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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
Author(s) -
COLLINS P.,
FARADJI A.,
MORFINI M.,
ENRIQUEZ M. M.,
SCHWARTZ L.
Publication year - 2010
Publication title -
journal of thrombosis and haemostasis
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.947
H-Index - 178
eISSN - 1538-7836
pISSN - 1538-7933
DOI - 10.1111/j.1538-7836.2009.03650.x
Subject(s) - medicine , bleed , on demand , clinical endpoint , prophylactic treatment , crossover study , surgery , gastroenterology , clinical trial , multimedia , alternative medicine , pathology , computer science , placebo
Summary. Background : Hemarthroses in severe hemophilia precipitate physical, psychosocial and financial difficulties. Objective : To compare the effects of secondary prophylaxis with on‐demand sucrose‐formulated recombinant factor VIII (rFVIII‐FS) therapy in severe hemophilia A. 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 : Secondary prophylaxis with rFVIII‐FS significantly reduced the frequency of hemarthroses compared with on‐demand therapy in adult patients with severe hemophilia A.