Comparative Analysis of PFT’s and PROMIS Dyspnea in the INSPIRE IBM Trial


Clinical Trials

Poster Number: M236


Pola Gaid, BS, University of California, Irvine, Marie Wencel, BS, UCI Health, Namita Goyal, MD, UCI Health, Miriam Freimer, MD, Ohio State University, Olimpia Carbunar, MD, University of Miami Health Systems, Mazen Dimachkie, MD, University of Kansas Medical Center, Colin Quinn, MD, University of Pennsylvania, Thomas Lloyd, MD, PhD, Johns Hopkins University, Conrad Weihl, MD, PhD, Washington University in Saint Louis, Aziz Shaibani, MD, Nerve and Muscle Center of Texas, Tahseen Mozaffar, MD, UCI Health

OBJECTIVE: To investigate pulmonary function abnormalities in a large cohort of patients with inclusion body myositis (IBM) and to examine the relationship between pulmonary function test (PFT) results and PROMIS Dyspnea scores

Inclusion body myositis (IBM) is a rare muscle disorder causing progressive weakness primarily in skeletal muscles. Respiratory dysfunction is one of the leading causes for morbidity and mortality. Systematic investigations of pulmonary functions and their decline over time has not been studied in IBM. Furthermore, the correlation of PFTs to the symptoms of dyspnea reported by patients in the PROMIS Dyspnea questionnaire has not been studied. The PROMIS Dyspnea questionnaire specifically evaluates the severity and impact of shortness of breath or dyspnea experienced by individuals. Understanding correlations between PFT parameters and patient-reported dyspnea (PROMIS Dyspnea) can refine clinical interventions and may improve outcomes for IBM patients.

DESIGN/METHODS: This study will analyze PFT data, including sitting and supine FVC, MIP, and MEP, at baseline and over time in this 2-year natural history study in 150 IBM patients across 13 US sites. The relationship of these pulmonary functions to the PROMIS Dyspnea scores in 150 INSPIRE will be further examined. Correlations will be assessed using statistical analysis comparing PROMIS dyspnea scoring and PFT results. Regression analysis will be performed to identify if there’s a predictive relationship between PFT assessments and dyspnea symptoms.

RESULTS/CONCLUSION: Analysis will be done once enrollment closes in January 2024 at which time all patients will have completed their baseline visit. Results from the baseline visit will be analyzed and presented.