With treatment, it’s usually possible to manage chronic myeloid leukaemia (CML) and keep it under control.
Throughout your treatment, your medical team will always discuss your treatment options with you. You’ll be able to give your opinions and preferences and ask questions at any point.
Chronic myeloid leukamia (CML) treatment
The most common treatments for CML are drugs called tyrosine kinase inhibitors (TKI). The very first TKI to be made, which is still the most commonly used, is called imatinib. Once the diagnosis is confirmed, your doctors will prescribe a TKI. The aim of treatment is to get you into remission.
The usual drugs you’ll have for your first treatment, if you’re diagnosed in the chronic phase, are standard doses of one of the TKI licensed for first-line use, such as imatinib, dasatinib or nilotinib. These drugs allow most people with CML to return to a virtually normal lifestyle, including continuing to work and study.
Watch Kris talk about starting treatment for CML.
It’s really important that you take your TKI exactly as directed by your doctor (this is known as your regimen). Evidence shows that if you do, your response to the treatment will be better. You'll especially need to follow their advice around diet and getting pregnant, if this is something that may affect you.
Your healthcare team will be able to help you find techniques to stick to your regimen. Unlike most anti-cancer drugs, you’ll probably be taking your TKI for life. These drugs work by slowly destroying the leukaemia cells in your body and if you stop taking your TKI without being advised to do so by your doctor, your leukaemia will come back.
There are a few reports of patients stopping imatinib and remaining well, but this is in special circumstances where they’ve been on treatment for several years and have responded exceptionally well. So it’s very strongly advised that you don’t stop taking your TKI unless your doctor tells you to.
Most people start on imatinib. Imatinib (Glivec™ in the UK or Gleevec™ in the US) was introduced for treatment of CML in 1998. It was the first of a new class of drug called tyrosine kinase inhibitors (or TKI) which work by counteracting the effect of the protein that causes the leukaemia. Imatinib is taken as a pill once a day after food.
Newer TKI include nilotinib, bosutinib, dasatinib (called ‘second generation’ drugs) and ponatinib (a ‘third generation’ drug). Your doctor will decide, with you, whether you should take imatinib or nilotinib. The decision will be based on your disease phase, the potential risk of side effects, your risk score and any other conditions you may have.
Nilotinib is a stronger drug than imatinib, so if your risk scores are higher they may suggest you start on this drug. Nilotinib can cause your blood sugar to rise, which can cause problems for some patients, such as people with diabetes. If this is the case for you, you might start on imatinib and only move to nilotinib if imatinib isn’t effective.
The other TKI – bosutinib, dasatinib and ponatinib – are all currently available in the UK through the Cancer Drug Fund. You may take one of these drugs if neither imatinib nor nilotinib work for you.
All of these drugs are taken as a pill. Nilotinib is taken twice a day with a ‘fasting regimen’, meaning no food two hours before or one hour after taking the pill. Dasatinib and ponatinib are taken once a day (with or without food), and bosutinib is taken after a meal.
If you’re diagnosed in the blast phase
Very few patients will be in this phase when diagnosed. If you are, your CML will be treated more aggressively, like an acute leukaemia. Your doctor will discuss your treatment options with you.
What happens if initial treatment doesn’t work?
Doctors can usually tell which patients aren’t going to respond to imatinib within the first three months of treatment. If this is the case for you, you’ll be asked to try another TKI to try and get a better response. Most patients will respond to the second or third TKI.
Sometimes your doctor can identify a specific reason why you’re not responding to a TKI. Your leukaemia cells might have developed a genetic fault (mutation) that changes the shape of the BCR-ABL1 protein in a way that means that imatinib can’t attach (bind) and stop it working. The newer TKI were designed to be able to inhibit BCR-ABL1 proteins with mutations, so you ought to be able to find a drug that suits you.
There is one particular mutation (T315I) that responds only to ponatinib.
If CML progresses while you’re taking imatinib
If the disease progresses to one of the more advanced phases while you’re taking imatinib it shows that the drug isn’t working for you, so you’ll stop taking it. However, it may be possible to achieve a remission with another TKI such as dasatinib or nilotinib, as these have been shown to be effective against most types of imatinib resistance.
If your white cell count is very high (and it can be over 100 at diagnosis) and particularly if you have certain symptoms such as blurred vision, your doctors might advise removing some white blood cells in a mechanical process called pheresis.
In this procedure, a plastic tube will be inserted into your arm. Your blood will drain slowly into a machine (centrifuge) which will be spinning at high speed. The spinning separates the blood into white blood cells, red blood cells and plasma. The white blood cells are removed and discarded and the red cells and plasma are returned to your body. At any one time no more than a cupful of blood will be in the centrifuge and the procedure is very safe. You might be a bit bored as it can take 2–3 hours to reduce your white cell count in this way.
The pheresis will be done by a specially trained nurse, who will talk you through the procedure as they do it.
Watch Professor Jane Apperley, Consultant Haematologist at Hammersmith Hospital, talk about treatment for CML.
Watch Professor Jane Apperley, Consultant Haematologist at Hammersmith Hospital, talk about responses to treatment for CML.
Once your diagnosis is confirmed, your team will discuss your treatment options with you. The decision about what type of treatment any person with cancer has is based on guidelines produced by experts, which look at the very latest evidence.
The aim of treatment is to achieve remission. Remission is when leukaemia cells can’t be detected and you’re clinically well. There are several levels of remission with CML which you normally achieve one by one, based on how long you’ve been having treatment.
For more information see our follow up section below.
When will I start treatment?
When you first have a blood count that shows you have too many white blood cells and may have CML, your doctors will have to do a number of the other tests to confirm the diagnosis.
Almost all patients will be given a drug called allopurinol 24 hours before starting any chemotherapy. This is to prevent gout which may be caused by the rapid death and breakdown of the leukaemia cells at the start of your treatment.
If your white blood cell count is high and/or you have a lot of symptoms and feel unwell, they may give you a mild chemotherapy tablet, known as hydroxycarbamide. This will reduce your blood count and control your symptoms until the diagnosis is absolutely confirmed.
Watch Professor Jane Apperley, Consultant Haematologist at Hammersmith Hospital, talk about what happens at the beginning of treatment for CML.
Stem cell transplant
Stem cell transplants are now only recommended for people whose CML hasn’t responded to at least two TKI.
This is the case for around 5–10% of people with CML. Stem cell transplantation is a good treatment for these patients.
Even if your risk score is higher, you’re likely to try TKI first. The only exception normally would be if you’re diagnosed at an advanced stage, in good health otherwise, and a donor is available.
> For more information on stem cell transplants, download or order our booklet The seven steps: blood stem cell and bone marrow transplants
Watch Professor Jane Apperley, Consultant Haematologist at Hammersmith Hospital, talk about stem cell transplants for CML.
Side effects from imatinib and other TKI aren't usually severe. This is because they target the specific cause of the cancer – in this case the tyrosine kinase protein produced because of the fusion BCR-ABL1 gene.
However, you may notice some things which could be connected to the drugs you’re taking. Your healthcare team will be able to help you manage any side effects you have. The following side effects are common to all TKI:
fluid retention (this may cause swelling)
abnormal liver function (this will be monitored using blood tests)
low blood counts.
Some common side effects of bosutinib include:
diarrhoea, which can be particularly severe in the first few days.
Some common side effects of dasatinib include:
very rarely, blood in the stool
fluid retention, particularly in between the linings of the lung (this can be managed easily by stopping taking the drug either temporarily or permanently – make sure you tell your doctor if you notice new fevers, a cough or pain in the chest when you take a deep breath).
Some common side effects of imatinib include:
fluid retention, particularly around the eyes
dry gritty eyes
haemorrhages into the white of the eye; these are not dangerous or harmful to your sight but can look unpleasant
nausea, if you don’t take the drug on a full stomach.
Some common side effects of nilotinib include:
rash (more common than with bosutinib, dasatinib and imatinib)
changes in the chemicals made by the liver
increase in blood glucose (sugar levels)
increases in cholesterol levels
rarely, clots in the arteries of the heart, brain and lower legs (this usually happens in patients who already have a higher risk of clots, such as heavy smokers, patients with high blood pressure and previous history of clots).
Some common side effects of ponatinib include:
high blood pressure
inflammation of the pancreas, an organ in the abdomen (stomach area); if this happens you’d notice severe pain
clots in the arteries of the heart, brain and lower legs (this is slightly more common than with nilotinib, but again this usually happens in patients who already have a higher risk of clots).
Watch Professor Jane Apperley, Consultant Haematologist at Hammersmith Hospital, talk about the side effects of treatment for CML.
Fertility and CML
If you’re considering having children at the time when you’re diagnosed or in the future, you should consider talking to your doctor, who’ll be able to refer you to a specialist. There is no evidence that any TKI affects fertility (your ability to have a baby). However, you should avoid becoming pregnant while you’re taking imatinib and other TKI, and use reliable contraception. This is because a number of babies born to mothers who were taking imatinib while they were pregnant have suffered from abnormalities.
Although some mothers taking imatinib have had healthy babies, doctors strongly recommend that you don’t become pregnant, because the risk is too high. Because it’s currently thought that people with CML will have to take TKI for the rest of their life, this may impact your plans to have children.
There are options available in terms of egg storage and coming off treatment for a period of time. It’s best to discuss your individual circumstances with your doctor, who might make certain recommendations based on how you’re responding to treatment.
There isn’t much information about whether other TKI are also harmful. They are more potent than imatinib however, so it’s likely that they will be. Doctors recommend that you follow the same advice, and take care not to become pregnant. If you’re diagnosed with CML while you’re pregnant, or if you become pregnant after being diagnosed, your doctors will be able to discuss your options with you. This might – if appropriate for you – include delaying or adapting your treatment until the baby is born. This is something you’ll need to think very carefully about and discuss with your healthcare team.
Imatinib is present in breast milk, so women taking imatinib shouldn’t breastfeed. If you don’t respond to a TKI and are planning to have a transplant, it is quite likely that the drugs used for the transplant will cause an early menopause. Discuss options for preserving your fertility after the transplant with your doctor, and consider starting hormone replacement therapy soon after your transplant.
There’s no evidence at the moment about any harmful impact if the father was taking imatinib at the time of conception. However, some TKI are relatively new and there isn’t much evidence so your doctor might suggest coming off treatment – if appropriate for you – while you conceive.
Again, this is something you’d need to think very carefully about and discuss with your healthcare team. Most CML centres will recommend storing some of your sperm at the time when you’re diagnosed.
Watch Professor Jane Apperley, Consultant Haematologist at Hammersmith Hospital, talk about becoming pregnant while treated for CML, and being diagnosed with CML while pregnant.
Because TKI are relatively new drugs, the effects of taking them for life aren’t yet known. There may be late effects, but we’ll only find out about these after many years of follow up on patients who’ve been taking TKI for a long time. Current evidence suggests that TKI are likely to bring a good quality of life for a long time, with no known late effects so far.
Imatinib and other drugs
There’s some evidence that taking imatinib or other TKI in combination with other treatments may cause more side effects than if you take it on its own. Clinical trials are currently being carried out, to find out if the benefits of adding other drugs outweigh the side effects.
Follow-up and remission
Your doctor will measure your response to treatment, and whether you’re in remission, at your follow-up appointments. It’s really important that you attend these.
Remission is when leukaemia cells can’t be detected and you’re clinically well. There are several levels of remission with CML which you normally achieve one by one, based on how long you’ve been having treatment.
When your blood counts return to normal, you’re said to be in complete haematological remission (CHR). This normally happens around three months after you start treatment.
Although your blood count is normal, the disease isn’t necessarily well controlled. If you were to stop treatment as soon as your blood count returns to normal, it’s likely that your white cell count would increase rapidly again.
This is because a blood test is a relatively insensitive test, which can’t detect a small number of residual leukaemia cells. This means that there may still be large numbers of leukaemia cells remaining.
A more sensitive test is when doctors look for the amount of Philadelphia chromosome in your bone marrow.
If the Philadelphia chromosome can’t be detected, this is called a complete cytogenetic response (CCyR). This is performed on a sample of your bone marrow.
Today, this follow-up test is often replaced by the molecular test described below. It differs from hospital to hospital. There’s thought to be some benefit in having the bone marrow test at least annually, as it can detect other genetic changes that may develop. However, the benefit hasn’t been proven yet.
Molecular remission (PCR negativity)
A PCR test (a polymerase chain reaction test) is the recommended test for monitoring patients who are responding to treatment for CML. It’s carried out every three months, using a blood sample, to measure how you’re responding and let your doctor know if you need to change drugs or dosages. This is a very reliable and sensitive test which can detect one leukaemia cell in up to one million normal blood cells.
It’s used to detect tiny amounts of a product (called a transcript) made by the abnormal BCR-ABL1 fusion gene.
If the level of the BCR-ABL1 transcripts is reduced at least 1,000-fold, then it’s called a major molecular response (MMR). If no BCR-ABL1 transcripts can be detected you might hear your result referred to as ‘undetectable transcripts’, because there may be leukaemia cells present somewhere in your body, even when the test is negative.
In a clinical trial, the best current treatment is compared to one that could be better. You’ll still get normal treatment while you’re taking part in the trial, and your safety and well-being is always the first priority.
Taking part in a clinical trial does come with uncertainties, and you may prefer not to take part in one. If you don’t want to be in a trial, or there isn’t a suitable trial available, you’ll be offered the best treatment available at that time which is suitable for your individual condition.
Thanks to imatinib and other TKI, the outlook for the majority of people with CML is generally positive. The drugs usually stop CML from progressing, survival rates have improved and you can have a good quality of life.
While we haven’t seen the same improvements for people who don’t respond well to TKI, there are still treatment options available.
Talking about your prognosis
You may find it hard to ask or talk about your prognosis. Sometimes those close to you might want to know your prognosis even if you don’t. However, your healthcare team aren’t allowed to give this or any other information to anyone – not even family members – without your permission. Try to decide early on who you want to know about your condition, then tell your healthcare team – you can change your mind at any time.
Remember that your outlook might change, for example if you respond well to treatment. If there’s a change in your condition, or if you’ve finished all or part of your treatment, you might want to consider asking if your prognosis is still the same.
If you’re taking TKI
Imatinib and other TKI have been used to treat CML for 16 years. If – like most patients – you’re diagnosed in the early chronic phase, these drugs will usually stop the disease progressing and you’ll stay in a prolonged chronic phase.
It’s currently thought that patients treated with TKI will need to take them for the rest of their lives, although this may change in the future as more data becomes available.
If TKI don’t work for you
If you don’t achieve a good response to TKI, your outlook will depend on how you respond to alternative treatments such as a stem cell transplant. If this is the case for you, you should discuss treatment options and your likely prognosis with your specialist.
If you’re in blast phase
Imatinib and other TKI haven’t brought the same benefits for the small number of patients diagnosed in the blast phase, or patients who enter the blast phase. If this is the case for you, you may be invited to take part in a clinical trial. Sometimes patients will respond to intensive therapy and enter a second chronic phase, so have an improved outlook. If this isn’t the case for you, you should discuss your likely prognosis with your specialist.
Watch Professor Jane Apperley, Consultant Haematologist at Hammersmith Hospital, talk about the outlook for the patients with CML.
Questions to ask your healthcare team
It’s easy to forget the questions you wanted to ask when you’re sitting with your healthcare team and trying to take in lots of new information. Some people find it useful to write down the questions they want to ask, before they get there.
What tests will I have?
What will these tests show?
Where will I have the tests done?
Are there any risks associated with the tests?
Will any of the tests be painful?
Do I need to know anything about preparing for the tests, for example, not eating beforehand?
How long will it take to get the results?
Who will explain the results?
What is my exact diagnosis?
Will I need to have treatment? If so, when?
What does the treatment do?
Is there a choice of treatments?
Is there a clinical trial that I could join?
What’s likely to happen if I decide not to have the treatment my healthcare team recommended?
If I don’t need to start treatment straight away, how will I know when I need to start it?
Who do I contact if I take a turn for the worse?
Who can I contact if I have any questions?
Is there any written information available or any recommended websites?
My main treatment
What type of treatment will I have?
Will I have to stay in hospital?
If not, how often will I need to go to hospital as an outpatient?
What drug regimen will I be given? Will I be given it by mouth, injection or drip (into a vein)?
Will my treatment be continuous or in blocks of treatment with a break in between?
How long will my treatment last?
What side effects could I get from my treatment?
Can side effects be treated or prevented?
Will they affect me all the time or only while I’m taking certain drugs?
What are the fertility risks with treatment and what options are available to me to protect my fertility?
What effect is the treatment likely to have on my daily life?
Will I be able to carry on working or studying?
Will I need to take special precautions, for example against infection?
Will I need to change my meal times or work my drugs around these?
Stem cell transplant
Is a transplant an option for me?
If I’m having a transplant:
How long will I be in hospital for?
Do I have to be in isolation?
How long will it be before I get back to normal?
Choosing the right treatment for you
If you’re asked to choose between treatments, you might like to ask your consultant these questions about each one:
What’s the best outcome I can hope for?
How might the treatment affect my quality of life?
How will the cancer be monitored after my treatment?
How often will I need to have follow-up appointments?
Is there anything I need to watch out for after my treatment?
Who can I contact if I have any questions or worries?
How will doctors know if the cancer is progressing?
What are the options for more treatment?
What will the treatment involve? Will it be different from my initial treatment?
Will there be any side effects from more treatment?
Is my prognosis likely to change with more treatment?
Your healthcare team
If you’re diagnosed with a blood cancer, your hospital will give you the names and contact details of your consultant, clinical nurse specialist and other members of your healthcare team. You can then use these details to contact your team if you have any questions you want to ask when you’re not in the hospital.
Most people with blood cancer have a haematologist as their main doctor. A haematologist specialises in treating people with blood diseases. Your consultant will be an expert in treating your specific condition.
Your clinical nurse specialist
They are your key point of contact with the rest of your healthcare team. You may like to see your clinical nurse specialist when you’re first diagnosed, to talk about blood cancer and your care.
Your clinical nurse specialist will be with you right through your treatment, and you can always go to them with any worries or questions. They get to know you well, and can support you and your family and friends, and help you access resources that could help you through your treatment. Many patients say it can be really helpful to have them by their side at every step.
Talking to other patients
You might like to ask your consultant or key worker if you can talk to someone who’s had the same diagnosis and treatment as you. If you do this, remember that someone else’s experience won’t always be the same as yours. For example, some patients have side effects from a drug and others don’t.
You may also want to contact a support organisation – many provide patient meetings or further online support.
When you’re diagnosed with a blood cancer, your care is discussed at a multidisciplinary team (MDT) meeting. An MDT brings together doctors, nurses and any other specialist staff who’ll be looking after you. A senior consultant usually leads the meetings, which are held regularly. They’ll discuss the best treatment for you and every aspect of your care, including any changes in your condition.
Your other healthcare professionals
It’s definitely worth telling other healthcare professionals you see – like your dentist or optician – about your diagnosis and any medication you’re taking.
Finding out more
After you've been diagnosed, it’s worth taking some time to think about what information you want to know, when and how.
For some people, this is a way to have some control over what’s happening.
Write down any questions you have and keep them handy for when you see your consultant or key worker. If they can’t answer your questions, they’ll be able to tell you who to speak to.
You might prefer to ask your clinical nurse specialist questions rather than your consultant, but do whatever works for you.
Most patients say they find it useful taking someone with them to consultations.
If you’d find it helpful, you could ask them to take notes while you listen. You can choose who to take; it doesn’t have to be a family member.
If you’re staying in hospital it might be harder to have someone with you when you speak to your consultant. It might be useful to ask in advance what time the consultant is likely to speak to you, so you can try to arrange for someone to be with you at that time.
When you’re in the clinic or staying in the hospital you may be looked after by a more junior doctor, a senior house officer or a registrar. These doctors have left medical school but are still training to be consultants. They’ll be able to answer many of your questions, but if they can’t then they’ll ask the consultant. All doctors in training are supervised closely by more senior colleagues.
Some people find that joining a patient support group is helpful. It may be easier to talk to someone outside of your family about your situation and being able to share similar experiences might also help you.