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Infant acute lymphoblastic leukaemia: the decisions you may face

4 treatment settings, 4 with more than one named option. Each section lays out the options the standard of care names, what each is for, the trials behind them with their recorded results, the side effects and cautions on record, and questions to ask. Built from the cancer page's standard-of-care rows; nothing here is advice for your case.

Interfant backbone: a week of prednisone, then dexamethasone, vincristine, cytarabine, daunorubicin and asparaginase with intrathecal therapy.

The options, in plain words

Prednisone is the everyday steroid tablet in cancer care. It is the P in CHOP and MOPP for lymphoma, part of childhood leukaemia treatment, and taken with abiraterone in prostate cancer.

Dexamethasone is a long-acting glucocorticoid steroid that kills lymphoid cancer cells directly, which is why it sits in nearly every myeloma regimen and in childhood leukaemia and lymphoma protocols. It is also the standard drug for preventing chemotherapy sickness and for brain swelling and spinal cord compression; high blood sugar, insomnia, muscle wasting and infection follow prolonged use.

A plant-derived chemotherapy from the Madagascar periwinkle that has been in almost every childhood leukaemia and lymphoma regimen since the 1960s.

Cytarabine is the core chemotherapy for acute myeloid leukaemia, given with an anthracycline as the classic 7+3 induction and at high doses for consolidation; it is also injected into the spinal fluid to treat or prevent leukaemia in the brain.

Daunorubicin is the anthracycline most often combined with cytarabine to bring acute myeloid leukaemia into remission; it is also part of induction for acute lymphoblastic leukaemia.

An enzyme that starves leukaemia cells of an amino acid they cannot make; a mainstay of childhood ALL therapy for 50 years, with new versions solving allergy and supply problems.

The 1948 antifolate that produced the first chemotherapy remissions in childhood leukaemia and the first cure of a solid tumour; still essential in ALL, lymphoma, osteosarcoma and CNS lymphoma.

The evidence behind it
  • Infant ALL (<1 year): standard vs AML-type early intensification; transplant for high-risk KMT2A-rearranged infants
    Show survival figures (1)

    Averages across everyone diagnosed, often years ago. Your stage, subtype, age, fitness and the treatment you receive matter more than the average, and the numbers are improving quickly.

    • 6-year EFS 46.1%; intensification no benefit.
    Event-free survival at 6 years (%): All infants 46.1 (n=651) · source
The main trade-offs on record
  • Fatal if given intrathecally: label all syringes.
  • High-dose methotrexate requires normal renal function, hydration, urine alkalinisation and leucovorin rescue with level monitoring.
Questions to ask about this decision
  1. Between Prednisone, Dexamethasone, Vincristine and the other options, which do you recommend for me, and what about my case would make you choose differently?
    Why: Guidelines list several reasonable options; the choice turns on details of your tumour, your health and your priorities.
  2. What is each option trying to achieve: cure, long control, or relief of symptoms, and over what time?
    Why: The aim shapes how much side effect and disruption is worth accepting.
  3. How closely do I match the people in Interfant-06, and does that change what the results mean for me?
    Why: Trial populations are selected; age, fitness, prior treatment and biomarkers all affect how far results carry.
  4. What happens if I delay, or decline this step for now? Is the decision reversible?
    Why: Some decisions can wait for a second opinion or a trial slot; others cannot. Knowing which is part of the choice.
  5. Does your recommendation follow the current guideline (NCI PDQ: Childhood Acute Lymphoblastic Leukemia Treatment (health professional version)), and if it departs from it, why?
    Why: Departures from guidelines are sometimes right for an individual; they should be explained.
  6. For my situation (induction), which of the standard options do you recommend and why?
    Why: Guideline options include: Interfant backbone: a week of prednisone, then dexamethasone, vincristine, cytarabine, daunorubicin and asparaginase with intrathecal therapy.
  7. Am I a candidate for Prednisone, Dexamethasone, Vincristine or related drugs, and what side effects should I expect?
    Why: Knowing the expected toxicities helps you plan work, family, and supportive care.
  8. How do the results of Interfant-06 apply to someone like me?
    Why: Trial populations differ from individual patients; ask how closely you match.

Add these to your appointment list, or take the full question set for this cancer.

Maintenance

Post-induction, KMT2A-rearranged

One 28-day course of blinatumomab after induction (Interfant-21), then Interfant-06 consolidation and maintenance.

The options, in plain words

Blinatumomab was the first T-cell engager (2014), and is now given to children and adults with leukaemia even when in remission, because it improves survival.

Cytarabine is the core chemotherapy for acute myeloid leukaemia, given with an anthracycline as the classic 7+3 induction and at high doses for consolidation; it is also injected into the spinal fluid to treat or prevent leukaemia in the brain.

The 1948 antifolate that produced the first chemotherapy remissions in childhood leukaemia and the first cure of a solid tumour; still essential in ALL, lymphoma, osteosarcoma and CNS lymphoma.

Mercaptopurine is a daily tablet, or a liquid for children, that keeps acute lymphoblastic leukaemia in remission during the long maintenance phase of treatment.

The evidence behind it
  • Infant ALL (<1 year): standard vs AML-type early intensification; transplant for high-risk KMT2A-rearranged infants
    Show survival figures (1)

    Averages across everyone diagnosed, often years ago. Your stage, subtype, age, fitness and the treatment you receive matter more than the average, and the numbers are improving quickly.

    • 6-year EFS 46.1%; intensification no benefit.
    Event-free survival at 6 years (%): All infants 46.1 (n=651) · source
The main trade-offs on record
Side effectAny gradeGrade 3+
Febrile neutropenia · Relapsed/refractory adult ALL31%28%
Infections · Relapsed/refractory adult ALL28%15%
Neurological toxicities · Relapsed/refractory adult ALL65%13%
Pyrexia · Relapsed/refractory adult ALL55%6%

Rates from the label or pivotal trial as recorded on the product page; each grade 3+ figure links to its source.

  • High-dose methotrexate requires normal renal function, hydration, urine alkalinisation and leucovorin rescue with level monitoring.
Questions to ask about this decision
  1. Between Blinatumomab, Cytarabine, Methotrexate and the other options, which do you recommend for me, and what about my case would make you choose differently?
    Why: Guidelines list several reasonable options; the choice turns on details of your tumour, your health and your priorities.
  2. What is each option trying to achieve: cure, long control, or relief of symptoms, and over what time?
    Why: The aim shapes how much side effect and disruption is worth accepting.
  3. How closely do I match the people in Interfant-06, and does that change what the results mean for me?
    Why: Trial populations are selected; age, fitness, prior treatment and biomarkers all affect how far results carry.
  4. Which side effects of Blinatumomab are most likely for me, which are reversible, and which would make us stop?
    Why: The recorded rates below come from labels and trials; your own risk depends on dose, other medicines and your health.
  5. What happens if I delay, or decline this step for now? Is the decision reversible?
    Why: Some decisions can wait for a second opinion or a trial slot; others cannot. Knowing which is part of the choice.
  6. Does your recommendation follow the current guideline (NCI PDQ: Childhood Acute Lymphoblastic Leukemia Treatment (health professional version)), and if it departs from it, why?
    Why: Departures from guidelines are sometimes right for an individual; they should be explained.
  7. For my situation (post-induction, kmt2a-rearranged), which of the standard options do you recommend and why?
    Why: Guideline options include: One 28-day course of blinatumomab after induction (Interfant-21), then Interfant-06 consolidation and maintenance.
  8. Am I a candidate for Blinatumomab, Cytarabine, Methotrexate or related drugs, and what side effects should I expect?
    Why: Knowing the expected toxicities helps you plan work, family, and supportive care.
  9. How do the results of Interfant-06 apply to someone like me?
    Why: Trial populations differ from individual patients; ask how closely you match.

Add these to your appointment list, or take the full question set for this cancer.

2 options

Allogeneic transplant in first remission after blinatumomab and consolidation.

The options, in plain words

Allogeneic stem cell transplantation replaces a patient's blood system with a donor's after conditioning chemotherapy, so donor immune cells hunt down leukaemia left behind. It remains the only cure for adverse-risk acute myeloid leukaemia, high-risk acute lymphoblastic leukaemia and Richter transformation, at the price of graft-versus-host disease.

  • Curative for otherwise incurable leukaemia
  • Graft-versus-leukaemia is an antigen-agnostic immune therapy

Blinatumomab was the first T-cell engager (2014), and is now given to children and adults with leukaemia even when in remission, because it improves survival.

The evidence behind it

No trial record is attached to this row yet. The trials tab lists what is recruiting and the landmark trials for this cancer.

The main trade-offs on record
  • Treatment-related mortality 10-20%
  • Chronic GVHD
  • Relapse remains the main cause of failure
Side effectAny gradeGrade 3+
Febrile neutropenia · Relapsed/refractory adult ALL31%28%
Infections · Relapsed/refractory adult ALL28%15%
Neurological toxicities · Relapsed/refractory adult ALL65%13%
Pyrexia · Relapsed/refractory adult ALL55%6%

Rates from the label or pivotal trial as recorded on the product page; each grade 3+ figure links to its source.

Questions to ask about this decision
  1. Between Allogeneic stem cell transplantation and Blinatumomab, which do you recommend for me, and what about my case would make you choose differently?
    Why: Guidelines list several reasonable options; the choice turns on details of your tumour, your health and your priorities.
  2. What is each option trying to achieve: cure, long control, or relief of symptoms, and over what time?
    Why: The aim shapes how much side effect and disruption is worth accepting.
  3. Which side effects of Blinatumomab are most likely for me, which are reversible, and which would make us stop?
    Why: The recorded rates below come from labels and trials; your own risk depends on dose, other medicines and your health.
  4. What happens if I delay, or decline this step for now? Is the decision reversible?
    Why: Some decisions can wait for a second opinion or a trial slot; others cannot. Knowing which is part of the choice.
  5. Does your recommendation follow the current guideline (NCI PDQ: Childhood Acute Lymphoblastic Leukemia Treatment (health professional version)), and if it departs from it, why?
    Why: Departures from guidelines are sometimes right for an individual; they should be explained.
  6. For my situation (high risk), which of the standard options do you recommend and why?
    Why: Guideline options include: Allogeneic transplant in first remission after blinatumomab and consolidation.
  7. Am I a candidate for Blinatumomab, and what side effects should I expect?
    Why: Knowing the expected toxicities helps you plan work, family, and supportive care.

Add these to your appointment list, or take the full question set for this cancer.

Third line and beyond

Relapsed or refractory

Revumenib (approved from the age of one) or trials of menin inhibitors; blinatumomab or CD19 CAR T-cells; transplant.

The options, in plain words

Revumenib (Revuforj) is the first menin inhibitor (2024), for acute leukaemias with KMT2A rearrangements or NPM1 mutations.

Ziftomenib is the second menin inhibitor for leukaemia, approved in November 2025 as a once-daily pill for relapsed NPM1-mutated AML.

Blinatumomab was the first T-cell engager (2014), and is now given to children and adults with leukaemia even when in remission, because it improves survival.

Tisagenlecleucel was the first CAR-T therapy ever approved (2017), for children and young adults whose leukaemia had come back after everything else.

Allogeneic stem cell transplantation replaces a patient's blood system with a donor's after conditioning chemotherapy, so donor immune cells hunt down leukaemia left behind. It remains the only cure for adverse-risk acute myeloid leukaemia, high-risk acute lymphoblastic leukaemia and Richter transformation, at the price of graft-versus-host disease.

  • Curative for otherwise incurable leukaemia
  • Graft-versus-leukaemia is an antigen-agnostic immune therapy
The evidence behind it
The main trade-offs on record
  • Known QT prolongation. Avoid other QT-prolonging drugs where possible; check ECG and correct potassium and magnesium before and during treatment.
Side effectAny gradeGrade 3+
Febrile neutropenia · Relapsed/refractory adult ALL31%28%
Infections · Relapsed/refractory adult ALL28%15%
Neurological toxicities · Relapsed/refractory adult ALL65%13%
Pyrexia · Relapsed/refractory adult ALL55%6%

Rates from the label or pivotal trial as recorded on the product page; each grade 3+ figure links to its source.

Side effectAny gradeGrade 3+
Cytokine release syndrome · ELIANA, Penn grading77%46%

Rates from the label or pivotal trial as recorded on the product page; each grade 3+ figure links to its source.

  • Treatment-related mortality 10-20%
  • Chronic GVHD
  • Relapse remains the main cause of failure
Questions to ask about this decision
  1. Between Revumenib, Ziftomenib, Blinatumomab and the other options, which do you recommend for me, and what about my case would make you choose differently?
    Why: Guidelines list several reasonable options; the choice turns on details of your tumour, your health and your priorities.
  2. What is each option trying to achieve: cure, long control, or relief of symptoms, and over what time?
    Why: The aim shapes how much side effect and disruption is worth accepting.
  3. How closely do I match the people in AUGMENT-101, and does that change what the results mean for me?
    Why: Trial populations are selected; age, fitness, prior treatment and biomarkers all affect how far results carry.
  4. Which side effects of Blinatumomab or Tisagenlecleucel are most likely for me, which are reversible, and which would make us stop?
    Why: The recorded rates below come from labels and trials; your own risk depends on dose, other medicines and your health.
  5. What happens if I delay, or decline this step for now? Is the decision reversible?
    Why: Some decisions can wait for a second opinion or a trial slot; others cannot. Knowing which is part of the choice.
  6. Does your recommendation follow the current guideline (NCI PDQ: Childhood Acute Lymphoblastic Leukemia Treatment (health professional version)), and if it departs from it, why?
    Why: Departures from guidelines are sometimes right for an individual; they should be explained.
  7. For my situation (relapsed or refractory), which of the standard options do you recommend and why?
    Why: Guideline options include: Revumenib (approved from the age of one) or trials of menin inhibitors; blinatumomab or CD19 CAR T-cells; transplant.
  8. Am I a candidate for Revumenib, Ziftomenib, Blinatumomab or related drugs, and what side effects should I expect?
    Why: Knowing the expected toxicities helps you plan work, family, and supportive care.
  9. How do the results of AUGMENT-101 apply to someone like me?
    Why: Trial populations differ from individual patients; ask how closely you match.

Add these to your appointment list, or take the full question set for this cancer.

How to read this page. Options and results come from OnCo records with their sources; the settings are the standard-of-care rows on the cancer page, and the lines of therapy are on the sequencing grid. Where a setting names one path, the choice is usually about timing, trials and where to be treated: see expert centres. OnCo is orientation, not medical advice.