RSS-Feed abonnieren
DOI: 10.1055/s-0037-1614159
Endogenous Clotting Factor Activity and Long-term Outcome in Patients with Moderate Haemophilia
This study was partly supported by Zonne-projects of the WKZ, UMCU, Grant # 5.Publikationsverlauf
Received
30. Dezember 1999
Accepted after resubmission
10. Juli 2000
Publikationsdatum:
13. Dezember 2017 (online)
![](https://www.thieme-connect.de/media/10.1055-s-00035024/200006/lookinside/thumbnails/10-1055-s-0037-1614159-1.jpg)
Summary
In order to address the question of the optimum target level for prophylactic treatment of severe haemophilia patients, the association between endogenous clotting factor activity and outcome was studied in a cohort of 46 patients with moderate haemophilia. Data on treatment and outcome were collected annually.
Median follow-up was 8.0 years (range 1-26). Median joint bleed frequency was 1 per year and median radiological score according to Pettersson was 1 point (max. 78) at the age of 25 years. One percent increase in clotting factor level was associated with a 4 months later onset of joint bleeds (95% Confidence Interval (CI): 2-6 months) and a 16% lower Pettersson score (95% CI: 3-27%). No statistically significant effect of clotting factor activity on joint bleed frequency could be demonstrated.
These findings confirm that patients with moderate haemophilia experience only mild arthropathy, and provide evidence for a protective effect of higher clotting factor levels on joint damage.
-
References
- 1 Ramgren O. Haemophilia in Sweden III. Symptomatology, with special reference to differences between haemophilia A and B. Acta Med Scan 1962; 171: 237-42.
- 2 DePalma AF. Hemophilic arthropathy. Clin Orthop 1967; 52: 145-62.
- 3 Soreff J, Blombåck M. Arthropathy in children with severe hemophilia A. Acta Paediatr Scand 1980; 69: 667-73.
- 4 Editorial. Prophylaxis in haemophilia. Lancet 1971; 705: 118-9.
- 5 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.
- 6 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.
- 7 Bouma BN, Starkenborg AE. Dilution of haemophilic plasma used as a reagent in the determination of anti-haemophilic factor A (factor VIII). Haemostasis 1974; 03: 94-7.
- 8 Mannucci PM, Pareti FI, Ruggeri ZM. 1-Deamino-8-D-arginine vasopressin: a new pharmacological approach to the management of hemophilia and von Willebrand’s disease. Lancet 1977; 01: 869-72.
- 9 Gilbert MS. Prophylaxis: musculoskeletal evaluation. Semin Hematol 1993; 30: 3-6.
- 10 Pettersson H, Nilsson IM, Hedner U, Norehn K, Ahlberg A. Radiologic evaluation of prophylaxis in severe haemophilia. Acta Paediatr Scand 1981; 70: 565-70.
- 11 Pettersson H. Radiographic scores and implications. Semin Hematol 1993; 30: 7-9.
- 12 Aledort LM, Haschmeyer RH, Pettersson H. A longitudinal study of orthopaedic outcomes for severe factor-VIII-deficient haemophiliacs. The Orthopaedic Outcome Study Group. J Int Med 1994; 236: 391-9.
- 13 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 1965; 77 (suppl) 5-99.
- 14 Janco RL, Maclean WE, Perrin JM, Gortmaker SL. A prospective study of patterns of bleeding in boys with haemophilia. Haemophilia 1996; 02: 202-6.