Longer-acting factor VIII to overcome limitations in haemophilia management: The PEGylated liposomes formulation issue

G. Di Minno, A. M. Cerbone, A. Coppola, E. Cimino, M. Di Capua, F. Pamparana, A. Tufano, M. N D Di Minno

Research output: Contribution to journalArticlepeer-review

Abstract

Injected factor VIII (FVIII), the current treatment for haemophilia A, leads to major improvements in the quality of life and life expectancy of individuals with this disorder. However, because injected FVIII has a short half-life in vivo, this strategy has major limitations for highly demanding regimens (e.g. prophylaxis, immune tolerance induction, surgery). Newer formulations of longer-acting FVIII are presently under investigation. The use of low molecular weight polyethylene glycol (PEG)-containing liposomes as carriers for recombinant FVIII (rFVIII) results in the prolongation of haemostatic efficacy. Data from preclinical experiments in mice, early clinical evaluations, and pharmacokinetics and pharmacodynamics results indicate that an rFVIII pegylated liposomal formulation may provide potential clinical benefit to patients with severe haemophilia A by prolonging the protection from bleeding. In light of this potential clinical benefit, a multicentre, randomized, active-controlled, non-inferiority phase II trial with two parallel treatment arms and equal randomization after stratification for the presence or absence of target joints in patients and for ages ≥18 years vs.

Original languageEnglish
Pages (from-to)2-6
Number of pages5
JournalHaemophilia
Volume16
Issue numberSUPPL. 1
DOIs
Publication statusPublished - Jan 2010

Keywords

  • Clinical trial
  • Efficacy
  • Haemophilia A
  • PEGylated liposomes
  • rFVIII
  • Safety

ASJC Scopus subject areas

  • Hematology
  • Genetics(clinical)

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