Clinical evaluation of an advanced category antihaemophilic factor prepared using a plasma/albumin-free method: Pharmacokinetics, efficacy, and safety in previously treated patients with haemophilia A1

Michael D. Tarantino, Peter W. Collins, Charles R M Hay, A. D. Shapiro, Ralph A. Gruppo, Erik Berntorp, G. L. Bray, S. A. Tonetta, P. C. Schroth, A. D. Retzios, S. S. Rogy, M. G. Sensel, B. M. Ewenstein, Thomas Abshire, Wing Yen Wong, Jorgen Ingerslev, Amy Shapiro, Peter W. Marks, Roshni Kulkarni, Margaret V. RagniAnne Marie Hurlet-Jensen, Arthur Thompson, Paul Giangrande, Christine Lee, Pier Mannuccio Manucci, Claude Negrier, Jozef Vermylen, Joan Gill, Ingrid Pabinger, Claire Philipp, Rachelle Nuss, Donna Di Michele

Research output: Contribution to journalArticle

Abstract

The efficacy and safety of an advanced category recombinant antihaemophilic factor produced by a plasma- and albumin-free method (rAHF-PFM) was studied in 111 previously treated subjects with haemophilia A. The study comprised a randomized, double-blinded, crossover pharmacokinetic comparison of rAHF-PFM and RECOMBINATE rAHF (R-FVIII); prophylaxis (three to four times per week with 25-40 IU kg-1 rAHF-PFM) for at least 75 exposure days; and treatment of episodic haemorrhagic events. Median age was 18 years, 96% of subjects had baseline factor VIII -1 year-1) than subjects who were more adherent (4.4 episodes subject-1 year-1; P <0.03). One subject developed a low titre, non-persistent inhibitor (2.0 BU) after 26 exposure days. These data demonstrate that rAHF-PFM is bioequivalent to R-FVIII, and suggest that rAHF-PFM is efficacious and safe, without increased immunogenicity, for the treatment of haemophilia A.

Original languageEnglish
Pages (from-to)428-437
Number of pages10
JournalHaemophilia
Volume10
Issue number5
DOIs
Publication statusPublished - Sep 2004

Keywords

  • Factor VIII
  • Haemophilia A
  • Recombinant antihaemophilic factor plasma-free method
  • Recombinant factor VIII

ASJC Scopus subject areas

  • Hematology

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    Tarantino, M. D., Collins, P. W., Hay, C. R. M., Shapiro, A. D., Gruppo, R. A., Berntorp, E., Bray, G. L., Tonetta, S. A., Schroth, P. C., Retzios, A. D., Rogy, S. S., Sensel, M. G., Ewenstein, B. M., Abshire, T., Wong, W. Y., Ingerslev, J., Shapiro, A., Marks, P. W., Kulkarni, R., ... Di Michele, D. (2004). Clinical evaluation of an advanced category antihaemophilic factor prepared using a plasma/albumin-free method: Pharmacokinetics, efficacy, and safety in previously treated patients with haemophilia A1. Haemophilia, 10(5), 428-437. https://doi.org/10.1111/j.1365-2516.2004.00932.x