Being diagnosed with blood cancer or a blood disorder can be a stressful and worrying time for both you and your loved ones. Many people become anxious about an uncertain future, but others can feel a sense of relief that their recent health problems have been recognised and treatment can start. Everybody reacts to news like this in their own way and it can take some people longer than others to come to terms with their situation.
You might have had a recent diagnosis and you’re now looking for information about different treatments. You might be a concerned partner, family member or friend who is trying to plan ahead for the future. No matter what your situation, we are here to help and answer your questions about stem cell or bone marrow transplants.
This section focuses on some of the more common types of blood cancers and blood disorders. It will give you a better understanding of your situation and hopefully answer some of your most important questions including:
Your blood is made up of different cell types including red blood cells for carrying oxygen, platelets to help blood clot and white blood cells that fight infections. They all originally come from stem cells, which have the potential to develop into any type of blood cell as they divide and mature. Problems in this process, known as ‘differentiation’, are at the root of all blood cancers. Different types of blood cancer depend on when and how these problems occur.
These problems often lead to your body producing large numbers of immature blood cells that can’t perform their job properly. They can also ‘clog up’ your bone marrow, which prevents other types of blood cells from doing their job too.
Blood Cancer Types
Acute myeloid leukaemia (AML) occurs in your bone marrow, when a type of blood cell called a myeloid cell starts growing abnormally and in an uncontrolled way. Normally your blood stem cells develop into myeloid cells, which then become red blood cells, platelets and certain white blood cells (called neutrophils).
If you have AML, your myeloid cells can’t fully mature and they remain as young, immature cells called myeloblasts. These cells divide too quickly which can ‘clog up’ your bone marrow and stop other blood cells from doing their job properly too.
AML is ‘acute’ because it usually develops quickly, sometimes within a few days or weeks. There are different types of AML – your symptoms will depend on the type you have, the number of leukaemia cells in your body, and where they are.
AML can be classified into ‘high’, ‘standard’ and ‘low’ risk – but this only relates to the type of treatment you will be given, not to the chance of it working.
AML facts
Name: Acute Myeloid Leukaemia (AML)
Cell type: Myeloid cells
Risk: More common in people over 70
Treatment: Chemotherapy and sometimes a stem cell transplant
Other information: AML symptoms develop quickly; treatment often starts straight away
AML and stem cell transplants
If you have AML, it is likely that you’ll need to start treatment quickly after you’re diagnosed. You’ll normally be offered chemotherapy as your main treatment option, although this can vary because certain types of AML are treated differently.
Normally, after chemotherapy, your doctors will consider further treatment to stop the leukaemia coming back. This is where a stem cell or bone marrow transplant comes in. A high dose of chemotherapy, followed by a stem cell transplant, may be the best way to stop the cancer coming back.
Most transplants for AML are allograft transplants – when stem cells are donated to you by someone else. It’s much rarer for AML to be treated with an autologous transplant – when doctors use your own stem cells. This is usually only considered if a stem cell donor cannot be found and your medical team decide it’s a better option than other non-curative treatments.
Acute lymphoblastic leukaemia (ALL) is a blood cancer that stops the white blood cells of your immune system growing properly. It involves blood cells called lymphocytes that develop from stem cells in your bone marrow.
Before these cells are fully formed, they’re called lymphoblasts (immature lymphocytes). Your body needs to make new lymphocytes – but when you have ALL, this process doesn’t work properly. Instead, the lymphoblasts grow too quickly and cannot function properly. These cells can also ‘clog up’ your bone marrow and prevent it from making other blood cells.
You will probably hear ALL referred to as either ‘B cell’ or ‘T cell’ ALL. This relates to the type of lymphocyte that is affected, and can help your doctor decide on the best course of treatment.
Some types of ALL are characterised by the ‘Philadelphia Chromosome’, which is also found in chronic myeloid leukaemia (CML). The presence of this genetic alteration enables doctors to give Tyrosine Kinase Inhibitors (TKIs), a targeted treatment that should be effective.
Anybody can get ALL, but it’s the most common type of childhood cancer. It’s an acute condition, meaning symptoms can develop quickly – so it’s very important that treatment is started as soon as possible.
ALL facts
Name: Acute Lymphoblastic Leukaemia (ALL)
Cell type: Lymphocytes, a type of white blood cell
Risk: Nearly half of all ALL patients are under 10 years old
Treatment: A combination of chemotherapy drugs and possibly a stem cell transplant
Other information: ALL is more common in men than women
ALL and stem cell transplants
In some cases ALL can be treated with chemotherapy alone – however, depending on the type of ALL you have, how it responds to treatment and your general health, you may be offered a stem cell transplant. Your doctor will talk to you about whether a transplant is the best option for you or your child.
If you’re a parent who’s supporting a child through a transplant, we have more advice here.
Most transplants for ALL are allograft transplants – when stem cells are donated to you by someone else. It’s very rare for ALL to be treated with an autologous transplant – when doctors use your own stem cells. This is usually only considered if a stem cell donor cannot be found and your medical team decide it’s a better option than other non-curative treatments.
Chronic myeloid leukaemia (CML) affects a type of white blood cell called a myeloid cell. These cells are a middle step between stem cells and fully-formed white blood cells.
People with CML produce too many granulocytes, a specific type of myeloid cell, that are not fully formed. This is why you may hear CML referred to as ‘chronic granulocytic leukaemia’ (CGL). Over time, these abnormal cells fill the bone marrow, which reduces the number of normal cells in the blood.
CML is not classified into different stages, but it does have three recognised phases: ‘chronic’, ‘accelerated’ and ‘blast crisis’. Very few patients progress beyond the chronic phase today due to the effectiveness of modern treatment.
CML is defined by the presence of a ‘Philadelphia Chromosome’ which forms when parts of your DNA are exchanged between two chromosomes. This causes two different genes (called ‘ABL’ and ‘BCR’) that are normally completely separate, to fuse together and promote uncontrolled cell growth.
CML facts
Name: Chronic Myeloid Leukaemia (CML)
Cell type: Myeloid cells, in particular granulocytes
Risk: More common in people over 65
Treatment: Tyrosine Kinase Inhibitors (TKI) and possibly a stem cell transplant
Other information: TKIs allow most CML patients to live long and healthy lives
CML and stem cell transplants
Most patients with CML are diagnosed during the chronic phase, when treatment with a type of drug called Tyrosine Kinase Inhibitors (TKIs) is possible. Some TKIs are designed to stop cancer cells growing by targeting the Philadelphia Chromosome.
Stem cell transplants are only recommended:
– if you have CML that hasn’t responded to TKIs, or
– if you are diagnosed with ‘blast crisis’ CML and treatment gets you back to ‘chronic’ phase CML.
If you do need a transplant, it will be an allograft transplant – when stem cells are donated to you by someone else.
Chronic lymphoblastic leukaemia (CLL) is a blood cancer that stops the white blood cells of your immune system growing properly. It involves blood cells called B lymphocytes that develop from stem cells in your bone marrow.
Before these cells mature into lymphocytes, they’re called lymphoblasts (immature lymphocytes). Your body needs to make new lymphocytes – but when you have CLL, this process doesn’t work properly. Instead, the immature cells grow too quickly and cannot function properly. Over time, they accumulate in the lymphatic system and may cause large, swollen lymph nodes. They also fill the bone marrow, reducing the number of normal white blood cells, red blood cells and platelets that can be made.
CLL is a chronic condition that usually develops very slowly – many people don’t need treatment for months or years. However, some people may need to have treatment straight away.
CLL facts
Name: Chronic lymphoblastic leukaemia
Cell type: B Lymphocytes, a type of white blood cell
Risk: More common in people over 70
Treatment: Chemotherapy or immunotherapy (when drugs stimulate the immune system) may be needed but not always straight away
Other information: CLL progresses very slowly and very few people will need a stem cell transplant
CLL and stem cell transplants
You will normally only be offered a bone marrow or stem cell transplant if your CLL hasn’t responded to initial treatment, or if your doctors think it’s unlikely to.
Most transplants for CLL are allograft transplants – when stem cells are donated to you by someone else.
It only happens very rarely, but some people with CLL have an autologous transplant – when doctors use your own stem cells.
Other types of leukaemia include:
If you have lymphoma, it means your body is making too many lymphocytes, which is a type of white blood cell. Your lymphocytes also live longer than they should. This overload compromises your immune system and stops other cells in your blood from doing their job. There are two types of lymphocytes, B cells and T cells. Non-Hodgkin Lymphoma can develop from T cells, but a B cell origin is more common.
Lymphomas are grouped into stages based on how far they have spread in the body. This enables doctors to give the most effective treatment, because lymphomas of different stages will respond differently. In general, staging is applied to all types of lymphoma; however different systems are used for skin lymphomas and non-Hodgkin lymphomas in children.
Non-Hodgkin lymphoma refers to any lymphoma that doesn’t have abnormal cells called “Reed-Sternberg” cells. These distinct, large cells are only seen in blood samples of patients with Hodgkin lymphoma.
Name: Non-Hodgkin lymphoma
Cell type: Lymphocytes, white blood cells that fight infections
Risk: More common in elderly people – 50% of cases are in people over 70
Treatment: Chemotherapy, radiotherapy and a stem cell transplant
Other information: Classified into ‘high’ and ‘low’ grades based on how quickly they develop
Non-Hodgkin lymphoma and stem cell transplants
Doctors will normally only offer you a transplant if you’ve had other treatment and the lymphoma has come back. You’re more likely to have an autologous transplant – when doctors use your own stem cells. This provides the best chance of keeping your lymphoma in remission for longer, and causes fewer complications, because a stem cell donor isn’t needed.
You may then have a second transplant if you relapse. This will be an allograft transplant – when stem cells are donated by someone else.
Your medical team may consider offering you anallograft transplant straight away if:
If you have lymphoma, it means your body is making too many lymphocytes, a type of white blood cell. Your lymphocytes also live longer than they should. This overload compromises your immune system and stops other cells in your blood from doing their job too. There are two types of lymphocytes, B cells and T cells; Hodgkin lymphoma (sometimes called Hodgkin’s disease) develops from B cells.
Lymphomas are grouped into stages based on how far they have spread in the body. This enables doctors to give the most effective treatment because lymphomas of different stages will respond differently. In general, staging is applied to all types of lymphoma – however, there are a few exceptions. A different system is used for skin lymphomas.
Hodgkin lymphoma can be identified by the presence of abnormal cells called ‘Reed-Sternberg’ cells. These cells have a distinct large shape when looked at under a microscope.
Name: Hodgkin lymphoma
Cell type: B cells, a type of white blood cell
Risk: Most common in people aged between 15-25, and over 50
Treatment: Chemotherapy, radiotherapy and a stem cell transplant
Other information: Defined by the presence of Reed-Sternberg cells
Hodgkin lymphoma and stem cell transplants
Doctors will normally only offer you a transplant if you’ve had other treatment first and the lymphoma has come back. You’re more likely to have an autologous transplant – when doctors use your own stem cells. This provides the best chance of keeping your lymphoma in remission for longer, and causes fewer complications, because a stem cell donor isn’t needed.
You may then have a second transplant if you relapse. This will be an allograft transplant – when stem cells are donated by someone else.
Your medical team may consider offering you an allograft transplant straight away if:
You have a type of lymphoma that doctors feel isn’t likely to respond well to normal chemotherapy
Stem cells divide and mature into the different cell types that make up our blood. This includes red blood cells (for carrying oxygen), platelets (to help blood clot) and white blood cells (for fighting infections). If you have a blood disorder, it’s likely that there is a problem with a stage in this process. This means that some of the cells in your blood cannot properly perform the job they are designed to do.
Many of these problems occur because of a faulty gene that was inherited at birth. However, others, such as myelodysplastic syndromes and autoimmune diseases, are not inherited and can occur without an obvious reason.
There are lots of different blood disorders and they are treated in different ways depending on their type and severity. A few of them can develop into blood cancers over time. Only some people with blood disorders will need a stem cell transplant – usually only if other treatments have been unsuccessful.
Blood Disorder Types
Myelodysplastic syndromes (MDS) are a group of blood disorders that cause your bone marrow to produce too many blood cells. These cells are often unhealthy (dysplastic) and are destroyed soon after leaving the bone marrow. People with MDS feel very tired, weak and bleed or bruise more easily because they cannot produce enough healthy blood cells.
Although MDS are not cancers themselves, they can sometimes develop into acute myeloid leukaemia (AML). All types of MDS are placed into low and high risk groups based on how likely this is to happen. It also helps your doctor select the most effective treatment option for you.
MDS facts
Name: Myelodysplastic Syndromes (MDS)
Cell type: Bone marrow and its production of blood cells
Risk: More common in people over 70
Treatment: Blood transfusions and possibly a stem cell transplant in aggressive cases
Other information: Not classed as a blood cancer but can develop into acute myeloid leukaemia (AML)
Stem cell transplants and MDS
If you have low risk MDS, you may not need treatment straight away but regular blood transfusions and medication can help manage your symptoms. Some people with more severe MDS have chemotherapy and a small number of people may need to have a stem cell transplant. This will be an allograft transplant – when your new stem cells are donated by someone else.
If you have aplastic anaemia, your bone marrow doesn’t make enough blood cells, including red blood cells that transport oxygen to the rest of your body. This leads to anaemia, which can cause fatigue, headaches and concentration problems – alongside more serious issues.
Most cases of aplastic anaemia are acquired, meaning that it develops after an event such as viral infection or taking certain medications. The main cause of developing aplastic anaemia is after an autoimmune reaction, where your immune system targets your own cells. Aplastic anaemia can also be inherited at birth, through faulty genes passed on by your parents, although that is much rarer.
Aplastic anaemia facts
Name: Aplastic anaemia
Cell type: Red blood cells
Risk: More common in 10-20-year olds and people over 60
Treatment: Blood transfusions and immunosuppression; a stem cell transplant may then be needed
Stem cell transplants and aplastic anaemia
If you have severe aplastic anaemia, your doctors may decide that you need a stem cell transplant, especially if other treatments haven’t helped. This will be an allograft transplant – when your new stem cells are donated by someone else.
Children with aplastic anaemia are more likely to need a stem cell transplant. If you’re a parent who’s supporting a child through a transplant, please read our page for parents.
If you have a sickle cell disease your blood is unable to carry oxygen around your body effectively. This is because your red blood cells are an abnormal shape (sickle or crescent-shaped) instead of disc-shaped – so they can’t bind as much oxygen as a normal cell. This will often leave you feeling tired, short of breath and prone to infections.
Sickle cell diseases are genetic. To be affected, both your parents need to have the sickle cell gene. If you get the gene from one parent, you will be a carrier (also known as sickle cell trait) but you won’t have any symptoms. The diseases are more common in people of African and Mediterranean descent.
Sickle cell diseases facts
Name: Sickle cell disease (SCD)
Cell type: Red blood cells
Risk: Inherited from your parents
Treatment: Blood transfusions and, rarely, stem cell transplants
Other information: Pregnant women are routinely offered screening to see if they are a carrier
Sickle cell disease and stem cell transplants
A stem cell transplant would only be considered if other treatments have not been successful.
It is rare for adults with sickle cell disease to be considered for stem cell transplant. It would usually only be considered if you have severe sickle cell disease and if it’s possible for you to have a sibling transplant (where your new stem cells are donated by your brother or sister).
Children with sickle cell disease are considered for stem cell transplant more often than adults. This would most likely be a sibling transplant but it could also be a haploidentical transplant (where stem cells are donated by one of your parents) or an allograft transplant (where your new stem cells are donated by an unrelated donor).
All autoimmune diseases are caused by an overactive immune system that targets your own cells rather than foreign bodies such as bacteria or viruses. There are over 80 known autoimmune diseases including type I diabetes, rheumatoid arthritis and lupus. Not all autoimmune diseases can be treated with a stem cell transplant, but they can help people with systemic sclerosis and multiple sclerosis, among other conditions.
Although autoimmune diseases are more common in some families, they are not inherited directly (like primary immunodeficiency diseases). They can also be caused by environmental factors, and can sometimes develop after an infection.
Autoimmune diseases and stem cell transplants
A stem cell transplant would only be offered as an alternative treatment if other options have been unsuccessful or if your symptoms are becoming unmanageable.
It will likely be an autologous transplant – when doctors use your own stem cells. Some conditions can be treated with an allograft transplant – where new stem cells are donated from an unrelated donor, but this is rare.
Please speak to your transplant team for more information about your own situation, as they will be able to give you personalised, specific advice.
If you have thalassemia, you have an abnormal type of haemoglobin – the protein in red blood cells that carries oxygen. This destroys more of your red blood cells than normal, leading to anaemia, which can cause fatigue, headaches and concentration problems. Sometimes it can cause more serious symptoms like shortness of breath.
Thalassaemia is hereditary, meaning that the genes that cause it are passed on from your parents. Roughly 7% of people are thalassaemia carriers (also known as thalassaemia trait) but it’s only passed on to children if both parents are carriers.
Thalassaemia facts
Name: Thalassaemia
Cell type: Red blood cells
Risk: Inherited from your parents
Treatment: Blood transfusions and chelation therapy; stem cell transplants are considered in very rare situations
Other information: Pregnant women are routinely offered screening to see if they are a carrier
Thalassaemia and stem cell transplants
A stem cell transplant can help to treat some people with thalassaemia, especially children, although they are only performed very rarely.
If you’re a parent who is supporting a child through a transplant, please read our page for parents for more advice.
If a stem cell transplant is an option for you, it will likely be an allograft transplant – where your new stem cells are donated from an unrelated donor.
There are many inherited conditions (also known as genetic disorders) that can affect your blood and bone marrow.
People inherit these conditions through the genes they receive from their parents. They are often very rare and can sometimes be identified from a parent or newborn’s blood sample. However, some conditions remain undetected at birth and do not develop until a little later in life. They include mucopolysaccharide and related diseases (MPS), Wiskott-Aldrich syndrome and chronic granulomatous disease.
There are two main groups of inherited conditions: Primary immunodeficiency diseases (PID) and inborn errors of metabolism (IEM).
There are many different types of PID, with different symptoms, but they all share one similarity – they cause problems with the development of the immune system. Your immune system protects you from infection, so if you have a PID, you are more prone to infections.
Treatment for PIDs aims to control your symptoms and minimise the effect they have on your quality of life. A stem cell transplant may be considered as a curative treatment if your symptoms become unmanageable.
This is a very rare group of inherited diseases that cause problems with your metabolism (the chemical reactions that occur in the cells of your body, which allow your body to break down nutrients and create energy). If you have an IEM, your metabolism doesn’t work as well as it could, which can cause serious health problems.
Inherited conditions and stem cell transplants
A stem cell transplant will probably be offered as an alternative treatment if other options are unsuccessful. Many inherited conditions are detected at birth, which means stem cell transplants may have to be given to small children.
If you are a parent who’s supporting a child through a transplant, please read our page for parents for more advice.
The stem cell transplant will likely be an allograft transplant – where new stem cells are donated from an unrelated donor. A small number of conditions can be treated with an autologous transplant – when doctors use a patient’s own stem cells.
Please speak to your transplant team for more information about your own situation, as they will be able to give you personalised, specific advice.
The What are my treatment options? section looks at other possible ways that your medical team may decide to treat your blood disorder – either before or instead of a stem cell transplant.
A stem cell transplant is one of many possible treatment options for blood cancers and blood disorders. Some of these treatments may provide a cure for your condition while others will help to relieve your symptoms on a day-to-day basis.
There are lots of common questions around treatments. Here we provide some of the answers and link to websites where you can find more information.
When it comes to deciding on the best treatment, there are many factors that need to be considered, including:
Some conditions can be managed through non-curative treatments such as regular blood transfusions – especially if you have a blood disorder. If you have a blood cancer you might be given chemotherapy or radiotherapy in the first instance to get your body into remission.
A stem cell transplant will be considered if treatments have been unsuccessful, your symptoms are becoming difficult to manage or if there is a high risk of relapse.
Your medical team will be able to give you specific and personalised advice about your own situation.
All chemotherapy drugs are designed to target and destroy dividing cells. Cancerous or abnormal cells divide more quickly than normal cells and so chemotherapy is more effective against them. Unfortunately, these drugs also attack healthy cells, which is why many patients experience side effects such as nausea, tiredness and hair loss during treatment.
Chemotherapy aims to get your condition into remission, where abnormal cells are no longer growing and dividing. Doctors will often combine multiple drugs, as well as radiotherapy, to give the best chance of remission occurring.
Chemotherapy is also given in preparation for a stem cell transplant. It removes the cells of your immune system and makes space for the new stem cells that you receive from your donor. This treatment is known as conditioning therapy.
Radiotherapy works by using high energy X-rays to kill cancer cells. It’s an effective first line of treatment for some leukaemia and lymphomas. Radiotherapy is delivered by specialists who can target the correct dose to a precise area of the body.
Unfortunately, radiotherapy also damages normal cells, which can cause side effects. These vary greatly for each person; some people experience mild symptoms, such as tiredness, while for others it can be more debilitating. These side effects normally pass within a few weeks.
As with chemotherapy, radiotherapy is used to prepare a patient for a stem cell transplant as part of the conditioning therapy. It will usually be given to your whole body, when it is known as total body irradiation (TBI).
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