Personalizing Patient-Reported Outcomes for Hemophilia
Source: Hemophilia News Today, September 12, 2016
In a recent paper, a group of experienced hematologists reviewed two tools used for assessing patient-reported outcomes (PROs). While standard modes of outcome measurement, such as annualized bleeding rate and joint health score, are relatively effective in evaluating larger groups of bleeding disorders patients, they are not designed to capture the unique healthcare needs, perspectives and goals of each individual. The need for a personalized approach for people with hemophilia is even greater in light of dramatic changes in the clinical landscape over the last two decades, including the proliferation of progressively safer and more effective therapies, and the availability of preventive treatment regimens like prophylaxis.
The article, “Recognizing the Need for Personalization of Haemophilia Patient-Reported Outcomes in the Prophylaxis Era,” was published August 31, 2016, in the journal Haemophilia. The lead author of the study was Michael Recht, MD, a pediatric hematologist-oncologist at the Hemophilia Center at the Oregon Health & Science University in Portland. Recht and his colleagues assessed a pair of tools: the patient-reported outcomes measurement information system (PROMIS®) and the goal attainment scaling (GAS).
First launched in 2004 by the US National Institutes of Health (NIH), PROMIS takes the traditional questionnaire-based approach and employs modern measurement theory to develop new and enhanced PRO tools. The system improves on the traditional PRO techniques in part through the use of a psychometric method, which measures knowledge, abilities, attitudes and personality traits among individuals to produce scores associated with probable answers to questions.
These scores can then be used by computerized adaptive testing (CAT) to pinpoint the most informative follow-up question to an initial question from a predetermined bank of questions. According to investigators, PROMIS allows clinicians and researchers access to “efficient, precise, valid and responsive adult- and child-reported measures” in the physical, mental and social areas. These can then be applied across a broad range of chronic conditions, including hemophilia.
GAS involves a truly collaborative approach whereby a patient works with his or her physician to select important personal goals that are linked to the clinical or functional impacts of the patient’s condition. An assessment of actual goal attainment is made after a predetermined amount of time. According to the authors, GAS has been applied successfully in both clinical practice and research in patients with a broad range of other chronic conditions, including dementia, diabetes, acquired brain injury and various types of physical disabilities. One primary advantage of GAS is that it allows for the discovery of seemingly minor, though clinically and practically significant, changes, with real-world implications for a unique individual’s quality of life.
“We believe that this approach most directly addresses the need for personalization of outcome measurement in hemophilia,” concluded the authors. “Adoption of this or a similarly qualified patient-centric outcome measure will provide clinicians and researchers with an important innovation that addresses the contemporary challenge of measuring incremental but clinically meaningful improvement in hemophilia patients.”