First Ever Daily Quality Of Life Measurements Studied In Hemophilia Patients

Published Sep 17, 2012
Princeton, New Jersey – A new study reports for the first time daily quality of life (QoL) assessments in a chronic disease state. Results found that overall QoL of hemophilia inhibitor patients is often impaired, both on days when they experience a bleeding episode and days when they do not. While its not suprising that frequent joint bleeding episodes in patients with hemophilia result in acute pain and impaired QoL from childhood, the day-to-day experience of adolescents and adults with chronic joint damage highlights the burden of this disease. QoL in patients with hemophilia and arthropathy has typically been assessed on a one time or periodic basis with standard and/or disease specific questionnaires. This study was conducted to increase understanding about the impact of the disease on the daily QoL of patients and caregivers and to obtain additional information about the burden of living with hemophilia with inhibitors. For patients with hemophilia with antibodies (inhibitors) to factor VIII or IX replacement, bleeding episodes are more difficult to control or prevent, resulting in joint damage with repeated bleeds. A recent study, "Effect of Acute Bleeding on Daily Quality of Life (QOL) Assessments in Patients With Congenital Hemophilia With Inhibitors and Their Families: Observations From the Dosing Observational Study in Hemophilia (DOSE)", published in Value in Health, reviews the impact of hemophilia on the patient’s perceived health and pain, ability to complete routine activities, and family anxiety and stress. The study was co-authored by Drs. Ellis Neufeld, Michael Recht, Hernan Sabio, Kapil Saxena, Caitlyn Solem, Simon Pickard, Robert Gut, and David Cooper and sponsored by Novo Nordisk Inc. Across individual patient data, the percentage of patients with any problems with QOL was high on all days. QoL health assessment and pain scores were significantly worse on days with bleeding episodes, from days without bleeding episodes, with more family anxiety/stress and changes in family activities. Overall, few patients noted a perfect QoL (no pain) on days with or without a bleeding episode. “Changes in quality of life with progressive joint damage over decades have been well documented in patients with hemophilia and inhibitors. However, the day-to-day variability in pain and disablity in adolescents and adults has never been observed on this level before,“ says Dr. Neufeld, Associate Chief of the Division of Hematology/Oncology at Children's Hospital, Boston, and Professor of Pediatrics at Harvard Medical School. “This study shed light on the need for researchers to develop and validate new methods to assess this impact, which will allow us to compare outcomes as we try to develop treatment strategies that will be more beneficial for our patients.“ Congenital hemophilia results from a deficiency of clotting factor VIII or IX. The DOSE study enrolled 52 of the ~ 800 patients in the US with hemophilia complicated by antibodies (inhibitors) to factor VIII or IX treatment. The quality of life data is discussed in Value in Health, the official journal of the International Society of Pharmacoeconomics and Outcomes Research (ISPOR). The sponsor of this study, Novo Nordisk., provided editorial content for this press release.

Value in Health (ISSN 1098-3015) publishes papers, concepts, and ideas that advance the field of pharmacoeconomics and outcomes research as well as policy papers to help health care leaders make evidence-based decisions. The journal is published bi-monthly and has over 8,000 subscribers (clinicians, decision-makers, and researchers worldwide).

International Society for Pharmacoeconomics and Outcomes Research (ISPOR) is a nonprofit, international, educational and scientific organization that strives to increase the efficiency, effectiveness, and fairness of health care resource use to improve health. For more information:

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