Prophylaxis Therapy

Randomized, Controlled, Parallel-Group Trial of Routine Prophylaxis Versus On-Demand Treatment With rFVIII-FS in Adults With Severe Hemophilia A (SPINART).

J Thromb Haemost. 2013 Mar 25. doi: 10.1111/jth.12202. [Epub ahead of print]

Randomized, Controlled, Parallel-Group Trial of Routine Prophylaxis Versus On-Demand Treatment With rFVIII-FS in Adults With Severe Hemophilia A (SPINART).
Manco-Johnson MJ, Kempton CL, Reding MT, Lissitchkov T, Goranov S, Gercheva L, Rusen L, Ghinea M, Uscatescu V, Rescia V, Hong W.
Source

University of Colorado, Aurora, CO, USA.
Abstract

BACKGROUND:

The benefits of routine prophylaxis versus on-demand treatment with factor VIII (FVIII) products have not been evaluated in controlled clinical trials in older patients with hemophilia A.
OBJECTIVES:

To report results from a preplanned analysis of data from the first year of the 3-year SPINART study, which compares routine prophylaxis versus on-demand treatment with sucrose-formulated recombinant FVIII (rFVIII-FS) PATIENTS/METHODS: SPINART is an open-label, randomized, controlled, parallel-group, multinational trial. Males aged 12 to 50 years with severe hemophilia A, ≥150 exposure days to FVIII, no FVIII inhibitors, no prophylaxis for >12 consecutive months in the past 5 years, and 6 to 24 bleeding episodes in the preceding 6 months were randomized 1:1 to rFVIII-FS prophylaxis (25 IU/kg 3x/wk) 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 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.
RESULTS:

Eighty-four patients were enrolled and analyzed (n=42/group; mean age, 30.6 years; median treatment duration, 1.7 years). Median number of total bleeding episodes and total bleeding episodes/year were significantly lower with prophylaxis versus on demand (total, 0 vs 54.5; total/year, 0 vs 27.9; both P<0.0001). No treatment-related adverse events occurred, and no patients developed FVIII inhibitors.
CONCLUSIONS:

Routine prophylaxis with rFVIII-FS leads to a significant reduction in bleeding compared with on-demand treatment. Adverse events were consistent with the established rFVIII-FS safety profile. © 2013 International Society on Thrombosis and Haemostasis.

© 2013 International Society on Thrombosis and Haemostasis.

Copyright © 2018 PPTA. All rights reserved. (202) 789-3100