Update in Work Package 2
Patient recruitment for international PCD recruitment continues
(Progress in Work Package 2)
Bestcilia Work Package 2 (WP2) has noted considerable progress this year. The main objective of WP2 is establishing an international registry of PCD patients which will allow for systematic collection of data on incidence as well as general trends in diagnostics and treatment regimens for this rare disease. That aim has been realized to a large extent and the works continue to progress as scheduled.
With the international registry already up and running (accessible online under www.pcdregistry.eu), the current primary goal of WP2 is the recruitment of PCD patients for the international registry; in the recent months 75 new subject were enrolled, bringing the total to 227. Moreover, four centers are working on entering data into the registry and six additional centers are in the process of obtaining ethical approval to participate.
With a view to raising awareness about PCD, WP2 team has also drafted a manuscript which provides a detailed description of the registry and its features. The manuscript has been submitted for publication and will soon be made available for the general public.
Current activities in WP2 also involve a sideline task of introducing technological improvements to the registry and its software. After a few months of preparatory works, a platform for multiple identical registries has recently been put in place and is available for the public online. A meeting of Bestcilia partners as well as potential new registry contributors is being organized; teams from all WPs will attempt to arrive at a consortium consensus as to whether the registies should be managed jointly by the IT partner involved with WP2 or individually by each research center.
Through PCD registry-related works, the WP2 team continues to make consistent efforts to faciliate collecting data on the clinical presentation as well as to prepare a patient cohort for future multicenter clinical studies. The next step on the way will be the cross-sectional analysis of the data obtained from the registry in the form of annual registry reports, intended to complement the general endeavor of the consortium, i.e. improved quality of life and survival of PCD sufferers.