Subcutaneous immunoglobulin for maintenance treatment in chronic inflammatory demyelinating polyneuropathy (The PATH Study): study protocol for a randomized controlled trial

van Schaik, Ivo N.; van Geloven, Nan; Bril, Vera; Hartung, Hans-Peter; Lewis, Richard A.; Sobue, Gen; Lawo, John-Philip; Mielke, Orell; Cornblath, David R.; Merkies, Ingemar S. J.

doi:10.1186/s13063-016-1466-2

Trials

Table 2 Analyses performed at each study period for secondary outcomes, IgG levels, and health-related quality of life variables

From: Subcutaneous immunoglobulin for maintenance treatment in chronic inflammatory demyelinating polyneuropathy (The PATH Study): study protocol for a randomized controlled trial

	Period (analysis set)
	IVIg restabilization (PSDS)	SC treatment (ITTS, PPS)	IVIg rescue (RSDS)
INCAT
Total	Overall: - Descriptive statistics - Change from reference visit - Time to first improvement on IgPro10: Kaplan-Meier estimates	- By treatment and by treatment and subgroup: - Descriptive statistics - Comparison between treatments of changes from reference visit: exact Jonckheere-Terpstra test^a - Comparisons between each IgPro20 dose group and placebo group, and the comparison between the two IgPro20 dose groups of changes from reference visit: Wilcoxon rank sum test^b - Time to CIDP relapse or withdrawal for any other reason: Kaplan-Meier estimates^c	Overall: - Descriptive statistics - Change from reference visit - Time to improvement after CIDP relapse with IgPro10 rescue: Kaplan-Meier estimates
Mean grip strength (Dominant/non-dominant hand), MRC sum score, I-RODS
	Overall: - Descriptive statistics - Change from reference visit - Time to first improvement on IgPro10: Kaplan-Meier estimates	By treatment and by treatment and subgroup: - Descriptive statistics - Comparison between treatments of changes from reference visit; exact Jonckheere-Terpstra test^a - Comparisons between each IgPro20 dose group and placebo group, and the comparison between the two IgPro20 dose groups of changes from reference visit; Wilcoxon rank sum test^b	Overall: - Descriptive statistics - Change from reference visit
Electrophysiological parameters (average distal latency, average proximal latency, overall average conduction velocity, average conduction block, and average compound muscle action potential amplitude)
		By treatment and by treatment and subgroup: - Descriptive statistics - Change from reference visit - Between-treatment comparisons of the changes from baseline: analysis of variance (ANOVA) model^d
IgG level
	Overall: - Descriptive statistics - Change from reference visit	By treatment and by treatment and subgroup: - Descriptive statistics - Change from reference visit	Overall: - Descriptive statistics - Change from reference visit
EQ-5D dimensions, TSQM, WPAI-GH
	Overall: - Descriptive statistics	By treatment: - Descriptive statistics - Change from reference visit - Comparison between treatments of changes from reference visit; exact Jonckheere-Terpstra test^a - Comparisons between each IgPro20 dose group and placebo group, and the comparison between the two IgPro20 dose groups of changes from reference visit; Wilcoxon rank sum test^b	Overall: - Descriptive statistics
Patient preference for treatment questionnaire
		By treatment: - Descriptive statistics

EQ-5D EuroQoL 5-Dimension Questionnaire, IgG immunoglobulin G, ITTS intention-to-treat set, IVIg intravenous immunoglobulin, MRC Medical Research Council, PSDS pre-randomization safety data set, I-RODS Inflammatory-Rasch-built Overall Disability Scale, RSDS, rescue safety data set, SC subcutaneous, TSQM Treatment Satisfaction Questionnaire for Medication, WPAI-GH Work Productivity and Activity Impairment Questionnaire for General Health
^aExact Jonckheere-Terpstra test see ref [54]
^bFor each pair-wise comparison, the one-sided p value from the Wilcoxon rank sum test and the Hodges-Lehmann estimate of the median difference between treatments will be presented together with the corresponding two-sided 95 % Moses confidence interval
^cOverall between-treatment comparison will be performed using the log-rank test for trend, all pair-wise comparisons will be performed using the log-rank test
^dTreatment and region (Japan, non-Japan) will be used as explanatory variables. Within the framework of this ANOVA model, comparisons of each IgPro20 dose with placebo, and the comparison of the two IgPro20 doses will be performed. For these comparisons, the least squares mean for each treatment group, an estimate of the difference between treatments (if applicable), corresponding 95 % confidence interval and 2-sided p-values will be presented

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ISSN: 1745-6215

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