Need for prophylactic treatment in adult haemophilia A patients

Transfusion Medicine and Hemotherapy

Volumn 36, Issue 4, 2009, Pages 283-288

  Siegmund, Berthold ^a   Richter, Heinrich ^a   Pollmann, Hartmut ^a  

^a Institut für Thrombophilie und Hämostaseologie   (Germany)

Author keywords

Continuous prophylaxis; Haemophilia A; On demand therapy

Indexed keywords

BLOOD CLOTTING FACTOR 8;

ADOLESCENT; ADULT; ARTICLE; BLEEDING TENDENCY; BODY WEIGHT; CONTROLLED STUDY; HEMARTHROSIS; HEMOPHILIA A; HOME CARE; HUMAN; MAJOR CLINICAL STUDY; THERAPY EFFECT; TREATMENT OUTCOME; TREATMENT RESPONSE;

EID: 69549114635     PISSN: 16603796     EISSN: None     Source Type: Journal    
DOI: 10.1159/000225965     Document Type: Article

References (28)

1. Klinge J, Ananyeva NM, Hauser CAE, Saenko EL: Hemophilia A - from basic science to clinical practice. Semin Thromb Hemost 2002;28:309-322. (Pubitemid 34634346)
2. Hoyer LW: Hemophilia A. N Engl J Med 1994;330:38-47.
3. Bolton-Maggs PHB, Pasi KJ: Haemophilias A and B. Lancet 2003;361:1801-1809.
4. Yee TT, Beeton K, Griffioen A, Harrington C, Miners A, Lee CA, Brown SA: Experience of prophylaxis treatment in children with severe haemophilia. Haemophilia 2002;8:76-82. (Pubitemid 35238059)
5. 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. (Pubitemid 34498628)
6. Manco-Johnson MJ, Abshire TC, Shapiro AD, Riske B, Hacker MR, Kilcoyne R, Ingram JD, Manco-Johnson ML, Funk S, Jacobson L, Valentino LA, Hoots WK, Buchanan GR, DiMichele D, Recht M, Brown D, Leissinger C, Bleak S, Cohen A, Mathew P, Matsunaga A, Medeiros D, Nugent D, Thomas GA, Thompson AA, McRedmond K, Soucie JM, Austin H, Evatt BL: Prophylaxis versus episodic treatment to prevent joint disease in boys with severe hemophilia. N Engl J Med 2007;357:535-544. (Pubitemid 47236278)
7. Berntorp E, Boulyjenkov V, Brettler D, Chandy M, Jones P, Lee C, Lusher J, Mannucci P, Peak I, Rickard K: Modern treatment of haemophilia. Bull World Health Org 1995;73:691-701.
8. Naraine VS, Risebrough NA, Oh P, Blanchette VS, Lee S, Stain A-M, Hedden D, Teitel JM, Feldman BM: Health-related quality-of-life treatments for severe haemophilia: utility measurements using the Standard Gamble technique. Haemophilia 2002;8:112-120. (Pubitemid 35238064)
9. Bohn RL, Schramm W, Bullinger M, Van Den Berg M, Blanchette V: Outcome measures in haemophilia: more than just factor levels. Haemophilia 2004;10(suppl 1):2-8.
10. Bullinger M, Von Mackensen S: Quality of life assessment in haemophilia. Haemophilia 2004;10(suppl 1):9-16.
11. Remor E, Young NL, Von Mackensen S, Lopatina EG: Disease-specific quality-of-life measurement tools for haemophilia patients. Haemophilia 2004;10(suppl 4):30-34.
12. Medical and Scientific Advisory Council (MASAC). MASAC Recommendation Concerning Prophylaxis (Regular Administration of Clotting Factor Concentrate to Prevent Bleeding). MASAC document #170; 2006. www.hemophilia.org/NHFWeb/ Resource/StaticPages/menu0/menu5/menu57/170.pdf.
13. Bundesärztekammer: Leitlinien zur Therapie mit Blutkomponenten und Plasmaderivaten, 3. Aufl. Köln, Deutscher Ärzte-Verlag, 2003. www.bundesaerztekammer. de/downloads/Blutkomponentenpdf.pdf.
14. Schramm W: Experience with prophylaxis in Germany. Semin Hematol 1993;30:12-15.
15. Fischer K, Astermark J, Van Der Bom JG, Ljung R, Berntorp E, Grobbee DE, Van Den Berg HM: Prophylactic treatment for severe haemophilia: comparison of an intermediate-dose to a high-dose regimen. Haemophilia 2002;8:753-760. (Pubitemid 35446627)
16. 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;236:391-399.
17. 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.
18. Pollmann H, Externest D, Ganser A, Eifrig B, Kreuz W, Lenk H, Pabinger I, Schramm W, Schwarz TF, Zimmermann R, Zavazava N, Oldenburg J, Klamroth R: Efficacy, safety and tolerability of recombinant factor VIII (REFACTO®) in patients with haemophilia A: interim data from a postmarketing surveillance study in Germany and Austria. Haemophilia 2006;12:1-13. (Pubitemid 46237944)
19. Carcao MD, Aledort L: Prophylactic factor replacement in hemophilia. Blood Rev 2004;18:101-113.
20. Hoots WK, Nugent DJ: Evidence for the benefits of prophylaxis in the management of hemophilia A. Thromb Haemost 2006;96:433-440.
21. 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. (Pubitemid 36622045)
22. Abshire TC, Brackmann HH, Scharrer I, Hoots K, Gazengel C, Powell JS, Gorina E, Kellermann E, Vosburgh E; the International Kogenate-FS Study Group: Sucrose formulated recombinant human antihemophilic factor VIII is safe and efficacious for treatment of hemophilia A in home therapy - International Kogenate-FS Study Group. Thromb Haemost 2000;83:811-816.
23. Battistella LR: Maintenance of musculoskeletal function in people with haemophilia. Haemophilia 1998;4(suppl 2):26-32. (Pubitemid 28423432)
24. Su Y, Wong W-Y, Lail A, Donfield SM, Konzal S, Gomperts E: Long-term major joint outcomes in young adults with haemophilia: interim data from the HGDS. The Hemophilia Growth and Development Study. Haemophilia 2007;13:387-390.
25. 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;22:251-258. (Pubitemid 23000361)
26. Smith PS, Teutsch SM, Shaffer PA, Rolka H, Evatt B: Episodic versus prophylactic infusions for hemophilia A: a cost-effectiveness analysis. J Pediatr 1996;129:424-431. (Pubitemid 27484150)
27. The ESCHQoL Study, European Study of Clinical, Health Economic and Quality of Life Outcomes in Haemophilia treatment. www.eschqol.lmu.de/sys/cgi/ project.html.
28. Hobbins P, Anderson L, Cunningham N, Carnahan M, Park J, Denholm J, Newell C, McPherson J: Liberal eugenics: in defence of human enhancement J Bioethical Inquiry 2005;2:106-115.