Collecting data from patients with hemophilia is important not only for advocacy purposes, but also to answer important questions about how hemophilia is managed in the clinical setting. For example, how to best tailor prophylaxis to each individual, how to treat inhibitors, and what is the best ways to use alternative therapies like desmopressin. Individual studies are already looking at many of these questions but since hemophilia is such a rare disease, the number of patients enrolled in them is often too small to make the results conclusive, or to make the study sufficiently powerful to satisfy the increasing demands of governments and policy-makers.
As a global organization with access to a network of hemophilia treatment centres (HTCs) and patients in countries with varying levels of access to care, the WFH is uniquely positioned to develop a global registry that could be used to systematically document the care that people with hemophilia receive around the world. That is the vision of the WFH’s epidemiological research program.
The WFH has been successfully collecting and publishing aggregate demographic data on people with inherited bleeding disorders for over a decade through its Annual Global Survey. With the epidemiological research program, the aim is to collect individual patient data from an international network of treatment centres and create a World Bleeding Disorders Registry, which would serve as a platform for directed epidemiological and outcomes research.
In 2016, we will be initiating a pilot project in a few centres around the world, with the goal of progressively expanding both the amount of data we collect, and the number of centres in which we collect it, over the long term. We will also be publishing a comprehensive data collection questionnaire, the Universal Case Report Form, which outlines all the data that would ideally be collected in the clinic, at diagnosis and at each follow-up visit. While we know that very few centres in the world, if any at all, will be able to complete the questionnaire in its entirety, it will give them a roadmap to systematically collect data in a way that can be shared at a global level.
For more information on the WFH Epidemiological Research Program, contact Jennifer Laliberté at [email protected]