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Paediatric Acute Lymphogenous Leukemia

Dr. Samantha A. Vergano, MD - Clinical Genetics, Genomics, Cytogenetics, Biochemical Genetics Specialist. Dr. Samantha A. Vergano, MD - Clinical Genetics, Genomics, Cytogenetics, Biochemical Genetics Specialist.
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Rx Autoimmune, Genetic and Rare Diseases (A - Z)
  • Other names
  • Types
  • Causes and risk factors
  • Symptoms and signs
  • Diagnostic tests
  • Non-Pharmacological Treatments (Therapies and Others)
  • Major Drug Treatments (Chemotherapy and Targeted Drugs)
  • Dietary Molecular Supplements (Supportive, Not a Cure)
  • Immunity-Booster / Regenerative / Stem-Cell–Related Drugs
  • Surgeries and Invasive Procedures
  • Prevention (Mainly Prevention of Complications and Relapse)
  • When to See Doctors or Go to Emergency
  • What to Eat and What to Avoid
  • Frequently Asked Questions (FAQs)

Paediatric acute lymphogenous leukaemia (usually called childhood acute lymphoblastic leukaemia or ALL) is a blood cancer in children. In this disease, the bone marrow makes too many very young white blood cells called lymphoblasts. These cells are abnormal and do not work properly. They crowd out normal blood cells, so the child has fewer healthy red cells, platelets, and mature white cells. Without treatment, ALL grows quickly and is life-threatening, but with modern care most children can be cured. NewYork-Presbyterian+1

Pediatric acute lymphoblastic (lymphogenous) leukemia (ALL) is a blood cancer that starts from very young white blood cells called lymphoblasts in the bone marrow of children. These abnormal cells grow too fast and crowd out healthy blood cells. This causes anemia, infections, and bleeding problems. Modern treatment is very successful, and many children are cured when treatment is given in a specialized pediatric cancer center using strict international protocols and supportive care.Cancer.gov+1

Other names

Doctors use several names for the same disease. Understanding them helps you not feel confused if you see different words in reports. Childhood acute lymphoblastic leukaemia is also called acute lymphocytic leukaemia, acute lymphoid leukaemia, or simply ALL. When it happens in children, it may also be written as paediatric ALL or childhood ALL in medical texts and guidelines. NewYork-Presbyterian+1

Types

Types are based on the kind of lymphocyte involved and special tests on the leukaemia cells.

  1. B-cell ALL (B-ALL) – This is the most common type in children. The cancer cells come from early B lymphocytes (B cells). It usually responds very well to standard treatment. Medscape+1

  2. T-cell ALL (T-ALL) – Here, the cancer cells come from early T lymphocytes (T cells). It may present with a large mass in the chest and higher white blood counts, and treatment is adjusted for this type. Medscape+1

  3. Philadelphia chromosome–positive ALL (Ph+ ALL) – In this type, the leukaemia cells have a special change in their chromosomes called BCR::ABL1 (Philadelphia chromosome). Children with this type usually need targeted drugs (like tyrosine kinase inhibitors) along with chemotherapy. PubMed+1

  4. Early T-cell precursor ALL (ETP-ALL) – This is a high-risk form of T-ALL with features of both T cells and early blood stem cells. It needs strong and carefully planned treatment. PubMed+1

  5. Infant ALL – ALL that appears in babies, usually under one year old. It often has changes in a gene called KMT2A (MLL) and behaves differently, so treatment plans are special for this age group. Medscape+1

  6. Relapsed or refractory ALL (by history) – This is not a separate biological type, but doctors may describe ALL as “relapsed” if it comes back after treatment, or “refractory” if it never goes into full remission. Type and genetics still guide therapy. NCBI+1

Causes and risk factors

For most children with ALL, no clear single cause is found. Doctors talk about risk factors, which are things that can increase the chance of the disease. Having one or more risk factors does not mean a child will definitely get leukaemia. Many children with ALL have no known risk factor. NewYork-Presbyterian+1

  1. Genetic syndromes such as Down syndrome – Children with certain chromosome problems, especially Down syndrome (trisomy 21), have a higher chance of developing ALL, probably because of changes in how blood cells grow and repair DNA. NewYork-Presbyterian+1

  2. Other inherited conditions – Rare genetic disorders like neurofibromatosis type 1, Li–Fraumeni syndrome, or familial DNA-repair problems can increase leukaemia risk because the cells’ genetic material is more likely to get damaged. NCBI+1

  3. Abnormal genes in the leukaemia cells – The leukaemia itself often carries gene fusions and chromosome changes (for example, BCR::ABL1, TEL-AML1, or hyperdiploidy). These are not always inherited but happen in bone marrow cells and make them grow out of control. Cancer.gov+1

  4. Previous chemotherapy for another cancer – Children treated earlier with certain strong chemotherapy drugs (especially alkylating agents or topoisomerase II inhibitors) have a higher risk of developing a second leukaemia years later. NCBI+1

  5. Previous radiation therapy – High-dose radiation to treat other cancers or conditions can damage bone marrow cells. Over time this damage can sometimes lead to leukaemia, including ALL, as a late effect of treatment. NCBI+1

  6. High-dose environmental radiation – Very high radiation exposure, such as being near nuclear accidents or atomic bomb blasts, is linked with a higher risk of childhood leukaemia, though this is rare worldwide. Cleveland Clinic+1

  7. Benzene and some other chemicals – Long-term exposure to benzene, a chemical used in some industries and found in petrol fumes and cigarette smoke, is linked to leukaemia risk. In children this is less common but still a concern. Cleveland Clinic

  8. Pesticide exposure – Some studies suggest that heavy exposure to certain pesticides in the home or in farms may raise the risk of childhood ALL, but the evidence is not fully clear and may vary between studies. Frontiers

  9. Maternal smoking in pregnancy – Smoking while pregnant may slightly increase a baby’s risk of leukaemia. Chemicals from cigarette smoke can cross the placenta and may affect the baby’s developing blood cells. Cleveland Clinic+1

  10. High birth weight or fast growth in early life – Some research shows that babies with higher birth weight or rapid early growth may have a slightly greater risk of ALL, possibly because of higher levels of growth factors that also stimulate blood cells. Frontiers

  11. Planned (elective) caesarean section – Recent studies suggest that babies born by planned caesarean section (before labour starts) may have a slightly increased risk of ALL, although the overall risk remains very low. Live Science

  12. Immune system problems – Children with weakened immune systems (for example, from HIV infection, medicines that suppress immunity, or inherited immune defects) have a higher chance of developing blood cancers like ALL. NewYork-Presbyterian+1

  13. Very early infections in a special pattern – One theory suggests that lacking normal early-life infections and then meeting certain infections later may disturb the immune system and trigger leukaemia in some children who are already at risk genetically. Frontiers

  14. Male sex – Boys have a slightly higher rate of childhood ALL than girls. The exact reasons are unclear and may relate to both hormones and genetic factors. htct.com.br+1

  15. Age between 2 and 5 years – ALL is most common in young children between about 2 and 5 years. This pattern suggests that changes in the immune system and bone marrow during these years may make this age a peak risk time. htct.com.br+1

  16. Family history of leukaemia – A close relative with leukaemia can slightly increase risk, especially if the leukaemia occurred at a young age. This may show shared genes or shared environmental exposures. Cleveland Clinic

  17. Obesity and poor diet – In some studies, overweight children or mothers, and diets high in processed foods and low in fruits and vegetables, may show a higher risk of childhood leukaemia, but the evidence is still being studied. Frontiers

  18. Parental exposure to chemicals at work – Parents who work with solvents, some industrial chemicals, or pesticides may bring small amounts home or have exposures that slightly raise their children’s risk. Data are mixed but this is under study. Frontiers

  19. Lack of breastfeeding (possible) – Some research suggests that breastfeeding may protect slightly against childhood leukaemia, so not being breastfed might remove this small protection. The link is not strong but is being explored. Frontiers

  20. Unknown or random DNA changes – In most children, doctors cannot find a clear cause. Many cases seem to come from random DNA changes in one early blood cell during pregnancy or early life that later grow into leukaemia. NewYork-Presbyterian+1

Symptoms and signs

Symptoms of paediatric ALL mostly come from too few healthy blood cells and too many leukaemia cells in the bone marrow or other organs. Symptoms often develop over days to weeks. Many signs are similar to common childhood illnesses, so tests are needed to be sure. American Cancer Society+1

  1. Tiredness and weakness – Children often feel very tired, weak, or lacking in energy. This happens because there are not enough red blood cells to carry oxygen around the body, causing anaemia. American Cancer Society+1

  2. Pale skin – The child may look more pale than usual, especially on the face, lips, and inner eyelids. This is another clear sign of anaemia due to low red blood cell counts. American Cancer Society+1

  3. Shortness of breath – The child may breathe faster, get breathless when playing, or feel like they cannot catch their breath, again because the blood carries less oxygen. American Cancer Society+1

  4. Frequent infections and fevers – Because normal white blood cells are low and poor in quality, the child gets infections more often. They may have fevers that do not go away or keep coming back. American Cancer Society+1

  5. Easy bruising and bleeding – With low platelets, the child bruises easily, may have nosebleeds, bleeding gums, or small red or purple spots on the skin called petechiae, even after mild knocks. American Cancer Society+1

  6. Bone or joint pain – Children can complain of pain in legs, arms, or joints. This pain is caused by leukaemia cells pressing inside the bone marrow and on the hard outer bone. American Cancer Society+1

  7. Swollen lymph nodes – Painless lumps may appear in the neck, under the arms, or in the groin. These are enlarged lymph nodes filled with leukaemia cells and reacting immune cells. American Cancer Society+1

  8. Swollen abdomen (tummy) – The liver and spleen may become large because of leukaemia cells building up. The child’s belly can look swollen or feel full or uncomfortable. American Cancer Society+1

  9. Night sweats and fevers – Some children have drenching night sweats and fevers, even without a clear infection. This reflects the body’s response to cancer cells and the release of inflammatory substances. American Cancer Society+1

  10. Loss of appetite and weight loss – Children may not feel like eating and may lose weight. A large liver or spleen pressing on the stomach can also make them feel full quickly. American Cancer Society+1

  11. Headaches and nervous system problems – If leukaemia spreads to the brain or spinal fluid, a child can have headaches, vomiting, blurred vision, seizures, or trouble with balance or concentrating. American Cancer Society+1

  12. Chest pain or breathing trouble from a chest mass – In T-ALL, there may be a big cluster of cells in the chest (mediastinal mass). This can cause cough, chest pain, swelling of the face, or serious breathing problems. Medscape+1

  13. Bone fractures from weak bones (rare) – In some children, the bones become thin or weak from disease and can break more easily, causing sudden pain and difficulty moving. American Cancer Society+1

  14. General unwell feeling – Many children just feel “not right” with vague aches, low energy, irritability, or not wanting to play. These early signs can be easily mistaken for viral infections. American Cancer Society+1

  15. Swelling of gums or skin rashes (less common in ALL) – Sometimes white blood cells can build up in the gums causing swelling and bleeding, or appear as skin spots or lumps, though this is more typical of other leukaemias. American Cancer Society+1

Diagnostic tests

Doctors use several groups of tests to confirm paediatric ALL, find its type, and plan treatment. These include physical examination, manual bedside checks, lab and pathological tests, electrodiagnostic tests, and imaging tests. Usually a combination is used, not just one test. Cancer Research UK+1

Physical examination tests

  1. Full body physical examination – The doctor looks at the child’s overall condition, checks temperature, heart rate, breathing rate, and blood pressure, and looks for signs like pallor, bruising, and rashes. This helps decide if leukaemia is likely and what tests to do next. American Cancer Society+1

  2. Lymph node examination – The doctor gently feels the neck, underarms, and groin for enlarged lymph nodes. Firm, painless, multiple swollen nodes can suggest blood cancer and guide further testing. American Cancer Society+1

  3. Abdominal exam for liver and spleen – By feeling and tapping the abdomen, the doctor checks if the liver and spleen are enlarged. A big liver and spleen are common in ALL and help in staging the disease. American Cancer Society+1

Manual bedside tests

  1. Manual bone and joint tenderness check – The doctor presses gently over bones and joints to see if they are painful. Bone or joint pain with tenderness is a clue that leukaemia cells are inside the bone marrow. American Cancer Society+1

  2. Manual neurological exam (strength and reflexes) – The doctor tests muscle strength, reflexes, sensation, and coordination. Changes can suggest that leukaemia has spread to the brain or spinal cord, or that there are effects from anaemia or high cell counts. American Cancer Society+1

  3. Manual respiratory and heart exam – By listening with a stethoscope and feeling the chest, the doctor checks for abnormal breath sounds, signs of fluid, or effects of a mediastinal mass on breathing and blood flow. Medscape+1

Lab and pathological tests

  1. Complete blood count (CBC) with differential – This blood test counts red cells, white cells, and platelets and shows the types of white cells present. In ALL, counts may be high, normal, or low, but blasts (very young cells) are often seen, which raises strong suspicion. American Cancer Society+1

  2. Peripheral blood smear – A drop of blood is spread on a glass slide and looked at under a microscope. Doctors can see blasts and other abnormal cells. The shape and appearance of cells give important early clues about leukaemia. Cancer Research UK+1

  3. Bone marrow aspiration – This key test uses a needle to draw liquid bone marrow, usually from the hip bone, under local anaesthesia and sometimes sedation. In ALL, more than 25% of the cells in the marrow are lymphoblasts, confirming the diagnosis. Cancer Research UK+1

  4. Bone marrow biopsy – A small core of bone and marrow is taken with a thicker needle. This shows how packed the marrow is with leukaemia cells and helps assess how well normal blood formation is preserved. bestpractice.bmj.com+1

  5. Flow cytometry / immunophenotyping – Special markers on cell surfaces are measured using antibodies and a machine called a flow cytometer. This test confirms whether the blasts are B-cell or T-cell type and helps classify the exact subtype of ALL. bestpractice.bmj.com+1

  6. Cytogenetic analysis (karyotype and FISH) – Chromosomes of leukaemia cells are examined to look for extra, missing, or rearranged pieces, such as the Philadelphia chromosome. These findings help predict risk and guide targeted treatments. Cancer.gov+1

  7. Molecular genetic tests (PCR/NGS and MRD tests) – Very sensitive methods like PCR or next-generation sequencing look for specific gene changes and can measure minimal residual disease (MRD). MRD shows how many leukaemia cells remain after treatment and is a strong guide for therapy intensity. Cancer.gov+1

  8. Blood chemistry tests (liver, kidney, uric acid, LDH, electrolytes) – These blood tests show how well organs are working and look for tumour lysis (cell breakdown) markers like high uric acid and LDH. They are vital both at diagnosis and during treatment. NewYork-Presbyterian+1

  9. Coagulation profile (clotting tests) – Tests like PT, aPTT, and fibrinogen levels check blood clotting. They help detect bleeding or clotting problems that may happen in leukaemia or during intensive treatment. NCBI+1

  10. Cerebrospinal fluid (CSF) analysis via lumbar puncture – A thin needle is used to take fluid from around the spinal cord. This fluid is checked for leukaemia cells. Finding blasts in CSF means there is central nervous system involvement and affects treatment planning. NCCN+1

Electrodiagnostic tests

  1. Electrocardiogram (ECG) – This test records the heart’s electrical activity using small stickers on the chest and limbs. It is done before and during treatment to check for heart rhythm problems, especially when using drugs that may affect the heart. NCCN+1

  2. Electroencephalogram (EEG) (if seizures occur) – EEG measures brain electrical activity using electrodes on the scalp. In a child with seizures or suspected brain involvement, it helps assess how the brain is functioning and whether there are abnormal electrical patterns. American Cancer Society+1

Imaging tests

  1. Chest X-ray – A simple X-ray picture of the chest can show a mediastinal mass (a collection of cells in the middle of the chest) or fluid around the lungs. This is important in T-ALL because a large mass can cause serious breathing problems. Cancer Research UK+1

  2. MRI or CT scan (especially of brain or body) – MRI and CT use strong magnets or X-rays to create detailed images of the brain, spine, chest, or abdomen. They help show enlarged organs, masses, or bleeding and are used when doctors suspect central nervous system or organ involvement. NCCN+1

Non-Pharmacological Treatments (Therapies and Others)

1. Psychological counseling for child and parents
Counseling is talking with a trained psychologist or counselor about feelings, fears, and stress during leukemia treatment. The purpose is to reduce anxiety, sadness, and behavior problems, and to help the whole family cope. The counselor uses simple language, stories, and age-appropriate activities. The mechanism is emotional support and teaching coping skills such as deep breathing, problem-solving, and positive thinking. This support can improve sleep, cooperation with treatment, and quality of life for the child and the caregivers.Frontiers

2. Play therapy
Play therapy uses toys, dolls, drawing, and games to help the child express feelings that are hard to say with words. The purpose is to make the hospital less frightening and to help the child feel more in control. The therapist watches how the child plays and gently guides the play. The mechanism is that play is the natural “language” of children. Through play, they process fear, pain, and confusion, which can reduce anxiety and improve cooperation with procedures.

3. Art and music therapy
Art and music therapy use drawing, painting, crafts, singing, or playing simple instruments. The purpose is to give the child a safe way to express feelings and distract from pain or nausea. The mechanism is that creative activities activate brain areas linked with pleasure and relaxation. This lowers stress hormones, eases pain perception, and improves mood and coping during long treatment days.Frontiers

4. Physical exercise and physiotherapy
Exercise and physiotherapy programs are specially designed for children with ALL. Simple walking, stretching, balance work, and light games are used. The purpose is to maintain muscle strength, balance, and heart fitness, and to reduce treatment-related fatigue. The mechanism is that safe, supervised exercise improves circulation, muscle function, and energy levels and can reduce cancer-related fatigue and improve quality of life. Studies show exercise improves endurance and functional mobility in children with ALL.PMC+1

5. Occupational therapy (energy saving and daily skills)
Occupational therapists help the child manage daily activities like dressing, bathing, writing, and playing with less effort. The purpose is to keep independence and reduce frustration. The mechanism is teaching energy-saving techniques, using special tools (like grip aids), and adjusting tasks to match the child’s strength level. This reduces fatigue and supports normal development during treatment.

6. School support and home tutoring
School support may include hospital school, home tutoring, or online classes. The purpose is to keep the child connected with education and friends and reduce the feeling of being “left behind.” The mechanism is adapting school tasks to the child’s energy, immune status, and hospital schedule. This maintains learning, routine, and self-esteem, which are very important for long treatments.

7. Family education sessions
Healthcare teams give structured teaching sessions for parents and older children about leukemia, medicines, side effects, infection prevention, and nutrition. The purpose is to empower families to care safely for the child at home. The mechanism is clear, repeated information in simple words, with written plans and checklists. This improves adherence to treatment, early detection of complications, and reduces fear.Sheba Medical Center

8. Infection-prevention training and home hygiene
Nurses teach hand hygiene, mask use when needed, safe food handling, and how to avoid crowded or high-risk places during low immunity periods. The purpose is to reduce dangerous infections when white cells are low. The mechanism is breaking the chain of germ transmission by good handwashing, cleaning surfaces, avoiding raw or contaminated food, and limiting contact with sick people.

9. Pain management techniques (non-drug)
Non-drug pain methods include distraction (games, videos), relaxation, guided imagery, positioning, warm or cold packs (only if allowed), and gentle massage. The purpose is to decrease pain from procedures and bone pain. The mechanism is that attention is moved away from pain, muscles relax, and the nervous system becomes less sensitive, so pain signals feel weaker and more tolerable.

10. Sleep hygiene and routine planning
Good sleep hygiene means regular bedtimes, quiet dark room, limited screen time before sleep, and calming evening routines. The purpose is to improve sleep quality, which affects mood, immunity, and healing. The mechanism is supporting the body’s natural circadian rhythm and melatonin production, so the child feels less tired and irritable and copes better with treatment.

11. Nutritional counseling by dietitian
A pediatric oncology dietitian checks the child’s weight, appetite, and lab tests, and makes an individual eating plan. The purpose is to prevent malnutrition, support growth, and reduce treatment side effects like constipation or diarrhea. The mechanism is giving enough calories, protein, vitamins, and fluids through suitable foods or supplements, and adjusting texture when the mouth is sore or nausea is present.Sheba Medical Center

12. Social work and financial counseling
Social workers help families find financial aid, transport support, school adjustments, and community resources. The purpose is to reduce stress from money and practical issues. The mechanism is linking families with government programs, charity groups, and hospital funds, and helping with paperwork. Lower stress helps parents focus on the child’s care.

13. Behavioral therapy for anxiety and procedures
Behavioral therapy uses step-by-step exposure, coping plans, and rewards to help children face needles, scans, and other procedures. The purpose is to reduce extreme fear and struggle during medical care. The mechanism is teaching the child to link procedures with coping skills instead of panic, which over time lowers anxiety and improves cooperation.Frontiers

14. Rehabilitation programs after intensive phases
After strong chemotherapy blocks, children may be very weak. Multi-disciplinary rehab programs combine physiotherapy, occupational therapy, and sometimes speech therapy. The purpose is to rebuild strength, coordination, and daily function. The mechanism is gradual, supervised training that gently stresses muscles and nerves so they recover without over-fatigue.

15. Palliative care and symptom-management team
Palliative care is not only for end of life. It is a support team that focuses on comfort, pain control, nausea control, and emotional support at any stage. The purpose is to improve quality of life during and after treatment. The mechanism is expert symptom assessment and holistic care, so children can play, sleep, and interact better despite illness and treatment.

16. Spiritual or meaning-centered support (if family wishes)
Some families find strength in their own spiritual or meaning-based beliefs. Hospital chaplains or similar support persons may offer listening and comfort, respecting each family’s culture and choices. The purpose is to help families find hope and meaning. The mechanism is emotional support, shared reflection, and respect for values, which can reduce distress.

17. Digital health tools and diaries
Families may use apps or simple paper diaries to track temperatures, symptoms, medicine times, and clinic visits. The purpose is to stay organized and to spot problems early. The mechanism is regular recording and reminders, which improves adherence and helps doctors quickly understand what is happening between visits.

18. Oral care programs
Nurses and dentists teach gentle mouth care, soft toothbrush use, and sometimes special mouth rinses. The purpose is to prevent mouth sores and infections that worsen pain and reduce eating. The mechanism is keeping the mouth clean and moist, reducing bacterial load, and protecting the fragile lining of the mouth during chemotherapy.

19. Safe play and infection-aware recreation
Staff help plan safe indoor games and activities that do not expose the child to germs or injury. The purpose is to allow normal play and joy while respecting medical limits. The mechanism is careful choice of clean toys, small groups, and avoiding rough contact or crowded areas, so the child can still have fun and social contact.

20. Parent support groups
Support groups connect parents who are going through similar experiences. The purpose is to share practical tips, coping strategies, and emotional support. The mechanism is peer-to-peer communication, which reduces isolation, provides realistic hope, and can improve mental health and coping for caregivers.


Major Drug Treatments (Chemotherapy and Targeted Drugs)

(Doses and exact schedules are always decided by pediatric oncologists using specialized protocols. The information here is general and educational, not a dosing guide.)

Chemotherapy for pediatric ALL usually uses a backbone of glucocorticoids (prednisone or dexamethasone), vincristine, asparaginase, methotrexate, and 6-mercaptopurine, with other drugs added for risk groups.PMC+1

1. Vincristine
Vincristine is a chemotherapy drug from the vinca alkaloid class. It blocks microtubules in dividing cells, so leukemia cells cannot divide and die. It is given by intravenous injection on specific days in each treatment phase. The dose is calculated from body size. Purpose is to kill fast-growing leukemia cells and help reach remission. Side effects may include nerve damage (tingling, weakness), constipation, jaw pain, and hair loss, so doctors watch very carefully.FDA Access Data+1

2. Prednisone
Prednisone is a glucocorticoid (steroid) used in induction therapy. It causes leukemia lymphoblasts to die (apoptosis) by changing cell signals. It is given by mouth at set times each day for some weeks, then tapered. Purpose is rapid reduction in leukemia cell number and symptom relief. Side effects can include increased appetite, weight gain, mood changes, high blood sugar, and infection risk, so monitoring is essential.PMC

3. Dexamethasone
Dexamethasone is another glucocorticoid, often used instead of prednisone in some protocols because it may penetrate the brain and spinal fluid better. It is taken orally or intravenously on a schedule. Purpose is strong leukemia cell kill and central nervous system protection. Mechanism is similar to prednisone but with different potency. Side effects include muscle weakness, mood and sleep changes, bone thinning, and higher infection risk, so doctors balance benefit and risk.Cancer.gov+1

4. Pegaspargase (PEG-asparaginase, e.g., Oncaspar, Asparlas)
Pegaspargase is an enzyme drug that removes the amino acid asparagine from the blood. Leukemia cells depend on asparagine to grow, so when it is removed, they die. It is part of combination chemotherapy and is given intravenously or intramuscularly every few weeks. Purpose is to starve leukemia cells. Side effects include allergic reactions, pancreatitis, liver toxicity, and blood-clotting problems, so children are monitored closely during and after infusions.FDA Access Data+2FDA Access Data+2

5. L-asparaginase (Erwinia or other forms, e.g., Rylaze)
When children cannot tolerate E. coli-based asparaginase or pegaspargase, Erwinia asparaginase (such as Rylaze) may be used. It has the same purpose: remove asparagine to kill leukemia cells. It is given as repeated injections. Side effects are similar: allergic reactions, pancreatitis, and liver changes, so dosing and monitoring are very strict.FDA Access Data

6. Methotrexate (systemic and intrathecal)
Methotrexate is an antimetabolite that blocks folate pathways needed for DNA synthesis. It is used in many phases: intravenous high-dose, oral low-dose maintenance, and intrathecal (into spinal fluid) to protect the brain and spinal cord. Purpose is to kill hidden leukemia cells in the body and central nervous system. Side effects include mouth sores, liver enzyme elevation, low blood counts, and rarely kidney or lung problems; rescue medicines and careful monitoring reduce risk.Cancer.gov+1

7. 6-Mercaptopurine (6-MP)
6-MP is an oral antimetabolite used mainly in the maintenance phase. It gets built into DNA and RNA of fast-growing cells, causing damage and cell death. Children usually take it every day for many months or years as part of long maintenance therapy. Purpose is to keep leukemia away after remission. Side effects include low blood counts, liver toxicity, and, in some children, severe toxicity if they have certain enzyme gene variants, so blood tests guide dosing.Cancer Therapy Advisor+1

8. Cytarabine (Ara-C)
Cytarabine is a nucleoside analog that disrupts DNA synthesis in dividing cells. It is given intravenously or under the skin in certain blocks (consolidation and intensification). Purpose is to deeply clean the bone marrow of remaining leukemia cells. Side effects include low blood counts, nausea, fever, and in higher doses, neurologic effects or eye irritation, so protective eye drops and monitoring may be used.PMC+1

9. Cyclophosphamide
Cyclophosphamide is an alkylating agent that damages DNA in cancer cells. It is used in some protocols for higher-risk disease. Purpose is to provide strong additional leukemia cell kill. It is given intravenously, with fluids and bladder-protective medicine to reduce urine-related side effects. Side effects include low blood counts, hair loss, nausea, and risk of infertility later in life, so use is carefully balanced and limited in modern protocols.Cancer.gov+1

10. Doxorubicin or Daunorubicin (anthracyclines)
These are anthracycline chemotherapy drugs that intercalate into DNA and block topoisomerase II, causing breaks in DNA. They are used mainly in induction and consolidation, especially for higher-risk patients. Purpose is powerful leukemia cell kill. Side effects include low blood counts, hair loss, nausea, and possible long-term heart damage, so total lifetime dose is limited and heart function is monitored with echocardiograms.PMC+1

11. Thioguanine
Thioguanine is a purine analog similar to 6-MP, sometimes used in intensification phases. It becomes part of DNA and interferes with cell division. Purpose is additional marrow “deep cleaning” in some protocols. Side effects include low blood counts and possible liver toxicity, so dosing is carefully controlled.PMC+1

12. Nelarabine (for T-cell ALL)
Nelarabine is a nucleoside analog mainly used for T-cell ALL, especially relapsed or high-risk disease. It is given intravenously in specific courses. Purpose is targeted killing of T-cell leukemia cells that do not respond well to standard drugs. Main side effect risk is nerve toxicity (weakness, numbness), plus low blood counts and infections, so close neurological checks are needed.Medscape

13. Imatinib (for Philadelphia chromosome–positive ALL)
Imatinib is a tyrosine kinase inhibitor (TKI) that blocks the BCR-ABL protein made by the Philadelphia chromosome. It is given by mouth with chemotherapy for Ph+ ALL. Purpose is to directly target the abnormal signal that drives leukemia cell growth. Side effects may include nausea, swelling, muscle cramps, and liver test changes, but it greatly improves outcomes for this subgroup.Cancer.gov+1

14. Dasatinib (second-generation TKI)
Dasatinib is another BCR-ABL TKI used for Ph+ ALL, sometimes instead of or after imatinib. It may work when leukemia is resistant to earlier TKIs. Purpose and mechanism are similar: block abnormal signaling in leukemia cells so they die. Side effects can include low blood counts, fluid around the lungs, and bleeding risk, so imaging and blood tests are monitored.Cancer.gov+1

15. Blinatumomab
Blinatumomab is a bispecific T-cell engager antibody that links T cells to CD19-positive B-cell leukemia cells, helping the immune system kill them. It is given as a continuous intravenous infusion over several weeks, often for relapsed or minimal residual disease-positive ALL. Purpose is to clear very small amounts of leukemia. Side effects include cytokine release syndrome (fever, low blood pressure) and neurologic symptoms, so treatment is done in experienced centers.PMC+1

16. Inotuzumab ozogamicin
Inotuzumab is an antibody-drug conjugate that targets CD22 on leukemia cells and delivers a toxic drug into them. It is used mainly for relapsed or refractory B-cell ALL. Purpose is to give highly targeted chemotherapy directly to leukemia cells. Side effects include low blood counts and risk of liver problems, including veno-occlusive disease, especially around stem cell transplantation.PMC+1

17. Tisagenlecleucel (CAR-T cell therapy)
Tisagenlecleucel is a form of CAR-T therapy where a child’s T cells are taken, genetically changed in a lab to recognize CD19 on leukemia cells, and then returned to the child. Purpose is to give a living “drug” that hunts and destroys leukemia cells, used for some children with relapsed or refractory ALL. Side effects can be serious, including cytokine release syndrome and neurologic events, so it is given only in highly specialized centers.PMC

18. Intrathecal chemotherapy (methotrexate ± cytarabine, hydrocortisone)
Intrathecal medicine is injected into the fluid around the spinal cord to prevent or treat leukemia in the central nervous system. The drugs are similar to intravenous medicines but at doses and combinations designed for the brain and spinal cord. Purpose is to protect the nervous system from relapse. Side effects include headache and temporary back pain; serious complications are rare but monitored.Cancer.gov+1

19. Supportive anti-infective drugs
Although not leukemia-killing chemotherapy, antibiotics, antifungals, and antivirals are essential drug treatments in ALL. They prevent or treat serious infections when immunity is low. They are given orally or intravenously, sometimes for long periods. Side effects depend on the drug but may include stomach upset, liver changes, or allergic reactions; doctors select carefully to balance risk and benefit.Cancer.gov+1

20. Growth factor support during chemotherapy (e.g., G-CSF)
Granulocyte colony-stimulating factor (G-CSF) drugs are sometimes used to help the bone marrow recover white cells faster after intense chemotherapy. Purpose is to reduce the length of severe neutropenia and lower infection risk. These injections stimulate the bone marrow to make more neutrophils. Side effects can include bone pain and rare spleen problems, so use is protocol-based and closely monitored.Cancer.gov


Dietary Molecular Supplements (Supportive, Not a Cure)

(Always ask the oncology team before giving any supplement. Some interact with chemotherapy.)

  1. Vitamin D – Supports bone health and immune function, especially important because steroids and limited sunlight can weaken bones. Dose is based on blood levels; too much can harm kidneys. Mechanism: helps calcium absorption and bone mineralization.

  2. Calcium – Often paired with vitamin D to protect bones from steroid-related thinning. Dose depends on age and diet. Mechanism: provides the mineral needed for strong bones and normal muscle and nerve function.

  3. Omega-3 fatty acids (fish oil) – May support heart and brain health and reduce inflammation. Dose is usually a small capsule or liquid measured by weight. Mechanism: changes cell membranes and signaling molecules to a more anti-inflammatory profile, but must be checked because of possible bleeding risk.

  4. Probiotics (only if doctor agrees) – In some patients, carefully chosen probiotics may help gut balance after antibiotics. Dose is usually measured in colony-forming units (CFU). Mechanism: support beneficial gut bacteria, which may aid digestion and possibly immune function; they are not safe for all immunocompromised children, so oncologist approval is essential.

  5. Multivitamin without iron (if recommended) – A simple multivitamin can cover small gaps in diet when appetite is low. Dose is age-appropriate once daily. Mechanism: provides a mix of vitamins and trace minerals that support normal body functions.

  6. Folate / folinic acid (only when prescribed) – Sometimes used as “rescue” after high-dose methotrexate or to correct deficiency. Dose and timing are strictly controlled by the oncologist. Mechanism: restores safe folate levels in healthy cells without helping leukemia cells too much in the specific rescue setting.Cancer.gov

  7. Iron (only if proven deficiency) – Given when tests show true iron-deficiency anemia, not just any anemia. Dose is based on weight and closely monitored. Mechanism: supplies iron to make hemoglobin in red blood cells; wrong use can cause side effects or feed infections, so it must be doctor-directed.

  8. Zinc – May be used if tests show low zinc and poor wound healing or appetite. Mechanism: supports immune function and skin repair. Dose is small and time-limited; too much can disturb other minerals.

  9. Vitamin B12 – Corrects deficiency due to poor intake or absorption. Mechanism: supports normal red blood cell production and nerve health. Dose may be oral or injection, depending on levels.

  10. High-energy, high-protein oral nutrition drinks – These are complete liquid feeds with protein, carbohydrates, fat, vitamins, and minerals. Mechanism: provide concentrated energy and nutrients when the child cannot eat enough solid food, helping to maintain weight and muscle during treatment.


Immunity-Booster / Regenerative / Stem-Cell–Related Drugs

(These are powerful medical treatments, not simple supplements. They are used only by specialists.)

  1. Granulocyte colony-stimulating factor (G-CSF, e.g., filgrastim) – Stimulates the bone marrow to produce more neutrophils. Used after certain chemotherapy blocks to shorten low-white-cell periods. Mechanism: binds to receptors on marrow precursor cells, pushing them to grow and mature.

  2. Pegylated G-CSF (e.g., pegfilgrastim) – Long-acting form of G-CSF given as a single injection per cycle in some protocols. Mechanism and purpose are similar but with longer effect, so fewer injections are needed.

  3. Intravenous immunoglobulin (IVIG) – Pooled antibodies from donors given by vein to children with very low antibody levels or recurrent infections. Mechanism: provides ready-made antibodies to fight infections and support immune function temporarily.

  4. Erythropoiesis-stimulating agents (e.g., epoetin alfa, rarely used) – Sometimes used to stimulate red blood cell production when anemia is severe and transfusions are difficult. Mechanism: mimics natural erythropoietin hormone to push marrow to make red cells.

  5. Thrombopoietin receptor agonists (e.g., eltrombopag, in special cases) – In selected situations, used to increase platelet production. Mechanism: stimulates megakaryocyte cells in marrow to make more platelets.

  6. Hematopoietic stem cell transplantation (HSCT, “bone marrow transplant”) drugs – Before stem cell transplant, conditioning chemotherapy ± radiation and immune-suppressive drugs prepare the child to receive donor stem cells; later, drugs like cyclosporine prevent graft-versus-host disease. Mechanism: replaces diseased marrow with healthy donor stem cells that can make new, normal blood cells and immune cells.Cancer.gov


Surgeries and Invasive Procedures

  1. Central venous catheter (port or Hickman line) insertion
    A surgeon places a long-term central line into a large chest vein under anesthesia. Purpose: safe, repeated access for chemotherapy, blood tests, and transfusions without constant needle sticks.

  2. Lumbar puncture with intrathecal chemotherapy
    A needle is placed into the spinal fluid in the lower back under sterile conditions, often with sedation. Purpose: to check for leukemia in the central nervous system and to give protective chemotherapy directly into spinal fluid.

  3. Bone marrow aspiration and biopsy
    A needle is inserted into the hip bone under local anesthesia and often sedation. Purpose: to diagnose leukemia, assess response to treatment, and check minimal residual disease.

  4. Feeding tube (nasogastric or gastrostomy) placement
    If a child cannot eat enough, a tube is placed through the nose into the stomach or through the abdominal wall. Purpose: to provide reliable nutrition and medicines when oral intake is inadequate.

  5. Hematopoietic stem cell transplantation procedure
    Although much of transplant is medical, there are surgical and procedural parts including central line placement and sometimes bone marrow harvest from donors under anesthesia. Purpose: to allow infusion of donor stem cells to rebuild the child’s blood and immune system in high-risk or relapsed ALL.Cancer.gov


Prevention (Mainly Prevention of Complications and Relapse)

  1. Follow the full treatment plan exactly as the oncology team prescribes.

  2. Attend all clinic visits and lab checks, even when the child seems well.

  3. Use good hand hygiene for the child and all visitors.

  4. Avoid contact with people who have fever, cough, or known infections.

  5. Keep vaccines up to date according to the oncologist’s schedule (some live vaccines are delayed).

  6. Maintain good oral care to reduce mouth infections.

  7. Give medicines, including antibiotics and mouth rinses, exactly as instructed.

  8. Provide a balanced diet and enough fluids to support healing and drug metabolism.

  9. Protect the child from extreme sun or injury when blood counts are low.

  10. Quickly report any new worrying symptom (fever, bruising, breathing trouble) to the care team.Clinical Advisor+1


When to See Doctors or Go to Emergency

You should contact the child’s oncology team immediately or go to emergency if:

  • Fever (often ≥38.0 °C) or feeling very hot and unwell.

  • Chills, shivering, or looking very pale and weak.

  • Trouble breathing, fast breathing, or chest pain.

  • New or rapidly increasing bruises, nosebleeds, or bleeding that will not stop.

  • Severe headache, confusion, seizures, or sudden change in behavior.

  • Severe stomach pain, vomiting many times, or no pee / very little pee.

  • Extreme sleepiness, not waking properly, or sudden change in walking or talking.

  • Any symptom that worries the parent’s “gut feeling” that something is seriously wrong.

On regular days, see the oncology team for planned visits, vaccine planning, school letters, and long-term follow-up of growth, learning, and late effects.Cancer.gov+1


What to Eat and What to Avoid

(Always ask the hospital dietitian; food rules vary with local germs and the child’s immunity.)

  1. Eat: Well-cooked meats, fish, eggs, and pulses for protein; Avoid: Raw or undercooked meats, raw eggs, and sushi.

  2. Eat: Freshly washed and peeled fruits and well-cooked vegetables; Avoid: Unwashed raw vegetables and salad from unknown places.

  3. Eat: Whole grains like rice, bread, pasta for energy; Avoid: Very spicy or fried foods if they worsen nausea or diarrhea.

  4. Eat: Yogurt or pasteurized dairy if allowed; Avoid: Unpasteurized milk or cheese.

  5. Eat: Plenty of safe fluids (water that is boiled or bottled, oral rehydration solution); Avoid: Sugary soft drinks in large amounts and energy drinks.

  6. Eat: Small, frequent meals and snacks to keep energy up; Avoid: Forcing large meals that cause vomiting.

  7. Eat: Foods rich in fiber (if no gut restriction) to prevent constipation; Avoid: Very tough, dry foods that are hard to chew when the mouth is sore.

  8. Eat: Simple, bland foods (rice, banana, toast) during nausea; Avoid: Strong smells and very heavy meals at those times.

  9. Eat: Only food prepared in a clean kitchen with clean hands; Avoid: Street food or buffet food that may have sat out for hours.

  10. Eat: Any special therapeutic food or drink recommended by the dietitian; Avoid: Herbal or “miracle” supplements not approved by the oncology team, because they can interact with chemotherapy.Sheba Medical Center


Frequently Asked Questions (FAQs)

1. Is pediatric acute lymphoblastic leukemia curable?
Yes. Many children with ALL can be cured with modern multi-drug chemotherapy and, in some cases, targeted therapy or stem cell transplant. Cure rates in high-income settings are now around 85–90% for many risk groups when treatment is given correctly.Cancer.gov+1

2. How long does treatment usually last?
Treatment commonly lasts about 2–3 years, including induction, consolidation/intensification, and long maintenance therapy. The exact length depends on the protocol and risk group.Cancer.gov+1

3. Why are so many different chemotherapy drugs used?
Different drugs attack leukemia cells in different ways and at different stages of the cell cycle. Using several drugs together gives stronger killing power and reduces the chance that the leukemia will become resistant to any one drug.

4. Will my child lose hair?
Many chemotherapy drugs used in ALL cause hair loss, especially anthracyclines like doxorubicin. Hair usually grows back after treatment, though sometimes the texture or color may change slightly.

5. Can my child go to school during treatment?
Many children attend school part-time or use home or online teaching when blood counts and energy levels allow. The oncology team and school can work together to create a safe plan.

6. Is pain normal during treatment?
Some pain, such as bone pain, mouth sores, or procedure pain, can occur. However, pain should always be reported and treated. Both medicines and non-drug methods are used to control pain.

7. What about infections?
Infections are a major risk because chemotherapy lowers white blood cells. Families learn strict fever rules and infection-prevention steps. Early treatment of fever can save life.Cancer.gov+1

8. Will treatment affect growth and puberty?
Some drugs and steroids can slow growth or affect hormones. Doctors track height, weight, and puberty over time and may involve endocrinologists if needed.

9. Can my child play sports or exercise?
Light to moderate exercise, guided by the care team and physiotherapists, is often encouraged and can improve strength and mood. High-contact or risky sports are usually limited during periods of low blood counts.PMC+1

10. Will my child be able to have children in the future?
Some chemotherapy drugs, especially alkylating agents and radiation, can affect fertility. Modern pediatric protocols try to limit these. Discuss fertility risks and possible preservation options with the team before high-risk treatments.Cancer.gov

11. What is minimal residual disease (MRD)?
MRD is the very small number of leukemia cells that may remain after treatment, measured with sensitive lab tests. It helps doctors decide if the child is responding well or needs stronger therapy.

12. When is stem cell transplant needed?
Stem cell transplant is usually considered for high-risk, relapsed, or certain genetic types of ALL. It replaces diseased marrow with donor marrow and carries extra risks, so it is used only when benefits are greater than risks.Cancer.gov+1

13. Are “natural” or alternative cures safe?
There is no proven natural cure for ALL. Some herbs or supplements can interfere with chemotherapy or increase side effects. Always discuss any non-prescribed product with the oncology team before use.

14. How can parents cope emotionally?
Seeking counseling, joining support groups, taking turns for rest, and accepting help from friends and family are important. Caring for a child with leukemia is very stressful, and getting help is a sign of strength, not weakness.Frontiers

15. What is the most important thing I can do as a parent or caregiver?
The most important things are: follow the treatment plan, keep good communication with the care team, watch carefully for warning signs, and provide love, comfort, and stability for the child. Your presence and calm support are powerful “medicines” alongside chemotherapy.

Disclaimer: Each person’s journey is unique, treatment plan, life style, food habit, hormonal condition, immune system, chronic disease condition, geological location, weather and previous medical  history is also unique. So always seek the best advice from a qualified medical professional or health care provider before trying any treatments to ensure to find out the best plan for you. This guide is for general information and educational purposes only. Regular check-ups and awareness can help to manage and prevent complications associated with these diseases conditions. If you or someone are suffering from this disease condition bookmark this website or share with someone who might find it useful! Boost your knowledge and stay ahead in your health journey. We always try to ensure that the content is regularly updated to reflect the latest medical research and treatment options. Thank you for giving your valuable time to read the article.

The article is written by Team RxHarun and reviewed by the Rx Editorial Board Members

Last Updated: December 31, 2025.

PDF Documents For This Disease Condition

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  5. History of rare diseases and their genetic.[rxharun.com]
  6. health-care-and-rare-disorders.[rxharun.com]
  7. Rare Disease Registries.[rxharun.com]
  8. autoimmune-Rare-Genetic-Diseases.[rxharun.com]
  9. Rare Genetic Diseases.[rxharun.com]
  10. rare-disease-day.[rxharun.com]
  11. Rare_Disease_Drugs_e.[rxharun.com]
  12. fda-CDER-Rare-Diseases-Public-Workshop-Master.[rxharun.com]
  13. rare-and-inherited-disease-eligibility-criteria.[rxharun.com]
  14. FDA-rare-disease-list.pdf-rxharun.com1 Human-Gene-Therapy-for-Rare Diseases_Jan_2020fda.[rxharun.com]
  15. FDA-rare-disease-lists.[rxharun.com]
  16. 30212783fnl_Rare Disease.[rxharun.com]
  17. FDA-rare-disease-list.[rxharun.com]
  18. List of rare disease.[rxharun.com]
  19. Genome Res.-2025-Steyaert-755-68.[rxharun.com]
  20. uk-practice-guidelines-for-variant-classification-v4-01-2020.[rxharun.com]
  21. PIIS2949774424010355.[rxharun.com]
  22. hidden-costs-2016.[rxharun.com]
  23. B156_CONF2-en.[rxharun.com]
  24. IRDiRC_State-of-Play-2018_Final.[rxharun.com]
  25. IRDR_2022Vol11No3_pp96_160.[rxharun.com]
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  33. ENG_White paper_A4_Digital_FINAL.[rxharun.com]
  34. UK_Strategy_for_Rare_Diseases.[rxharun.com]
  35. MalaysiaRareDiseaseList.[rxharun.com]
  36. EURORDISCARE_FULLBOOKr.[rxharun.com]
  37. EMHJ_1999_5_6_1104_1113.[rxharun.com]
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  39. be-counted-052722-WEB.[rxharun.com]
  40. RDI-Resource-Map-AMR_MARCH-2024.[rxharun.com]
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