Comparative analysis of the pivotal studies of extended half-life recombinant FVIII products for treatment of haemophilia A

Pier Mannuccio Mannucci, Paolo Angelo Cortesi, Matteo Nicola Dario Di Minno, Mario Sanò, Lorenzo Giovanni Mantovani, Giovanni Di Minno

Research output: Contribution to journalReview articlepeer-review

Abstract

The need to reduce the burden of injections, and improve adherence and clinical outcomes in haemophilia A led to the development of recombinant FVIII products endowed with an extended plasma half-life (EHL-rFVIII) in comparison with standard half-life products (SHL-rFVIII). Lack of head-to-head studies makes difficult to grasp the relative value of each treatment option. We conducted a combined evaluation of the individual pivotal trials in order to assess between-product differences regarding the reported efficacy results and FVIII consumption. We evaluated 4 EHL-rFVIII products available to treat patients with haemophilia A without inhibitors and also a SHL-rFVIII as a comparator. In the frame of these clinical studies, all the EHL-rFVIII products showed a decrease in the injection burden coupled with good clinical efficacy, even though there were between-product differences in terms of reduction in injection frequencies. Further, between-product differences in terms of weekly/yearly consumption of rFVIII expressed in IU/Kg were identified, suggesting a different economic impact for the different EHL-rFVIII products in the context of comparable clinical efficacy. The present findings based upon the review of pivotal studies done in the frame of a highly selected clinical scenario should be integrated with real-life data.

Original languageEnglish
JournalHaemophilia
DOIs
Publication statusAccepted/In press - 2021

Keywords

  • clinical trial
  • costs and cost analysis
  • factor VIII
  • haemophilia A
  • pharmacokinetics
  • prophylaxis

ASJC Scopus subject areas

  • Hematology
  • Genetics(clinical)

Fingerprint

Dive into the research topics of 'Comparative analysis of the pivotal studies of extended half-life recombinant FVIII products for treatment of haemophilia A'. Together they form a unique fingerprint.

Cite this