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 of them markedly improved. After 6 months to 24 years of follow-up, 80 percent of the patients 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 doctors should be aware that they potentially have serious side effects. In many instances these can be minimized or prevented by careful monitoring by both the doctor and patient.
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 doctors regularly, both while they are on these medicines, as well as after the medicines have been stopped.
Prednisone
Prednisone is the most common glucocorticoid that doctors use. Prednisone is similar to cortisol, the natural glucocorticoid hormone produced by the body. 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. Doctors usually give prednisone as a single morning dose to try to imitate how the body normally secretes cortisol.
When the 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 in the disease, prednisone is gradually decreased and discontinued completely after approximately 6 to 12 months.
When prednisone is taken by mouth, the body stops making its own natural cortisol. As the prednisone dose is gradually reduced, the body will resume making cortisol again. It is extremely important that prednisone never be stopped suddenly because the body requires prednisone (or cortisol) to function and may not be able to immediately make what it needs.
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 can also cause weight gain, cataracts, brittle bones, diabetes, and changes in mood and personality.
Cyclophosphamide
Cyclophosphamide (Cytoxan) is the most commonly used cytotoxic drug used to treat Wegener's granulomatosis. People take cyclophosphamide once a day by mouth and must take the drug 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 doctor may adjust the dosage based on blood counts, which are monitored closely to be sure that the white blood cell count is maintained 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 given until remission and then switched to another medicine such as methotrexate or azathioprine (discussed below).
Cyclophosphamide is a powerful medicine that keeps the immune system from working normally. Doctors 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), bladder cancer, as well as other serious side effects.
Methotrexate
Methotrexate has been studied at NIH for the treatment of 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 people stay 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
Azathioprine (also called Imuran) is used 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) but it is not clear whether this risk exists in other situations. People with poor kidney function or liver disease can take azathioprine.
Other medicines
During the course of treating Wegener's granulomatosis, doctors often give their patients other medicines to prevent medicine-related side effects. These 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) are often given to people taking methotrexate
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