Treatment options

Treatment and care of people with cancer is usually provided by a team of health professionals, both medical and allied health, called a multidisciplinary team.

Some forms of leukaemia don’t need treatment straight away, and some may never need treatment ⁴⁶.

Treatment for leukaemia depends on:

the type and subtype of leukaemia
the stage of the disease
the location of the cancer
the severity of symptoms
your age ⁴⁷, general health and wishes.

Treatment may involve the following.

Surgery

Surgery is not useful in treating leukaemia because the cancer cells are spread throughout the bone marrow and often in many other organs.

However, surgery may be used to relieve symptoms. For example, in CLL, CML and hairy cell leukaemia, the spleen may remove too many blood cells from the bloodstream, or become enlarged and press on nearby organs. The spleen may be surgically removed (splenectomy) to relieve symptoms and reduce the severity of the anaemia or low platelet counts.

Chemotherapy

Some people with leukaemia may receive chemotherapy.

Chemotherapy is the main treatment for people with acute leukaemia (ALL and AML). It may be given over several years. People with ALL usually receive chemotherapy in 3 main phases over 1 to 3 years (the first 2 phases involve highdose chemotherapy) ⁴⁸. People with AML usually receive 2 phases of high-dose chemotherapy ⁴⁹.

Chemotherapy may be used before a stem cell transplant.

Chemotherapy may be used for CLL, sometimes combined with targeted therapy ⁵⁰. Different medicines and combinations may be used depending on your age, health and personal preferences ⁵¹.

Some people with CML may receive chemotherapy before starting targeted therapy, or as an alternative if they can’t take the targeted therapy medicines ⁵².

Using chemotherapy to treat leukaemia may cause a side effect called tumour lysis syndrome. This occurs when the large number of leukaemia cells in the body break open after they are killed by the chemotherapy medications, releasing their contents into the bloodstream. This can overwhelm the kidneys and cause serious problems. Extra fluids and certain medicines may be given to avoid this syndrome.

Steroids

Steroids are hormones naturally made in the body. They can also be made in a lab and used as drugs. Steroids may be used to increase the effect of chemotherapy, help destroy leukaemia cells, or to reduce allergic reactions to some chemotherapy drugs. They are usually given as an oral medication.

The most common steroids for ALL include prednisolone and dexamethasone ⁵³.

Steroids may also be used to treat CLL with chemotherapy, or if there is a sudden drop in the amount of red blood cells ⁵⁴.

Radiation therapy

Some people with leukaemia may receive radiation therapy.

Radiation therapy is not usually a major treatment for leukaemia. However, it may be used to treat leukaemia that has spread to the central nervous system or testicles, or to treat symptoms caused when swollen internal organs, such as the spleen, press on other organs. It may also be used before a stem cell transplant. Radiation therapy can be used to relieve bone pain where the leukaemia has spread to the bones

Targeted therapy

Targeted therapy refers to treatment with medicines that are designed to specifically attack cancer cells without harming normal cells. These types of medicines affect the way that cancer cells grow, divide, repair themselves or interact with other cells. Different types of targeted therapy may be used for different types of leukaemia.

Targeted therapy for ALL usually includes a group of drugs called tyrosine kinase inhibitors (TKIs). These drugs work by blocking a protein that leukaemia cells need to grow and divide ⁵⁵. Some of these medicines include imatinib, dasatinib, nilotinib, bosutinib and ponatinib. Some subtypes of ALL may be treated with a group of drugs called monoclonal antibodies. These work by targeting a protein on the surface of leukaemia cells. Monoclonal antibodies used to treat ALL include rituximab and blinatumomab.

Targeted therapy for AML include venetoclax, a BCL-2 inhibitory. Targeted therapy for some subtypes of AML may be available through clinical trials ⁵⁶.

The most common type of targeted therapy for CLL is monoclonal antibodies ⁵⁷, including the drugs rituximab, obinutuzumab, ofatumumab and alemtuzumab. Tyrosine kinase inhibitors may be used to treat CLL, including the drugs ibrutinib and idelalisib.

Targeted therapy using tyrosine kinase inhibitors is the main treatment to control CML symptoms (they can’t cure the disease) ⁵⁸. These include the drugs imatinib, dasatinib, nilotinib and ponatinib ⁵⁹.

CAR-T therapy is a term for a type of immunotherapy that changes your own T-cells so they can attack the cancer cells. Currently, CAR-T therapy is only used for some forms of acute lymphoblastic leukaemia (ALL).

During CAR-T some of your blood will taken and sent to a laboratory. In the laboratory, your cells will be changed so that they have structures called chimeric antigen receptors on the surface. After growing enough of the new cells, they are injected back into your body. In the body, the cells will replicate and grow. The ‘new’ T cells may be able to find and attack the cancer cells. The T cells are taken and re-injected intravenously (in a vein). ⁶⁰

Stem cell transplant

A stem cell transplant may be used if bone marrow has been destroyed by high doses of chemotherapy treatment. This is used to replace damaged or destroyed blood stem cells in the bone marrow with healthy ones. These can be your own blood stem cells (called an autologous stem cell transplant) or from a donor (called an allogeneic stem cell transplant). For acute leukaemia (ALL and AML), stem cells are usually collected from a donor ⁶¹ ⁶². Stem cell transplants aren’t suitable treatments for most people with chronic leukaemia (CLL and CML), but it may be used if the disease is spreading more quickly and has not responded to other treatments ⁶³ ⁶⁴.

A stem cell transplant from another person is only possible if that person has a tissue type that closely matches yours. The best donor is often a close relative. Stem cell transplants from the patient’s own body are more common in treating lymphoma. The cells are removed from the blood or bone marrow in the weeks before chemotherapy treatment and are then returned to the patient’s bloodstream.

Leukapheresis

If large numbers of leukaemia cells in the bloodstream are causing problems with normal circulation, a procedure called leukapheresis may be used. This involves passing the blood through a machine that removes white blood cells and then returns the treated blood back into the bloodstream.

Immunotherapy

Immunotherapy involves treatment with medicines that boost the ability of the immune system to attack cancer cells. Interferon-alpha may be used as immunotherapy to treat CML, although tyrosine kinase inhibitors are now preferred.

Follow-up

Treatment for leukaemia often lasts for years. Some people may require ongoing chemotherapy or radiation therapy to keep the leukaemia under control. CLL and CML are rarely cured, and treatment may continue at intervals for life.

After treatment for leukaemia, you will need regular follow-up examinations and tests to look for return of the cancer or side effects from treatment.

Advanced disease

If leukaemia treatment is unsuccessful, palliative treatment to control the symptoms of the disease may be the best option. This might include:

radiation therapy, chemotherapy, or both ⁶⁵
pain-relieving medicines to reduce pain from leukaemia in the bones
blood transfusions to treat fatigue
food supplements and medicines to treat nausea and loss of appetite.

Recurrent cancer

Leukaemia may recur (come back) after treatment. Secondary cancer is when the cancer spreads to another part of the body.

Treatment options for recurrent leukaemia include further chemotherapy, possibly using different medicines or higher doses, targeted treatment with blinatumomab (for some types of ALL) or a stem cell transplant.

Active monitoring

Some forms of leukaemia may not need to be treated straight away, or at all. Instead, you may have regular check-ups and blood tests to monitor the cancer ⁶⁶ (called active monitoring, watchful waiting, or watch and wait). Active monitoring can be better than treating the cancer straight away, because early treatment can cause side effects or make your body resistant to treatment.

Other treatments

Other treatments may be used for different types of leukaemia, but these are not used as often as the main treatments:

Treatment for acute promyelocytic leukaemia (APML) is different from most other AML treatments and consists of 3 phases – some people with APML may not need to go through all 3 phases ⁶⁷. This treatment involves different types of drugs, including chemotherapy drugs ⁶⁸.
Lowdose drug therapy may be used for AML if you aren’t healthy enough for intensive chemotherapy or a stem cell transplant. Drugs used for this therapy include azacytidine and cytarabine.
Allopurinol is drug that may be used to treat some symptoms of CML ⁶⁹.
Human leukocyte antigen (HLA) typing may be used if a stem cell transplant is an option for treatment. This test is used to make sure the donor is a suitable match ⁷⁰.