Abstract | BACKGROUND: OBJECTIVE: PATIENTS AND METHODS: This open-label study included patients aged 30-45 years with factor VIII (FVIII) coagulant activity < 1 IU dL(-1) who were using on-demand FVIII treatment. Patients were treated with rFVIII-FS on demand for 6 months, followed by 7 months prophylaxis (20-40 IU kg(-1), three times per week, with the first month considered a run-in). The primary endpoint was the number of hemarthroses. RESULTS: Twenty patients were enrolled (n = 19 completed); the mean age was 36.4 years, and 16 had target joints. The median (25-75%) number of joint bleeds decreased significantly with prophylaxis [0 (0-3)] vs. on-demand [15 (11-26); P < 0.001] therapy. The number of all bleeds was 0 (0-3) vs. 20.5 (14-37; P < 0.001), respectively. Median (range) total Gilbert scores improved after prophylaxis [18 (3-39)] compared with on-demand [25 (4-46)] therapy, predominantly reflecting the improved bleeding score. Median time from last prophylactic infusion to bleed was 2 days; 82.5% of bleeds occurred 2-3 days after the last infusion. Median 48-h and 72-h FVIII trough levels measured during months 10 and 13 were consistently > 6 and > 4 IU dL(-1), respectively. Treatment was well tolerated, and no inhibitor formation was observed. CONCLUSION:
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Authors | P Collins, A Faradji, M Morfini, M M Enriquez, L Schwartz |
Journal | Journal of thrombosis and haemostasis : JTH
(J Thromb Haemost)
Vol. 8
Issue 1
Pg. 83-9
(Jan 2010)
ISSN: 1538-7836 [Electronic] England |
PMID | 19817995
(Publication Type: Clinical Trial, Comparative Study, Journal Article, Multicenter Study, Research Support, Non-U.S. Gov't)
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Chemical References |
- Coagulants
- recombinant FVIII, sugar formulated
- Sucrose
- Factor VIII
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Topics |
- Adult
- Chemistry, Pharmaceutical
- Coagulants
(administration & dosage, adverse effects, chemistry)
- Cross-Over Studies
- Drug Administration Schedule
- Europe
- Factor VIII
(administration & dosage, adverse effects, chemistry)
- Hemarthrosis
(blood, drug therapy, etiology, prevention & control)
- Hemophilia A
(blood, complications, drug therapy)
- Home Infusion Therapy
- Humans
- Infusions, Intravenous
- Male
- Middle Aged
- Prospective Studies
- Secondary Prevention
- Severity of Illness Index
- Sucrose
(administration & dosage, adverse effects, chemistry)
- Time Factors
- Treatment Outcome
- United States
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