A controlled trial of interferon gamma to prevent infection in chronic granulomatous disease

Article Abstract:

Chronic granulomatous disease is the name given to several rare, inherited disorders characterized by serious, recurrent pus-forming infections. The disease begins in early childhood and may cause death early in life. People with chronic granulomatous disease have defective phagocytes (cells that ingest and kill bacteria and other foreign agents) that are able to ingest microorganisms, but cannot destroy them. This failure results from an abnormal form of an enzyme, NADPH oxidase, that is essential for the phagocyte to complete its task. Since interferon gamma, a cytokine (factor produced by cells when they are stimulated), activates macrophages (phagocytic white blood cells found in organs and tissues), it is possible that it could help patients with chronic granulomatous disease fight infection. This was tested in a randomized, controlled study in 13 medical centers in four countries. One hundred twenty-eight patients received either interferon gamma (63 patients) or a placebo injection (63 patients) three times weekly for varying time periods (average, 8.9 months). They were observed to determine the amount of time from study onset to development of a serious infection (requiring hospitalization and antibiotics), as well as the number and severity of such infections. In addition, tests were performed to evaluate the ability of the patients' blood cells to kill bacteria; other aspects of cell function were also noted. Results showed that 46 percent of the placebo group developed at least one serious infection, compared with 22 percent in the interferon gamma group. More patients in the interferon gamma group (77 percent) than the placebo group (30 percent) were infection-free after one year, indicating a lasting effect. Children younger than 10 received the greatest benefit; 81 percent were infection-free after 12 months of treatment. The side effects were minimal and consisted of fever, chills and headache. Blood cells from the two groups of patients were not shown to have different bacterial killing power or other properties. Interferon gamma seems to be an effective therapy for chronic granulomatous disease, and is recommended for prophylactic use in patients with this condition. (Consumer Summary produced by Reliance Medical Information, Inc.)

Health aspects, Physiological aspects, Interferon gamma

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High-dose chemotherapy with hematopoietic stem-cell rescue for multiple myeloma

Article Abstract:

High-dose chemotherapy followed by a hematopoietic stem cell transplant may be an effective treatment for a type of cancer called multiple myeloma, according to a study of 401 patients. Forty-four percent of the patients who received this treatment had a complete response, compared to 8% of those who received standard chemotherapy with no transplant. Hematopoietic stem cells are used to help the patient's immune system recover from chemotherapy.

Evaluation, Dosage and administration, Chemotherapy, Multiple myeloma

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What is the best way to treat inherited disorders?

Article Abstract:

Many inherited diseases may be cured in the future by giving patients a stem cell transplant that carries a copy of the normal gene. A study published in 2001 showed that stem cell transplants were effective in some patients with an inherited immune deficiency called chronic granulomatous disease.

Editorial

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