Analysis of relapse by inflammatory Rasch-built overall disability scale status in the PATH study of subcutaneous immunoglobulin in chronic inflammatory demyelinating polyneuropathy

I.S.J. Merkies*, I.N. van Schaik, V. Bril, H.P. Hartung, R.A. Lewis, G. Sobue, J.P. Lawo, O. Mielke, D.R. Cornblath, PATH Study Grp

*Corresponding author for this work

Research output: Contribution to journalArticleAcademicpeer-review

Abstract

Clinical trials in chronic inflammatory demyelinating polyneuropathy (CIDP) often assess efficacy using the ordinal Inflammatory Neuropathy Cause and Treatment (INCAT) disability score. Here, data from the PATH study was reanalyzed using change in Inflammatory Rasch-built Overall Disability Scale (I-RODS) to define CIDP relapse instead of INCAT. The PATH study comprised an intravenous immunoglobulin (IVIG) dependency period and an IVIG (IgPro10 [Privigen]) restabilization period; subjects were then randomized to weekly maintenance subcutaneous immunoglobulin (SCIG; IgPro20 [Hizentra]) 0.2 g/kg or 0.4 g/kg or placebo for 24 weeks. CIDP relapse was defined as >= 1-point deterioration in adjusted INCAT, with a primary endpoint of relapse or withdrawal rates. This retrospective exploratory analysis redefined relapse using I-RODS via three different cut-off methods: an individual variability method, fixed cut-off of >= 8-point deterioration on I-RODS centile score or >= 4-point deterioration on I-RODS raw score. Relapse or withdrawal rates were 47% for placebo, 34% for 0.2 g/kg IgPro20 and 19% for 0.4 g/kg IgPro20 using the raw score; 40%, 28% and 15%, respectively using the centile score, and 49%, 40% and 27%, respectively using the individual variability method. IgPro20 was shown to be efficacious as a maintenance therapy for CIDP when relapse was defined using I-RODS. A stable response pattern was shown for I-RODS across various applied cut-offs, which could be applied in future clinical trials.
Original languageEnglish
Pages (from-to)159-165
Number of pages7
JournalJournal of the Peripheral Nervous System
Volume27
Issue number2
Early online date15 Mar 2022
DOIs
Publication statusPublished - Jun 2022

Keywords

  • chronic inflammatory demyelinating polyneuropathy
  • CIDP
  • IgPro20
  • I-RODS
  • SCIG
  • subcutaneous immunoglobulin
  • INCAT
  • SCORE
  • RODS
  • SUM

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