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Acute promyelocytic 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.

Advanced, first line

Suspected APL, first hours

2 options

Start all-trans retinoic acid on morphological suspicion; transfuse platelets and fibrinogen to targets; avoid invasive procedures until coagulopathy is controlled.

The options, in plain words

The vitamin-A derivative that made acute promyelocytic leukaemia the first cancer cured by differentiation therapy rather than by killing cells.

Red cell and platelet transfusions, iron and erythropoiesis-stimulating agents keep patients safe through chemotherapy and marrow failure; restrictive thresholds and ESA caution reflect trials showing more is not better.

  • Life-saving in acute leukaemia and transplant
  • Restrictive strategies proven safe and cheaper
  • New MDS agents reduce transfusion burden
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
  • Blood supply shortages, especially in LMICs
  • ESA safety restricts use
  • Iron overload and alloimmunisation with chronic transfusion
Questions to ask about this decision
  1. Between Tretinoin (all-trans retinoic acid, ATRA) and Transfusion support and anaemia management, 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. 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.
  4. Does your recommendation follow the current guideline (NCCN Guidelines: Acute Myeloid Leukemia), and if it departs from it, why?
    Why: Departures from guidelines are sometimes right for an individual; they should be explained.
  5. For my situation (suspected apl, first hours), which of the standard options do you recommend and why?
    Why: Guideline options include: Start all-trans retinoic acid on morphological suspicion; transfuse platelets and fibrinogen to targets; avoid invasive procedures until coagulopathy is controlled.
  6. Am I a candidate for Tretinoin (all-trans retinoic acid, ATRA), 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.

Other settings

Low- and intermediate-risk

All-trans retinoic acid plus arsenic trioxide induction and consolidation without chemotherapy (APL0406); dexamethasone prophylaxis or treatment for differentiation syndrome.

The options, in plain words

The vitamin-A derivative that made acute promyelocytic leukaemia the first cancer cured by differentiation therapy rather than by killing cells.

An ancient poison turned cure: with retinoic acid it cures more than 95% of acute promyelocytic leukaemia without conventional chemotherapy.

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.

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

No side-effect rates or interaction flags are recorded for these options yet. The side-effect lookup and interaction checker cover the products that have them.

Questions to ask about this decision
  1. Between Tretinoin (all-trans retinoic acid, ATRA), Arsenic trioxide and Dexamethasone, 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. 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.
  4. Does your recommendation follow the current guideline (NCCN Guidelines: Acute Myeloid Leukemia), and if it departs from it, why?
    Why: Departures from guidelines are sometimes right for an individual; they should be explained.
  5. For my situation (low- and intermediate-risk), which of the standard options do you recommend and why?
    Why: Guideline options include: All-trans retinoic acid plus arsenic trioxide induction and consolidation without chemotherapy (APL0406); dexamethasone prophylaxis or treatment for differentiation syndrome.
  6. Am I a candidate for Tretinoin (all-trans retinoic acid, ATRA), Arsenic trioxide, Dexamethasone, 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.

All-trans retinoic acid plus arsenic trioxide with idarubicin or gemtuzumab ozogamicin added during induction to control the white count.

The options, in plain words

The vitamin-A derivative that made acute promyelocytic leukaemia the first cancer cured by differentiation therapy rather than by killing cells.

An ancient poison turned cure: with retinoic acid it cures more than 95% of acute promyelocytic leukaemia without conventional chemotherapy.

Idarubicin is an anthracycline chemotherapy approved in 1990 for adult acute myeloid leukaemia, given with cytarabine in 3+7 induction and FLAG-Ida salvage and in acute promyelocytic leukaemia protocols. Randomised trials showed more complete remissions than with daunorubicin, which is why centres prefer it; heart damage, marrow suppression and extravasation injury are the class risks.

Gemtuzumab ozogamicin (Mylotarg) is an anti-CD33 antibody linked to the DNA-cutting payload calicheamicin, the first ADC approved, in 2000 for relapsed acute myeloid leukaemia. An unstable linker and no benefit in a confirmatory trial led to withdrawal in 2010; it returned in 2017 at a lower fractionated dose, with liver toxicity, including veno-occlusive disease, its defining risk.

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

No side-effect rates or interaction flags are recorded for these options yet. The side-effect lookup and interaction checker cover the products that have them.

Questions to ask about this decision
  1. Between Tretinoin (all-trans retinoic acid, ATRA), Arsenic trioxide, Idarubicin 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. 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.
  4. Does your recommendation follow the current guideline (NCCN Guidelines: Acute Myeloid Leukemia), and if it departs from it, why?
    Why: Departures from guidelines are sometimes right for an individual; they should be explained.
  5. For my situation (high-risk), which of the standard options do you recommend and why?
    Why: Guideline options include: All-trans retinoic acid plus arsenic trioxide with idarubicin or gemtuzumab ozogamicin added during induction to control the white count.
  6. Am I a candidate for Tretinoin (all-trans retinoic acid, ATRA), Arsenic trioxide, Idarubicin or related drugs, 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.

Second line

Molecular relapse

3 options

Arsenic-based salvage to molecular remission, then autologous transplant if PML::RARA negative, allogeneic transplant if not.

The options, in plain words

An ancient poison turned cure: with retinoic acid it cures more than 95% of acute promyelocytic leukaemia without conventional chemotherapy.

Autologous stem cell transplant collects the patient's own blood-forming stem cells, gives melphalan or another chemotherapy at a dose that would otherwise destroy the marrow, then returns the cells to rebuild it. It is standard consolidation in myeloma and for relapsed lymphoma, though CAR-T has displaced it in early-relapsing large B-cell lymphoma.

  • Permits dose intensity impossible otherwise
  • Decades of outcome data
  • Widely available

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

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 ~1-2% (myeloma) to higher in children
  • Infertility, second malignancies, prolonged cytopenias
  • Being challenged by CAR-T and MRD-guided deferral
  • Treatment-related mortality 10-20%
  • Chronic GVHD
  • Relapse remains the main cause of failure
Questions to ask about this decision
  1. Between Arsenic trioxide, Autologous stem cell transplant (high-dose therapy) and Allogeneic stem cell transplantation, 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. 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.
  4. Does your recommendation follow the current guideline (NCCN Guidelines: Acute Myeloid Leukemia), and if it departs from it, why?
    Why: Departures from guidelines are sometimes right for an individual; they should be explained.
  5. For my situation (molecular relapse), which of the standard options do you recommend and why?
    Why: Guideline options include: Arsenic-based salvage to molecular remission, then autologous transplant if PML::RARA negative, allogeneic transplant if not.
  6. Am I a candidate for Arsenic trioxide, 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.

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.