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Sucrose-Formulated Recombinant Factor VIII Dosing Flexibility in Prophylaxis Regimens: Experience from Postmarketing Surveillance Studies.

Humphries TJ, Rauchensteiner S, Tückmantel C, Pieper A, Maas Enriquez M, Mathew P - Adv Hematol (2015)

Bottom Line: Methods.Data from 3 postmarketing studies were pooled.Results.

View Article: PubMed Central - PubMed

Affiliation: Bayer HealthCare, 100 Bayer Boulevard, P.O. Box 915, Whippany, NJ 08981-0915, USA.

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.

No MeSH data available.


Related in: MedlinePlus

Annualized bleeding rate for all bleeds by dosing group. Q1 = quartile 1; Q3 = quartile 3.
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fig1: Annualized bleeding rate for all bleeds by dosing group. Q1 = quartile 1; Q3 = quartile 3.

Mentions: The median (quartile 1; quartile 3 [Q1; Q3]) 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 (Figure 1); mean ± SD ABR was 4.1 ± 6.4 and 7.0 ± 10.7, respectively. Similarly, the median ABRs for joint bleeds, spontaneous bleeds, and trauma-related bleeds were numerically lower in the 1-2 prophylaxis injections/wk group compared with the ≥3 prophylaxis injections/wk group (Table 2).


Sucrose-Formulated Recombinant Factor VIII Dosing Flexibility in Prophylaxis Regimens: Experience from Postmarketing Surveillance Studies.

Humphries TJ, Rauchensteiner S, Tückmantel C, Pieper A, Maas Enriquez M, Mathew P - Adv Hematol (2015)

Annualized bleeding rate for all bleeds by dosing group. Q1 = quartile 1; Q3 = quartile 3.
© Copyright Policy
Related In: Results  -  Collection

License
Show All Figures
getmorefigures.php?uid=PMC4556870&req=5

fig1: Annualized bleeding rate for all bleeds by dosing group. Q1 = quartile 1; Q3 = quartile 3.
Mentions: The median (quartile 1; quartile 3 [Q1; Q3]) 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 (Figure 1); mean ± SD ABR was 4.1 ± 6.4 and 7.0 ± 10.7, respectively. Similarly, the median ABRs for joint bleeds, spontaneous bleeds, and trauma-related bleeds were numerically lower in the 1-2 prophylaxis injections/wk group compared with the ≥3 prophylaxis injections/wk group (Table 2).

Bottom Line: Methods.Data from 3 postmarketing studies were pooled.Results.

View Article: PubMed Central - PubMed

Affiliation: Bayer HealthCare, 100 Bayer Boulevard, P.O. Box 915, Whippany, NJ 08981-0915, USA.

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.

No MeSH data available.


Related in: MedlinePlus