A 6-year follow-up of dosing, coagulation factor levels and bleedings in relation to joint status in the prophylactic treatment of haemophilia

Haemophilia

Volumn 10, Issue 6, 2004, Pages 689-697

  Ahnstrom J ^a,b   Berntorp, E ^a   Lindvall, K ^a   Björkman, Sven ^a,b  

^a LUND UNIVERSITY HOSPITAL   (Sweden)

^b UPPSALA UNIVERSITY   (Sweden)

Author keywords

Dosing; Factor IX; Factor VIII; Haemophilia; Pharmacokinetics; Prophylactic treatment

Indexed keywords

BLOOD CLOTTING FACTOR 8; BLOOD CLOTTING FACTOR 9;

ADOLESCENT; ADULT; ARTHROPATHY; BLEEDING; BLOOD SAMPLING; CALCULATION; CHEMOPROPHYLAXIS; CHILD; DISEASE SEVERITY; DISTRIBUTION VOLUME; DRUG BLOOD LEVEL; DRUG CLEARANCE; DRUG HALF LIFE; DRUG MEGADOSE; FOLLOW UP; HEMARTHROSIS; HEMOPHILIA A; HEMOPHILIA B; HUMAN; INCIDENCE; INFANT; MEDICAL RECORD; PATIENT CODING; PRIORITY JOURNAL; RETROSPECTIVE STUDY; REVIEW; SCORING SYSTEM;

ADOLESCENT; ADULT; AGED; CHILD; CHILD, PRESCHOOL; FACTOR IX; FACTOR VIII; FOLLOW-UP STUDIES; HEMARTHROSIS; HEMOPHILIA A; HEMORRHAGE; HUMANS; INFANT; INFANT, NEWBORN; MIDDLE AGED; RETROSPECTIVE STUDIES;

EID: 11144330901     PISSN: 13518216     EISSN: None     Source Type: Journal    
DOI: 10.1111/j.1365-2516.2004.01036.x     Document Type: Review

References (39)

1. Nilsson IM, Berntorp E, Löfqvist T, Pettersson H. Twenty-five years' experience of prophylactic treatment in severe haemophilia A and B. J Intern Med 1992; 232: 25-32.
2. Skolnick AA. Hemophilia foundation recommends prophylactic use of clotting factors. J Am Med Assoc 1994; 272: 1153-4.
3. Ljung R. Prophylactic treatment in Sweden - over-treatment or optimal model? Haemophilia 1998; 4: 409-12.
4. Berntorp E. Guidelines on treatment of haemophilia in Sweden. Haemophilia 1998; 4: 425-6.
5. 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.
6. 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.
7. 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.
8. van den Berg HM, Fisher 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.
9. 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): 43-6.
10. Björkman S. Prophylactic dosing of factor VIII and factor IX from a clinical pharmacokinetic perspective. Haemophilia 2003; 9 (Suppl. 1): 101-10.
11. Carlsson M, Berntorp, E, Björkman S, Lindvall K. Pharmacokinetic dosing in prophylactic treatment of haemophilia A. Eur J Haematol 1993; 51: 247-52.
12. Björkman S, Carlsson M. The pharmacokinetics of factor VIII and factor IX: methodology, pitfalls and applications. Haemophilia 1997; 3: 1-8.
13. Carlsson M, Berntorp E, Björkman S, Lethagen S, Ljung R. Improved cost-effectiveness by pharmacokinetic dosing of factor VIII in prophylactic treatment of haemophilia A. Haemophilia 1997; 3: 96-101.
14. Carlsson M, Björkman S, Berntorp, E. Multidose pharmacokinetics of factor IX: implications for dosing in prophylaxis. Haemophilia 1998; 4: 83-8.
15. Björkman S, Berntorp E. Pharmacokinetics of coagulation factors. Clinical relevance for patients with haemophilia. Clin Pharmacokinet 2001; 40: 815-32.
16. Rainsford SG, Hall A. A three-year study of adolescent boys suffering from haemophilia and allied disorders. Br J Haematol 1973; 24: 539-51.
17. Aledort LM, Haschmeyer RH, Pettersson H and The Orthopaedic Outcome Study Group. A longitudinal study of orthopaedic outcomes for severe factor-VIII-deficient haemophiliacs. J Intern Med 1994; 236: 391-99.
18. Ghosh K, Shetty S, Mohanty D. Milder clinical presentation of haemophilia A with severe deficiency of factor VIII as measured by one-stage assay. Haemophilia 2001; 7: 9-12.
19. Berntorp E. Methods of haemophilia care delivery: regular prophylaxis versus episodic treatment. Haemophilia 1995; 1 (Suppl. 1): 3-7.
20. Liesner RJ, Khair K, Hann IM. The impact of prophylactic treatment on children with severe haemophilia. Br J Haematol 1996; 92: 973-8.
21. Löfqvist T, Nilsson IM, Berntorp E, Pettersson H. Haemophilia prophylaxis in young patients - a long-term follow-up. J Intern Med 1997; 241: 395-400.
22. Nilsson IM, Hedner U, Ahlberg Å. Haemophilia prophylaxis in Sweden. Acta Paediatr Scand 1976; 65: 129-35.
23. Nilsson IM. Experience with prophylaxis in Sweden. Semin Hematol 1993; 30 (Suppl. 2): 16-9.
24. Pettersson H, Gilbert M. Diagnostic Imaging in Hemophilia Berlin: Springer-Verlag, 1985: 56-68.
25. Björkman S, Carlsson M, Berntorp E, Stenberg P. Pharmacokinetics of factor VIII in humans. Obtaining clinically relevant data from comparative studies. Clin Pharmacokinet 1992; 22: 385-95.
26. Björkman S, Carlsson M, Berntorp E. Pharmacokinetics of factor IX in patients with haemophilia B. Methodological aspects and physiological interpretation. Eur J Clin Pharmacol 1994; 46: 325-32.
27. Sheiner LB, Beal S, Rosenberg B, Marathe VV. Forecasting individual pharmacokinetics. Clin Pharmacol Ther 1979; 26: 294-305.
28. Ruffo S, Messori A, Grasela TH et al. A calculator program for clinical application of the Bayesian method of predicting plasma drug levels. Comput Methods Programs Biomed 1985; 19: 167-77.
29. Björkman S, Shapiro AD, Berntorp E. Pharmacokinetics of recombinant factor IX in relation to age of the patient: implications for dosing in prophylaxis. Haemophilia 2001; 7: 133-9.
30. Fischer K, van der Bom JG, Mauser-Bunschoten EP et al. Endogenous clotting factor activity and long-term outcome in patients with moderate haemophilia. Thromb Haemost 2000; 84: 977-80.
31. Fischer K, van der Bom JG, Mauser-Bunschoten EP et al. Changes in treatment strategies for severe haemophilia over the last 3 decades: effects on clotting factor consumption and artropathy. Haemophilia 2001; 7: 446-52.
32. 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.
33. Ingerslev J, Poulsen LH, Sørensen B. Potential role of the dynamic properties of whole blood coagulation in assessment of dosage requirements in haemophilia. Haemophilia 2003; 9: 348-52.
34. Sørensen B, Ingerslev J. Whole blood clot formation phenotypes in hemophilia A and rare coagulation disorders. Patterns of response to recombinant factor VIIa. J Thromb Haemost 2004; 2 102-10.
35. Walsh PN, Rainsford SG, Biggs R. Platelet coagulant activities and clinical severity in haemophilia. Thromb Haemost 1973; 29: 722-9.
36. Arbini AA, Mannucci PM, Bauer KA. Low prevalence of the factor V Leiden mutation among 'severe' hemophiliacs with a 'milder' bleeding diathesis. Thromb Haemost 1995; 74: 1255-8.
37. Lee DH, Walker IR, Teitel J et al. Effect of the factor V Leiden mutation on the clinical expression of severe hemophilia A. Thromb Haemost 2000; 83: 387-91.
38. Escuriola Ettingshausen C, Halimeh S, Kurnik K et al. Symptomatic onset of severe haemophilia A in childhood is dependent on the presence of prothrombotic risk factors. Thromb Haemost 2001; 85: 218-20.
39. Novak-Göttl U, Escuriola C, Kurnik K et al. Haemophilia and thrombophilia. What do we learn about combined inheritance of both genetic variations? Hämostaseologie 2003; 23: 36-40.