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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.
J Thromb Haemost 2012; 10(1):81-9JT

Abstract

BACKGROUND

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.

PATIENTS/METHODS

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.

RESULTS AND CONCLUSIONS

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 (

REGISTRATION NUMBER

NCT00486278).

Authors+Show Affiliations

Hematology and Hemotherapy Center, University of Campinas, São Paulo, Brazil. erich@unicamp.brNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info available

Pub Type(s)

Journal Article
Multicenter Study
Randomized Controlled Trial
Research Support, Non-U.S. Gov't

Language

eng

PubMed ID

22470921

Citation

de Paula, Erich V., et al. "Recombinant Factor VIIa Analog (vatreptacog Alfa [activated]) for Treatment of Joint Bleeds in Hemophilia Patients With Inhibitors: a Randomized Controlled Trial." Journal of Thrombosis and Haemostasis : JTH, vol. 10, no. 1, 2012, pp. 81-9.
de Paula EV, Kavakli K, Mahlangu J, et al. Recombinant factor VIIa analog (vatreptacog alfa [activated]) for treatment of joint bleeds in hemophilia patients with inhibitors: a randomized controlled trial. J Thromb Haemost. 2012;10(1):81-9.
de Paula, E. V., Kavakli, K., Mahlangu, J., Ayob, Y., Lentz, S. R., Morfini, M., ... Chuansumrit, A. (2012). Recombinant factor VIIa analog (vatreptacog alfa [activated]) for treatment of joint bleeds in hemophilia patients with inhibitors: a randomized controlled trial. Journal of Thrombosis and Haemostasis : JTH, 10(1), pp. 81-9.
de Paula EV, et al. Recombinant Factor VIIa Analog (vatreptacog Alfa [activated]) for Treatment of Joint Bleeds in Hemophilia Patients With Inhibitors: a Randomized Controlled Trial. J Thromb Haemost. 2012;10(1):81-9. PubMed PMID: 22470921.
* Article titles in AMA citation format should be in sentence-case
TY - JOUR T1 - Recombinant factor VIIa analog (vatreptacog alfa [activated]) for treatment of joint bleeds in hemophilia patients with inhibitors: a randomized controlled trial. AU - de Paula,Erich V, AU - Kavakli,Kaan, AU - Mahlangu,Johnny, AU - Ayob,Yasmin, AU - Lentz,Steven R, AU - Morfini,Massimo, AU - Nemes,László, AU - Šalek,Silva Z, AU - Shima,Midori, AU - Windyga,Jerzy, AU - Ehrenforth,Silke, AU - Chuansumrit,Ampaiwan, AU - ,, PY - 2012/4/4/entrez PY - 2012/4/4/pubmed PY - 2012/12/21/medline SP - 81 EP - 9 JF - Journal of thrombosis and haemostasis : JTH JO - J. Thromb. Haemost. VL - 10 IS - 1 N2 - BACKGROUND: 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. PATIENTS/METHODS: 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. RESULTS AND CONCLUSIONS: 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 ( REGISTRATION NUMBER: NCT00486278). SN - 1538-7836 UR - https://www.unboundmedicine.com/medline/citation/22470921/Recombinant_factor_VIIa_analog__vatreptacog_alfa_[activated]___for_treatment_of_joint_bleeds_in_hemophilia_patients_with__inhibitors:_a_randomized_controlled_trial_ L2 - https://doi.org/10.1111/j.1538-7836.2011.04549.x DB - PRIME DP - Unbound Medicine ER -