What is Wegener's granulomatosis?
Wegener's granulomatosis (WG) is a rare disease of
uncertain cause. It is characterized by inflammation in a variety of tissues,
including blood vessels (vasculitis). Inflammation damages vital organs of the body.
WG primarily affects the upper respiratory tract
(sinuses, nose, trachea [upper air tube]), lungs, and kidneys. Any other organ
in the body can be affected as well.
Who is affected by the disease?
WG can occur at any age. The peak age groups affected
are from 40-60 years. It affects men and women equally.
What are the symptoms?
The symptoms of WG and their severity vary among patients. General signs of the disease
- Loss of appetite
- Weight loss
Most patients first notice symptoms in the respiratory
tract. Symptoms may include:
- Persistent runny nose (also called rhinorrhea) or the formation of nasal
crusts and sores
- Nasal or facial pain
- Nose bleeds or unusual nasal discharge, caused by inflammation of the
nose or sinuses
- Cough that might include bloody phlegm caused by upper airway or lower
airway (lung) inflammation
- Chest discomfort with or without shortness of breath
- Middle ear inflammation (also called otitis media), pain, or hearing loss
- Voice change, wheezing, or shortness of breath caused by inflammation of
Other possible symptoms include:
- Eye inflammation and/or bulging, with or without loss of vision
- Joint pain (arthritis) or muscle pain
- Rashes or skin sores
- Kidney inflammation*
*Although kidney inflammation is common, it is not usually associated with symptoms, such as pain.
How is the disease diagnosed?
WG has symptoms similar to a number of other
disorders, which may make it difficult to diagnose. However, for the most
effective and successful treatment, early diagnosis is critical.
It is the combination of symptoms, results of physical
examinations, laboratory tests, X-rays, and sometimes a biopsy (sample) of
affected tissue (skin, nose, sinus, lung, kidney or other sites) that together
prove the diagnosis of WG. Following treatment, these factors are also critical
in judging whether the disease is active or in remission.
A positive blood test for antineutrophil cytoplasmic
antibodies (ANCA) can support a suspected diagnosis of the disease. However,
this blood test does not by itself prove the diagnosis of WG or determine
To help your doctor judge disease activity, he or she may order:
- A red blood cell count (to look for signs of anemia)
- Sedimentation rate (the speed in which blood cells settle in a vertical
- Chest or sinus X-rays
Sometimes the lungs may be abnormal even though there
are no symptoms such as cough or shortness of breath. If symptoms of WG are
apparent, but not from the lungs, one quarter to one third of patients may still
have unexpected lung abnormalities detected on imaging tests (conventional
X-rays or a CT scan). Therefore, it is important to have lung images performed
if active WG is suspected -- even if you don't have any symptoms of lung disease.
How is the disease treated?
Because WG is often a life-threatening disease, it is
treated with a variety of powerful drugs that have been shown to be life-saving.
Treatment usually includes corticosteroid medicines,
such as prednisone, and other drugs that also suppress immune function. These
include cyclophosphamide, methotrexate, azathioprine, or mycophenolate mofetil.
Such drugs usually induce remission (the complete absence of all signs of the
disease). Recent studies have also shown that a biologic agent called rituximab
is as effective as cyclophosphamide and does not have some of the toxic effects
of that drug. Improvement with these therapies usually occurs within days to
weeks. When WG is in remission, the dosage of prednisone is reduced or often
completely stopped. Some patients do require a low dose of prednisone to sustain
remission. The ideal duration of treatment with the other immunosuppressive
medications is uncertain. However, it is well established that WG is associated
with relapse that may be as high as 60% in the first year and 80% by the end of
the second year after stopping treatment. It must be borne in mind that these
therapies can provide excellent control of disease, but are not cures.
The old practice of using cyclophosphamide over
extended periods of time is no longer considered the "standard of care." Most
patients with severe disease can be placed into remission with cyclophosphamide
and corticosteroids within 3-4 months. Switching from cyclophosphamide to either
methotrexate or azathioprine is usually effective in sustaining improvement or
remission. This approach is a major change in strategy that has spared most
patients the risks of long-term cyclophosphamide therapy. Indeed many patients
who have milder forms of WG can be treated without cyclophosphamide; being able
to achieve remission with agents such as methotrexate, mycophenolate mofetil or rituximab.
The treatment used for WG has also been successfully
applied to other vasculitic diseases.
What are the side effects of treatment?
Because these drugs suppress the immune system, there
is an increased risk of developing serious infections. Prednisone can also cause
weight gain, cataracts, brittle bones, high blood pressure, diabetes, and
changes in mood and personality. Cyclophosphamide can cause sterility, bladder
irritation and bleeding, and even cancer of the bladder when used over extended
periods of time. Thus every effort is made to control severe disease with
cyclophosphamide and prednisone and then stop cyclophosphamide after about 3-6
months and substitute other agents that may be effective, although are less
powerful. Methotrexate can cause liver irritation. Cyclophosphamide and
methotrexate can each cause changes in blood counts and sometimes lung inflammation.
Because the medicines used to treat WG can have
serious side effects, patients are monitored closely by their doctor. The dosage
of medicine is adjusted as needed throughout the course of treatment.
The risk of side effects has stimulated research to
discover other effective treatments that have less risk. An excellent example is
rituximab, an agent recently demonstrated to be as effective as cyclophosphamide.
Experience with this new therapy is far less than the others noted above. The
precise role that it will play in treatment of WG is still being defined.
Because all of these drugs have some risk, laboratory
tests that may detect side effects are regularly monitored in patients with WG.
These tests, although not perfect, may also reflect worsening of disease
activity, even in patients that feel well. Thus, regular monitoring checks are
important for medication tolerance and are also one measure of disease activity.
What is the outlook for people with Wegener's granulomatosis?
After treatment and improvement occurs, relapses of
the disease are common, especially if maintenance treatments are fully
withdrawn. Flare-ups might follow reductions of the doses of prednisone,
methotrexate, azathioprine, or cyclophosphamide. Flare-ups can usually be
controlled by increasing the dose of these medicines.
WG is a very serious disease, and its treatment
carries significant risks. However, treatment is life-saving for almost everyone
when the diagnosis is made in a timely fashion and appropriate treatment
instituted. This includes regular monitoring of disease activity and possible
Prior to recognizing effective therapy in the 1970s,
half of all patients with this illness died within 5 months of diagnosis. Today,
more than 75% of treated patients are alive at least eight years later. For many
people with WG, long term survival has been seen with many also being able to
lead relatively normal lives. If past is prelude to the future, ongoing research
will lead to further discoveries and even better treatment.
Schilder AM. Wegener's Granulomatosis vasculitis and granuloma. Autoimmun Rev 2010;9:483–487
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