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

Cell Rep Med. 2020 Dec 22;1(9):100158. doi: 10.1016/j.xcrm.2020.100158.


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.

Trial registration: NCT02817997.

Keywords: Castleman disease; direct-to-patient; natural history registry; orphan disease; patient-powered.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adolescent
  • Adult
  • Aged
  • Aged, 80 and over
  • Castleman Disease / diagnosis
  • Castleman Disease / therapy
  • Child
  • Child, Preschool
  • Data Collection* / standards
  • Female
  • Humans
  • Infant
  • Male
  • Middle Aged
  • Rare Diseases / diagnosis
  • Rare Diseases / therapy*
  • Registries / statistics & numerical data*
  • Research Design* / standards
  • Young Adult

Associated data