Randomized, controlled, parallel‐group trial of routine prophylaxis vs. on‐demand treatment with sucrose‐formulated recombinant factor VIII in adults with severe hemophilia A (SPINART). Issue 6 (3rd July 2013)

• Record Type: Journal Article
• Authors: Manco‐Johnson, M. J.; Kempton, C. L.; Reding, M. T.; Lissitchkov, T.; Goranov, S.; Gercheva, L.; Rusen, L.; Ghinea, M.; Uscatescu, V.; Rescia, V.; Hong, W.
• Is Part Of: Journal of thrombosis and haemostasis. Volume 11:Issue 6(2013)
• Page Start: 1119
• Page End: 1127
• Publication Date: 2013-07-03
• Subjects: Thrombosis -- Periodicals; Hemostasis -- Periodicals; Blood coagulation disorders -- Periodicals; 616.1
• DOI: 10.1111/jth.12202
• Languages: English
• ISSNs: 1538-7933

Abstract

<abstract abstract-type="main" id="jth12202-abs-0001"> <title>Summary</title> <sec id="jth12202-sec-0001" sec-type="section"> <title>Background</title> <p>The benefits of routine prophylaxis vs. on‐demand treatment with factor VIII products have not been evaluated in controlled clinical trials in older patients with hemophilia A.</p> </sec> <sec id="jth12202-sec-0002" sec-type="section"> <title>Objectives</title> <p>To report results from a preplanned analysis of data from the first year of the 3‐year SPINART study, which compares routine prophylaxis with on‐demand treatment with sucrose‐formulated recombinant FVIII (rFVIII‐FS).</p> </sec> <sec id="jth12202-sec-0003" sec-type="section"> <title>Patients/Methods</title> <p>SPINART is an open‐label, randomized, controlled, parallel‐group, multinational trial. Males aged 12–50 years with severe hemophilia A, ≥ 150 days of exposure to FVIII, no FVIII inhibitors, no prophylaxis for > 12 consecutive months in the past 5 years and 6–24 bleeding episodes in the preceding 6 months were randomized 1 : 1 to rFVIII‐FS prophylaxis (25 IU kg<sup>−1</sup>, three times weekly) or on‐demand treatment. The primary efficacy endpoint, number of total bleeding episodes in the intent‐to‐treat population, was analyzed after the last patient had completed 1 year of follow‐up. A negative binomial model was used for the primary endpoint analysis; analysis of variance was used for confirmatory analysis of annualized bleeding rates.</p> </sec> <sec id="jth12202-sec-0004" sec-type="section"> <title>Results</title> <p>Eighty‐four patients were enrolled and analyzed (<italic>n</italic> = 42 per group; mean age, 30.6 years; median treatment duration, 1.7 years). The median number of total bleeding episodes and total bleeding episodes per year were significantly lower with prophylaxis than with on‐demand treatment (total, 0 vs. 54.5; total per year, 0 vs. 27.9; both <italic>P </italic>< 0.0001). No treatment‐related adverse events occurred, and no patients developed FVIII inhibitors.</p> </sec> <sec id="jth12202-sec-0005" sec-type="section"> <title>Conclusions</title> <p>Routine prophylaxis with rFVIII‐FS leads to a significant reduction in bleeding as compared with on‐demand treatment. Adverse events were consistent with the established rFVIII‐FS safety profile.</p> </sec> </abstract>