Chronic Granulomatous Disease (CGD)
Chronic granulomatous disease is an inherited disorder characterized by a defect in immune cells called phagocytes. The primary function of phagocytes is to recognize, engulf, and destroy microbial pathogens. In patients with CGD, phagocytes fail to generate sufficient levels of the enzymes responsible for killing ingested microorganisms. As a result, patients have an increased susceptibility to severe recurrent infections which often require hospitalization and aggressive therapy.
ACTIMMUNE® helps to reduce the frequency and severity of serious infections in patients with CGD. 1 Although the precise mechanism of action is not fully understood, ACTIMMUNE® appears to work by enhancing the immune response to pathogens. In clinical trials, patients treated with ACTIMMUNE® experienced significantly fewer serious infections and less frequent hospitalization compared to patients treated with placebo.2
Severe, Malignant Osteopetrosis
Severe, Malignant Osteopetrosis is an inherited disorder characterized by a defect in cells called osteoclasts. The primary role of osteoclasts is to support the maintenance of healthy bone by removing old bone cells in order to make room for the deposition of new bone cells. In patients with osteopetrosis, a defect in the osteoclasts prevents the efficient removal of old bone cells, which results in increased bone mass and reduced bone marrow function.
ACTIMMUNE® reduces bone volume and improves bone marrow function in patients with osteopetrosis by enhancing the function of defective osteoclasts.3 This restores the balance between resorption of old bone and deposition of new bone, which increases the size of the bone marrow cavity, creating more space for the production of immune cells. Clinical trials have demonstrated that treatment with ACTIMMUNE® significantly delays disease progression and decreases the risk of serious infections in patients with severe, malignant osteopetrosis.4,5
The health information contained herein is provided for educational purposes only and is not intended to replace discussions with a healthcare provider. All decisions regarding patient care must be made with a healthcare provider, considering the unique characteristics of patients.
For U.S. resident use only. The product discussed herein may have different product labeling in different countries. Please see the full U.S. Prescribing Information (pdf file).
References
1. Bemiller LS, Roberts DH, Starko KM, Curnutte JT. Safety and effectiveness of long-term interferon gamma therapy in patients with chronic granulomatous disease. Blood Cells Mol Dis. 1995;21:239-247.
2. The International Chronic Granulomatous Disease Cooperative Study Group. A controlled trial of interferon-gamma; to prevent infection in chronic granulomatous disease. N Engl J Med. 1991;324:509–516.
3. Beard CJ, Key L, Newburger PE, et al. Neutrophil defect associated with malignant infantile osteopetrosis. J Lab Clin Med. 1986;108:498-505.
4. Key LL, Ries WL, Rodriguiz RM, Hatcher HC. Recombinant human interferon gamma for osteopetrosis. J Pediatrics. 1992;121:119-124.
5. Key LL. A phase III randomized controlled open-label trial of Interferon-gamma-1b in patients with severe congenital (malignant) osteopetrosis. J Bone Min Res. 2004;19:1372
