Recombinant factor VIIa analog (vatreptacog alfa [activated]) for treatment of joint bleeds in hemophilia patients with inhibitors: a randomized controlled trial

Erich V de Paula; Kaan Kavakli; Johnny Mahlangu; Yasmin Ayob; Steven R Lentz; Massimo Morfini; László Nemes; Silva Z Šalek; Midori Shima; Jerzy Windyga; Silke Ehrenforth; Ampaiwan Chuansumrit; 1804 (adept(TM)1) Investigators

doi:10.1111/j.1538-7836.2011.04549.x

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Recombinant factor VIIa analog (vatreptacog alfa [activated]) for treatment of joint bleeds in hemophilia patients with inhibitors: a randomized controlled trial

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Recombinant factor VIIa analog (vatreptacog alfa [activated]) for treatment of joint bleeds in hemophilia patients with inhibitors: a randomized controlled trial

Erich V de Paula, Kaan Kavakli, Johnny Mahlangu, Yasmin Ayob, Steven R Lentz, Massimo Morfini, László Nemes, Silva Z Šalek, Midori Shima, Jerzy Windyga, …

Journal of thrombosis and haemostasis, Vol.10(1), pp.81-89

01/2012

DOI: 10.1111/j.1538-7836.2011.04549.x

PMID: 22470921

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Abstract

A recombinant factor VIIa analog (NN1731; vatreptacog alfa [activated]) was developed to provide safe, rapid and sustained resolution of bleeds in patients with hemophilia and inhibitors. This global, prospective, randomized, double-blinded, active-controlled, dose-escalation trial evaluated and compared one to three doses of vatreptacog alfa at 5, 10, 20, 40, and 80 lg kg(-1) with one to three doses of recombinant FVIIa (rFVIIa) at 90 lg kg(-1) in the treatment of acute joint bleeds in hemophilia patients with inhibitors. The primary endpoint comprised adverse events; secondary endpoints were evaluations of immunogenicity, pharmacokinetics, and efficacy. Overall, 96 joint bleeds in 51 patients (> 12 years of age) were dosed. Vatreptacog alfa was well tolerated, with a low frequency of adverse events. No immunogenic or thrombotic events related to vatreptacog alfa were reported. A high efficacy rate of vatreptacog alfa in controlling acute joint bleeds was observed; 98% of bleeds were controlled within 9 h of the initial dose in a combined evaluation of 20–80 lg kg(-1) vatreptacog alfa. The efficacy rate observed for rFVIIa (90%) is consistent with data from published clinical trials. The trial was not powered to compare efficacy, and further trials are needed to investigate the efficacy of vatreptacog alfa as compared with that of rFVIIa. The trial was registered at ClinicalTrials.gov ( NCT00486278).

Recombinant Proteins - therapeutic use

Double-Blind Method

Autoantibodies - blood

Hemophilia A - immunology

Humans

Recombinant Proteins - pharmacokinetics

Treatment Outcome

Factor VIIa - therapeutic use

Hemophilia A - complications

Dose-Response Relationship, Drug

Young Adult

Hemarthrosis - drug therapy

Adolescent

Adult

Factor VIIa - pharmacokinetics

Hemophilia A - drug therapy

Details

Title: Subtitle: Recombinant factor VIIa analog (vatreptacog alfa [activated]) for treatment of joint bleeds in hemophilia patients with inhibitors: a randomized controlled trial
Creators: Erich V de Paula - Hematology and Hemotherapy Center, University of Campinas, São Paulo, Brazil. erich@unicamp.br
Kaan Kavakli
Johnny Mahlangu
Yasmin Ayob
Steven R Lentz
Massimo Morfini
László Nemes
Silva Z Šalek
Midori Shima
Jerzy Windyga
Silke Ehrenforth
Ampaiwan Chuansumrit
1804 (adept(TM)1) Investigators
Resource Type: Journal article
Publication Details: Journal of thrombosis and haemostasis, Vol.10(1), pp.81-89
DOI: 10.1111/j.1538-7836.2011.04549.x
PMID: 22470921
NLM abbreviation: J Thromb Haemost
ISSN: 1538-7933
eISSN: 1538-7836
Language: English
Date published: 01/2012
Academic Unit: Hematology, Oncology, and Blood & Marrow Transplantation; Internal Medicine
Record Identifier: 9984094641002771

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