Abstract | AIM: A multicenter randomized open-label crossover prospective trial was designed to compare the efficacy, safety, and cost of standard and high dosages of recombinant factor VIIa ( rFVIIa) for home treatment of hemarthroses in hemophiliacs with inhibitors. METHODS: Patients were instructed to treat, within 6 h from the onset of bleeding, four consecutive hemarthroses of ankles, knees, or elbows, either with the rFVIIa standard dose of 90 microg kg(-1) (repeated as necessary every 3 h) or with a single high dose of 270 microg kg(-1). Patients who did not achieve a clinical success within 9 h continued rFVIIa treatment with repeated standard doses. Response to treatment was assessed for up to 48 h by patients/caregivers, who reported on a Visual Analogue Scale (VAS) graded from 0 to 100 the improvement in symptoms and also rated the responses as effective, partially effective or ineffective. Success was defined a treatment course rated as effective and with a VAS score > or =70 and failure a treatment course rated as ineffective and VAS score < or =30, whereas treatment courses that did not fulfill these criteria were considered partial responses. RESULTS: Twenty hemophiliacs with inhibitors were originally enrolled (median age: 27 years), 18 of them treated 32 hemarthroses assigned to the standard-dosage and 36 to the high-dosage regimen, during the study period of 18 months. Forty-eight hemarthroses (71%) occurred in target joints. Success rates for standard- and high-dosage regimens were similar: 31% and 25% at 9 h, 53% and 50% at 24 h, 66% and 64% at 48 h, the end point for outcome assessment. The median number of rFVIIa infusions needed to achieve a successful course was significantly greater for the standard-dosage (n = 3) than for the high-dosage regimen (n = 1), and the median amount of rFVIIa ultimately used per successful course was identical (270 microg kg(-1)). CONCLUSION: Our results indicate that a high-dosage regimen with rFVIIa for home treatment of hemarthroses is effective, safe, does not imply an increased consumption of rFVIIa and requires the infusion of a smaller number of rFVIIa doses. Its convenience is particularly relevant in cases with difficult venous access and in hemorrhages into target joints.
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Authors | E Santagostino, M E Mancuso, A Rocino, G Mancuso, F Scaraggi, P M Mannucci |
Journal | Journal of thrombosis and haemostasis : JTH
(J Thromb Haemost)
Vol. 4
Issue 2
Pg. 367-71
(Feb 2006)
ISSN: 1538-7933 [Print] England |
PMID | 16420567
(Publication Type: Journal Article, Multicenter Study, Randomized Controlled Trial, Research Support, Non-U.S. Gov't)
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Chemical References |
- Recombinant Proteins
- Factor VIIa
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Topics |
- Adolescent
- Adult
- Child
- Child, Preschool
- Cross-Over Studies
- Factor VIIa
(administration & dosage, adverse effects, antagonists & inhibitors, therapeutic use)
- Hemarthrosis
(drug therapy, etiology)
- Hemophilia A
(complications, drug therapy)
- Humans
- Infant
- Male
- Middle Aged
- Prospective Studies
- Recombinant Proteins
(administration & dosage, adverse effects, therapeutic use)
- Safety
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