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Granulomatosis with polyangiitis (Wegener's), also known as GPA, is a rare blood vessel disease. It can cause symptoms in the sinuses, lungs, and kidneys as well as other organs. It is a potentially serious disease. However, with prompt diagnosis, granulomatosis with polyangiitis can be treated effectively.
GPA is abnormal inflammation of the blood vessels, which is also called a vasculitis. GPA involves the small and medium-sized blood vessels throughout the body. The inflammation in the blood vessel does not allow blood to flow properly, so the cells do not get the oxygen they need. This causes tissue injury called granulomatous inflammation. A granuloma is a type of cellular inflammation that can usually be seen on biopsies of affected organs. The cause of GPA is not known.
Most commonly, GPA affects the sinuses, lungs, and kidneys, but also can affect the eyes, ears, skin, nerves, joints, and other organs. Symptoms can develop over days to months. The first symptoms generally appear in the respiratory tract (e.g., nose, sinuses, and lungs). The symptoms include nasal congestion, frequent nosebleeds, shortness of breath, and cough that may produce bloody phlegm. Other early symptoms can include joint pain, decreased hearing, skin rashes, eye redness and/or vision changes, fatigue, fever, appetite and weight loss, night sweats, and numbness or loss of movement in the fingers, toes, or limbs.
If not treated quickly and aggressively, organ tissue can be damaged, sometimes permanently. There are several medications that help treat GPA.
This rare disease is estimated to affect 3 out of every 100,000 people. It affects men and women equally. GPA can occur at any age, but most often between the ages of 40 and 65. It is rare in children.
There is not one test for diagnosing GPA. Many things play an important role in diagnosing GPA, including: symptoms, physical examination, laboratory testing and imaging studies such as X-rays, CT scans, or MRI. If the doctor suspects GPA, he/she may order a urinalysis and blood test to measure kidney function (creatinine) and lung imaging through a chest X-ray or CT scan.
The diagnosis of GPA is based on the all the results of the blood tests, X-rays and CT findings, and physical exam findings. Most physicians will recommend a biopsy of the tissues involved. A biopsy of the affected tissue can help confirm the diagnosis.
The most common blood test is an antibody test called ANCA (anti-neutrophil cytoplasmic antibody). This test checks for this antibody in your bloodstream. A blood test positive for ANCA supports the need for further examination for GPA. A positive test does not confirm a diagnosis. A tissue biopsy may still be needed. A negative test does not rule out GPA. Up to 20% of people with GPA can have a negative ANCA test.
Active GPA causes inflammation within the blood vessels and organs. Ongoing inflammation in the blood vessels or organs can cause organ failure. Without treatment, GPA can worsen rapidly, leading to potentially life-threatening kidney or lung failure. Treatment choices are based on the organs affected, disease severity, and individual medical factors.
Those with active severe disease often receive a glucocorticoid (steroid) medication such as prednisone combined with cyclophosphamide (Cytoxan), a chemotherapy type of medication. Prednisone is started at a high dose and gradually reduced over weeks to months. Cyclophosphamide is given for three - six months; it can be given orally or by injection. Those who improve are then switched to another medication such as methotrexate (Rheumatrex, Trexall) or azathioprine (Imuran, Azasan) for two or more years.
Another option for the treatment of severe granulomatosis with polyangiitis (GPA) is rituximab (Rituxan) combined with glucocorticoids. Rituximab is a medication, given by injecting into the vein, which stops the blood vessel and organ inflammation. The patient and his/her doctor will work together to decide which treatment will be best for the patient. Those with less active disease may be treated with prednisone and methotrexate. Each medication has potential side effects, which need to be considered as part of the treatment plan.
The impact of GPA can vary greatly, depending on its severity, the organs involved, and any complications related to the disease or its treatment. People with GPA need ongoing checkups that include laboratory studies, imaging tests, and clinic visits with their doctor. Follow-up monitoring is very important for patients with GPA. It is important for the doctor to look for signs of disease recurrence, and look for and prevent short or long-term treatment-related complications.
Even with effective treatment, recurrences (relapses) are common. Relapses may resemble or differ from the initial onset, so new symptoms should be reported to the medical practitioner as soon as possible. Regular doctor visits together with monitoring of laboratory tests and imaging studies can help to detect relapses early.
Depending on the organ sites involved, patients with GPA frequently require care from several specialists, each of whom contributes in a valuable way. Within this team of doctors, however, it remains important to find a lead doctor to prescribe and check for problems with related medications. This is typically done by the rheumatologist, who is trained to use these medications to effectively treat GPA while minimizing the potential for side effects.
Updated May 2017 by Jennifer Murphy, MD and reviewed by the American College of Rheumatology Committee on Communications and Marketing.
This information is provided for general education only. Individuals should consult a qualified health care provider for professional medical advice, diagnosis and treatment of a medical or health condition.
© 2017 American College of Rheumatology