A Review of Patient-Reported Outcomes in Neuromuscular Diseases: What's Available and What's Missing
What is it about?
This review examined how patients with neuromuscular diseases (NMDs) report their health and quality of life using patient surveys called Patient-Reported Outcome Measures (PROMs). PROMs are questionnaires that help patients describe their symptoms, daily functioning, and well-being without a doctor's input. This scoping review aimed to compile all the available PROMs used for adults and children with NMDs. The researchers examined 247 studies and identified 190 different PROMs. They found that most PROMs focused on specific diseases rather than being general. The physical functioning aspect was the most commonly assessed area. However, many of these surveys only partially captured all the essential aspects of patients' experiences, like social participation or the impact of treatment. The study highlighted the need for more research on specific areas, especially the ability of PROMs to measure changes in health over time and interpret results meaningfully. The findings will help healthcare professionals choose the best tools to monitor patient health and involve patients in treatment decisions.
Why is it important?
This review is unique and important because it carefully examines patient surveys that help people with neuromuscular diseases (NMDs) share their health experiences. By looking at 190 different surveys, it reveals gaps in how well these surveys measure important aspects like social participation and the impact of treatment. This insight helps healthcare professionals understand their patients' needs better and choose the most accurate surveys for better, more personalized care. It ensures that patient voices are heard clearly so that treatment decisions can be tailored to improve their quality of life.