Vasculitis Information for patients and families

Great Ormond Street Hospital for Children NHS Trust This booklet explains about the various forms of , and what to expect when your child comes to Great Ormond Street Hospital for treatment.

What is vasculitis? Vasculitis is a word used to describe various diseases that involve inflammation of the blood vessels. The system is made up of various sizes of blood vessels, arteries (which carry blood away from the ), veins (which carry blood back to the heart) and capillaries (tiny Heart blood vessels) through which the blood travels to all tissues and organs. Kidneys When a small blood vessel Large artery becomes inflamed, it can break and bleed into the surrounding . This causes small red or purple dots on the skin. If Small arteries a larger blood vessel becomes inflamed, it may swell to produce Small vessels a lump that you feel under the skin. The inside of the blood vessel may also narrow, which reduces the amount of blood able to flow through it or it may become blocked by a blood clot. If the blood flow through the blood vessels is reduced or stops, the tissue may begin to die. On rare occasions, vasculitis may Medium sized artery cause the wall of a blood vessel to weaken and develop a bulge () that can rupture and bleed. There are many types of The booklet describes vasculitis, affecting different sizes different types of of blood vessel and different . parts of the body. The different kinds of vasculitis are defined in The most common types of different ways. For example, from vasculitis affecting children: the size of the affected blood ■ Henoch-Schönlein vessels or the way they show up most often. ■

Some types of vasculitis affect Other types of vasculitis: predominantly the skin and others can affect internal organs ■ ANCA associated vasculitis, with more serious complications. which includes microscopic Those mainly affecting the polyangiitis, Wegener’s skin include conditions granulomatosis and renal such as: ; limited vasculitis leucocytoclastic vasculitis; panniculitis which involves the ■ fat tissue deep to the skin and ■ Takayasu’s . pityriasis lichenoides. These are relatively rare disorders and There are other types of vasculitis can be associated with some where it is not clear into which systemic (affecting the whole category they fit, but these are body) symptoms. Children with very rare in children and will these skin conditions are usually be recognised by specialised referred to a skin specialist children’s doctors. Other types (dermatologist). Often a skin of vasculitis mainly affect older is needed to help confirm patients are not included in this the diagnosis. Treatment varies booklet. according to the diagnosis and any associated medical problem, but for those with mainly skin vasculitis the outlook is usually good. What are the symptoms of vasculitis? How is vasculitis diagnosed? The symptoms of vasculitis depend on which organs are Henoch-Schönlein Purpura and affected. In most cases, children have other symptoms of Kawasaki disease are often easy to general illness, including fatigue, , weight loss and diagnose because of the fairly typical aches and pains all over. symptoms. The other diseases, which are much less common, have The symptoms associated with some types of vasculitis symptoms that can look very similar include: to those of other diseases. This may mean it takes a while to achieve a firm ■ skin - red or purple pinprick spots called ‘petechiae’. diagnosis. As it affects various parts of If the spots are larger (about the size of your the body, a multidisciplinary approach fingertip) they are called ‘purpura’. These are the is often needed, with input from most common skin symptoms associated with rheumatologists (specialists of the vasculitis, but others occur including itching, musculoskeletal system), nephrologists (nettle ) or wheals on the skin, and painful lumps (kidney specialists), neurologists ■ joints - aching joints are very common with (brain and nerve specialists), vasculitis. They may swell up and feel warm too cardiologists (heart specialists), dermatologists (skin specialists) and so ■ kidneys - vasculitis often leads to kidney damage on. At Great Ormond Street Hospital, which shows up as high blood pressure or blood and these doctors meet regularly to hold protein in the urine clinics to diagnose and treat patients with vasculitis. ■ brain - are a symptom of vasculitis affecting the brain In some cases, the doctors can only decide on a diagnosis once other ■ peripheral nerves (in the limbs) - numbness, conditions have been ruled out. In tingling, and loss of strength in a other cases, specific blood tests, x-rays or from affected organs are ■ intestines - if the blood flow to the intestines is helpful. reduced, this can cause tummy pain and bloating

■ heart - the heart is a muscle that is supplied with blood by the . If the blood supply What causes vasculitis? is affected, it can cause when exercising, called angina. Pre-school children may not be able to We do not know what causes describe this symptom vasculitis to develop. The most likely reason is that the white blood cells ■ lungs - symptoms of fever, , chest pain and attack healthy cells instead of foreign tend to show up on x-rays as invaders like bacteria and viruses, but pneumonia we do not know why this happens. It may also be related to other ■ eyes - the blood vessels in the back of the eye autoimmune disorders like (retina) can be affected, which leads to blurring or and systemic even loss of sight. It may be accompanied by a severe (SLE). too. How is vasculitis treated?

Many types, especially Henoch- ■ Steroids – Most severe cases of vasculitis Schönlein Purpura, do not will need treatment with steroids. It will need treatment. In most cases often be started as injections of high doses of the uncommon and most of methylprednisolone in ‘pulses’ or blocks severe vasculitis diseases, of treatment. After that the child will often quite strong treatment with need high doses of as tablets different medicines is needed, or medicines, which we gradually reduce for instance, steroids that or ‘wean’ to as low a dose as possible. Long reduce the inflammation and term treatment, for a year or more, with low damp down the overactive doses of prednisolone is often needed. The parts of the . steroids are often very good at reducing the Other medicines, like inflammation and damp down the immune , may also be system. Unfortunately, as with many powerful used alongside steroids to damp medicines, there are side effects. Most side down the immune system further. effects relate to the cumulative total dose Less powerful medicines or lower received. For more information, please see our doses of stronger medicines leaflet on steroids. will then be used to maintain improvement. ■ Immunosuppressant medicines – A number of different immunosuppressant medicines are used in the treatment of severe vasculitis. The most common is cyclophosphamide, which will often be started as injections in ‘pulses’ or blocks of treatment. After that the child will often need high doses of cyclophosphamide as tablets or medicines, which we gradually reduce or ‘wean’ to as low a dose as possible. Sometimes, other medicines may be used for maintenance, including , cyclosporin A, MMF, colchicine, methotrexate and thalidomide. As in steroid medicines, these can be effective but do have side effects. More information is available in our leaflets. Plasma exchange is also used in severe cases. Please see our leaflet for more information.

There are newer, even more powerful medicines under trial, which will be fully explained to you. Henoch-Schönlein Purpura What is Henoch-Schönlein Purpura? What are the symptoms? It is a disease where small blood vessels called In most cases, symptoms occur as above, but capillaries become inflamed and damaged. often not at the same time. Very typically, the It is named after the two doctors who first symptoms come and go for several weeks. described the disease and is often referred to as HSP for short. It mainly affects four How is it diagnosed? organs: In most cases, doctors will be able to diagnose it easily as the symptoms are very similar ■ the skin causing a purpuric rash, which from child to child. in severe cases can become swollen and ulcerated; How is it treated? ■ the digestive system causing severe There is no specific treatment for HSP, only stomach pains and blood in the faeces treatment for the symptoms. If the joints are (poo). In a small number of children, this uncomfortable, then a mild pain relieving is so severe that surgery is needed. medicine should be taken to reduce any pain. Steroids can also help some children with ■ the joints and the tissue around the joints severe symptoms. causing severe pain and there may be difficulty in walking; What is the outlook for people with HSP? ■ inflammation in the kidneys causing blood and protein in the urine and sometimes The outlook for most children overall is increased blood pressure. very good. The symptoms tend to disappear within a few weeks, although in about half of In a few cases, other parts of the body can be the children affected they may return weeks affected too. or months later. If the kidneys have been affected, in a small number of cases, this can How common is it? lead to kidney failure, which would mean It occurs mainly in young children of school that your child might need dialysis later. age, but has also occurred in younger children and adults. It seems to affect slightly higher numbers of males than females. It is the most common type of childhood vasculitis and occurs in about 14 out of every 100,000 children. Kawasaki disease

What is Kawasaki disease? How is it diagnosed? It is a disease that affects young children, The doctors will need to rule out other named after the doctor who first reported it. , but in most cases, the doctors will be It usually starts with a fever and a rash like a able to diagnose Kawasaki disease because the ‘normal’ , but it tends to last more symptoms are rather similar from child to child. than two weeks. Other symptoms develop As about 20 to 40 per cent of children develop during this time as well. vasculitis in the blood vessels around the heart, an echocardiogram (ECHO) or an angiogram is How common is it? often needed. If the blood vessels are affected it Kawasaki disease is the second most common may lead to a bulge developing in the wall of a type of childhood vasculitis. The exact heart blood vessel (coronary aneurysm). incidence is not known, but reports suggest How is it treated? that it affects eight in every 100,000 children under five years old in the UK. It tends to Kawasaki disease is usually treated aggressively affect children under the age of five, and is in hospital with high doses of a more common in males than females. It has called given directly into a been reported in all racial groups, although it vein (intravenously or IV) through a ‘drip’. occurs more often in Japan, where it was first If this is given early enough in the course of reported. the disease, it can reduce the chance of future heart problems. Your child may also be given What are the symptoms? , which reduces the chance of blood The main symptoms include fever (over clots developing. 38°C), redness of the insides of the eyelids, Long term follow up treatment is usual for lips, and inside the mouth. The most children, to check how the heart is lymph nodes in the neck may also become working and catch any problems that may swollen and there may be a rash, especially on occur, early. the chest. The palms of the and soles of the feet may turn a bright red colour and What is the outlook for become puffy. The skin at the ends of the people with Kawasaki Disease? fingers may also turn bright red and start to peel. There may also be swelling of the joints It usually takes children a few weeks to start (arthritis) in older children, which usually to feel better. Children who do not have any affects large and small joints on both sides heart problems usually recover fully. A second of the body. Other symptoms can include attack of the disease is rare, but if it happens, diarrhoea and , stomach pain and steroid therapy is often started. The outlook . Some children develop symptoms for children whose heart has been affected like breathlessness and chest pain if the heart varies from child to child, depending on the is affected. level of damage sustained. ANCA associated vasculitis ANCA associated vasculitis is a group of What are the symptoms? uncommon cases of vasculitis affecting the The generalised symptoms include fever, small blood vessels in many parts of the body. fatigue, muscle aches and pains and weight Most of these cases have ‘ANCA ’. loss. The other symptoms depend to an These are antibodies attacking the kind of extent on which part of the body is affected. white blood cells that are called neutrophils. However, common symptoms include There are two main kinds of ANCA: P- purpura, coughing up blood, ANCA (also called MPO ANCA) and C- and kidney problems. ANCA (also called PR3 ANCA). There are three forms of ANCA vasculitis: How is it diagnosed? ■ ; It is diagnosed by the symptoms present. Most cases have high values of MPO ANCA. ■ Wegeners granulomatosis; A kidney biopsy will often show severe kidney ■ and renal limited vasculitis. inflammation.

These diseases are defined from their How is it treated? symptoms and the kind of ANCA antibodies Microscopic polyangiitis is treated with that are present. steroids and immunosupressants.

What is the outlook for people with microscopic polyangiitis? ■ Microscopic If the patient receives early treatment, the polyangiitis outlook is fairly good. Most patients go into What is microscopic polyangiitis? remission and a high proportion of these do not have a relapse, where the disease returns. It is a disease that affects small and medium Most patients continue to lead normal lives, blood vessels throughout the body. It usually but will have to continue to see their doctor affects the blood vessels in the skin, lungs, regularly to check for signs of a relapse. digestive system and kidneys. ■ Wegener’s How common is it? granulomatosis In the UK, it affects about two in every 100,000 people, usually more males than What is Wegener’s granulomatosis? females. It can appear at any age from It is a very rare disease that starts with childhood onwards, but is much more inflammation of the tissues in the nose, throat common in adults. and lungs, but may develop into vasculitis affecting blood vessels throughout the body including severe kidney inflammation. It is How is it diagnosed? named after the doctor who first reported It is diagnosed by the symptoms present and it. As with most types of vasculitis, we do a positive result for PR3 ANCA. not know exactly what causes the disease to develop. How is it treated? How common is it? The treatment is similar to that used for microscopic polyangiitis. Taking the The exact incidence is not known, but it can co-trimoxazole long term to occur at any age. It is twice as common in prevent infections has been show to be men than in women. It is also more common beneficial in preventing relapses. in white people.

What are the symptoms? What is the outlook for people with Wegener’s granulomatosis? The first symptoms tend to affect the nose, ears and windpipe and may include If the patient receives treatment, the outlook nosebleeds, sinusitis (inflammation of the is reasonably good. However, about half of sinuses), middle ear infections, and coughing. the people with the disease have a relapse A high temperature, loss of appetite, joint where the disease returns. This tends to pains and swelling, and inflammation of the happen within a couple of years of stopping eye or ear may occur as well. The disease may treatment but could occur anytime. Most also affect other blood vessels, especially the patients lead normal lives but will have to arteries to the heart, which can cause chest continue to see their doctor regularly to pain or a heart attack, or those leading to check for signs of a relapse. The outlook for the brain and spinal cord, which can cause children whose kidneys have been affected similar symptoms to a lot of neurological varies from child to child, depending on the diseases. Rarely the bowel is affected, causing level of damage sustained. pain and blood in the faeces (poo). ■ Renal limited vasculitis The disease may also spread causing inflammation of blood vessels throughout the What is renal limited vasculitis? body. These can affect the skin, causing sore Renal limited vasculitis might be a form of patches that spread and may leave scars. The microscopic polyangiitis with symptoms disease may also affect the kidneys, which only from the kidneys. The main feature could lead to kidney damage, although this is glomerulonephritis (inflammation in ranges from mild kidney damage to kidney their kidneys). The diagnosis is made on failure requiring dialysis. the kidney biopsy and with the ANCA antibodies. The treatment is similar to that of MPA. Other types of vasculitis ■ Polyarteritis nodosa indicate inflammation and when doctors have ruled out other diseases that can have these What is polyarteritis nodosa? symptoms. The diagnosis can be confirmed It is a disease where medium and small-sized by a biopsy of an affected blood vessel. This arteries become inflamed and damaged. involves taking a small sample of tissue This leads to problems with blood supply containing blood vessels to examine under to various parts of the body, depending a microscope. Other biopsies may also be on the arteries affected. We do not know needed to check the extent of any damage to exactly what causes the disease to develop. organs like the liver or kidneys. A specific test Sometimes an infection can seem to start called an angiogram can show up damage in the inflammation, especially in one type of the arteries. For more information about this polyarteritis nodosa where the skin shows test, please see our leaflet and symptoms but the internal organs do not. : information for families. How common is it? How is it treated? It tends to develop between the ages of 40 The treatment is similar to that used for and 50 in adults, but childhood polyarteritis microscopic polyangiitis and Wegener’s nodosa most commonly occurs around the granulomatosis. age of nine years. There are no exact figures What is the outlook for people for how many children have polyarteritis with polyarteritis nodosa? nodosa but we know that it is rare. There is a range of possible outcomes What are the symptoms? depending on the severity of the disease Fever is an early symptom along with tummy and how much damage is already there in pain, pins and needles in the hands and feet, vital organs such as the kidneys. Many will weakness and weight loss. Muscle and joint respond to the medicines and not have pain is also common, and the joints may further relapses. Others will have a more become inflamed and swollen. The blood chronic, relapsing course and require long- vessels near the surface of the skin may feel term maintenance treatment. This may lumpy and rarely, ulcers can develop on the include medicines to treat the damage caused skin over these blood vessels. by the disease before it is under control, such Children with polyarteritis nodosa develop as raised blood pressure from damage to the kidney damage. Other symptoms depend kidneys. Long-term side effects of both the on the arteries affected, which may be in the disease and the medicines used to treat it can digestive system, the heart, the brain or the occur. liver. ■ Takayasu’s arteritis How is it diagnosed? What is Takayasu’s arteritis? As with most types of vasculitis, it is It is a disease where the (large artery diagnosed by the symptoms and signs that leaving the heart) and the blood vessels kidneys can develop which can lead to high branching off of it become inflamed and blood pressure and kidney failure. The heart damaged. This usually leads to problems with and lungs can also be affected too. the blood supply to the head and arms, but How is it diagnosed? can affect other parts of the body. It is named after the doctor who first reported it. As with It is usually diagnosed by a physical most types of vasculitis, we do not know examination including checking the patient’s exactly what causes the disease to develop. pulse and blood pressure. If a person has the disease, the pulse may be very weak and How common is it? difficult to find, and the blood pressure in It tends to develop in people between the the arms is much lower than in the legs. The ages of 15 and 30, and affects about eight blood pressure may differ from one side of times as many women as men. It can affect the body to the other. Occasionally, there all races, although it seems more common in may be a murmur where the blood makes a women of Asian origin. noise as it rushes through a narrowed artery What are the symptoms? at high pressure. Some patients with the disease seem to start How is it treated? with an (or sudden) phase where there is The disease tends to be treated a general feeling of being unwell, fever, night with like steroids and sweats, weight loss, joint pain and tiredness. immunosuppressants, which damp down the Some people also have anaemia. This sudden immune system. If there is a risk of mini- phase seems to improve and the disease then strokes, then a blood-thinning medication has an ongoing phase. The main symptoms may be suggested. If blood pressure is high, of this phase are temperature changes, pins then medications to lower this will also be and needles and pain in the affected arm or suggested. leg. These symptoms are caused by damage If the arteries are narrowed to a dangerous to the aorta and other arteries as a result of extent, then they may need to be unblocked inflammation and narrowing of the vessel, (angioplasty), although clear discussion which reduces blood flow through it. Other about the possible risks and benefits of this patients with the disease do not seem to have procedure is needed. the sudden phase but develop the ongoing symptoms straightaway. What is the outlook for people with Takayasu’s arteritis? When blood flows through the aorta and other vessels is reduced, this can lead to fainting and The outlook depends on whether serious transient ischaemic attacks (TIAs or mini- complications of the arteries have occurred. strokes), along with cramps in the jaw or arms. In people with no serious complications, the This reduction in blood supply can also cause outlook is good. The outlook is also good the muscles in the face and arms to weaken if serious complications are treated quickly and grow smaller. Sometimes can before too much damage is done. Otherwise, develop, where the wall of the artery balloons people may need repeated operations or out and weakens. If the artery supplying procedures to correct any damage that occurs blood to the kidneys is affected, damage to the as well as medications for a long period. More information and support groups General information about vasculitis There is no support group in the Cleveland Clinic (USA) UK for vasculitis but the following Website: www.clevelandclinic.org/ organisations provide information arthritis/vasculitis/ and support: Johns Hopkins Vasculitis Center Arthritis Care Youth Service (USA) The Source Website: vasculitis.med.jhu.edu/ 18 Stephenson Way London NW1 2HD Youth Helpline: 0808 808 2000 for people up to 26 years of age. There are few support organisations Mondays to Fridays 10am to 2pm for the specific types of vasculitis, Website: www.arthritiscare.org.uk but the following organisations and Arthritis Research Campaign website might be useful: Copeman House Takayasu’s Arteritis Foundation St Mary’s Court International St Mary’s Gate Website: www.takayasu.org Chesterfield S41 7TD Tel: 0870 850 5000 Wegener’s Granulomatosis UK Website: www.arc.org.uk Email: wegeners.uk@btinternet. Stuart Strange Vasculitis Trust com 12 Acton Road Website: www.btinternet.com/ Mackworth ~wegeners.uk/ Derby DE22 4JF Tel: 01332 521595 Kawasaki Support Group Website: vasculitis-uk.org 13 Norwood Grove

Potters Green European Vasculitis Study Group Coventry CV2 2FR Website: www.vasculitis.org 2004 Tel/Fax 024 7661 2178

Please note: The mention of a particular support group or website does not constitute an endorsement by Great Ormond Street Hospital.

Ref: Ref: 2004F127 July © GOSH Trust NHS Department Compiled in by collaboration the Vasculitis with the Child and Information Group Family

Great Ormond Street Hospital for Children NHS Trust Great Ormond Street London WC1N 3JH Tel: 020 7405 9200 www.goshfamilies.nhs.uk www.childrenfirst.nhs.uk