Statistics.
WHIM
CNS INFECTIONS
Participant sample size of 20 was defined from retrospective frequency and severity data on infections in 11 patients with WHIM treated with G-CSF at the NIH-CC for 1 year (as detailed in the We used a noninferiority test to assess sustained ANC improvement because both drugs are known to increase the ANC, and we used a superiority test to assess sustained ALC improvement because plerixafor, but not G-CSF, is known to elevate the ALC. The effect parameter for ANC is the proportion of successes (at least 75% of measured ANC above 500 cells/μL) while on G-CSF minus the proportion of successes while on plerixafor, and we prespecified a noninferiority margin of 0.40, which was estimated to be about 50% of the size of the effect of G-CSF versus placebo. For quantifying CIs on differences in success proportions for maintaining the ANC and ALC above predetermined levels on drug, we used a method that accounts for the pairing due to the crossover design and is valid for small samples (