Seventy-nine patients were evaluable for safety, and data from 70 patients were used for efficacy analysis.
Analysis of the FDA Table of Pharmacogenomic Biomarkers in Drug Labels (96 drugs in total) demonstrated that 62% of the biomarkers are used to exclude patients or adjust dose based on safety-related concerns, and 31% are used for efficacy.[4,5].[4,5]
Descriptive statistics were used for efficacy and safety data.
WHO evaluation criteria were used for efficacy analysis (World Health Organization, 1979).
The intention-to-treat (ITT) population was used for efficacy evaluations, per prospective Statistical Plan.
Only data before rescue were used for efficacy and safety analyses, which may have also affected the results.
For patients who received rescue therapy, the last post-baseline value prior to initiation of rescue therapy was used for efficacy analyses.
Therefore, a total of 91 patients (group A, n=45 and group B, n=46) were included in the FAS that was used for efficacy and safety analyses.
Six weeks old female SCID Beige mice, weighing approximately 20 g, were purchased from Charles River Laboratories (Wilmington, MA) and were used for efficacy studies.
All patients randomized to treatment, except those with severe protocol violations, comprised the full analysis set, which was used for efficacy analyses.
