Chronic Granulomatous Disease Clinical Trial
Official title:
A Retrospective Analysis of Efficacy and Safety of Interleukin-1 Receptor Inhibitor for the Treatment of Granulomatous Complications in Patients With Chronic Granulomatous Disease
"Kineret" (INN: Anakinra) neutralizes the biological activity of interleukin-1α (IL-1α) and interleukin-1β (IL-1β) by the concurrent inhibition of binding to interleukin-1 receptor I (IL-1RI). Interleukin-1 (IL-1) is the main pro-inflammatory cytokine that mediates many cellular responses. Anakinra inhibits the reactions caused by IL-1 in vitro, including the induction of nitric oxide and prostaglandin E2 and / or the formation of collagenase by synovial cells, fibroblasts and chondrocytes. According to published data, patients with the chronic granulomatous disease have an increased secretion of interleukin-1, which contributes to the development of granulomatous inflammation. Blocking interleukin-1 reduces the activity of the main pro-inflammatory complex - the inflammasomes, and also restores the autophagy process impaired in patients with chronic granulomatous disease. In this way, inhibition of the IL-1 receptor prevents the activation of innate immunity cells and prevents the maintenance of pathological pro-inflammatory signaling in conditions of IL-1 overproduction. The efficacy and safety of therapy with the above drug is based on the results of international studies on the using of anakinra in patients with chronic granulomatous disease.
The research will include a group of patients with a molecular-genetic confirmed diagnosis of
chronic granulomatous disease, which has granulomatous complications on the basis of an
initial comprehensive survey.
This examination will include clinical data; laboratory tests - clinical and biochemical
analysis of blood (with an assessment of inflammatory activity); molecular-genetic methods
for detecting mutations in the genes CYBB, CYBA, NCF2, NCF1 or NCF4; methods for studying the
functional activity of neutrophils (chemiluminescence of neutrophils, test with rhodamine);
methods for assessing pro-inflammatory interleukins; microbiological testing of
bronchoalveolar lavage; histological studу of lung biopsies; as well as the results of
visualization techniques (ultrasound investigation of the abdominal organs, CT scan of the
chest and abdominal organs). Negative galactomannan and lack of microorganism growth in
bronchoalveolar lavage and/or lack of response to complex antibacterial and antifungal
therapy for two to three weeks confirm the presence of granulomatous complications in
patients. The next step is therapy with an inhibitor of IL-1 receptor (Anakinra). Evaluation
of the efficacy and safety therapy is based on the results of control examinations after 3-6
months from the start of treatment and includes an assessment of the level of C-reactive
protein and pro-inflammatory cytokines, as well as the results of CT scan of the chest and
abdominal organs.
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