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Matching-adjusted indirect comparisons of annualized bleeding rate and utilization of BAY 94-9027 versus three recombinant factor VIII agents for prophylaxis in patients with severe hemophilia A

Authors Batt K, Gao W, Ayyagari R, Deschaseaux C, Vashi PB, Yao Z, Wang Y, Kessabi S, Klamroth R

Received 26 February 2019

Accepted for publication 2 May 2019

Published 20 June 2019 Volume 2019:10 Pages 147—159


Checked for plagiarism Yes

Review by Single-blind

Peer reviewer comments 2

Editor who approved publication: Dr Martin Bluth

Katharine Batt,1 Wei Gao,2 Rajeev Ayyagari,2 Céline Deschaseaux,3 Parth B Vashi,4 Zhiwen Yao,2 Yao Wang,2 Sophia Kessabi,3 Robert Klamroth5

1Department of Internal Medicine, Section on Hematology/Medical Oncology, Wake Forest University Baptist Medical Center, Winston-Salem, NC, USA; 2Analysis Group, Inc., Boston, MA, USA; 3Global Market Access Hematology, Bayer Consumer Care AG, Basel, Switzerland; 4US Data Generation and Observational Studies, Bayer Corporation, Whippany, NJ, USA; 5Department for Internal Medicine - Vascular Medicine and Coagulation Disorders, Vivantes Klinikum im Friedrichshain, Berlin, Germany

Background: BAY 94-9027 is an extended half-life recombinant factor VIII (rFVIII) that prevents bleeding in persons with hemophilia A at twice-weekly, 5-day, and 7-day dosing intervals. In rare diseases such as hemophilia, where small populations preclude head-to-head comparisons in randomized controlled trials, outcomes from different studies can be compared by matching-adjusted indirect treatment comparisons (MAICs) via matched summary statistics of individual patient data. This study compared MAIC-adjusted outcomes of BAY 94-9027 with other FVIII agents for prophylaxis of hemophilia.
Methods: Weighted patient data from the BAY 94-9027 PROTECT VIII trial were used to compare annualized bleeding rates (ABRs), percentage of patients with zero bleeds, and factor utilization against published data on rFVIII–Fc fusion protein (rFVIIIFc), BAX 855, and recombinant antihemophilic factor/plasma/albumin-free method (rAHF-PFM).
Results: After matching BAY 94-9027 and comparators, the mean BAY 94-9027 utilization was significantly lower than rFVIIIFc pre- and post-matching (66.2 vs 82.2 IU/kg/week; 66.5 vs 82.2 IU/kg/week; both P<0.001). Median BAY 94-9027 utilization (IU/kg/week) trended lower than BAX 855 (64.3 vs 87.4) and rAHF-PFM (2004 study: 64.0 vs 107.5; 2012 study: 63.6 vs 109.9). Mean ABRs and percentages of patients with zero bleeds were similar post-matching between BAY 94-9027 and comparators.
Conclusion: BAY 94-9027 demonstrated similar MAIC-adjusted ABR with lower utilization than rFVIIIFc, BAX 855, and rAHF-PFM.

Keywords: rFVIII, annualized bleeding rate, utilization

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