Monitoring Inflammation Biomarkers in Duchenne Muscular Dystrophy


Pre-Clinical Research

Poster Number: S11


Ahmed Naveed, Binghamton University, Chiara Degan, Leiden University, Roula Tsonaka, Leiden University Medical Center, Utkarsh Dang, PhD, Carleton University, Rebecca Tobin, Carleton University, ON, Canada, Cristina Al-Khalili, KTH Royal Institute, Albert Jimenéz Requena, KTH Royal Institute, Pietro Spitali, PhD, Leiden University Medical Center, Jordi Diaz-Manera, MD, PhD, John Walton Muscular Dystrophy Research Centre, Newcastle University, Newcastle-Upon-Tyne, UK, Michela Guglieri, Newcastle University and Newcastle Hospitals NHS Foundation Trust, Yuri van der Burgt, Leiden University Medical Center, VBP15-004 Investigators, CINRG, FOR-DMD Investigators, MD, University of Rochester

We have previously shown that some inflammation biomarkers including IL-6, CXCL-10 and CCL2 are elevated in blood and muscle of glucocorticoid naïve Duchenne muscular dystrophy (DMD) patients compared to age matched healthy controls. These inflammation biomarkers can be useful in assessing disease progression and responds to glucocorticoid treatment in DMD. In this study we used a validated ELISA assay to measure levels of inflammation biomarkers (e.g., CCL2, CCL3, CCL4, CCL13, CXCL10, Eotaxin, Eotaxin 3, TARC, MDC, and IL-8) in longitudinal serum samples collected from DMD patients enrolled in FOR-DMD clinical trials.

Serum samples from 12 patients (age range 4-9 years old) were collected at baseline, year 1, and year 2 following treatment with Prednisone or Deflazacort. Our aim was to investigate the association of the longitudinal trajectory of inflammation biomarkers and clinical outcomes (e.g., 6 min walk test, 10 m run/walk and time to stand from supine position) in this subset of patients using mixed linear model. Preliminary analysis showed that CXCL10 negatively correlated with the “reciprocal of time to rise from the floor” test after adjusting for age and corticosteroid treatment (p-value < 0.05). This suggests that CXCL10 might a potential biomarker candidate to monitor disease progression and treatment response in DMD. We are currently validating the data using larger samples size that includes more than 180 samples from 55 DMD patients receiving different glucocorticoids regimen.