Melissa Conrad Stöppler, MD, is a U.S. board-certified Anatomic Pathologist with subspecialty training in the fields of Experimental and Molecular Pathology. Dr. Stöppler's educational background includes a BA with Highest Distinction from the University of Virginia and an MD from the University of North Carolina. She completed residency training in Anatomic Pathology at Georgetown University followed by subspecialty fellowship training in molecular diagnostics and experimental pathology.
Treatment with medication can bring long-term remission for most people.
Remission means the disease disappears or its progression is slowed, but the
disease is not cured.
Without medical treatment, a person diagnosed with Wegener's granulomatosis has
a high risk of dying of the disease within two years (90%), usually from lung
(respiratory) or kidney (renal) failure.
People with severe Wegener's granulomatosis need urgent care because their condition is life-threatening.
less powerful form, called limited Wegener's granulomatosis, is not as severe.
Both types are treated with similar medications. Surgery may also be needed.
Because Wegener's granulomatosis affects so many different parts of the body,
care may require a team of doctors who specialize in lung disease (pulmonologists),
kidney disease (nephrologists), arthritis and similar conditions
(rheumatologists), and ear, nose, and throat diseases (otolaryngologists).
The goal of treatment is to bring about remission, maintain remission, and
treat the disease if it becomes active again (relapse).
likely in most people when treated with medications. Almost 90% respond to the
drug cyclophosphamide, and about 75% have complete remission.
In 35-50% of people who respond to a course of medications, the disease
eventually becomes active again (relapse), and then therapy is begun again.
bring about remission, people with severe Wegener's granulomatosis may be given
corticosteroid medication (such as prednisone) for the first few months and
cyclophosphamide (Cytoxan), which is taken by pill form for up to a year.
Sometimes cyclophosphamide may be given once per month through an IV injection
(called pulse therapy). Blood tests are needed every two weeks to check for a
low white blood cell count, which could weaken the immune system and cause
A person who has a relapse may be
given cyclophosphamide and prednisone and possibly an antibiotic.
powerful medications and may cause toxic side effects. It is important to know
what to expect during treatment and discuss all side effects with the doctor.
With careful monitoring, side effects can be reduced.
Side effects of these drugs:
Prednisone: Ideally, corticosteroids are used
for only a short time in order to bring sudden flares in symptoms under control.
Long-term use is associated with serious side effects, such as osteoporosis,
glaucoma, cataracts, mental changes, abnormal blood glucose levels, or arrested
bone growth in children before puberty. After prolonged use, the corticosteroid
dose must be gradually decreased over weeks to months to avoid corticosteroid
Cyclophosphamide: Blood cell counts are monitored
regularly to watch for toxic effects. The urine is examined regularly for red
blood cells, which may be the first sign of hemorrhagic cystitis (severe
inflammation of the bladder that causes blood in the urine). It is important to
drink lots of fluids while on this drug to prevent hemorrhagic cystitis.
Methotrexate: To guard against problems, kidney and liver function are monitored
on a regular basis, as are blood cell counts. Methotrexate may cause toxic
effects on blood, kidneys, liver, lungs, and gastrointestinal and nervous
Inflamed tissue may show necrosis (localized tissue death) and need to be
surgically removed. Such tissue would be located in the nose, throat, and lungs.
Problems with middle ear infections can be remedied by surgically inserting
ear tubes through the eardrum to allow the ear to drain.
For people with
severe kidney failure, a kidney transplant may be a lifesaving option.