Prophylaxis in the haemophilia population-optimizing therapy

Haemophilia

Volumn 13, Issue 3, 2007, Pages 227-232

  Carcao, Manuel D ^a   Aledort, L M ^a  

^a HOSPITAL FOR SICK CHILDREN   (Canada)

Author keywords

[No Author keywords available]

Indexed keywords

BLOOD CLOTTING INHIBITOR;

BLEEDING; CLINICAL EFFECTIVENESS; CONFERENCE PAPER; COST EFFECTIVENESS ANALYSIS; DIAGNOSTIC VALUE; DRUG DOSE ESCALATION; DRUG MEGADOSE; DRUG WITHDRAWAL; HEMARTHROSIS; HEMOPHILIA; HEPATITIS C; HUMAN; HUMAN IMMUNODEFICIENCY VIRUS INFECTION; JOINT INJURY; LOW DRUG DOSE; NUCLEAR MAGNETIC RESONANCE IMAGING; PRIORITY JOURNAL; TREATMENT DURATION; TREATMENT PLANNING; TREATMENT RESPONSE;

ADOLESCENT; ADULT; BLOOD COAGULATION FACTORS; CHILD; CHILD, PRESCHOOL; HEMARTHROSIS; HEMOPHILIA A; HUMANS; INFANT; INFANT, NEWBORN;

EID: 34248549384     PISSN: 13518216     EISSN: 13652516     Source Type: Journal    
DOI: 10.1111/j.1365-2516.2007.01457.x     Document Type: Conference Paper

References (50)

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. 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.
3. Liesner RJ, Khair K, Hann IM. The impact of prophylactic treatment on children with severe haemophilia. Br J Haematol 1996; 92: 973-8.
4. Smith PS, Teutsch SM, Shaffer PA, Rolka H, Evatt B. Episodic versus prophylactic infusions for hemophilia A: A cost-effectiveness analysis. J Pediatr 1996; 129: 424-31.
5. 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.
6. Stobart K, Iorio A, Wu JK. Clotting factor concentrates given to prevent bleeding and bleeding-related complications in people with hemophilia A or B. Cochrane Database Syst Rev 2005; 18: CD003429.
7. Aronstam A, Arblaster PG, Rainsford SG et al. Prophylaxis in haemophilia: A double-blind controlled trial. Br J Haematol 1976; 33: 81-90.
8. Gringeri A. Prospective controlled studies on prophylaxis: An Italian approach. Haemophilia 2003; 9(Suppl. 1): 38-42; discussion 43.
9. Manco-Johnson M, Abshire T, Brown D et al. Initial results of a randomized prospective trial of prophylaxis to prevent joint disease in young children with factor VIII (FVIII) deficiency. Blood 2005; 106: 6a.
10. Mannucci PM, Mendolicchio L, Gringeri A. Use of prophylaxis to prevent complications of hemophilia. Ad Exp Med Biol 2001; 489: 59-64.
11. Szucs TD. Economic issues in European self-sufficiency. Blood Coag Fibrinol 1994; 5(Suppl. 4): S51-S55.
12. Ljung RC. Prophylactic infusion regimens in the management of hemophilia. Thromb Haemost 1999; 82: 525-30.
13. Roosendaal G, Tekoppele JM, Vianen ME, van den Berg HM, Lafeber FP, Bijlsma JW. Articular cartilage is more susceptible to blood induced damage at young than at old age. J Rheumatol 2000; 27: 1740-4.
14. 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.
15. van den Berg HM, Fischer K, van der Bom JG, Roosendaal G, Mauser-Bunschoten EP. Effects of prophylactic treatment regimens in children with severe haemophilia: A comparison of different strategies. Haemophilia 2002; 8(Suppl. 2): 43-46.
16. Carcao MD, Aledort L. Prophylactic factor replacement in hemophilia. Blood Rev 2004; 18: 101-13.
17. Petrini P, Lindvall N, Egberg N, Blomback M. Prophylaxis with factor concentrates in preventing hemophilic arthropathy. Am J Ped Hemat Oncol 1991; 13: 280-7.
18. Funk M, Schmidt H, Escuriola-Ettingshausen C et al. Radiological and orthopedic score in pediatric hemophilic patients with early and late prophylaxis. Ann Hematol 1998; 77: 171-4.
19. 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.
20. 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.
21. 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.
22. 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.
23. Molho P, Rolland N, Lebrun T et al. Epidemiological survey of the orthopaedic status of severe haemophilia A and B patients in France. The French Study Group. secretariat.haemophilesγch.ap-hop-paris.fr. Haemophilia 2000; 6: 23-32.
24. Onwuzurike N, Warrier I, Lusher JM. Types of bleeding see during the first 30 months of life in children with severe haemophilia A and B. Haemophilia 1996; 2: 137-40.
25. Pollmann H, Richter H, Ringkamp H, Jurgens H. When are children diagnosed as having severe haemophilia and when do they start to bleed? A 10-year single-centre PUP study. Eur J Pediatr 1999; 158(Suppl. 3): S166-70.
26. 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.
27. Berntorp E, Astermark J, Bjorkman S et al. Consensus perspectives on prophylactic therapy for haemophilia: Summary statement. Haemophilia 2003; 9(Suppl. 1): 1-4.
28. Blanchette VS, McCready M, Achonu C, Abdolell M, Rivard G, Manco-Johnson MJ. A survey of factor prophylaxis in boys with haemophilia followed in North American haemophilia treatment centres. Haemophilia 2003; 9(Suppl. 1): 19-26.
29. Ljung R, Aronis-Vournas S, Kurnik-Auberger K et al. Treatment of children with haemophilia in Europe: A survey of 20 centres in 16 countries. Haemophilia 2000; 6: 619-24.
30. Chambost H, Ljung R. Changing pattern of care of boys with haemophilia in western European centres. Haemophilia 2005; 11: 92-9.
31. Ljung R. Central venous catheters in children with haemophilia. Blood Rev 2004; 18: 93-100.
32. Journeycake JM, Quinn CT, Miller KL, Zajac JL, Buchanan GR. Catheter-related deep venous thrombosis in children with hemophilia. Blood 2001; 98: 1727-31.
33. Blanchette V, Rivard G, Israels S et al. Results from the Canadian hemophilia dose escalation prophylaxis trial: An update. J Thromb Haemostas 2005; 3(Suppl. 1): PO227.
34. van Dijk K, Fischer K, van der Bom JG, Grobbee DE, van den Berg HM. Variability in clinical phenotype of severe haemophilia: The role of the first joint bleed. Haemophilia 2005; 11: 438-43.
35. Ahnstrom J, Berntorp E, Lindvall K, Bjorkman S. A 6-year follow-up of dosing, coagulation factor levels and bleedings in relation to joint status in the prophylactic treatment of haemophilia. Haemophilia 2004; 10: 689-97.
36. van't Veer C, Golden NJ, Kalafatis M, Simioni P, Bertina RM, Mann KG. An in vitro analysis of the combination of hemophilia A and factor V (LEIDEN). Blood 1997; 90: 3067-72.
37. Escuriola C, Ettingshausen C, Halimeh S et al. Symptomatic onset of severe hemophilia A in childhood is dependent on the presence of prothrombotic risk factors. Thromb Haemost 2001; 85: 218-20.
38. van Dijk K, van der Bom JG, Fischer K, Grobbee DE, van den Berg HM. Do prothrombotic factors influence clinical phenotype of severe haemophilia? A review of the literature. Thromb Haemost 2004; 92: 305-10.
39. Ghosh K, Shankarkumar U, Shetty S, Mohanty D. Chronic synovitis and HLA B27 in patients with severe haemophilia. Lancet 2003; 361: 933-4.
40. 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.
41. van Dijk K, Fischer K, van der Bom JG, Scheibel E, Ingerslev J, van den Berg HM. Can long-term prophylaxis for severe haemophilia be stopped in adulthood? Results from Denmark and the Netherlands. Br J Haematol 2005; 130: 107-12.
42. Arnold WD, Hilgartner MW. Hemophilic arthropathy. Current concepts of pathogenesis and management. J Bone Joint Surg Am 1977; 59: 287-305.
43. Pettersson H, Ahlberg A, Nilsson IM. A radiologic classification of hemophilic arthropathy. Clin Orthop 1980; 149: 153-9.
44. Lundin B, Babyn P, Doria AS et al. Compatible scales for progressive and additive MRI assessments of haemophilic arthropathy. Haemophilia 2005; 11: 109-15.
45. Doria AS, Lundin B, Kilcoyne RF et al. Reliability of progressive and additive MRI scoring systems for evaluation of haemophilic arthropathy in children: Expert MRI Working Group of the International Prophylaxis Study Group. Haemophilia 2005; 11: 245-53.
46. Srivastava A. Dose and response in haemophilia - optimization of factor replacement therapy. Br J Haematol 2004; 127: 12-25.
47. Heemstra HE, Zwaan T, Hemels M et al. Cost of severe haemophilia in Toronto. Haemophilia 2005; 11: 254-60.
48. Fischer K, Astermark J, van der Bom JG et al. Prophylactic treatment for severe haemophilia: Comparison of an intermediate-dose to a high-dose regimen. Haemophilia 2002; 8: 753-60.
49. Carlsson KS, Hojgard S, Lethagen S, Lindgren A, Berntorp E, Lindgren B. Willingness to pay for on-demand and prophylactic treatment for severe haemophilia in Sweden. Haemophilia 2004; 10: 527-41.
50. Kavakli K, Nisli G, Aydinok Y et al. Prophylactic therapy for hemophilia in a developing country, Turkey. Pediatr Hematol Oncol 1997; 14: 151-9.