Sickle cell disease (SCD) and stem cell transplant

Introduction

If you have been told that a stem cell transplant may now be a treatment option for you, you might have lots of questions. There is lots to think about and consider. This webpage should give you plenty of information and support that you can use to help make the best decisions for your own health and future.

Your medical team should know your unique situation best, but we hope this information can guide you through this potentially tricky process.

This information is also available as a leaflet. You can download it now (PDF 393KB) or order free copies from our publications page.

What is sickle cell disease (SCD)?

Sickle cell disease is a group of inherited blood disorders that affect the red blood cells in your body. It mostly affects people from African and Caribbean backgrounds and can be diagnosed soon after you are born — or even earlier during pregnancy. You can find out more about sickle cell disease on the NHS and Sickle Cell Society websites.

People with sickle cell disease produce red blood cells in the shape of a ‘sickle’. These unhealthy cells stick together and get stuck in blood vessels. This blocks the flow of healthy blood cells, which can cause various health complications and acute pain episodes (sometimes called sickle cell crises).

For many people, this pain can be severe. It can affect their day-to-day life and result in multiple stays in hospital. People with severe sickle cell disease can also be at risk of serious complications, which could result in death.

There are treatments for sickle cell disease but not all people respond well to these treatments.

Can I have a stem cell transplant if I have sickle cell disease?

Yes, you might be able to have a stem cell transplant to cure your sickle cell disease. You can read more about what a stem cell transplant involves in the Understanding stem cell transplants section on our website. This treatment has been an option on the NHS for adults in the UK since 2020.

You are likely to only be offered a stem cell transplant if the benefits outweigh the risks. In this case, you may have severe sickle cell disease which:

• cannot be controlled with pain management or other treatments
• means you spend a lot of time in hospital to treat your acute pain episodes
• affects your quality of life so you cannot go to work, take part in education, socialise, or carry out normal day-to-day activities
• causes or puts you at risk of complications such as a stroke, organ dysfunction (where your organs don't work properly), acute chest syndrome (a lung condition causing chest pain and breathing difficulties), and infections
• puts you at risk of death.

You can only have a stem cell transplant if you have a matched related donor — a brother or sister who is a full match and can donate their stem cells to you. Your sibling can still donate their stem cells if they have the sickle cell trait, but not if they also have sickle cell disease.

There isn’t enough evidence yet to support having a matched unrelated donor, like someone from the Anthony Nolan stem cell register, or a haploidentical donor (a family member who is a half-match). This may change in the future as more research is carried out and more improvements are made in the transplant process.

You can always ask your medical team if a stem cell transplant might be a treatment option for you. If it hasn’t been brought up in any appointments and you’re interested to find out more, do ask for more information.

Remmy's story

Remmy (Elizabeth), from Essex, had a stem cell transplant in 2021 to cure her sickle cell disease. Watch her story to find out about her experience. You can find chapters below the video.

What are the risks and benefits?

A stem cell transplant can cure blood cancers and blood disorders, like sickle cell disease, which is simply fantastic. But a transplant is an intensive treatment which comes with lots of risks and benefits to consider.

For someone with a blood cancer, like leukaemia, a stem cell transplant can be their last chance of survival. Therefore, patients and their medical teams are likely to take more risks as it is a final treatment option.

For people with sickle cell disease, there are more risks to consider as it may not be a treatment option for survival. It will more likely be a treatment option to have a better quality of life. So it’s important for you to consider all the risks and benefits with your medical team to see if this is the best option for your individual health and future.

Benefits

• A stem cell transplant can cure your sickle cell disease.
• You can expect your pain crises to stop but this may take some time. You might experience chronic pain due to the effects of your sickle cell disease. Your pain management will be carefully controlled after transplant if this applies to you. This will all depend on your individual situation.
• A stem cell transplant should prevent further complications caused by your sickle cell disease, like problems with your eyes and organ dysfunction.
• You can have a better quality of life. You may be offered a stem cell transplant if your severe sickle cell disease affects your day-to-day life and you don't response well to any other treatments.

Risks

• Any current complications you have from your sickle cell disease, like liver failure or heart conditions, could put you at risk during a stem cell transplant. Your medical team can talk you through how well your body could cope with a transplant.
• Graft versus host disease (GvHD) is a potentially life-threatening side effect of a stem cell transplant. This is where your new stem cells start to attack other cells in your body. You can read lots more about GvHD on our webpage. GvHD won't always be life-threatening and the risk of GvHD is much lower for people with sickle cell disease. But it is still a risk to consider.
• You will need to have conditioning therapy before your transplant. Currently, for people with sickle cell disease, this is made up of an immunosuppressive drug and low dose radiotherapy. Conditioning therapy prepares your body for your new stem cells and you're more at risk of infection during this time. However, sickle cell patients receive a less intense form of conditioning therapy which lessens the risk of complications. You can read more about conditioning therapy on our webpage.

What are the other treatments for sickle cell disease?

A stem cell transplant is currently the only treatment that can cure sickle cell disease. But there are other treatments available which can help to manage your symptoms.

• Pain management can ease symptoms and help to give you a better quality of life.
• Long-term antibiotics, like penicillin, can help to prevent infections.
• Long-term blood transfusions can help to increase oxygen being carried around your body, which can prevent strokes and acute chest syndrome, as well as reduce pain episodes.
• Red Cell Exchange is similar to a blood transfusion but removes your red blood cells first, before replacing them with red blood cells from a donor.
• Hydroxycarbamide is a drug which can help to reduce the number of pain episodes and other complications.
• Voxelotor is a drug which can help to reduce the need for blood transfusions.

Not all people with sickle cell disease can have these treatments, and they come with their own side effects. If your sickle cell disease is severe and you aren’t able to have these treatments, or they simply don’t work, you may be offered a stem cell transplant.

You can find out more about all treatments for sickle cell disease on the NHS website.

What happened to crizanlizumab (Adakveo)?

Crizanlizumab (Adakveo) was a drug treatment introduced in 2020. It has now, since 10 January 2024, been withdrawn. This is due to concerns about how effective the drug is in reducing painful crises. This means no new patients will be prescribed with crizanlizumab. However, this does not mean the treatment is unsafe.

If you are currently taking crizanlizumab, please speak to your medical team to discuss alternative treatment options.

You can read more about crizanlizumab being withdrawn on the Sickle Cell Society website.

Adewale's story

Adewale, from Cambridge, had a stem cell transplant after having a severe stroke caused by sickle cell disease. Watch his story to find out more about his journey. You can find chapters below the video.

Why could I not have a stem cell transplant before 2020?

Adults with sickle cell disease could not have a stem cell transplant before 2020 as there was not enough evidence to prove that it was a suitable treatment option.

People with sickle cell disease are more likely to pick up infections and have other health conditions. This can put them at higher risk of complications during or after a stem cell transplant.

Thankfully, there have been recent improvements in transplant procedures. For example, the conditioning therapy someone with sickle cell disease needs to have before their transplant is now less intense. This means there is now less risk of complications if someone with sickle cell disease has a stem cell transplant.

There have also been more research studies. This evidence shows that if you have a sibling who is a full match and can donate their stem cells, a stem cell transplant can be a successful treatment option for someone with sickle cell disease.

Iggie's story

Iggie had a stem cell transplant in the USA in 2019 to cure her sickle cell disease. Watch her story to find out more about her 'tough but rewarding' stem cell transplant journey. You can find chapters below the video.

What else do I need to think about when considering a stem cell transplant?

There are a number of things to consider when it comes to having a stem cell transplant. The below may be things you want to think about for your own situation and bring up with your medical team.

• Currently, you can only have a stem cell transplant to cure your sickle cell disease if you have a matched related donor (a brother or sister). If your brother or sister is a full match, that's fantastic. But they still need to be happy and agree to donate their stem cells for you. You can read more about donating your stem cells to a relative on our webpage.
• A stem cell transplant can cure your sickle cell disease. However, you will still carry the sickle cell gene. This means you could still pass it on to any children you have after transplant. You and your partner can have a test before getting pregnant to find out how likely it is that your child will have the sickle cell gene. Your GP can help organise this test.

• Having a stem cell transplant can be a long, challenging process. You will need to have conditioning therapy beforehand, and spend a long time recovering afterwards. Read more about recovering from a stem cell transplant on our recovery webpages.
• Consider your individual situation and current risk to your life. Have a chat with your medical team to discuss the risks to your life if you don’t have a stem cell transplant, compared to the risks to your life if you do.

• Is your sickle cell disease having a negative effect on your day-to-day life? If other treatments are not working, or aren’t giving you the quality of life you deserve, it may be worth considering a stem cell transplant.

Key facts about sickle cell disease

• About 15,000 people in the UK have sickle cell disease.
• Almost 300 babies each year in the UK are born with sickle cell disease.
• You can have a blood test during pregnancy (as well as before — see above) to check if your baby is at risk of having sickle cell disease.
• When your baby is born, they can have a sickle cell screening as part of their newborn blood spot test.
• Sickle cell disease mainly affects people from African and Caribbean backgrounds.

FAQs

Where can I find more information and support?

The Anthony Nolan Patient Services team is here for you before, during and after your stem cell transplant. We can answer questions you have about the process, support with any worries you have, and help to give any practical help you need.

You can call our helpline on 0303 303 0303 or email patientinfo@anthonynolan.org. Our opening hours are 9am to 5pm, Monday to Friday.

• The Sickle Cell Society supports and represents people with sickle cell disease. You can find lots of information and advice on their website, including their A Parent’s Guide to Sickle Cell Disease. You can also get in touch with their helpline or mentoring scheme to share experiences, and find other support groups and local services.
• ACLT (African Caribbean Leukaemia Trust) is a charity spreading awareness of donor registers, including the stem cell register. They particularly encourage people from the Black and Black mixed-race community to become potential donors. They also assist people with sickle cell disease who need matched blood for transfusions or blood exchanges. You can find information and support, and get involved in their work, on their website.
• The NHS also has information about sickle cell disease, including symptoms, treatments, and living with sickle cell disease, on their website.