title: Sucrose-Formulated Recombinant Factor VIII Dosing Flexibility in Prophylaxis Regimens: Experience from Postmarketing Surveillance Studies.
abstract: Objectives. Prophylaxis regimens for severe hemophilia A allowing more flexible dosing while maintaining efficacy may improve adherence and decrease the cost of prophylaxis. Here, we compared the clinical effectiveness of once- or twice-weekly versus ≥3-times-weekly prophylaxis with sucrose-formulated recombinant factor VIII (rFVIII-FS) in a "real-world" practice setting. Methods. Data from 3 postmarketing studies were pooled. Patients with severe hemophilia A receiving ≥1 prophylaxis infusion/wk of rFVIII-FS for ≥80% of a prophylaxis observation period (≥5 months) were included. Patients were categorized based on physician-assigned treatment regimens of 1-2 prophylaxis injections/wk (n = 63) or ≥3 prophylaxis injections/wk (n = 76). Descriptive statistics were determined for annualized bleeding rates (ABRs). Results. Median (quartile 1; quartile 3) ABR for all bleeds was 2.0 (0; 4.0) in the 1-2 prophylaxis injections/wk group and 3.9 (1.5; 9.3) in the ≥3 prophylaxis injections/wk group. Median ABRs for joint, spontaneous, and trauma-related bleeds were numerically lower with 1-2 prophylaxis injections/wk. As an estimate of prophylaxis success, 63% (≥3 prophylaxis injections/wk) to 84% of patients (1-2 prophylaxis injections/wk) had ≤4 annualized joint bleeds. Conclusions. Dosing flexibility and successful prophylaxis with rFVIII-FS were demonstrated. Very good bleeding control was achieved with both once-twice-weekly and ≥3-times-weekly prophylaxis dosing regimens.
authors.affiliation: Bayer HealthCare, 100 Bayer Boulevard, P.O. Box 915, Whippany, NJ 08981-0915, USA.
authors.forename: Thomas J
authors.initials: TJ
authors.lastname: Humphries
authors.affiliation: Bayer Pharma AG, Global Medical Affairs Therapeutic Areas (GMA), Muellerstrasse 178, 13353 Berlin, Germany.
authors.forename: Stephan
authors.initials: S
authors.lastname: Rauchensteiner
authors.affiliation: Bayer Pharma AG, Aprather Weg 18a, Building 470, 42096 Wuppertal, Germany.
authors.forename: Claudia
authors.initials: C
authors.lastname: Tückmantel
authors.affiliation: M.A.R.C.O. GmbH & Co. KG, Moskauer Strasse 25, 40227 Düsseldorf, Germany.
authors.forename: Alexander
authors.initials: A
authors.lastname: Pieper
authors.affiliation: Bayer Pharma AG, Global Clinical Development Therapeutic Area NOHI, Aprather Weg, 42096 Wuppertal, Germany.
authors.forename: Monika
authors.initials: M
authors.lastname: Maas Enriquez
authors.affiliation: Bayer HealthCare, 100 Bayer Boulevard, P.O. Box 915, Whippany, NJ 08981-0915, USA.
authors.forename: Prasad
authors.initials: P
authors.lastname: Mathew