Mixing and administration times of bypassing agents: observations from the Dosing Observational Study in Hemophilia (DOSE)

Abstract

Jennifer Maahs,1 Jennifer Donkin,2 Michael Recht,3 David L Cooper4 1Indiana Hemophilia and Thrombosis Center, Indianapolis, IN, 2Hemostasis and Thrombosis Center, Children's Hospital Los Angeles, Los Angeles, CA, 3Oregon Hemophilia Center, Oregon Health and Science University, Portland, OR, 4Novo Nordisk, Princeton, NJ, USA Abstract: DOSE (Dosing Observational Study in Hemophilia) was a prospective, observational diary study designed to evaluate the use of bypassing agents in patients prescribed recombinant activated factor VII (rFVIIa) as first-line treatment in the home setting. Patients with congenital hemophilia with inhibitors and caregivers participated, and as part of the study, the time spent preparing and administering product was recorded for bypassing agent (BPA) infusions. The aim of this manuscript is to present the results of the analysis of the time spent preparing and administering a single dose of either rFVIIa or plasma-derived activated prothrombin complex concentrate (pd-aPCC). Diaries were completed for 18 adult patients and 19 caregivers of 21 children with 176 BPA-treated bleeding episodes and 1,350 BPA infusions (1,270 rFVIIa, 80 pd-aPCC). The median preparation and administration times were 5.0 minutes and 5.0 minutes for rFVIIa and 29.0 minutes and 24.5 minutes for pd-aPCC, respectively. Preparation and administration times were significantly shorter with rFVIIa than pd-aPCC (P<0.0001). The significantly shorter combined preparation and administration time of rFVIIa, taking into consideration the faster-than-recommended aPCC infusion rates, suggests that rFVIIa permits a rapid and safe initiation of treatment once a bleeding episode is identified and a decision is made to treat at home. Keywords: congenital hemophilia, inhibitors, bypassing agent, recombinant-activated factor VIIa, home treatment