The Penn Relapsing Polychondritis (RP) Program at the Penn Vasculitis Center provides comprehensive, multidisciplinary treatment for patients with relapsing polychondritis, and conducts clinical and translational research in this complex disease.
What is Relapsing Polychondritis?
Relapsing polychondritis is a rare, multisystem, multifactoral disease with an estimated incidence of 1- 3.5 per million per year with heterogeneous clinical manifestations and without any biomarker for diagnosis or monitoring.
The rarity of the disease, multiple clinical manifestations, heterogeneous presentations, and absence of diagnostic markers present various challenges in conducting research.
A Hub for Polychondritis Care at Penn Medicine
The Penn Relapsing Polychondritis Program evaluates, treats and actively follows the cases of nearly 100 patients with this rare disease — a number that continues to grow steadily.
Penn Medicine has emerged as a hub and destination for patients with relapsing polychondritis from around the nation. Half of the patients are local to the region and from neighboring states, and other patients travel from international locations to be seen by our multispecialty team.
Penn Medicine provides patients with highly experienced clinicians who regularly diagnosis and treat this condition. And Penn's ability to provide multispecialty care at a single location is critical for these patients, as the vast majority require multidisciplinary evaluation and comprehensive treatment.
The Vasculitis Clinical Research Consortium (VCRC) at Penn Medicine
The Penn Relapsing Polychondritis Program has benefited from funding from the National Institutes of Health (NIH) and partnership and support from the Relapsing Polychondritis Foundation and private donors.
The program uniquely integrates research with clinical care. The cornerstone of the program's research is the Vasculitis Clinical Research Consortium (VCRC) and The Relapsing Polychondritis (RP) Longitudinal Cohort study.
Founded in 2003, the Vasculitis Clinical Research Consortium is the largest international network dedicated to research in vasculitis. The VCRC conducts translational and clinical research studies in vasculitis, including observational cohort studies, biomarker development, studies of genetics and genomics, clinical outcomes research, pilot clinical projects, and multi-centered, randomized clinical trials.
Longitudinal Study of Relapsing Polychondritis
The VCRC Study is currently being conducted at Penn and the NIH with plans for future expansion. The VCRC-RP longitudinal observational cohort is a unique study which was founded with the aims of understanding the etiology of relapsing polychondritis, gaining an insight of the disease's natural history and stages, and learning what factors are associated with remission and/or active disease.
Under this study protocol, our clinical research team systematically collects data and biological samples from the patients enrolled in the study, while they are getting clinical care. At each study visit a comprehensive set of clinical data is collected and linked to biosamples.
To date, more than 150 patients have been enrolled in the VCRC Study, making it one of the world's largest clinical database and biobank resources for relapsing polychondritis. The study is an outstanding research asset that is already in use for novel projects, including the development of classification criteria, analyses of clinical outcomes to aid in the validation and development of outcome measures, various studies of new biomarkers, and genomic studies.
This cohort and these projects will be the foundation for a future clinical trial. Other current projects include an analysis of immunosuppressive medications used in relapsing polychondritis and a systematic literature review of immunomodulatory drugs in RP.
Clinical cohort studies such as the VCRC Longitudinal Study in Relapsing Polychondritis are essential for drug development by providing data to guide the choice of candidate patient subpopulations and treatment interventions for a Phase II trial.
We aim for our data to generate preliminary data essential when seeking funding support for larger projects from national funding agencies (NIH, PCORI) and industry partners.
The Future of the Penn Relapsing Polychondritis Program
Going forward, the Penn Relapsing Polychondritis Program has plans to conduct clinical trials evaluating the efficacy of new treatments for the disease based on the insights gained from its current projects.
And these exciting accomplishments are just the beginning. The potential is substantial for this work to improve the lives of patients with relapsing polychondritis and to make strides in our scientific understanding of this complex disease.