Efficacy and safety of secondary prophylactic vs. on-demand sucrose-formulated recombinant factor VIII treatment in adults with severe hemophilia A: Results from a 13-month crossover study

Journal of Thrombosis and Haemostasis

Volumn 8, Issue 1, 2010, Pages 83-89

  Collins, P ^a   Faradji, A ^b   Morfini, M ^c   Enriquez, M M ^d   Schwartz, L ^e  

^a UNIVERSITY HOSPITAL OF WALES   (United Kingdom)

^b HÔPITAL DE HAUTEPIERRE   (France)

^c University Hospital   (Italy)

^d BAYER PHARMA AG   (Germany)

^e BAYER CORPORATION   (United States)

Author keywords

Factor VIII; Hemophilia; On demand therapy; Secondary prophylaxis

Indexed keywords

BLOOD CLOTTING FACTOR 8 INHIBITOR; RECOMBINANT BLOOD CLOTTING FACTOR 8; SUCROSE;

ADULT; ARTICLE; CLINICAL ARTICLE; CLINICAL TRIAL; CONTROLLED CLINICAL TRIAL; CONTROLLED STUDY; CROSSOVER PROCEDURE; DOSAGE SCHEDULE COMPARISON; DRUG EFFICACY; DRUG INDUCED HEADACHE; DRUG SAFETY; DRUG TOLERABILITY; DYSGEUSIA; HEALTH ECONOMICS; HEMARTHROSIS; HEMOPHILIA A; HUMAN; HYPERHIDROSIS; JOINT FUNCTION; MALE; PATIENT COMPLIANCE; PRIORITY JOURNAL; QUALITY OF LIFE; SECONDARY PREVENTION;

ADULT; CHEMISTRY, PHARMACEUTICAL; COAGULANTS; CROSS-OVER STUDIES; DRUG ADMINISTRATION SCHEDULE; EUROPE; FACTOR VIII; HEMARTHROSIS; HEMOPHILIA A; HOME INFUSION THERAPY; HUMANS; INFUSIONS, INTRAVENOUS; MALE; MIDDLE AGED; PROSPECTIVE STUDIES; RECURRENCE; SECONDARY PREVENTION; SEVERITY OF ILLNESS INDEX; SUCROSE; TIME FACTORS; TREATMENT OUTCOME; UNITED STATES;

EID: 73049092113     PISSN: 15387933     EISSN: 15387836     Source Type: Journal    
DOI: 10.1111/j.1538-7836.2009.03650.x     Document Type: Article

References (22)

1. 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.
2. van den Berg HM, Fischer K, van der Bom JG. Comparing outcomes of different treatment regimens for severe haemophilia. Haemophilia 2003, 9:27-31.
3. Ahlberg A. Haemophilia in Sweden. VII. Incidence, treatment and prophylaxis of arthropathy and other musculo-skeletal manifestations of haemophilia A and B. Acta Orthop Scand Suppl 1965, (Suppl. 77):3-132.
4. 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.
5. Lofqvist T, Nilsson IM, Berntorp E, Pettersson H. Haemophilia prophylaxis in young patients-a long-term follow-up. J Intern Med 1997, 241:395-400.
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. Prophylaxis versus episodic treatment to prevent joint disease in boys with severe hemophilia. N Engl J Med 2007, 357:535-44.
7. 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.
8. Liesner RJ, Khair K, Hann IM. The impact of prophylactic treatment on children with severe haemophilia. Br J Haematol 1996, 92:973-8.
9. Medical and Scientific Advisory Council (MASAC) recommendations concerning prophylaxis (regular administration of clotting factor concentrate to prevent bleeding). Document #179. http://www.hemophilia.org/NHFWeb/Resource/StaticPages/menu0/menu5/menu57/masac179.pdf, Accessed 4 May 2009
10. Berntorp E. Guidelines on treatment of haemophilia in Sweden. Haemophilia 1998, 4:425-6.
11. Berntorp E, Boulyjenkov V, Brettler D, Chandy M, Jones P, Lee C, Lusher J, Mannucci P, Peak I, Rickard K, Seremetis S. Modern treatment of haemophilia. Bull World Health Organ 1995, 73:691-701.
12. 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.
13. Fischer K, van der Bom JG, Prejs R, Mauser-Bunschoten EP, Roosendaal G, Grobbee DE, van den Berg HM. Discontinuation of prophylactic therapy in severe haemophilia: incidence and effects on outcome. Haemophilia 2001, 7:544-50.
14. Hay CR. Prophylaxis in adults with haemophilia. Haemophilia 2007, 13:10-5.
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, 8:753-60.
16. Fischer K, van der Bom JG, Molho P, Negrier C, Mauser-Bunschoten EP, Roosendaal G, De Kleijn P, Grobbee DE, van den Berg HM. Prophylactic versus on-demand treatment strategies for severe haemophilia: a comparison of costs and long-term outcome. Haemophilia 2002, 8:745-52.
17. Gilbert MS. Prophylaxis: musculoskeletal evaluation. Semin Hematol 1993, 30:3-6.
18. Rentz A, Flood E, Altisent C, Bullinger M, Klamroth R, Garrido RP, Scharrer I, Schramm W, Gorina E. Cross-cultural development and psychometric evaluation of a patient-reported health-related quality of life questionnaire for adults with haemophilia. Haemophilia 2008, 14:1023-34.
19. Miesbach W, Alesci S, Krekeler S, Seifried E. Comorbidities and bleeding pattern in elderly haemophilia A patients. Haemophilia 2009, 15:894-9.
20. Loverin JA, Mensah P, Nokes TJC. Limited prophylaxis in adults with severe hemophilia: a pilot study [abstract]. Haemophilia 2000, 6:274-5.
21. Collins PW, Blanchette VS, Fischer K, Bjorkman S, Oh M, Fritsch S, Schroth P, Spotts G, Astermark J, Ewenstein B. Break-through bleeding in relation to predicted factor VIII levels in patients receiving prophylactic treatment for severe hemophilia A. J Thromb Haemost 2009, 7:413-20.
22. Barnes C, Lillicrap D, Pazmino-Canizares J, Blanchette V, Stain AM, Clark D, Hensmen C, Carcao M. Pharmacokinetics of recombinant factor VIII (Kogenate-FS) in children and causes of inter-patient pharmacokinetic variability. Haemophilia 2006, 12:40-9.