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
