Proposed Core Set of Items for Measuring Disease Activity in Systemic Juvenile Idiopathic Arthritis.

Objective: To date, there are no standardized disease activity tools for systemic juvenile idiopathic arthritis (sJIA). We developed a core set of disease activity measures for sJIA.

Methods: We conducted a validation study in patients with sJIA recruited from 3 Canadian institutions.

Experience with central venous access devices (CVADs) in the Canadian hemophilia primary prophylaxis study (CHPS).

Introduction: Haemophilia A treatment with factor VIII concentrates requires frequent venipunctures; a central venous access device (CVAD) may be required to facilitate reliable venous access, especially in young children. While CVADs provide reliable venous access, complications such as infection and thrombosis may occur.

Aim: The aim of this study was to assess CVAD use in the Canadian Hemophilia Primary Prophylaxis Study (CHPS), a single-arm, multi-centre prospective study whereby factor use is tailored to individual prophylactic need.

Using value-of-information methods when the disease is rare and the treatment is expensive--the example of hemophilia A.

Background: Hemophilia A is a rare, sex-linked genetic disorder treated with intravenous administration of factor VIII (FVIII) to prevent bleeding; however, approaches vary across and within countries. Value-of-information (VOI) methods identify situations in which the cost-benefit evidence is sufficient to adopt one treatment strategy over another; when the evidence is insufficient, VOI methods provide the optimal sample size for additional research.

Objective: The objective of the study was to use VOI methods in a cost-benefit decision context to evaluate the current evidence in support of using (1) alternate day prophylaxis (AP), (2) tailored prophylaxis (TP) or (3) on-demand treatment (OD) with FVIII to prevent arthropathy in children with severe hemophilia A.

Musculoskeletal health of subjects with hemophilia A treated with tailored prophylaxis: Canadian Hemophilia Primary Prophylaxis (CHPS) Study.

Background: Full-dose prophylaxis is very effective at minimizing joint damage but is costly. Tailored prophylaxis has been proposed as a way of reducing costs while still protecting joints.

Objective: To report detailed findings in index joints of 56 subjects with severe hemophilia A entered into the Canadian Hemophilia Prophylaxis Study, and treated with tailored prophylaxis, after 13 years.