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ACCELERATE: A Patient-Powered Natural History Study Design Enabling Clinical and Therapeutic Discoveries in a Rare Disorder

2020; Elsevier BV; Volume: 1; Issue: 9 Linguagem: Inglês

10.1016/j.xcrm.2020.100158

2666-3791

Sheila K. Pierson, Johnson S. Khor, Jasira Ziglar, Amy Liu, Katherine Floess, Erin Napier, Alexander Gorzewski, Mark-Avery Tamakloe, Victoria Powers, Faizaan Akhter, Eric Haljasmaa, Raj Jayanthan, Arthur H. Rubenstein, Mileva Repasky, Kojo S.J. Elenitoba‐Johnson, Jason R. Ruth, Bette Jacobs, Matthew Streetly, Linus Angenendt, J.L. Patier de la Peña, Simone Ferrero, Pier Luigi Zinzani, Louis Terriou, Corey Casper, Elaine S. Jaffe, Christian Hoffmann, Éric Oksenhendler, Alexander Fosså, Gordan Srkalović, Amy Chadburn, Thomas S. Uldrick, Megan S. Lim, Frits van Rhee, David C. Fajgenbaum,

Chronic Lymphocytic Leukemia Research

Geographically dispersed patients, inconsistent treatment tracking, and limited infrastructure slow research for many orphan diseases. We assess the feasibility of a patient-powered study design to overcome these challenges for Castleman disease, a rare hematologic disorder. Here, we report initial results from the ACCELERATE natural history registry. ACCELERATE includes a traditional physician-reported arm and a patient-powered arm, which enables patients to directly contribute medical data and biospecimens. This study design enables successful enrollment, with the 5-year minimum enrollment goal being met in 2 years. A median of 683 clinical, laboratory, and imaging data elements are captured per patient in the patient-powered arm compared with 37 in the physician-reported arm. These data reveal subgrouping characteristics, identify off-label treatments, support treatment guidelines, and are used in 17 clinical and translational studies. This feasibility study demonstrates that the direct-to-patient design is effective for collecting natural history data and biospecimens, tracking therapies, and providing critical research infrastructure.