Current issues in prophylaxis

Long-acting concentrates

Is 1% sufficient to prevent sub-clinical bleeding, or is it based on historical supply constraints, economics, and treatment protocol burdens?

New formulations of clotting factor concentrates with extended half-lives (they remain at higher levels for longer periods in the body) are designed to sustain the level of clotting factor concentrates in the blood for longer periods of time, meaning that less frequent and/or fewer injections will be needed to achieve the same result. The availability of these medications could change how prophylaxis is administered. They have the potential to increase the adoption of prophylaxis among patients, to improve patient adherence, and to improve outcomes.

Personalized prophylaxis

We are entering an era in which it is becoming possible to treat an individual patient, rather than treating their disease. To be most effective, a prophylaxis protocol should be tailored to the individual based on their age, bleeding pattern, joint health, the level and timing of physical activity they engage in, their clotting factor levels, and their ability to adhere to a protocol. Prophylactic regimens should also be flexible enough to change with time as the individual patient’s circumstances change.

Knowledge of a patient’s pharmacokinetics – i.e. how the body absorbs, distributes, and eliminates a drug – is likely to help personalize prophylaxis when combined with other information.

New panel discussion: New Developments in Treatment for Bleeding Disorders – Personalized Prophylaxis, will prolonged half-life products mean tailored treatment for all?
With a number of new clotting factor concentrate products in the pipeline and entering the market in various countries, leading hematologists and patient advocates from around the world debate what this might mean for the standards of care, access to care, and quality of life for all people with hemophilia.

Panel: David Lillicrap, Edward Tuddenham, Glenn Pierce, Declan Noone, chaired by David Page.

The opinions expressed in this video are those of the individual panelists and not the official position of the World Federation of Hemophilia.

What is prophylaxis?
This patient handbook contains basic information about prophylaxis, the regular infusion of clotting factor concentrates in order to prevent bleeding. Prophylaxis is the goal of treatment for people with severe hemophilia, allowing them to remain active and participate more fully in daily life.

This booklet is also available in French, Russian, Arabic, Simplified Chinese, and Spanish.

Updated March 2016