Prophylactic versus on-demand treatments for hemophilia: advantages and drawbacks

ABSTRACT

Introduction: Early long-term prophylaxis is the standard of care to prevent joint bleeding and chronic arthropathy in patients with severe hemophilia.

Areas covered: Despite the obvious prophylaxis advantages upon the clinical outcomes, there are still several drawbacks to be addressed for the optimal patients’ compliance. Frequency of treatment due to short half-life of conventional FVIII and FIX concentrates, difficult venous access, adherence to the prescribed therapy and costs may represent significant critical issues. The development of inhibitors also makes replacement therapy ineffective, preventing patients from receiving long-term prophylaxis. This paper will review these drawbacks and the tools to overcome these limitations, mainly thanks to the use of extended half-life products and the development of novel non-conventional therapeutic approaches.

Expert commentary: The use of extended half-life products may help in reducing the burden of the frequent intravenous administration and in better tailoring an individualized prophylaxis. The development of novel therapeutic approaches, like the bi-specific antibody mimicking the coagulation function of FVIII or inhibition of anticoagulant proteins may facilitate prophylaxis treatment not only in patients with inhibitors, but also in severe hemophilia patients without inhibitors. Exciting opportunities are emerging for improving prophylaxis in hemophilia.

KEYWORDS:

• Hemophilia
• bleeding
• replacement therapy
• prophylaxis
• challenge
• extended half-life concentrates
• novel therapeutic approaches

Declaration of interest

G Castaman declares advisory board membership for Bayer, Shire, Kedrion, Sobi, Pfizer, Uniqure, Roche, Novo Nordisk, CSL Behring, and research grants to his institute from Pfizer and CSL Behring. S Linari declares advisory board membership for Novo Nordisk, Sobi, Genzyme, and Shire. The authors have no other relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript apart from those disclosed.

Reviewer disclosures

Peer reviewers on this manuscript have no relevant financial or other relationships to disclose.

Additional information

Funding

This paper was not funded.