Measuring Patient Reported Outcomes in Hemophilia

Pain Functional Impairment and Quality of Life (P-FIQ): An Assessment of Reliability of Five Patient Reported Outcome (PRO) Instruments in Adult People with Hemophilia

ISPOR 2015 (Philadelphia, May 16-20) A recently completed study of 381 adult patients with hemophilia of any severity with a history of joint pain or bleeding examined the reproducibility and reliability of 5 different patient reported outcome measures (surveys) in patients with congenital hemophilia. Data presented at the International Society of Pharmacoeconomics and Outcomes Research (ISPOR) 2015 Meeting reported on one hundred and sixty four patients completed these surveys at the beginning and at the end of a comprehensive care visit in a similar non-bleeding state with all 164 of them completing the surveys twice. The median age of the patients included in the study was 33.9 years. Majority of the patients had hemophilia A (74.4%) and were white-non-Hispanic (72.6%). In addition, 48.7% of these patients were married, 62.6% had some college or graduate-level education, 80.7% were employed and 61.0% self-reported arthritis/bone/joint problems. The average percentage of pre- and post-test item responses that were exactly the same for the associated outcome measures were reported as follows: EQ-5D-5L (measure of health outcomes): 79.1%; Brief Pain Inventory (measure of pain severity and interference): 58.9%; SF-36V2 Health Survey (measure of quality of life): 76.4%; International Physical Activity Questionnaire (measure of activity in the last week): 100% and Hemophilia Activities list (measure of activities in the last month): 75.9%.


The PFIQ Study (NCT01988532) was sponsored by Novo Nordisk Inc.