Medicines for Granulomatosis with polyangiitis

What it is

Granulomatosis with polyangiitis (GPA) is a rare autoimmune condition, and a type of vasculitis — a group of conditions in which the immune system causes inflammation of blood vessels. In GPA, the inflammation particularly affects small and medium-sized blood vessels, and characteristically involves the respiratory tract (nose, sinuses and lungs) and the kidneys, though it can affect many other parts of the body too. Symptoms vary depending on which areas are involved, and can include: persistent nose and sinus problems (such as a blocked or crusty nose, nosebleeds, sinus pain, and sometimes changes to the nasal structure), ear problems, a cough (sometimes with blood), breathlessness, and, if the kidneys are affected, changes that may only show on tests initially but can, if untreated, seriously affect kidney function. There are often also general symptoms such as fever, tiredness, weight loss, joint and muscle aches, and sometimes skin, eye or nerve involvement. GPA is a serious condition because, untreated, the inflammation can damage vital organs (particularly the kidneys and lungs). However — importantly — it is treatable, and modern treatment can control the disease effectively, so early diagnosis and specialist care are key.

How it is treated

GPA is diagnosed and managed by specialists (often involving rheumatologists, kidney and respiratory doctors), using a combination of tests — including blood tests (notably a marker called ANCA, which is often positive in GPA), urine tests and kidney function, imaging, and often a biopsy of an affected area to confirm the diagnosis. Because untreated GPA can damage organs, treatment is started promptly and has two phases. First, to bring the disease under control (induce remission), intensive treatment is used to strongly suppress the overactive immune system — typically steroids combined with another powerful immune-suppressing or targeted (biologic) medicine, chosen according to the severity and organs involved; this is often very effective at controlling the inflammation. Second, once the disease is under control, ongoing "maintenance" treatment (usually with immune-modulating medicines at lower intensity) is continued for a period to keep it in remission and prevent relapse, as GPA can flare or relapse. Careful monitoring — of the disease, the organs (especially the kidneys), and the treatment (which requires monitoring for side effects and infections) — is central to long-term care. With modern treatment, many people achieve good control. The reassuring message is that granulomatosis with polyangiitis, though a serious autoimmune condition, is treatable — prompt, specialist-guided treatment to suppress the immune inflammation can control the disease and protect the organs, with ongoing treatment and monitoring to maintain remission.