Prophylaxis in the haemophilia population‐optimizing therapy
2007; Wiley; Volume: 13; Issue: 3 Linguagem: Inglês
10.1111/j.1365-2516.2007.01457.x
ISSN1365-2516
AutoresManuel Carção, Louis M. Aledort,
Tópico(s)Coagulation, Bradykinin, Polyphosphates, and Angioedema
ResumoHaemophiliaVolume 13, Issue 3 p. 227-232 Prophylaxis in the haemophilia population-optimizing therapy M. D. CARCAO, M. D. CARCAO Division of Haematology/Oncology, Department of Paediatrics, Hospital for Sick Children, University of Toronto, Toronto, CanadaSearch for more papers by this authorL. M. ALEDORT, L. M. ALEDORT Division of Haematology/Oncology, Department of Paediatrics, Hospital for Sick Children, University of Toronto, Toronto, CanadaSearch for more papers by this authorTHE ROUND TABLE GROUP, THE ROUND TABLE GROUP Division of Haematology/Oncology, Department of Paediatrics, Hospital for Sick Children, University of Toronto, Toronto, CanadaSearch for more papers by this author M. D. CARCAO, M. D. CARCAO Division of Haematology/Oncology, Department of Paediatrics, Hospital for Sick Children, University of Toronto, Toronto, CanadaSearch for more papers by this authorL. M. ALEDORT, L. M. ALEDORT Division of Haematology/Oncology, Department of Paediatrics, Hospital for Sick Children, University of Toronto, Toronto, CanadaSearch for more papers by this authorTHE ROUND TABLE GROUP, THE ROUND TABLE GROUP Division of Haematology/Oncology, Department of Paediatrics, Hospital for Sick Children, University of Toronto, Toronto, CanadaSearch for more papers by this author First published: 16 March 2007 https://doi.org/10.1111/j.1365-2516.2007.01457.xCitations: 6 M. D. Carcao, Division of Haematology/Oncology, Department of Paediatrics, Hospital for Sick Children, University of Toronto, Toronto, Canada.Tel.: 416-813-5367; fax: 416-813-5327;e-mail: [email protected] Members participating in round table: Carcao MD (Rapporteur, Canada), Aledort LM (Chair, USA), Blanchette VS (Canada), Doria AS (Canada), Feldman BM (Canada), Fischer K (Netherlands), Gomperts E (USA), Kavakli K (Turkey), Ljung RC (Sweden), Petrini P (Sweden), Schramm W (Germany) and Taki M (Japan). Read the full textAboutPDF ToolsRequest permissionExport citationAdd to favoritesTrack citation ShareShare Give accessShare full text accessShare full-text accessPlease review our Terms and Conditions of Use and check box below to share full-text version of article.I have read and accept the Wiley Online Library Terms and Conditions of UseShareable LinkUse the link below to share a full-text version of this article with your friends and colleagues. Learn more.Copy URL Share a linkShare onEmailFacebookTwitterLinkedInRedditWechat References 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. [email protected] . 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. Citing Literature Volume13, Issue3May 2007Pages 227-232 ReferencesRelatedInformation
Referência(s)