Regulatory T cells suppress novel skeletal muscle macrophages identified by single-cell RNA sequencing


The Future of Neuromuscular care

Poster Number: 201


Laurey Brown, PT, DPT, PCS, Katie Hoffman, MPT, Kristin Kroschell, Christa Weigel, PT, DPT, Nancy Kuntz, MD, PhD, Vamshi Rao, MBBS, MD


1. Ann & Robert H Lurie Children's Hospital of Chicago, 2. Ann & Robert H. Lurie Children's Hospital of Chicago, 3. , 4. Ann & Robert H Lurie Children's Hospital of Chicago, 5. Lurie Children's Hospital - Chicago, 6. Lurie Childrens Hospital

Sprinaza is the first commercial drug treatment to be approved for treatment of Spinal Muscular Atrophy (SMA). In sites where Spinrza is administered, physical therapists perform standardized assessments to track drug efficacy and to advocate for lifelong coverage and delivery. As more patients are treated and sustainability of long-term drug delivery is considered, it is essential to monitor quality care delivery and impart strategies that assure equity and standardization. We offer our experience at one such site as a template.

The knowledge to perform necessary standardized measures is not provided in graduate level education and advanced training by an expert clinician is required. Using an annual training and refresher model, therapists were initially trained on primary outcome measures and tested to ensure inter-rater reliability. The refresher program reviewed and re-established reliability with previously trained therapists. This model demonstrates consistent reliability within the established standard deviation and reassures patients, parents and third-party payors that testing results are accurate.

To effectively meet the needs of parents and patients and assist in ease of caregiver burden, assessments were offered at multiple locations. This helped in modifying barriers to receiving care with currently 4 trained therapists at 3 different locations to serve approximately 50 patients and families.

Imparting quality improvement required analysis of practice performance. Monthly meetings were held to review the program’s current state and address emerging concerns.

Quality assurance in the delivery of standardized assessments is essential to tracking patient outcomes. Evaluating therapists should undergo structured training with peer-reviewed monitoring, provide multiple locations of therapy delivery and establish reliability and that is maintained over time.

We gratefully acknowledge the patients, families and providers participating in our Spinraza program.