Presentation Details
| Development of a Patient Reported Outcome Measure to Study Quality of Life in Bleeding Disorder of Unknown Cause Callie Berkowitz1, Supreet Goraya2, Alice Ma1, Nigel Key1, Angela Stover3. 1Division of Hematology and Blood Research Center, University of North Carolina, Chapel Hill, NC, USA.2Department of Maternal, Child, and Family Health, Gillings School of Public Health, University of North Carolina, Chapel Hill, NC, USA.3Department of Health Policy and Management, Gillings School of Public Health, University of North Carolina, Chapel Hill, NC, USA |
Abstract
Background: Bleeding of Disorder Unknown Cause (BDUC) is an increasingly recognized diagnostic category encompassing patients with a clear bleeding tendency but without identifiable abnormality on hemostatic testing. Phenotypically, BDUC resembles other mucocutaneous bleeding disorders including von Willebrand’s disease (vWD) and platelet function defects, and the National Hemophilia Foundation guidelines now recommend that patients with BDUC be followed in Hemophilia Treatment Centers. However, little is known about the Quality of Life (QoL) impact of the disorder and patient-specific needs. Existing validated bleeding disorder-specific patient reported outcome measures (PROMs) may fail to capture the social, emotional, and reproductive health impact of this disorder. Objective: The objective of this study is to develop a disease-specific QoL measure for patients with BDUC. Methods: We conducted a literature review of existing PROMs used in the study of bleeding disorders, with focus on vWD, hemophilia, and menorrhagia. Using results of the literature review and our clinical expertise in caring for BDUC patients, we drafted an initial set of candidate items and elicited expert feedback. Semi-structured interviews of patients with BDUC were conducted for purposes of both concept elicitation and cognitive debriefing on candidate items. Items were iteratively revised with a trained group of PRO researchers, and a literacy review was performed using Lexile Analyzer. Candidate items were subsequently piloted in an adult undiagnosed bleeding disorder clinic with endpoints of acceptability and feasibility, jointly administered with other validated questionnaires (PROMIS-29, Self-BAT, and the Menstrual Impact Questionnaire). Results: Review of existing PROMs in hemophilia showed emphasis on activity limitation due to joint disease limiting generalizability to BDUC. We identified 7 studies of QoL in vWD using disease-specific measures, however each study relied on ad-hoc questionnaires without validation procedure. While multiple validated questionnaires for menorrhagia exist, heavy menses is not a universal symptom in BDUC and may not drive QoL impact in an individual patient. Informed by literature review, an initial set of candidate items was developed including the following symptom domains: daily function, emotional health, sexual & reproductive health, and healthcare impact. Seven semi-structured interviews with BDUC patients were conducted, which included one male patient and one person of color. Iterative revisions throughout content validation procedure were systematically tracked. Reading level analysis of the PROM was consistent with 3rd-4th grade reading level. Thirty-two patients completed pilot testing in the clinic setting: 88% of patients found the items relevant to them and 84% of patients found the questions easily comprehensible. Conclusion: We developed and performed content validation of a novel PROM to study QoL in BDUC with further validation studies ongoing. Our work highlights the need for validated PROMs in the study of mucocutaneous bleeding disorders given limitations of existing tools.
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No part of this publication may be reproduced, distributed, or transmitted in any form or by any means, including photocopying, recording, or other electronic or mechanical methods, without the prior written permission of the author.