Treatment
With the appropriate treatment, the outlook is good for people with Wegener's granulomatosis. In a study of 158 patients who were treated with prednisone and cyclophosphamide at the National Institutes of Health (NIH), 91 percent markedly improved. After 6 months to 24 years of follow-up, 80 percent survived.
In most cases, treatment consists of a combination of a glucocorticoid (a steroid) and a cytotoxic medicine. Although these medicines are helpful in treating Wegener's granulomatosis, people and their healthcare professionals should be aware of the potentially serious side effects. In many instances healthcare professionals and patients can minimize or prevent side effects by careful monitoring.
Approximately half of people with Wegener's granulomatosis may experience a return of their disease. This occurs most frequently within 2 years of stopping medicine but can occur at any point both during treatment or after stopping treatment. Thus, it is extremely important that people continue to see their healthcare professionals regularly, both while they are on these medicines as well as after they have stopped taking the medicines.
Prednisone
Prednisone is the most common glucocorticoid that healthcare professionals use. Prednisone is similar to cortisol, the natural glucocorticoid hormone produced by the body. However, it is chemically different from the anabolic steroids that have been used by athletes and is given in doses much higher than the body normally produces. Healthcare professionals usually give prednisone as a single morning dose to try to imitate how the body normally secretes cortisol.
When a person's illness improves, the prednisone dose is gradually decreased and converted to an every-other-day dosing schedule, usually over a period of 3 to 4 months. With further improvement prednisone is gradually decreased and discontinued completely after approximately 6 to 12 months.
When a person takes prednisone by mouth, the body stops making its own natural cortisol. As the prednisone dose is gradually reduced, the body will resume making cortisol. It is extremely important that prednisone never be stopped suddenly because the body needs prednisone (or cortisol) to function and may not be able to immediately make what enough.
Prednisone can affect the body's ability to fight off infection. People taking this medicine should report immediately any symptoms of infection and specifically, any fever to their doctors. Prednisone also can cause weight gain, cataracts, brittle bones, diabetes, and changes in mood and personality.
Cyclophosphamide
Cyclophosphamide (Cytoxan) is the cytotoxic medicine most commonly used to treat Wegener's granulomatosis. People take cyclophosphamide once a day by mouth and must take the medicine all at once in the morning followed by drinking a large amount of liquid. Although the first dose of cyclophosphamide is based on the person's weight and kidney function, the healthcare professional may adjust the dosage based on blood counts, which are monitored closely to be sure the white blood cell count is kept at a safe level.
In the original regimen, cyclophosphamide was continued for a full year beyond that point at which the disease was in remission. The dose of cyclophosphamide was then decreased gradually and eventually stopped. In more recent treatment approaches, however, cyclophosphamide is either not used at all or given only to patients with severe disease manifestations. When cyclophosphamide is used, it is only administered until remission is achieved and then switched to another, less toxic medication such as methotrexate or azathioprine (discussed below).
Cyclophosphamide is a powerful medicine that keeps the immune system from working normally. Healthcare professionals must monitor their patients carefully and perform blood tests frequently. Cyclophosphamide can cause an increased risk of infection, bone marrow suppression (lowering of blood counts), sterility, hemorrhagic cystitis (bleeding from the bladder), and bladder cancer, as well as other serious side effects.
Methotrexate
Researchers at NIH have been studying methotrexate used for treating Wegener's granulomatosis since 1990. In people with active, but not immediately life threatening, Wegener's granulomatosis, methotrexate has been used in combination with prednisone to bring about remission. It also is used to maintain remission after a person has initially received cyclophosphamide. Methotrexate is usually given for 1 to 2 years, after which time if a person stays in remission, it is decreased and stopped.
Methotrexate is given once a week usually by mouth, but occasionally as an injection under the skin or in the muscle. People taking methotrexate need to have regular blood work to monitor their response and to watch for side effects.
The side effects of methotrexate include infection, lowering of the blood counts, nausea, soreness and ulceration of the mouth lining, irritation of the lungs (pneumonitis), and inflammation and scarring of the liver. People taking methotrexate cannot drink alcoholic beverages. Methotrexate cannot be given to people who have poor kidney function or who have underlying liver disease such as hepatitis.
Azathioprine
Healthcare professionals use azathioprine (also called Imuran) primarily to maintain remission in people who have initially been treated and gone into remission with cyclophosphamide. It is taken once a day by mouth. Similar to methotrexate, it is usually given for 1 to 2 years, after which time the dosage is lowered until it is stopped.
The side effects of azathioprine include infection, lowering of the blood counts, and rarely an allergic-type reaction. In people who receive azathioprine to prevent rejection of a transplanted organ, there has been a suggestion of an increased risk of blood cancers (leukemia and lymphoma). Whether this risk exists in other situations is unclear. People with poor kidney function or liver disease can take azathioprine.
Rituximab
Rituximab is an antibody that selectively reduces specific types of immune cells (B cells) circulating in the blood. It currently is used to treat certain types of lymphoma and rheumatoid arthritis. Recent studies of rituximab suggest this medicine, when given along with prednisone, effectively induces remission in select patients who have Wegener’s granulomatosis. In April 2011, the FDA approved rituximab, in combination with glucocorticoids, for treating Wegener's granulomatosis. Researchers are still studying the long-term effects of rituximab on maintaining remission.
Other medicines
During the course of treating Wegener's granulomatosis, healthcare professionals often give their patients other medicines to prevent medicine-related side effects. These medicines include
- Trimethoprim/sulfamethoxazole (also called bactrim or septra) is given three times a week to prevent Pneumocystis carinii infection (a lung infection)
- A medicine regimen is often given to prevent prednisone-related bone loss (osteoporosis)
- Folic acid or folinic acid (also called leucovorin) is often given to people taking methotrexate
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