24 May, 2017 Measurement Properties of the Rasch-built Overall Disability Scale (R-ODS) in patients with hATTR Amyloidosis with Polyneuropathy
We presented data evaluating the measurement properties of the Rasch-built Overall Disability Scale (R-ODS) in patients with symptomatic hereditary ATTR (hATTR) amyloidosis with polyneuropathy. R-ODS is a 24-item patient-reported outcome instrument that assesses activity and social participation limitation. R-ODS assessments were collected from two trials investigating patisiran. These data were presented at the International Society for Pharmacoeconomics and Outcomes Research annual meeting in Boston, Massachusetts.
The R-ODS was initially designed and validated to measure the activity and social participation limitations in patients with Guillain-Barré syndrome, chronic inflammatory demyelinating polyradiculoneuropathy, and gammopathy-related polyneuropathy. This study set out to generate evidence of the reliability and internal validity of the measurement properties of the R-ODS in patients with symptomatic hATTR amyloidosis with polyneuropathy.
R-ODS assessments were completed in 252 patients, 225 from the APOLLO trial and 27 from the phase 2 OLE trial. In total, 359 assessments were conducted, 225 from APOLLO at baseline and 134 from the OLE trial (27 patients at baseline and weeks 27, 54 and 81, and 26 at week 108).
hATTR amyloidosis is a multi-systemic disease with heterogeneous clinical presentation including sensory and motor, autonomic, and cardiac symptoms. Disability and loss of autonomy are major patient concerns. Based on the wide spectrum of activity and social participation limitations covered by R-ODS, we conclude the R-ODS is a reliable and valid measurement tool in patients with hATTR amyloidosis with polyneuropathy. We will continue research to understand longitudinal psychometric properties of the R-ODS, especially its ability to detect change in these limitations over time.