Measures to identify unmet medical needs of children and adults with autoimmune and autoinflammatory diseases

 

Patients with autoimmune and autoinflammatory diseases face tough challenges in terms of their physical, mental, and social health, and they have many medical needs that are not being fully addressed. To help these patients, it's important to clearly understand what these unmet needs are. One way to gather this information is through Patient-Reported Outcomes (PROs), which are reports from patients about their own health issues. These reports are very useful in understanding what patients truly need.

This article looks at established ways to measure PROs to determine which are best for identifying the unmet needs of patients with autoimmune and autoinflammatory diseases. Experts from the Ministry of Health, Labor, and Welfare met to discuss these measures and also considered the possibility of using technology to collect PRO data electronically.

The experts suggested 28 potential measures, both specific to certain diseases and general ones. They emphasized the importance of making sure the measures are clearly understood in different languages and noted that the results from adults and children should not be mixed. They also discussed the challenges of moving from paper-based PRO measures to digital ones, mainly because of concerns about accuracy and a lack of technical and financial resources.

For non-disease-specific PRO measures, the experts recommended KINDL® and EQ-5D-Y for children, and SF-36v2® and EQ-5D™ for adults.

Here’s a quick breakdown of what these terms refer to:

1. KINDL®: This is a questionnaire specifically designed to measure the quality of life in children. It looks at various aspects of a child's well-being, like their physical health, emotional state, and social life.

2. EQ-5D-Y: This is a version of the EQ-5D, which is used to assess the general health of children. The "Y" stands for "Youth," so it's tailored for younger patients. It focuses on five areas: mobility, self-care, usual activities, pain/discomfort, and anxiety/depression.

3. SF-36v2®: This is a survey used to measure health-related quality of life in adults. It covers a wide range of health aspects like physical functioning, pain, mental health, and social well-being. The "v2" indicates it's the second version, with updates for better accuracy and clarity.

4. EQ-5D™: This is a general health measure used for both adults and children. Like the EQ-5D-Y, it looks at five key areas (mobility, self-care, usual activities, pain/discomfort, and anxiety/depression) but is meant for a broader age group.

These tools help doctors and researchers understand how diseases and treatments affect a person's day-to-day life and well-being.

READ ORIGINAL JOURNAL REPORT: Consideration of useful patient-reported outcome measures to identify unmet medical needs of children and adults with autoimmune and autoinflammatory diseases published in Modern Rheumatology April 2025.