1) The blister protocol is being used to evaluate inflammation in vivo in several populations of patients with rare immunodeficiencies including STAT3 deficiency, CGD, and others. We continue to accrue subjects with CGD (currently n=4) in order to examine the development and progression of inflammation in vivo in this patient population. 2) Interferon-γprophyaxis is an FDA-approved therapy for CGD however it causes significant flu-like symptoms in some patients and anecdotal reports suggest that it might not benefit all patients. During FY14, we completed microarray evaluation of multiple samples from 2 subjects who completed our NIH Protocol #10-I-0123 Assessment of the Biochemical Response to IFN-gamma in Subjects with Specific Gene Mutations in Chronic Granulomatous Disease. Patients received several doses over the study duration and mRNA and protein expression was determined at each dose and during washout phases. Expression analysis identified a treatment-related signature that will be used to develop a new clinical protocol over the next FY.
