For people with sickle cell disease, a stem cell transplant may now give hope of a cure.
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.
- What is sickle cell disease (SCD)?
- Can I have a stem cell transplant if I have sickle cell disease?
- What are the risks and benefits?
- What else do I need to think about when considering a stem cell transplant?
- Where can I find more information and support?
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.
Iggie had a stem cell transplant in the USA in 2019 to cure her sickle cell disease. Watch her video below to find out her honest thoughts and feelings on what it was like to have a stem cell transplant.
If you would like to share your own story of having a stem cell transplant to cure sickle cell disease, please email firstname.lastname@example.org or call 0303 303 0303. We, and others in similar situations, would love to hear from you.
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.
Even though having a stem cell transplant can cure your sickle cell disease, you will still carry the sickle cell gene. This means that you can still pass it on to your children even after your transplant.
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.
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.
- 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.
- 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.
A stem cell transplant will not necessarily improve any complications caused by your sickle cell disease. You may still need treatment for these complications after transplant.
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.
- Crizanlizumab is a new treatment which, like hydroxycarbamide, can help to reduce the number of pain episodes. Currently, it is only available through a Managed Access Agreement. You can find out more about this on the NICE website.
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.
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.
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.
I thought only cancer patients had a stem cell transplant. Is it that serious?
A stem cell transplant may be a treatment option for someone who has a blood cancer, like leukaemia, or a blood disorder, like sickle cell disease. In the future, it might even be an option for someone with a different kind of condition, like Parkinson’s.
So, even though people with cancer do have stem cell transplants, it does not mean that you also have cancer or are on the same journey. Parts of the treatment that you may associate with cancer, like chemotherapy and radiotherapy, are different for sickle cell disease patients.
Having a stem cell transplant can be a long and tough journey. It’s not an easy treatment. But there is lots of support available from Anthony Nolan, your medical team, clinical nurse specialists, and other charities.
Will having a stem cell transplant affect me having children?
Fertility issues and early menopause can be side effects which may be a big factor in your decision whether or not to have a stem cell transplant. Conditioning therapy before transplant can cause difficulty in conceiving without some professional support, like IVF.
If your conditioning therapy doesn't include chemotherapy, you will be at lower risk of fertility issues.
Your medical team will talk about your risk of fertility issues with you. If you are interested in freezing your eggs, sperm or embryos for the future, they can talk you through this option.
You can read more about fertility issues and early menopause on our Bigger Issues webpage.
I'm worried about a stem cell transplant affecting my mental health
Having a stem cell transplant can be tough, both physically and mentally. You will have to spend lots of time on your own in isolation, which some people can find hard. You might also have some setbacks in your recovery, or might feel anxious about returning to work or school, changes in your body, picking up an infection, or the effect your transplant could have on your relationships. You can read our Recovery: Mind section on our website for more information and support.
It’s very common to find the transplant journey hard. Thankfully, there are lots of people to support you. You are not alone. From the Anthony Nolan helpline and Telephone Emotional Support Service, to clinical psychologists and financial grants, there are plenty of people and resources in place to support you throughout your transplant journey. We’re here for you.
How long will I take to recover after a transplant?
Everyone’s transplant recovery journey is unique. It’s hard to say how long you will take to recover.
Recovery can also mean different things for different people. Some people might be most keen about going back to work, but some may just want to leave hospital. You need to do things in your own time and remember that you may have some setbacks throughout your recovery. Setbacks are common so you need to be patient with yourself and your new immune system.
You may be able to leave hospital a few weeks after your transplant, but you will need lots of hospital check-ups. Your immune system will be like a newborn baby’s for a year or two after your transplant, so you need to be careful about the risk of infection for a while.
Important: You may not have to be admitted to hospital for your stem cell transplant. This is called ambulatory care. In this case, you will be treated as an outpatient (you’ll only go to hospital for your appointments and treatment) and will be carefully monitored at home otherwise.
You might still experience pain for a while after transplant. This should ease in time, and your pain management will be carefully controlled, but you may need to manage your expectations. You may also still experience complications caused by your sickle cell disease before your transplant. A stem cell transplant can cure you, but you may need to go easy on yourself while your body gets used to its new life.
How much time will I need to take off work?
You’ll likely need to take at least six months to a year off work. Exactly how much time you need to take off work, or from education, will entirely depend on your individual situation and how you are recovering.
It will also depend on the type of work or education you do. For example, being able to work from home means you will be at much less risk of infection. But working from home can also be tiring, so you must not rush back to work or education until you are fully ready.
You can read more about going back to work after a stem cell transplant on our webpage. You can also download our booklets for employees and employers:
Can I get any financial support?
Yes, there is support available to help ease any money worries you may have. It’s common to feel anxious about your financial situation. It’s a big reason some people rush into going back to work after transplant. But we’re here to help in any way we can.
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 email@example.com. 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 sickle cell patients 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.
Information published: 25/08/22
Next review due: 25/08/23