Granulomatosis with polyangiitis

This page explains about Granulomatosis with polyangiitis (GPA) (previously known as Wegener’s granulomatosis), what causes it and how it can be treated. It also gives details of what to expect when a child comes to Great Ormond Street Hospital (GOSH) for assessment and treatment.

Granulomatosis with polyangiitis (GPA) (previously known as Wegener’s granulomatosis) is a rare disease that affects the upper respiratory tract (sinuses, ears, nose and throat), lungs, kidneys, eyes, skin and other body organs.

As with most types of vasculitis, we do not know exactly what causes the disease to develop.

The exact frequency in children is not known, but it can and does occur at any age, including in children. It is slightly more common in males than females.

Granulomatosis with polyangiitis (GPA) is usually diagnosed by looking at a child’s history of symptoms and with a physical examination. It is diagnosed by the symptoms present and a positive blood test result for a marker called ANCA (anti-neutrophil cytoplasmic antibodies).

• Ears, nose and throat
  Nosebleeds are common along with crusting of blood and mucus in the nose, and sinusitis
  Recurrent ear infections and possible hearing loss can occur
• General health
  A fever or high temperature with weight loss, tummy pain, and joint pains
• Chest
  Chest pains and coughing, bringing up blood at times
• Kidneys
  Can be affected, shown by abnormal urine tests and/or high blood pressure when measured

The treatment is with steroids and an immunosuppressant drug, usually cyclophosphamide or mycophenolate mofetil which dampens down the body’s immune system. The child will also be asked to take a regular long-term antibiotic to help prevent infections. The possibility of joining in with a clinical trial may be offered. More information about these medicines is available in our general .

The key to a good outcome is early diagnosis, but GPA is still a serious disease with a small risk of death in some patients.

If a child receives treatment early, the overall outlook is good, however. Once he or she goes into remission (the disease gets better) and the treatment is reduced, about half of children with the disease may have a relapse (the disease returns).

Most children and adolescents lead normal lives but will have to continue to see their doctor regularly to check for signs of a relapse, take their medicines regularly, and have blood tests every few months. The outlook for children whose kidneys have been affected varies from child to child, depending on the level of damage sustained.