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. Our efforts to accrue subjects with CGD (currently n=3) in order to examine the development and progression of inflammation in vivo in this patient population have been slow. This year, we collaborated with the Human Immunologic Diseases Unit (LHD) to examine inflammation in a patient with a chemotactic defect. 2) Interferon-γ prophyaxis 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.
Aimi ng toward differentiating patients who may respond to IFNG from those who may not, we initiated a clinical protocol (#10-I-0123 Assessment of the Biochemical Response to IFN-gamma in Subjects with Specific Gene Mutations in Chronic Granulomatous Disease) in 2010. Due to the low level of patient interest in this time-intensive protocol, we have terminated this protocol (effective December, 2014) and are currently evaluating other approaches to address this important question
