Placebo effect in chronic inflammatory demyelinating polyneuropathy: The PATH study and a systematic review

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Lewis, RA
Cornblath, DR
Hartung, HP
Sobue, G
Lawo, JP
Mielke, O
Durn, BL
Bril, V
Merkies, ISJ
Bassett, P
Cleasby, A
van Schaik, IN
Sabet, A
et al.
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2020
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Abstract

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.

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Journal of the Peripheral Nervous System

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25

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3

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© 2020 CSL Behring. Journal of the Peripheral Nervous System published by Wiley Periodicals LLC on behalf of Peripheral Nerve Society. This is an open access article under the terms of the Creative Commons Attribution-NonCommercial License, which permits use, distribution and reproduction in any medium, provided the original work is properly cited and is not used for commercial purposes

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Neurosciences

CIDP

immunoglobulin

non-relapse

placebo

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Lewis, RA; Cornblath, DR; Hartung, HP; Sobue, G; Lawo, JP; Mielke, O; Durn, BL; Bril, V; Merkies, ISJ; Bassett, P; Cleasby, A; van Schaik, IN; Sabet, A; et al., Placebo effect in chronic inflammatory demyelinating polyneuropathy: The PATH study and a systematic review, Journal of the Peripheral Nervous System, 2020, 25 (3), pp. 230-237

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