Immune Thrombocytopenic Purpura (ITP)

What is ITP?

Immune thrombocytopenic purpura (ITP) is a condition that causes the number of platelets in your blood to be reduced. Platelets are what makes blood clot and they are needed to help you stop bleeding and bruising after injury. If you do not have enough platelets in your blood, you are likely to bruise very easily or may be unable to stop bleeding if you cut or injure yourself.

ITP causes your body’s immune system to destroy your platelets. White blood cells in your blood and your spleen (an organ in your abdomen) are part of your immune system. One of their actions is to produce antibodies, which can help your body fight infections. If you develop an autoimmune condition, your immune system can become overactive and the white blood cells will start to destroy things they shouldn’t, such as your own platelets. This happens mainly in the spleen. ITP is a type of autoimmune condition (‘auto’ means “against yourself”).

ITP in adults is more common in women than men. It is very different to ITP in children. Children usually get ITP after a viral infection and it almost always gets better on its own without any treatment. ITP in adults usually needs treatment.

There are two types of ITP: primary and secondary. Primary is where we do not know why the autoantibody has developed. Secondary means there is another condition predisposing to it. Some people with ITP have other autoimmune conditions, such as rheumatoid arthritis, or infections such as hepatitis or HIV. If you have any of these medical issues, your ITP may be treated slightly differently.

A normal platelet count is between 150 and 400 thousand million platelets per litre of blood. This is usually referred to by doctors just using the first three numbers, such as “150” or “400”. You are unlikely to get bleeding symptoms unless your platelet count is below 20 or even 10. If you needed to have an operation, this would be safe as long as your platelet count is more than 50.

The need for treatment is based on you and your symptoms, rather than a set platelet number. The goal of treatment is to reduce or stop any bleeding symptoms, rather than aiming for a specific numerical platelet target.

What is the treatment for ITP?

If your ITP needs treating, we usually prescribe steroids in the first instance. These work by stopping your immune system from destroying your platelets, by reducing the level of antibodies in your bloodstream. Steroids are often a very effective treatment for ITP and you will usually only needs a short course of treatment (less than six weeks).

Are there any side effects?

Over a short period of time, steroids usually cause no problems. However, steroids can have side effects, especially if you need repeated courses of treatment or have to take them for a long time. Some side effects are related to stopping the white cells working properly; this can increase your chance of getting infections, as the white cells will also be less able to fight off bacteria and viruses. Steroids also have other side effects, such as thinning of the bones (osteoporosis), stomach ulcers and high blood sugar levels (diabetes). They can change your facial appearance and cause thinning and bruising of the skin. People feel they want to eat more while they are on steroids and often put on weight. These side effects usually reverse when the steroids are stopped. If you take steroids for a longer course, you will be given a ‘steroid treatment card’ to carry with you to show it to other health professionals. This is because you should not stop taking steroids without medical supervision or you may need a different dose if you are sick from something else.

Are there any alternatives?

As steroids are a good treatment for ITP and many people only need to take them for a short time, you may have been given them already. However, your doctor may recommend that you try another treatment for your ITP. This could be because:

• You didn’t respond to steroids at all.
• You responded but you “relapsed” (your platelet count fell again) when the dose of steroids was cut down.
• You have responded to steroids in the past, but your ITP has come back and your doctor does not want to give you more steroids because of the side effects (or you may not want to take them again because of the side effects you had before).

If you are taking steroids it is very important that you do not stop them without advice from your doctor, as your body starts to rely on them. They may need to be cut down slowly so that your body has time to adjust, otherwise you might experience weakness and fatigue. If you are worried about possible side effects, please discuss your treatment with your doctor before making any changes to your medication.

Why don’t steroids cure everyone?

There are many reasons you might not respond to steroids. If this happens, your doctor will want to make sure there is no other reason for your low platelet count; this may involve some extra blood tests (including checking for infections if this has not already been done) or a bone marrow test. There is no test available that is completely accurate in diagnosing ITP.

For every ten adults with ITP who are treated with steroids, only three (at most) will not require further treatment. In the other seven people, the platelet count will drop again and more treatment may be needed.

As spontaneous bleeding (bleeding without any injury) only occurs when the platelet count is very low, your doctor may not recommend any further treatment for the time being. Many people with ITP have a platelet count that is below normal, but they do not come to any harm and do not require treatment. This is called being in “remission” from ITP, However, it is important that we discuss with you what treatment may be given if your platelet count falls further, you have bleeding symptoms, or you need an operation.

What other options are there?

Unfortunately, all treatments for ITP have possible side effects, which is why your doctor will not recommend treatment unless you have bleeding symptoms, or your platelet count is very low (usually below 20).

There are different treatments available, and it is important you understand a bit about them, so that you and your doctor can decide which would be most appropriate. The other possible treatments include:

• Intravenous immunoglobulin (IVIG).
• TPO receptor agonists
• Rituximab
• Fostamatinib
• Immunosuppressants
• Splenectomy
• H Pylori treatment

What are the possible side effects of these treatments?

Patients can respond differently to treatment. If you do have a reaction, it can then be treated quickly. Not everyone gets side effects, but it is important to be aware if any appear so if you feel unwell or have any of the following signs or symptoms, you can tell your clinical team straight away.

Risk of infection

Some of the treatments listed below can increase the risk of infection. This is because they can reduce the number of white blood cells in your blood (neutropenia) meaning you are more likely to get an infection. If you have an infection, it is important to treat it as soon as possible. Speak to your clinical team if you develop unexplained fever (high temperature above 37.5°C) or unexplained low temperature (below 36°C), fatigue (extreme tiredness), sore throat, unexplained diarrhoea (loose stools), ulcers in your mouth or anus, or infection site swelling, pain and rash.

Intravenous immunoglobulin (IVIG)

This is a medicine containing antibodies (immunoglobulin) that is given into a vein, usually in your arm, through a drip (intravenously). Antibodies are produced by white blood cells to fight infections. It is a human blood product, which means that the antibodies have been collected from numerous blood donors. This means that if you have received IVIG you will never be able to donate blood in the UK, even when you recover from ITP.

Nobody understands exactly how IVIG works to treat ITP, but it is thought that the extra antibodies stop your white blood cells from destroying your platelets.

IVIG is given as an infusion (liquid mixture) through a drip over a few hours.

What are the advantages of IVIG?

IVIG works quite quickly, usually within a few days. Unfortunately, the effect does not last long (a few weeks at most) and so it will not cure your ITP. It is generally given before surgery or if you have significant bleeding symptoms and your platelet count needs to be increased urgently.

What are the risks of IVIG?

There is a small risk of a reaction (such as fast heart rate or breathlessness) while the IVIG is being given as a drip, so you will be monitored closely by a nurse. There is also a small risk of developing a rare complication called aseptic meningitis. This causes a headache, neck stiffness and dislike of bright lights. This condition usually gets better on its own, but if you develop any of these symptoms after treatment with IVIG you must seek medical attention immediately.

IVIG can very rarely cause kidney damage and there is also an extremely small risk (less than one in many millions) of infections such as hepatitis and HIV (as it is made from donated blood).

Thrombopoietin receptor (TPOR) agonists: romiplostim (NplateTM), eltrombopag (RevoladeTM), Avatrombopag (DopteleteTM)

Thrombopoietin (TPO) receptors (or sensors in other words) are on the surface of the cells that make platelets in the bone marrow; these drugs use these receptors to tell the cell to make more platelets. The drugs can be used if you have had one previous treatment for ITP.

Romiplostim (NplateTM) is given by an injection under the skin, usually once per week. You can be taught how to give this injection yourself.

Eltrombopag (RevoladeTM) is a tablet that is taken once a day. It cannot be absorbed by the gut if there is calcium nearby, so you must not eat foods high in calcium for four hours before or after you take it. Most people find it easiest to take the medication early in the morning or just before going to sleep. Foods high in calcium include dairy products, cereals, tinned fish with bones and green leafy vegetables. Your pharmacist can give you more information about which foods to avoid.

Avatrombopag (DopteleteTM) is a tablet taken once per day. Unlike eltrombopag it has no dietary restrictions.

You normally take one of these medications until there is a satisfactory platelet count. In some patients it is then possible to reduce the dose or stop altogether after a period of time on the medication if responding well and between 10-30% of patients who stop altogether have long term response.

What are the advantages of the TPOR agonists?

If we gave ten people ITP (that have already been treated with steroids and another treatment) one of these drugs, we would expect eight or nine of them to have some response (their platelet count may increase or they have less bleeding). These effects will continue in the long term in about five of these people.

What are the risks of the TPOR agonists?

Most people have no side effects with these drugs, some people get headaches and a few people taking romiplostim have developed scarring of the bone marrow. The scarring of the bone marrow does not appear to stop the bone marrow from working properly.

There may be a small risk of blood clots (which can be in the legs or lungs, or cause heart attacks or strokes) in people whose platelet count goes up to high levels. Your doctor will monitor your platelet count carefully while you are on this medication, to help avoid this. Sometimes your platelet count can go up and down a lot when you start these medications. This means that you will need frequent blood tests and clinic visits when you start to take this medicine.

Rituximab

Rituximab is a drug that was first used to treat cancer but has also been used for over twenty years to treat ITP. Like steroids, it stops the immune system destroying platelets, but it has fewer side effects than steroids. It is a manufactured antibody (developed by a medicines company) that affects the white blood cells. It is not made from donated human blood.

Rituximab is given as an infusion through a drip (a small tube into a vein in your arm) once a week for four weeks. It takes a couple of hours for the dose to be given. Soon we may be able to give it as a slow-release injection under the skin.

It usually takes a few weeks for rituximab to work, although some people respond many months after treatment. If rituximab works well for you, the treatment can be repeated months or years later, if needed.

What are the advantages of rituximab?

Around two out of three people given rituximab will have some increase in their platelet count. Most people experience further episodes of ITP (their platelet count will drop again) but usually the platelet count stays at a good level for at least a year.

What are the risks of rituximab?

Most people who are treated with rituximab for ITP have no side effects. The most common problem is a reaction to the infusion (such as a fast heart rate or breathlessness), but you will be monitored closely while it is given.

Although rituximab works by stopping the white cells from making antibodies, you are not likely to have any problems with infections. There is an extremely rare viral infection that can affect the nervous system, which a few people treated with rituximab have had. The condition is even rarer among patients with ITP who have received rituximab.

Rituximab Alert card

You will be given an alert card to say that you have received rituximab treatment and an emergency contact number. It is recommended that you carry a biological therapy alert card at all times. Then anyone treating you will know that you’ve had rituximab and that your antibody levels may be low. You can get this card from your clinical team.

See: https://www.cuh.nhs.uk/patient-information/rituximab-treatment-in-autoimmune-disorders/

Fostamatinib (TavlesseTM)

Fostamatinib is a tablet treatment for ITP that works by reducing the removal of platelets by targeting an enzyme called spleen tyrosine kinase which is involved in platelet destruction. It can be taken by patients who have already had a TPOR or if a TPOR is not suitable.

What are the advantages?

Results of clinical trials with patients who had received previous treatment for ITP showed almost half of patients achieved an overall response with almost 1 in 5 a stable response over a longer time period..

What are the main side effects?

Common side effects include diarrhoea and high blood pressure. Other side effects include nausea, dizziness and abnormal liver function tests. Most side effects are mild and respond to reducing or withholding the dose for a period of time or other measures.

Immunosuppressants

These other medications can also be used to treat ITP. Before TPOR agonists, rituximab and fostamatinib became available, they were often used. They are used less often now. They may be tried if you have no response to the newer drugs.

Immunosuppressants are often used in cancer treatment, but the doses used in ITP are much lower than for cancer. They are useful treatments for some people with ITP and do not usually cause many side effects. However, they can stop bone marrow from working properly, which can lead to anaemia and low white blood cell counts. This increases the risk of infection.

Some of these drugs can also affect the kidneys, so you will be closely monitored while you are taking them. Examples of these drugs are azathioprine, mycophenolate mofetil (MMF), cyclophosphamide, vincristine or ciclosporin. All are given as a tablet, except vincristine, which is given as an injection into a vein. If your doctor recommends one of these drugs they will tell you more about it.

Splenectomy (removal of the spleen)

As your platelets are mainly being destroyed when they are in your spleen, removing your spleen can cure the condition. This will be carried out during an operation under general anaesthetic (where you are made to be asleep). The operation can usually be done laparoscopically (using very small cuts to carry out keyhole surgery), which means you should recover more quickly. You will usually be in hospital for a couple of days, although it can take six weeks to fully recover from the operation.

Sometimes the operation needs to be carried out using open surgery (a larger cut). Your surgeon will discuss this with you if they think you are likely to need this type of operation.

You may need some treatment to increase your platelet count before the operation.

What are the advantages of splenectomy?

Splenectomy has been used to treat ITP for decades. In the past studies carried out found that approximately 2 out of every 3 people who had a splenectomy didn’t need further treatment but that was before we had any of the newer treatments like the TPOR and we think that the response rates are lower for people who have had several lines of treatment before. It is difficult to predict whether you will be cured by this operation but sometimes a special scan is performed first which can show whether your spleen is the main place that your platelets are being destroyed. However it cannot completely predict whether you will be cured by the surgery.

What are the risks of splenectomy?

Any surgical operation has risks. If 500 people have the operation by keyhole surgery, one may die because of the operation, either at the time of surgery or from complications happening afterwards. This is nearer to one in 100 people who have open (non-keyhole) surgery.

Other risks include:

• Reaction to the general anaesthetic.
• Excessive bleeding at the time of surgery (which may happen even if you have a normal platelet count).
• Damage to other organs during the operation.
• Infection.

You may be more at risk of complications from surgery if you have other medical conditions, are very overweight, or if it is not possible to increase your platelet count before the operation. Your doctor will discuss your own situation and specific risks with you.

To reduce the risk of long-term infection you will need to have vaccinations (immunisations or “jabs”) before having surgery. After the surgery you may need to take long term low dose antibiotics to help prevent infection, or you may be given a packet of antibiotics to keep at home in case you become unwell. This is because some of the white cells that would normally help your body to fight infection would have been made in your spleen.

You must seek medical advice quickly if you develop symptoms of an infection and you should carry a card to say that you have had your spleen removed in case you are in an accident. Your doctor and surgeon will discuss these details with you.

There is also information produced by the ITP Support Association on splenectomy along with lots of other useful information about ITP and ITP treatments. You can view this via the ITP Support Association website (opens in a new tab)

Helicobacter Pylori treatment

Some people with ITP have an infection in their stomach, known as Helicobacter pylori. Sometimes, treating this infection with antibiotics and antacids for two weeks can cure or improve the ITP. Helicobacter Pylori is diagnosed using a breath test or stool sample. Improvements of platelet count following treatment of the infection are not always permanent, but the treatment is very safe and may be recommended by your doctor.

Why can’t I have platelet transfusions to treat my ITP?

The platelets made by your bone marrow are healthy and it is only because your immune system is destroying them that you have a low platelet count. If you were to receive other people’s platelets (given by transfusion) they would also be destroyed by your immune system. Platelets transfused to you would only last minutes or hours before being destroyed. Platelet transfusions can be useful as an emergency treatment if you have severe bleeding, as they can help you to form a clot, but they are not useful for long term prevention of bleeding.

What about tranexamic acid?

Tranexamic acid is a medication that helps blood clots to last longer once they have formed. The clots are more stable than usual and more resistant to being broken down. Tranexamic acid does not treat ITP but can be useful if you have bleeding while your platelet count is low. It is a tablet that is taken three or four times a day. It should not be taken if you have blood in your urine. It can sometimes cause indigestion, which may get better if you take a lower dose.

What happens now?

This leaflet tells you about different treatments for ITP. Your doctor may have recommended one or more of these treatments. You should discuss any questions you might have about these treatments with your doctor so that you can make a decision together about which one would be appropriate for you. If you need to make an appointment to discuss this information with your doctor, please phone the Haematology specialist nurse.

Contact details

If you have bleeding symptoms, please ask your GP to check your platelet count urgently, or contact:

Haematology Specialist Nurses:

01223 217717

Haematology Day Unit

Tel: 01223 217720

Haematology Secretaries

Tel: 01223 256059 or 0123 274652

Bleeding symptoms may include fresh bleeding from your nose, mouth, in vomit, stools (faeces) or urine, or passing black sticky stools. A purple rash, usually on the ankles and legs, which does not fade when you press it, can also be a sign of a low platelet count. If you have excessive bleeding or bruising, you must go to the Emergency Department at your nearest hospital.

Original document reproduced with kind permission of Oxford University Hospital NHS Trust.

Authors: Suzanne Hall, ST7 Haematology

Mike Murphy, Consultant Haematologist

Sue Pavord, Consultant Haematologist

Oxford University Hospitals NHS Trust

Oxford OX3 9DU

OUH - NHS WEBSITE (opens in a new tab)

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