Assessments and end points.
infection, wart, infections
WART, ADVERSE EVENTS, INFECTIONS, INFECTION, SECONDARY, WARTS, REGRESSION, BLOOD, INFECTION TOE
The prespecified primary efficacy endpoint was the difference between the two 12-month treatment periods for the TISS, a weighted composite of predefined infection frequency and severity inputs defined for this study. Records of each medical encounter for infection were collected from the treating health care provider and scored by the study team according to the predefined point system shown in The points for each parameter in each column were added for each infection to produce an infection severity score, which could range from 1 to 10. For each patient, infection severity scores for all infections occurring during a treatment phase were added to generate a TISS for that phase that was then compared with the TISS for the second treatment phase. Additional details are provided in the Ordered secondary endpoints included sustained ANC and ALC improvement; infection incidence; antibiotic treatment duration; wart regression; and quality of life based on the 36-Item Short Form Survey, version 2 instrument (Clinical photography and dermatologist assessments of warts were obtained at baseline on day 0 of the equilibration phase as well as at the month 0, 4, 8 and 12 visits during the treatment phase. Two masked dermatologists scored the percentage change in baseline wart area at each of the 4 visits during each treatment phase. A complete response was defined as visible absence at the month 12 visit of a wart area defined at the baseline visit. Because carryover effects on warts were possible during drug crossover, the prespecified statistical analysis plan compared improvement only for the first treatment phase. HPV identification was as previously reported (Blood for quantitative immunoglobulin and lymphocyte subset assessments was obtained at the NIH-CC before the first study drug dose on day 0 of each equilibration phase and approximately 3 hours after the morning dose at the month 0, 4, 8, and 12 visits of each treatment phase. Immunophenotyping was performed by the Department of Clinical Immunology at the NIH-CC using freshly isolated PBMCs. Cells were washed with PBS, fixed with 1.0% formaldehyde, resuspended in PBS, and then stained for cell surface expression of CD45, CD3, CD4, CD8, CD14, CD56, CD45RA, CD62L, and CD19 using directly labeled monoclonal antibodies (We report all adverse events for all study phases.