What is eltrombopag?

Eltrombopag is one of the newer treatments for aplastic anaemia. It is a thrombopoetin agonist, which means it works like the normal hormone that the body makes (called thrombopoetin) that stimulates the bone marrow to make platelets. It is likely that eltromobag works by stimulating the bone marrow stem cell (the seed cell of the bone marrow) but the complete mechanisms of action in aplastic anaemia are still being determined. Eltrombopag is a tablet which is taken at home.

What are the response rates?

Expected response rates in recent clinical trials in aplastic anaemia are between 35-70% ranging from a response in a single blood line (i.e. platelets) to a response in all three blood cell types (white cells, red cells and platelets). We would expect patients to respond to eltrombopag within 16 weeks, but it can be earlier than this. Your team will discuss whether you need a bone marrow sample to see what your bone marrow cells look like before/during treatment with eltrombopag.

Where is eltrombopag used in the treatment for aplastic anaemia?

Currently eltrombopag is not used in first line treatment for patients with severe aplastic anaemia, it is used when they have not responded to their first or subsequent treatment options. Your doctor will discuss treatment options with you as to where in the treatment algorithm eltrombopag is advised. The preferred dose of eltrombopag is 150mg once a day. It may require your medical team to complete an individual funding request to your local health authority. This may change in the future.

Stopping eltrombopag

If you have responded to the drug and this is sustained for more than 8 weeks, your dose will be reduced with a view to stopping it slowly. However, it is not safe to stop treatment without medical advice.

Dietary advice

Calcium affects the absorption of eltrombopag. Therefore, it is important to take eltrombopag at least two hours before or four hours after taking either calcium tablets or dairy products. The easiest time to take it may be before going to bed depending on your dietary habits. Convenient, once every 24 hours dosing. You do not need to follow a low-calcium diet if you take eltrombopag at least 4 hours after eating.

Common side effects

Eltrombopag is usually well tolerated, however common side effects include:

• Impaired liver function: you will receive close monitoring of your blood levels at the start of treatment
• There is a small risk of blood clots: your doctor will discuss this with you
• Darkening of skin tone: this is not harmful
• Nausea
• Skin rash
• Headache
• Muscle aches
• Small increased risk of cataracts - you should have regular eye examinations

Useful facts

• Patients with East Asian ancestry require a reduced starting dose of eltrombopag due to differences in the metabolism of the drug
• The treatment dose of eltrombopag in aplastic anaemia is 150mg a day, this is different from other conditions that it is used for

Patients’ experiences

My medical team told me that Eltrombopag works best on a completely empty stomach and so I was advised to take it at 3am. That was hard, but as I was part of the initial UK trial back in 2018 I wanted to give it all my effort and the best shot to make it work. - Gareth

I tolerated Eltrombopag fairly well. My face went a bit tanned but other than that, no issues. The main drawback for me was setting an alarm to take it at 2am every day to fit with my other medication and eating habits! - Claire

I was delighted when I was offered to take Eltrombopag as it’s not always available for everyone. Within a couple of weeks my platelets started to rise and other than having to get up through the night to take it and yellow eyes it was ok, very little side effects. - Zoe

Thank you to

Colleagues at the St James' Hospital Haematology Team, Leeds for their enthusiastic help and expertise in developing and reviewing this booklet. 

You can order this information as a printed booklet from our shop, or download it as a pdf here

Eltrombopag for the treatment of aplastic anaemia