Hemophilia Review Highlights Improved Patient-reported Outcome Measures

With the safety and effectiveness of treatment options available for patients with hemophilia significantly improving over the years, it has become more and more important to develop new and improved outcome measures that can capture clinically meaningful changes which a treatment can generate.

In an article titled “Recognizing the need for personalization of hemophilia patient-reported outcomes in the prophylaxis era,” published in the scientific journal Hemophilia, researchers reviewed two new tools that are used to measure patient-reported outcome measures.

The first one, called patient-reported outcomes measurement information system (PROMIS), which was launched in 2004 by the U.S. National Institute of Health (NIH), uses modern measurement theory to develop new and improved patient reported outcome measures. According to the authors of the review, PROMIS optimizes the value of the questionnaire-based approach common to most patient reported outcomes and improves the psychometric assessment of quality of life through more sophisticated items.

The second tool, called goal attainment scaling (GAS), uses a fundamentally different approach based on goals that are selected by patients from a condition-specific menu of options specific to their own condition, and measures the extent to which these goals are attained following treatment.

“We believe that this approach most directly addresses the need for personalization of outcome measurement in hemophilia,” the first author of the review, Dr. Michael Recht of The Hemophilia Center at the Oregon Health & Science University in Portland, Oregon, and his colleagues wrote.

The authors suggest that adopting this, or similar patient-centric outcome measures, can help researchers and clinicians address the challenges of measuring clinically meaningful improvements that different types of treatments can provide hemophilia patients.

Outcome measures that are currently used to measure the effectiveness of available hemophilia therapies such as annualized bleeding rate, joint health scores, and validated quality of life measures were designed to measure common endpoints for a large cohort of patients and do not constitute a personalized approach.

The two approaches that were covered in this review may increase the ability of outcome measures to reliably detect and quantify the effectiveness of available therapies in hemophilia patients on a more tailored basis.