Clinical outcomes in hemophilia: Towards development of a core set of standardized outcome measures for research. Issue 4 (9th April 2020)

• Record Type: Journal Article
• Authors: Dover, Saunya; Blanchette, Victor S.; Srivastava, Alok; Fischer, Kathelijn; Abad, Audrey; Feldman, Brian M.
• Editors: Newall, Fiona
• Is Part Of: Research and practice in thrombosis and haemostasis. Volume 4:Issue 4(2020)
• Page Start: 652
• Page End: 658
• Publication Date: 2020-04-09
• Subjects: core set -- hemophilia -- nominal groups process -- outcome assessment (health care) -- outcome measure; Thrombosis -- Periodicals; Hemostasis -- Periodicals; 616.135005
• DOI: 10.1002/rth2.12331
• Languages: English
• ISSNs: 2475-0379

Abstract

Abstract: Introduction: A lack of uniformity in the choice of outcome measurement in hemophilia care and research has led to studies with incomparable results. We identified a need to define core outcome measures for use in research and clinical care of persons with hemophilia. Objective: To move toward a core set of outcome measures for the assessment of persons with hemophilia in research and practice. Methods: A modified nominal groups process was conducted with an international group of hemophilia experts, including persons with hemophilia as follows. Step 1: item generation for all potential outcome measures. Step 2: survey where respondents voted on the relative importance and usefulness of each item. Steps 3/4: 2‐day meeting where attendees voted for items they valued, followed by open discussion and a second round of voting. Step 5: survey where respondents selected their top five items from those with >50% agreement at the meeting. Results: The highest ranked items for the pediatric core set (% agreement) are treatment satisfaction (92.7%), joint health (83.3%), a measure of access to treatment (82.5%), a measure of treatment adherence (72.5%), and generic performance based physical function (72.1%). The highest ranked items for the adult core set (% agreement) are total bleeding events (88.1%), EuroQol five dimensions (85.4%), treatment adherence (82.1%), joint health (79.1%), and number/location of bleeds per unit time (78.6%). Conclusion: This process generated a list of preferred outcome measures to consider for assessment in persons with hemophilia. This information now requires refinement to define optimal core sets for use in different clinical/research contexts.