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Principal Investigator: Ami Aalok Shah
Organization: JOHNS HOPKINS UNIVERSITY
Fiscal Year: 2024
Award: $169,411
Funding agency: National Institute of Arthritis and Musculoskeletal and Skin Diseases
PROJECT SUMMARY / ABSTRACT
This is a proposal for a K24 Midcareer Investigator Award in Patient-Oriented Research for Ami A. Shah, MD,
MHS of Johns Hopkins University School of Medicine. Dr. Shah is an Associate Professor of Medicine in the
Division of Rheumatology, Deputy Director for the Rheumatology Precision Medicine Centers of Excellence
clinical programs, and Co-Director of the Johns Hopkins Scleroderma Center. Dr. Shah has spent the majority
of her career and scholarship focused on patient-oriented research in systemic sclerosis (scleroderma) and in
investigating the relationship between cancer and autoimmunity. Scleroderma is a complex, multisystem
rheumatic disease that manifests very differently among patients with the same diagnosis. There is heterogeneity
in symptoms, trajectory of disease, timing of events, and response to therapy. While many risk factors have been
identified for specific scleroderma complications at the population level, these have not been easily translatable
to clinical practice at the patient level. This has been due to many factors including difficulty (i) capturing
multivariate patient-specific disease trajectories, (ii) modeling the complex interplay between organ system
parameters over time, and (iii) utilizing knowledge gained from trajectories of other patients who share
scleroderma subgroup characteristics. In this proposal, the applicant seeks to harness rich clinical data through
the Johns Hopkins precision medicine platform and develop novel computational methods to generate
personalized risk estimates of major clinical events in scleroderma. She will utilize an innovative strategy to apply
and test these new insights in a clinical setting. By embedding estimated trajectories and probabilities of major
events into a patient level data visualization tool that updates in “real-time,” she will test whether these new
discoveries can influence provider risk estimation and future diagnostic and therapeutic decision-making. Lastly,
she will utilize phenotypic trajectories as a platform to identify candidate biomarkers at baseline that associate
with long-term disease progression in scleroderma, as this may provide insight into patient subgroups who could
benefit from intensive screening and treatment strategies. These aims will serve as an outstanding vehicle for
career development and growth for Dr. Shah’s mentees, opening new fields of inquiry and developing novel
precision medicine approaches in scleroderma.
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