Cure SMA-funded researcher, Chad Heatwole, and his team, have published a paper, “What Matters Most: A Perspective From Adult Spinal Muscular Atrophy Patients”.
This project was a pilot study funded by a grant from Cure SMA, and focused on developing a reliable method for adults with SMA to report on clinical trail outcomes that are important to them.
Dr. Heatwole and his team interviewed 15 adults with SMA, aged 18-59, asking open-ended questions about the impact of SMA on their daily lives. Their responses were then grouped into four primary categories: physical health, mental health, social health and other disease-related issues.
The results were then analyzed to deteremine which symptoms were experienced most frequently, which were most meaningful to the individuals interviewed and which could be most amenable to treatment intervention. Specifically, many participants cited limited mobility, decreased independence associated with activities of daily living – such as difficulty dressing or difficulty with eating and nutrition – as well as the emotional and social impact of SMA.
This pilot study was then used to develop a survey of 350 adult SMA patients. The results of that larger survey are being used to develop a patient-reported outcome measure (often referred to as a PROM) for adults with SMA. We expect this work to be published in early 2017, and we hope it will facilitate the ability to conduct clinical trials in adults with SMA.
This work may also be used by researchers in several other ways. Researchers plan to match up these self-reported measures against formal testing of mobility and strength, looking for connections between the two. Researchers may also evaluate how advances in care, such as physical and occupational therapy, and advances in equipment may work alongside new treatments in development to produce the most meaningful impact for adults with SMA.
Our thanks to Dr. Heatwole and his team for their great work on this important topic.
The grant to Dr. Heatwole is supported by funding from The Spinal Muscular Atrophy Research Team (SMART), Buffalo, NY.