
28. Astermark J, Petrini P, Tengborn L, Schulman S, Ljung R, Berntorp E. Primary prophylaxis in severe haemophilia should be started at an early age but can be individualized. Br J Haematol 1999; 105: 1109-13.
29. 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.
30. Onwuzurike N, Warrier I, Lusher JM. Types of bleeding seen during the first 30 months of life in children with severe haemophilia A and B. Haemophilia 1996; 2: 137-40.
31. Petrini P, Lindvall N, Egberg N, Blomback M. Prophylaxis with factor concentrates in preventing hemophilic arthropathy. Am J Pediatr Hematol Oncol 1991; 13: 280-7.
32. 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.
33. Manco-Johnson MJ, Nuss R, Funk S, Murphy J. Joint evaluation instruments for children and adults with haemophilia. Haemophilia 2000; 6: 649-57.