Although acute lymphoblastic leukemia is a rare 

 of blood cancer, it is the most common form of leukemia in children. It also affects adults, who comprise 4 out of every 10 cases of acute lymphoblastic leukemia. The risk of developing acute lymphoblastic leukemia is highest in those under 5 years old or over 50 years old. Acute lymphoblastic leukemia usually develops quickly, over the course of days or weeks, and requires rapid 

Which blood cells are affected in acute lymphoblastic leukemia?

To understand which cells are affected in acute lymphoblastic leukemia, it helps to look at blood composition. Blood is broadly made up of four components:

Red blood cells, which carry oxygen and give blood its distinctive color.

White blood cells, which fight infection.

Platelets, which are small cells involved in clotting and prevention of excessive bleeding.

Plasma, the straw-colored fluid in which blood cells are suspended.

Under normal conditions, these components exist in relatively stable amounts in relation to each other. However, in acute lymphoblastic leukemia, the number of white blood cells is no longer controlled correctly. White blood cells are produced more rapidly than normal within the bone marrow and are released before they are fully mature (these cells are known as lymphoblasts). This means that although there are more white blood cells in the blood, they do not function correctly.

This overproduction of white blood cells reduces the amount of red blood cells and platelets that are produced, and this causes many of the 

Know ALL was pleased to speak with Lizzie Dean, a patient with acute myeloid leukemia (AML) and patient ambassador for Know AML, and Hayley Ellis, senior hematology research nurse, Queen Elizabeth Hospital, Birmingham, UK. They discussed: “What can patients expect when joining a clinical trial?”

Lizzie shares her experiences of participating in two clinical trials: when she was newly diagnosed with acute leukemia (in her case AML) and then again following two relapses. She discusses how she joined the trials, her experiences of attending hospital appointments and treatment side effects, and what her key concerns and questions were through the process.

Hayley provides information on where patients can find information about participating in clinical trials if they are interested in taking part; for example, by discussing with a healthcare professional, contacting cancer charities, or registering with organizations online. She also explains how research nurses and other healthcare professionals can support patients through their treatment and clinical trial journey following diagnosis and gives an overview of how clinical trials might be conducted. For example, the HOVON Foundation has established a network of clinical trials and treatment centers, to advance research in the field of hematologic disease and to expand access to clinical trials for patients across Europe and the UK.

Hayley and Lizzie also provide their key advice for patients considering enrolling in a clinical trial, encouraging patients to fully familiarize themselves with any patient information materials provided by healthcare professionals and undertake their own further research online. They highlight the importance of asking their team of healthcare professionals any questions they might have, so that they can make an informed choice about enrolling in a trial and be fully supported throughout the process.

This educational resource is independently supported by Syndax. All content was developed by SES in collaboration with an expert steering committee. Funders were allowed no influence on the content of this resource.

Hovon. About Hovon. https://hovon.nl/en/about-hovon (https://hovon.nl/en/about-hovon). Accessed September 29, 2025.

Lizzie Dean

What can patients expect when joining a clinical trial? 

When I was 10 years old, I was diagnosed with acute lymphoblastic leukemia for the first time. In 2003, I underwent treatment according to the BFM [Berlin-Frankfurt-Münster] 2002 chemotherapy protocol, which lasted until May 2005. As a child, I did not fully understand how serious and life-threatening the disease was. I simply accepted treatment as part of my life, and once the treatment ended, I returned to childhood. I remained under regular medical follow-up until 2008, after which my life continued without limitations.

Years later, life unfolded in the most beautiful way. I graduated from university, got married, and in 2017 became a mother to a wonderful baby boy. Leukemia felt like a distant chapter of my past – something I believed I had left behind forever.

But in 2019, my body sent me a quiet warning. Large, painless bruises began appearing on my legs. Because of my medical history, I trusted my instincts and asked for blood tests. The results showed the presence of blasts, and a bone marrow biopsy confirmed a relapse of B-cell acute lymphoblastic leukemia.

This time, everything was different. I was no longer a child protected by innocence. I understood exactly what this diagnosis meant – and still, I chose to fight.

I underwent six cycles of intensive chemotherapy according to the HOVON 100 protocol for acute lymphoblastic leukemia, a standardized hematology clinical trial regimen.

Of course, chemotherapy came with many side effects: high fevers, significant fluid retention, bloating, hair loss, and extreme physical exhaustion. The course was further complicated by allergic reactions to asparaginase and catheter-related thrombosis. Surprisingly, chemotherapy itself was not the hardest part for me. I had been through it before and knew what to expect. What hurt the most was being separated from my boys – my child and my partner. Not being able to be with them every day was incredibly painful, but at the same time, they became my greatest source of strength. I kept telling myself that chemotherapy was something I could endure and complete.

Transplantation was something entirely different.

In January 2020, I underwent an allogeneic stem cell transplantation from a matched unrelated donor while in complete remission, following TBF [thiotepa, busulfan, fludarabine] conditioning with ATG [anti-thymocyte globulin], cyclosporine, and mycophenolate mofetil. Altogether, I spent 51 days in the hospital, including 6 days of preparation before transplantation and 1 day of recovery, with the remainder of my hospitalization marked by uncertainty and severe complications.

Within the first 42 days after transplantation, I experienced multiple serious complications. These included heavy bleeding, severe bone and muscle pain, extreme muscle weakness requiring neurological evaluation, viral infection complicated by pneumonia, and ultimately severe acute graft-versus-host disease affecting both my skin and liver. High-dose corticosteroids were required, followed by additional immunosuppressive therapies, including ruxolitinib and extracorporeal photopheresis.

I was discharged from the hospital 42 days after transplantation, but treatment and recovery continued for many months. Immunosuppressive therapy was gradually adjusted and slowly reduced. Mycophenolate mofetil was discontinued first, followed by tapering and eventual cessation of corticosteroids. During this period, I developed BK virus-associated cystitis, which required treatment with intravenous immunoglobulins. Antifungal therapy was modified, and extracorporeal photopheresis continued alongside additional antifungal treatment.

Over time, my condition slowly stabilized. Ruxolitinib was discontinued, and photopheresis was gradually reduced from twice weekly to monthly sessions before being stopped completely. Attempts to taper cyclosporine were challenging and at times led to worsening symptoms, requiring careful dose adjustments. At 18 months after transplantation, in June 2021, cyclosporine was finally discontinued. Only then was I able to begin revaccination, marking an important step in rebuilding my immune system.

Several months later, new long-term consequences began to surface. In July 2020, I started experiencing pain in both hips and knees. After evaluation, I was diagnosed with bilateral femoral avascular necrosis. In July 2021, I underwent left hip arthroplasty, followed by right hip arthroplasty in June 2022. My reproductive health was also affected. I had my last natural menstruation in 2020, and in August 2023 I began hormone replacement therapy. In January 2025, osteopenia was diagnosed, and I started vitamin D supplementation to support bone health.

Despite all these challenges, I proved to myself that I am strong and capable. In 2022, I returned to work, and in 2025, I completed my master’s degree in economics, received the Dean’s Award, and celebrated a personal victory that felt even more meaningful because of all I had endured.

Although today I am medically considered “healthy”, survival did not bring an immediate return to normal life. I live with the psychological consequences of everything my body and mind have endured. I experience periods of depression, anxiety, and a persistent feeling that my body is constantly on alert – not because I fear the disease will return and not because I live in fear of illness. I truly believe that leukemia is behind me. Yet my body remembers. It remains vigilant, as if it is still expecting something to go wrong.

Life continued for everyone else while mine was put on pause. When I finally returned, the world had moved on, and I had to learn how to re-enter it. Adapting to everyday life after survival has been one of the most difficult parts of this journey. Learning how to live without constant tension, how to trust my body again, and how to function in “normal life” without anxiety is still a work in progress.

This is a side of survivorship that is rarely spoken about. We celebrate remission and the end of treatment, but we talk far less about the emotional and psychological aftermath. Healing does not end when treatment stops. For many of us, it is where the most delicate and complex part of the journey truly begins.

By sharing my story, I hope to support others who find themselves in a similar situation – to remind them that struggling after survival does not mean weakness, and that they are not alone.

Matea's story

1. Cancer Research UK. What is acute lymphoblastic leukaemia (ALL)? 

https://www.cancerresearchuk.org/about-cancer/acute-lymphoblastic-leukaemia-all/about

. Updated May 8, 2024. Accessed Mar 18, 2026.

2. American Cancer Society. Key Statistics for Childhood Leukemia. 

https://www.cancer.org/cancer/types/leukemia-in-children/key-statistics.html

. Updated Jul 22, 2025. Accessed Mar 18, 2026.

3. American Cancer Society. Key statistics for acute lymphocytic leukemia (ALL). 

https://www.cancer.org/cancer/acute-lymphocytic-leukemia/about/key-statistics.html

. Updated Jan 13, 2026. Accessed Mar 18, 2026.

What is acute lymphoblastic leukemia?

What causes acute lymphoblastic leukemia?

Acute lymphoblastic leukemia occurs because of damage (mutations) in the genetic material (DNA) of a cell. The cause of most cases of acute lymphoblastic leukemia is unknown; however, certain risk factors may increase the risk of developing acute lymphoblastic leukemia. People with some rare genetic conditions, such as Down syndrome, Fanconi anemia, Bloom syndrome, and ataxia-telangiectasia have a higher chance of developing acute lymphoblastic leukemia (ALL). Some environmental factors are known to increase the likelihood of mutations, such as exposure to very high levels of radiation or high levels of a gas called radon. Patients with a weak immune system, or those who have received chemotherapy for a prior malignancy, may also have an increased risk of developing leukemia.

What are the symptoms of acute lymphoblastic leukemia?

The symptoms (a patient’s own experience) and signs (observed by a doctor) of acute lymphoblastic leukemia can be divided into four categories. As acute lymphoblastic leukemia affects the production of the three types of blood cells, each group produces different symptoms.

1. Low red blood cells (anemia) may cause:

Shortness of breath during normal physical activities.

2. Low white blood cells (leukopenia) may cause:

3. Symptoms of low platelets (thrombocytopenia) can be:

Minor cuts bleeding for longer than normal.

Small red dots on the skin, called petechiae.

In women, periods may be heavier or more frequent.

4. General symptoms may occur, including:

A feeling of fullness or pain just below the ribs.

Lumps in the neck, armpits, abdomen, or groin, caused by swollen glands known as lymph nodes.

Acute lymphoblastic leukemia is a rare disease, and other conditions can cause similar symptoms; therefore, if a patient notices any of the symptoms listed above, or a parent/carer notices them in their child, they should speak to their physician.

1. American Cancer Society. What causes acute lymphocytic leukemia (ALL?). 

https://www.cancer.org/cancer/types/acute-lymphocytic-leukemia/causes-risks-prevention/what-causes.html

. Updated Aug 13, 2025. Accessed Mar 18, 2026. 

2. NHS inform. Causes of acute lymphoblastic leukaemia. 

https://www.nhsinform.scot/illnesses-and-conditions/cancer/cancer-types-in-adults/acute-lymphoblastic-leukaemia/#causes-of-acute-lymphoblastic-leukaemia

. Updated Feb 16, 2026. Accessed Mar 18, 2026.

3. Cancer Research UK. Risks and causes for acute lymphoblastic leukaemia (ALL). 

https://www.cancerresearchuk.org/about-cancer/acute-lymphoblastic-leukaemia-all/risks-causes

. Updated Aug 13, 2025. Accessed Mar 18, 2026 

4. Leukemia & Lymphoma Society. Signs and Symptoms. 

https://www.lls.org/leukemia/acute-lymphoblastic-leukemia/signs-and-symptoms

5. Cancer Research UK. Symptoms of acute lymphoblastic leukaemia (ALL). 

https://www.cancerresearchuk.org/about-cancer/acute-lymphoblastic-leukaemia-all/symptoms

. Updated May 18, 2024. Accessed Mar 18, 2026.

Causes & symptoms

What to expect on the journey to a diagnosis

Getting a diagnosis is important, as it tells doctors if a patient has acute lymphoblastic leukemia rather than another condition. If an acute lymphoblastic leukemia diagnosis is made, it can be used to predict how quickly the disease might progress and how it may respond to certain 

Diagnostic techniques can also show what 

  of acute lymphoblastic leukemia is present. Identifying the subtype increases the accuracy of the doctor’s predictions about the course and severity of disease.

, in themselves or their child, they should visit their doctor as soon as possible. The symptom(s) might not be caused by acute lymphoblastic leukemia, but it is important to have them checked. Identifying diseases early often makes them easier to manage and increases the chances of successful treatment.

During the appointment, a medical history will be taken and a description of any changes noted. The doctor might perform a physical examination of any areas that are swollen, bleeding, or bruised, and check for possible signs of infection.

Depending on the outcome of this assessment, the doctor may also want to take a blood sample to perform some initial blood tests. 

Shows the amount of specific chemicals in the blood to assess the health of the liver and kidneys.

Shows the number of each group of blood cells present (red blood cells, white blood cells, and platelets).

Identifies changes in the appearance of cells under a microscope, which may indicate the presence of disease.

If your doctor feels that further tests are needed, a referral to a specialist may be made. This will most likely be a hematologist (a doctor who has undertaken specialist training in diseases of the blood). The hematologist will go over the results and may suggest that further tests are performed.

A sample is taken from the spongy marrow in the middle of the bone, normally from the hip.

In the case of a bone marrow aspiration, the sample will be a liquid, whereas in a biopsy, a small section of bone filled with marrow will be collected.

Bone marrow is where many blood cells are produced. Taking a sample from here can allow a hematopathologist (a specialist who studies blood cell diseases by looking at samples of cells and other tissues) to see what type of cells are being made and in what proportions. Cells that look unusual can be spotted using this test and classified according to any changes seen.

This test is used if there is a concern that the leukemia may have spread into the brain and spinal cord. It assesses the number of cells in the fluid that bathes these organs. It is performed by inserting a needle into the base of the spine and drawing out some fluid.

Further laboratory tests may be performed on the blood and bone marrow samples to diagnose and/or determine the specific type of acute lymphoblastic leukemia.

Reveals information about the type of cells present by exposing the cells to chemical dyes that only react with certain types of leukemia cells. This causes a color change that is visible under a microscope.

Allows the identification of leukemia cells by detecting specific proteins (known as antigens) on the surface of your cells. Different types of cells have different proteins, and the identification of these markers shows scientists what type of acute lymphoblastic leukemia cells (B, T, or mixed) are present in the sample. This can tell doctors what specific type of acute lymphoblastic leukemia is present.

Acute lymphoblastic leukemia comprises several distinct genetic subtypes. Identifying which subtype is present can help doctors to make a diagnosis and inform treatment decisions.

A range of different techniques will be used to examine the leukemic cells for abnormalities affecting the chromosomes and/or genes. This will help determine the genetic subtype of acute lymphoblastic leukemia.

There are some serious conditions associated with cancer that can occur. More blood tests may be performed to monitor for the conditions listed below, so that the best treatment can be provided as soon as possible.

While imaging is not essential for a diagnosis of acute lymphoblastic leukemia, it can be used to give a clearer picture of the health of your organs and show if the cancer has spread. They can also help to diagnose infections. You may have one or several forms of imaging performed.

This, in turn, can help predict the response to treatment. You may have one or several forms of imaging performed.

This is ideal for assessing soft tissue and fluid within the body. It uses high frequency soundwaves to create a dynamic image. This may be used to check your liver, spleen, and lymph nodes. The function of the heart may also be checked using a specialized form of ultrasound called echocardiography.

A method used to visualize what is going on within the body and spot any changes from normal that might indicate how far the cancer has spread. This scan also provides information on the health of multiple organs, particularly the head and chest.

Like a CT, an MRI scan takes pictures of the whole body from multiple angles, using magnetism and radio waves.

For acute lymphoblastic leukemia, there is often no need for an MRI, but your doctor may suggest one if you have symptoms of:

Leukemia that has spread to the central nervous system.

Once you, or your child, have a diagnosis, some of these tests may be repeated to show how you are responding to 

1. Blood Cancer United. Acute lymphoblastic leukemia (ALL). 

https://bloodcancerunited.org/blood-cancer/leukemia/acute-lymphoblastic-leukemia-all

2. Cancer Research UK. Seeing your GP when you have symptoms of acute lymphoblastic leukaemia (ALL). 

https://www.cancerresearchuk.org/about-cancer/acute-lymphoblastic-leukaemia-all/getting-diagnosed/seeing-your-gp

. Updated May 17, 2024. Accessed Mar 18, 2026.

3. American Cancer Society. Tests for acute lymphocytic leukemia (ALL). 

https://www.cancer.org/cancer/types/acute-lymphocytic-leukemia/detection-diagnosis-staging/how-diagnosed.html

. Updated Aug 18, 2025. Accessed Mar 18, 2026.

4. Cancer Research UK. Referral to a specialist for acute lymphoblastic leukaemia (ALL). 

https://www.cancerresearchuk.org/about-cancer/acute-lymphoblastic-leukaemia-all/getting-diagnosed/referral-specialist

5. Cancer Research UK. CT scan for acute lymphoblastic leukaemia (ALL). 

https://www.cancerresearchuk.org/about-cancer/acute-lymphoblastic-leukaemia-all/getting-diagnosed/tests/ct-scan

. Updated May 6, 2025. Accessed Mar 18, 2026.

6. Cancer Research UK. MRI scan for acute lymphoblastic leukaemia (ALL). 

https://www.cancerresearchuk.org/about-cancer/acute-lymphoblastic-leukaemia-all/getting-diagnosed/tests/mri-scan

. Updated Apr 28, 2025. Accessed Mar 18, 2026.

Diagnosis

The World Health Organization (WHO) classification system is the most commonly used method to distinguish acute lymphoblastic leukemia subtypes.

Based on the type of white blood cell (lymphocyte) affected, two main subtypes can be found:

B-cell acute lymphoblastic leukemia, which is the most common type (75% of cases in adults; ~85% of cases in children)

Most B-cell acute lymphoblastic leukemia stems from abnormal immature (pre) B cells.

Sometimes B-cell acute lymphoblastic leukemia develops from malignant mature B cells. You may also hear it called Burkitt-type acute lymphoblastic leukemia, as it is similar to another type of cancer called Burkitt lymphoma.

T-cell acute lymphoblastic leukemia (25% of cases in adults; 15% of cases in children)

More frequent in young adults and in males.

Leukemia subtypes can be further categorized into around 25 different subtypes according to the genetic abnormalities present. These genetic subtypes are very important for patient management, helping doctors to confirm a diagnosis and select the optimal treatment pathway; some genetic subtypes indicate that the disease will respond to a specific drug while others can indicate the aggressiveness of the leukemia. Doctors will use the genetic subtype in combination with other factors, like age and response to treatment, to determine the level of chemotherapy required by each patient.

The frequency of genetic subtypes varies by patient age and time of diagnosis.

Philadelphia-positive acute lymphoblastic leukemia

The most common genetic abnormality in adult acute lymphoblastic leukemia is the formation of the Philadelphia (Ph) chromosome; this is caused by the exchange of genetic material between Chromosomes 9 and 22 in leukemic cells, resulting in the creation of the BCR::ABL1 fusion gene. The presence of this fusion gene confers a sensitivity to a class of drug called tyrosine kinase inhibitors. Ph-positive acute lymphoblastic leukemia occurs in 25% of adults, but only 3–4% of children. You can read more about this and other therapies on the 

Philadelphia-like acute lymphoblastic leukemia

Characterized by a similar gene expression profile to Ph-positive acute lymphoblastic leukemia but lacking the BCR::ABL1 gene fusion. Although Ph-like acute lymphoblastic leukemia can occur at any age, it most frequently occurs in adolescents and adults. This subtype comprises two subgroups: ABL1 class fusions and JAK–STAT abnormality. The presence of an ABL-class fusion, but not a JAK–STAT abnormality, confers a sensitivity to a type of drug called tyrosine kinase inhibitors, making these drugs more effective.

ABL-class gene fusion acute lymphoblastic leukemia

This genetic subtype comprises a series of gene fusions involving one of the following genes: ABL1, ABL2, CSF1R, and PDGFRB. Similar to patients with the BCR::ABL1 fusion gene, these patients are usually sensitive to tyrosine kinase inhibitors, with many patients treated with imatinib or dasatinib, alongside standard chemotherapy. This genetic subtype is quite rare, accounting for 3–4% of cases in children.

ETV6::RUNX1 fusion or t(12;21)(p13;q21) acute lymphoblastic leukemia

This is a common genetic abnormality in children with acute lymphoblastic leukemia, occurring in ~25% of cases; however, it is very rare in adults. Children with ETV6::RUNX1-positive acute lymphoblastic leukemia generally have good outcomes.

High hyperdiploid acute lymphoblastic leukemia

This is the most common genetic abnormality in children with acute lymphoblastic leukemia, occurring in 30% of cases and only occurring in around 10% of adults. It is characterized by the multiple gain of whole chromosomes. Most children with high hyperdiploid acute lymphoblastic leukemia have a favorable prognosis.

KMT2A or MLL gene fusion acute lymphoblastic leukemia

Abnormalities involving the KMT2A gene can occur at any age and are usually associated with aggressive disease. Around 80% of infant patients (aged <1 year) will have this genetic subtype. It is rare in children (5%) but starts to increase in frequency with age – accounting for around 10% of adults with acute lymphoblastic leukemia.

Hypodiploidy acute lymphoblastic leukemia

This genetic subtype is characterized by multiple chromosome losses and is associated with aggressive disease. There are two subgroups: near haploidy and low hypodiploidy. Both subgroups are rare in children, accounting for ~6% of cases. Near haploid acute lymphoblastic leukemia is extremely rare in adults, but low hypodiploidy occurs in ~6%, especially in older patients (aged >50 years).

This genetic subtype involves a very complex rearrangement of Chromosome 21 and gives rise to an aggressive form of the disease. It most frequently occurs in older children (aged 8–14 years) and is rare in other age groups.

Other B-cell acute lymphoblastic leukemia subtypes include t(5;14)(q31.1;q32.3)/IGH::IL3, t(1;19)(q23.3;p13.3)/TCF3::PBX1, TCF3::HLF fusion, MYC rearrangement, DUX4 rearrangement, MEF2D rearrangement, ZNF384(362) rearrangement, NUTM1 rearrangement, CDX2/UBTF, IKZF1 N159Y mutation, ETV6::RUNX1-like, PAX5 alteration, ZEB2 (p.H1038R) mutation/IGH::CEBPE, ZNF384-like, and KMT2A-like.

T-cell acute lymphoblastic leukemia can be further classified according to both immunophenotype and genetics. Whilst many subtypes have been described in the research literature, the clinical relevance and prognostic value are not well understood. Further research is needed before these subtypes are widely and consistently used in the clinical setting.

This list of genetic subtypes is not exhaustive – we have focused on the most common subtypes used by doctors to make treatment decisions. The official classification of acute lymphoblastic leukemia can be found in the WHO classification.

1. Alaggio R, Amador C, Anagnostopoulos I, et al. The 5th edition of the World Health Organization classification of haematolymphoid tumours: Lymphoid neoplasms. Leukemia. 2022;36(7):1720-1748. DOI: 

2. Leukaemia Care. B-cell acute lymphoblastic leukaemia (B-cell ALL). 

https://www.leukaemiacare.org.uk/about-leukaemia/types/acute-lymphoblastic-leukaemia-all/b-cell-acute-lymphoblastic-leukaemia-b-cell-all/

. Updated Jun 2023. Accessed Mar 27, 2026.

3. Cancer Research UK. What is acute lymphoblastic leukaemia (ALL)? 

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4. Lejman M, Chalupnik A, Chilimoniuk Z, et al. Genetic biomarkers and their clinical implications in B-cell acute lymphoblastic leukemia in children. Int J Mol. 2022;23(5):2755. DOI: 

5. Blood Cancer United. Acute lymphoblastic leukemia (ALL). 

https://bloodcancerunited.org/research/acute-lymphoblastic-leukemia-all

Types

There are a variety of different treatments that you or your child may receive for acute lymphoblastic leukemia. It is likely treatment will begin rapidly after receiving a 

Chemotherapy is the most common form of treatment for acute lymphoblastic leukemia. Chemotherapy works by killing rapidly dividing cells such as cancer cells. Treatment may involve the use of one or more chemotherapy drug at once, and often these agents are given straight into the bloodstream. The type of treatment you receive will depend on a number of factors such as your age and fitness levels.

1. Induction phase with high doses of therapy

May last 1 or 2 months. The aim of the induction phase is to reduce the number of leukemia cells to a point when they are no longer detectable in bone marrow samples (called complete remission). After this point, the normal blood cells in the marrow can regrow. While achieving a complete remission is a positive sign, it does not necessarily mean the disease is cured, as leukemia cells may be present at very low levels or in other parts of the body.

2. Consolidation with high doses of therapy

May last 1 or 2 months. The consolidation phase is designed to stop the acute lymphoblastic leukemia coming back and to destroy any leukemia cells that may be present in the blood but that are in such small numbers they do not show up on tests.

Maintenance is a less intense treatment phase that can last up to 2 years. It aims to maintain remission by killing the few leukemic cells that have evaded previous rounds of therapy. Maintenance therapy uses  lower doses of chemotherapy, so the side effects should be less during this phase, allowing a return to normal activities.

Common chemotherapy agents used include: 

Steroids such as dexamethasone, prednisolone, and hydrocortisone taken before or alongside chemotherapy

These types of treatment target specific parts of cancer cells and typically target cancer cells only if certain genetic changes are present. 

Examples of genetic changes for which targeted treatment is available: 

The Philadelphia chromosome is the name given to the product of swapping specific genetic material between chromosomes 9 and 22. This results in the creation of a leukemia-specific gene called 

. Leukemia cells with this change are  sensitive to treatment by tyrosine kinase inhibitors such as

Activation of various kinases can occur in some subtypes of acute lymphoblastic leukemia, including in what is referred to as ‘Philadelphia-like’ acute lymphoblastic leukemia, and heretyrosine kinase inhibitors can also be included in the treatment. Ruxolitinib is such an inhibitor that is currently being tested in clinical trials.

 gene, a protein called menin interacts with KMT2A proteins and promotes the growth of leukemia cells. Menin inhibitors are drugs that block the interaction between menin and KMT2A, preventing the growth of leukemia cells.

Revumenibis a menin inhibitor that is approved in the US for the treatment of patients who have acute leukemia and changes within their KMT2A gene, where the leukemia has returned or is no longer responding to treatments. Currently, menin inhibitors and revumenib are only used to treat patients whose leukemia has returned after multiple rounds of chemotherapy.

Immunotherapy can be considered to be a specialized form of targeted therapy that uses specifically designed antibodies to treat acute lymphoblastic leukemia. Immunotherapy aims to encourage the patient’s immune system to target and kill leukemia cells.

The two main immunotherapeutic agents you may be treated with are

Blinatumomab is a special antibody used to treat some subtypes of B-cell acute lymphoblastic leukemia. Blinatumomab binds to a molecule on the surface of leukemic cells called CD19, as well as a molecular on the patient’s own normal Tcells. This enables the patient’s own immune system to kill the leukemic cells. Treatment usually occurs in a hospital or clinic because it is a relatively new therapy and it is important to document the side effects. Treatment of patients with B-cell acute lymphoblastic leukemia with blinatumomab is rapidly becoming the gold standard treatment and is very likely to revolutionise the treatment of this disease.

Inotuzumab ozogamicin is another unique antibody which is used to treat some subtypes of B-cell acute lymphoblastic leukemia after unsuccessful prior chemotherapy. It works by delivering the chemotherapy drug to the leukemia cells and killing them. 

CAR T-cells are a recent advancement in the treatment of acute lymphoblastic leukemia. White blood cells from the patient are taken and engineered  to specifically kill leukemic cells. These white blood cells are now called CAR T-cells and are multiplied before being given back to the patient. Tisagenlecleucel is a CAR T-cell product that can be used to treat acute lymphoblastic leukemia,

 it has been approved for use in children and young adults up to age 25 to treat B-cell acute lymphoblastic leukemia that has returned or is no longer responding to treatment.

Brexucabtagene autoleucel is approved to treat adults with B-cell acute lymphoblastic leukemia that has returned or is no longer responding to treatments.

Obecabtageneautoleucel is a CAR T-cell therapy that is approved in the US to treat adults with B-cell acute lymphoblastic leukemia that has returned or is no longer responding to treatments.

High doses of targeted radiation, normally called X-rays, can be used to treat cancer, and this technique is called radiotherapy. This may be performed on the brain or spinal cord if the leukemia appears to have spread to these areas. Alternatively, radiotherapy may be used over the whole body (known as total body irradiation) prior to a stem cell transplant.

This procedure is used following chemotherapy or radiotherapy. High intensity treatment is used to kill the leukemic cells in the bone marrow (the spongy part of the bone); however, normal blood cells are killed too.

A transplant is used to replace the blood cells with healthy cells that function correctly. The healthy cells are normally provided by a donor. In a transplant, these healthy cells can be taken from the bone marrow or from the bloodstream.

Although many treatments for acute lymphoblastic leukemia are very good at killing cancer cells, some normal healthy cells may be damaged or killed during treatment as well. This may result in side effects.

For chemotherapy, traditional side effects associated with treatment are: 

Immunotherapy may increase the risk of infection along with: 

CAR T-cell therapy may result in serious side effects, for this reason it is only given in specialized medical centers by trained staff. The most common side effects include:

Cytokine release syndrome (CRS): symptoms including fever, chills, difficulty breathing, severe nausea, vomiting, diarrhea, severe muscle or joint pain, and feeling dizzy

Nervous system problems: can present as headaches, changes in consciousness, confusion or agitation, seizures, trouble speaking and understanding, or loss of balance

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https://www.cancerresearchuk.org/about-cancer/acute-lymphoblastic-leukaemia-all/treatment/chemotherapy/about

. Published May 8, 2018. Accessed Mar 20, 2025.

2. American Cancer Society. Typical treatment of acute lymphocytic leukemia (ALL). 

https://www.cancer.org/cancer/types/acute-lymphocytic-leukemia/treating/typical-treatment.html

. Published Oct 8, 2021. Accessed Mar 20, 2025.

3. American Cancer Society. Targeted therapy for acute lymphocytic leukemia (ALL). 

https://www.cancer.org/cancer/acute-lymphocytic-leukemia/treating/targeted-therapy.html

. Published Oct 17, 2018. Accessed Mar 20, 2025.