Collaborative Effort Powers Global Castleman Disease Patient Registry
Castleman Registry Surpasses One-year Goal of 100 Patients
Article ID: 690503
Released: 4-Mar-2018 4:05 PM EST
Source Newsroom: Perelman School of Medicine at the University of Pennsylvania
Newswise — PHILADELPHIA – The University of Pennsylvania’s Castleman Disease Research Program is collaborating with Pulse Infoframe Inc., a medical informatics company, to power its global ACCELERATE Natural History Registry platform, which is designed to help improve understanding of the rare disease, facilitate research and clinical studies, and identify effective therapies.
The international registry, which opened enrollment in late 2016, recently surpassed its first-year goal of 100 patients, with 120 Castleman Disease patients enrolled. Powered by Pulse’s system, the ACCELERATE Natural History Registry is enrolling patients from around the world. Medical record data are being reviewed, extracted, and analyzed to inform researchers who work on Castleman Disease.
“Registries are incredibly important for rare diseases because they help us collect data from around the world for in-depth analyses that can lead to insights that help patients,” said David Fajgenbaum, MD, MBA, MSc, a research assistant professor of Medicine in Penn’s Perelman School of Medicine, the principal investigator of Penn’s Castleman Program, and himself a Castleman disease patient. “With this unique registry, we hope to gain a greater understanding of the effectiveness of siltuximab, currently the only U.S. Food and Drug Administration-approved treatment for idiopathic multicentric Castleman disease, and also to identify new treatment approaches for patients without other options.”
Castleman disease is a rare inflammatory condition with three subtypes, which is diagnosed in about 5,000 people of all ages each year in the United States – approximately the same incidence as Lou Gehrig’s disease. About one third of patients with Fajgenbaum’s subtype, idiopathic multicentric Castleman disease, die within five years of diagnosis and another third die within 10 years of diagnosis.
The disease is so deadly because of how poorly understood it is by the medical community. Pulse Infoframe and the Castleman Program are working to change that. Pulse Infoframe creates online registries to support cancer and rare disease networks to unite research and patient communities.
The ACCELERATE registry marks one of two collaborations with Pulse. Last year, the Penn Orphan Disease Center announcedits collaboration with Pulse Infoframe to develop patient registries for a rare congenital epilepsy disorder called CDKL5 Deficiency and Crigler-Najjar syndrome, a familial disorder of the central nervous system.
“At Pulse we support the entire ecosystem that researchers, clinicians, and patients require, and we help with generating the real world evidence necessary to advance treatment using innovative clinical trial designs that ultimately facilitate treatment advances,” said Pulse’s Founder and CEO Femida Gwadry-Sridhar, RPh, PhD. Fajgenbaum is also the co-founder of the Castleman Disease Collaborative Network (CDCN), a global initiative dedicated to accelerating research and treatment by facilitating collaboration among the global research community, mobilizing resources, and strategically investing in high-impact research. In 2016, the CDCN, the University of Pennsylvania, and Janssen Pharmaceuticals joined together to establish the ACCELERATE Natural History Registry.
“There is no doubt that it takes collaboration to better understand and treat diseases,” Fajgenbaum said, “and we look forward to continuing to collect and analyze patient data with this registry to get us closer to that goal.”
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