A Pilot Project towards Establishment of a US/Canadian Diagnostic Registry of Children with WG and Related Vasculitides

Investigator: David A. Cabral, MBBS

Location: BC Children’s Hospital and University of British Columbia, Vancouver, Canada

Timeline: March 1, 2005 – February 28, 2006

Abstract:  The presentation and natural history of pediatric Wegener’s Granulomatosis (pWG) may differ significantly from adult disease; and pWG may be more difficult to distinguish from other related types of chronic vasculitis. One-third of pWG does not fulfill adult-derived classification criteria and two-thirds of chronic vasculitis in children is unclassifiable. Because study of pWG is limited by rarity of the disease, we propose to set up a pilot pediatric registry of all chronic small-medium vessel vasculitis to determine the validity, and specificity of adult classification criteria for use in pWG; feasibility of establishing an international (US/Canada) registry will thus be assessed during operation of the pilot registry and by survey of potential physician participants. In the long term, a registry will permit detailed characterization of pWG and identify homogeneous subgroups of patients to enhance pathophysiological understanding and treatment of the disease.

The first phase of the study will electronically survey pediatric rheumatologists throughout US/Canada who are members of CARRA (Childhood Arthritis and Rheumatology Research Alliance) to determine feasibility of establishing and maintaining an international registry for the short or long term; assessment will include estimates of patient numbers, local resources and commitment to participation.

Secondly, by Delphi survey of the membership we will determine optimal diagnostic features and disease markers for pWG, to be included in the pilot, and proposed international registry; this is a well established consensus formation methodology to assimilate ‘expert’ opinion (weighted for experience) through the use of sequential, iterative questionnaires rationalized by interim analysis.

Finally, information on all cases of pWG and related vasculitides over the preceding five years will be collected from CARRA centers in the Pacific-Northwest, entered into the ‘electronic’ pilot registry, and analyzed against adult-derived criteria. Demands on participating centers will be simultaneously evaluated as part of the feasibility assessment.

Specific aim:  The ultimate aim of this project is to identify the need and feasibility for a valid, secure, web-based international (US/Canadian) registry of children with Wegener’s Granulomatosis and related small-medium vessel chronic vasculitis that has the ongoing commitment of all pediatric rheumatology centers to ensure reliable collection of diagnostic and monitoring data.