Placebo effect in chronic inflammatory demyelinating polyneuropathy: The PATH study and a systematic review
Document Type
Article
Abstract
© 2020 CSL Behring. Journal of the Peripheral Nervous System published by Wiley Periodicals LLC on behalf of Peripheral Nerve Society. The Polyneuropathy And Treatment with Hizentra (PATH) study required subjects with chronic inflammatory demyelinating polyneuropathy (CIDP) to show dependency on immunoglobulin G (IgG) and then be restabilized on IgG before being randomized to placebo or one of two doses of subcutaneous immunoglobulin (SCIG). Nineteen of the 51 subjects (37%) randomized to placebo did not relapse over the next 24 weeks. This article explores the reasons for this effect. A post-hoc analysis of the PATH placebo group was undertaken. A literature search identified other placebo-controlled CIDP trials for review and comparison. In PATH, subjects randomized to placebo who did not relapse were significantly older, had more severe disease, and took longer to deteriorate in the IgG dependency period compared with those who relapsed. Published trials in CIDP, whose primary endpoint was stability or deterioration, had a mean non-deterioration (placebo effect) of 43%, while trials with a primary endpoint of improvement had a placebo response of only 11%. Placebo is an important variable in the design of CIDP trials. Trials designed to show clinical improvement will have a significantly lower effect of this phenomenon than those designed to show stability or deterioration.
Publication Date
9-1-2020
Publication Title
Journal of the Peripheral Nervous System
ISSN
10859489
E-ISSN
15298027
Volume
25
Issue
3
First Page
230
Last Page
237
PubMed ID
32627277
Digital Object Identifier (DOI)
10.1111/jns.12402
Recommended Citation
Lewis, Richard A.; Cornblath, David R.; Hartung, Hans Peter; Sobue, Gens; Lawo, John Philip; Mielke, Orell; Durn, Billie L.; Bril, Vera; Merkies, Ingemar S.J.; Bassett, Paul; Cleasby, Alexa; van Schaik, Ivo N.; Sabet, A.; George, K.; Roberts, L.; Carne, R.; Blum, S.; Henderson, R.; Van Damme, P.; Demeestere, J.; Larue, S.; D’Amour, C.; Kunc, P.; Valis, M.; Sussova, J.; Kalous, T.; Talab, R.; Bednar, M.; Toomsoo, T.; Rubanovits, I.; and Gross-Paju, K., "Placebo effect in chronic inflammatory demyelinating polyneuropathy: The PATH study and a systematic review" (2020). Neurology. 484.
https://scholar.barrowneuro.org/neurology/484