Acute promyelocytic leukaemia
Acute promyelocytic leukaemia is caused by a single fused gene that freezes blood cells at an immature stage and triggers dangerous bleeding. Two non-chemotherapy drugs, a vitamin A derivative and arsenic trioxide, make the cells mature and cure more than nine in ten patients.
Overview
The t(15;17) translocation fuses PML to the retinoic acid receptor alpha gene. The PML::RARA protein blocks differentiation at the promyelocyte stage, and the promyelocytes release procoagulants that cause a bleeding and clotting disorder responsible for most early deaths. Diagnosis is a clinical emergency: all-trans retinoic acid (ATRA) is started on suspicion, before the fusion is confirmed, together with aggressive platelet and fibrinogen replacement. Risk is set by the white count at presentation, above 10 x 10^9/L being high risk.
ATRA, introduced in Shanghai in 1988, was the first differentiation therapy for any cancer; with anthracycline chemotherapy it lifted cure rates above 70 percent. Arsenic trioxide, developed in Harbin and Shanghai in the 1990s and approved in the United States for relapsed disease in 2000, degrades the fusion protein itself. The Italian-German APL0406 trial, reported in 2013, randomised low- and intermediate-risk patients to ATRA plus arsenic or ATRA plus chemotherapy: two-year event-free survival 97 percent versus 86 percent, with fewer infections and no secondary leukaemias, and the chemotherapy-free regimen became the standard, approved for first-line use in Europe in 2016 and the United States in 2018. High-risk patients receive ATRA and arsenic with an anthracycline or gemtuzumab ozogamicin added for the first weeks.
Differentiation syndrome, fever, fluid retention and lung infiltrates as the cells mature, affects around a quarter of patients and is treated with dexamethasone; QT prolongation and liver toxicity are watched on arsenic. Molecular monitoring for PML::RARA after consolidation catches the rare relapse, which is treated with arsenic-based regimens and autologous transplant. The remaining deaths are early haemorrhagic deaths before treatment starts, so the research target is recognition and access rather than a new drug.
State of the art
- A chemotherapy-free regimen of all-trans retinoic acid and arsenic trioxide cures more than nine in ten patients with standard-risk disease.
- Oral arsenic formulations are replacing intravenous arsenic in China and in trials elsewhere.
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.
- Early death from bleeding, before or during the first days of treatment, is now the main cause of failure.
Red cards
From the labels and guidelines behind the standard of care. Your team's thresholds win.- Emergency services nowDifferentiation syndrome
Fever, cough or breathlessness, rapid weight gain or swelling, bone pain, low blood pressure or reduced urine; the labels say to start steroids and monitor at the first suspicion, and the syndrome has been fatal.
- Emergency services nowBleeding or bruising
Bleeding that will not stop, black or bloody stools, or unexplained bruising when platelets are expected to be low.
- Emergency services nowSkin reaction
Blisters, peeling, or sores in the mouth or eyes with a rash. Enfortumab vedotin carries a boxed warning for Stevens-Johnson syndrome and toxic epidermal necrolysis, mostly in the first cycle.
- Good to knowDifferentiation syndrome
When a targeted drug makes leukaemia cells mature all at once, causing fever, fluid in the lungs, and weight gain. Treatable with steroids if caught early.
- Good to knowQT prolongation
A change on the heart's electrical trace, caused by some drugs blocking a potassium channel (hERG), that in rare cases sets off a dangerous rhythm. Drugs that do this need ECG checks and care with other medicines and low potassium or magnesium.
See all on the product pages:Arsenic trioxideGemtuzumab ozogamicinIdarubicinTretinoin (all-trans retinoic acid, ATRA)·Printable cards in the navigator
Anatomy and lymph node drainage
- Bone marrow (leukaemia, MDS, MPN, myeloma)
- Lymph node germinal centre (lymphomas)
- Spleen
- Blood (leukaemic phase)
- Lytic bone lesions (myeloma)
- Skin and extranodal sites
- Nodes: cervical
- Nodes: axillary
- Nodes: mediastinal
- Nodes: para-aortic and mesenteric
- Nodes: inguinal
Leukaemias, myeloma and MDS live in the marrow and blood; lymphomas grow in lymph nodes and spleen. The node stations are the disease map, not a route of spread, and staging counts them.
- Bone marrow (leukaemia, MDS, MPN, myeloma)High-risk APL (white count above 10 x 10^9/L) · Variant APL with RARA fusions other than PML (ZBTB16, NPM1, STAT5B; some resist arsenic or ATRA)
- Lymph node germinal centre (lymphomas)
- SpleenVariant APL with RARA fusions other than PML (ZBTB16, NPM1, STAT5B; some resist arsenic or ATRA)
- Blood (leukaemic phase)
- Lytic bone lesions (myeloma)
- Skin and extranodal sites
- cervical
- axillary
- mediastinal
- para-aortic and mesenteric
- inguinal
In lymphoma the node stations are the disease itself; staging (Ann Arbor / Lugano) counts how many regions and sides of the diaphragm are involved.
Same organ: Marginal zone lymphoma, Cutaneous T-cell lymphoma (mycosis fungoides and Sezary syndrome), Primary mediastinal (thymic) large B-cell lymphoma, Leukaemia (all types), Acute myeloid leukaemia, Acute lymphoblastic leukaemia, Chronic lymphocytic leukaemia, Chronic myeloid leukaemia (CML), Diffuse large B-cell lymphoma, Follicular lymphoma, Hodgkin lymphoma, Mantle cell lymphoma, Multiple myeloma, Non-Hodgkin lymphoma (all types), Myelodysplastic syndromes / neoplasms (MDS), Myeloproliferative neoplasms (PV, ET, myelofibrosis), Polycythaemia vera (PV), Essential thrombocythaemia (ET), Waldenström macroglobulinaemia, Hairy cell leukaemia, Peripheral T-cell lymphomas (including cutaneous T-cell lymphoma), Blastic plasmacytoid dendritic cell neoplasm (BPDCN), Burkitt lymphoma, HIV-associated (AIDS-related) lymphomas, Chronic myelomonocytic leukaemia and MDS/MPN overlap neoplasms, Systemic mastocytosis, Erdheim-Chester disease, Rosai-Dorfman disease and other histiocytic neoplasms, Langerhans cell histiocytosis (LCH), Post-transplant lymphoproliferative disorder (PTLD), FLT3-mutated acute myeloid leukaemia, IDH1- and IDH2-mutated acute myeloid leukaemia, NPM1-mutated and KMT2A-rearranged acute myeloid leukaemia, Secondary and therapy-related acute myeloid leukaemia, Acute myeloid leukaemia in older or unfit patients, Smouldering multiple myeloma, Newly diagnosed multiple myeloma, transplant-eligible, Newly diagnosed multiple myeloma, transplant-ineligible, Relapsed or refractory multiple myeloma, Plasma cell leukaemia, Lower-risk myelodysplastic syndromes, Higher-risk myelodysplastic syndromes, Chronic lymphocytic leukaemia, first treatment, Relapsed or refractory chronic lymphocytic leukaemia, Richter transformation of chronic lymphocytic leukaemia, Chronic myeloid leukaemia, chronic phase, Chronic myeloid leukaemia, accelerated and blast phase, Primary myelofibrosis
About one in ten cases of acute myeloid leukaemia; once the most rapidly fatal, it is now the most curable, with more than nine in ten patients cured when early bleeding is prevented.
Cases by country
No country-level case numbers. This cancer is not mapped to a GLOBOCAN site.
One section per setting: the options named, what each is for, the trials behind them, the recorded trade-offs and the questions to ask.
Start all-trans retinoic acid on morphological suspicion; transfuse platelets and fibrinogen to targets; avoid invasive procedures until coagulopathy is controlled.
All-trans retinoic acid plus arsenic trioxide induction and consolidation without chemotherapy (APL0406); dexamethasone prophylaxis or treatment for differentiation syndrome.
All-trans retinoic acid plus arsenic trioxide with idarubicin or gemtuzumab ozogamicin added during induction to control the white count.
Arsenic-based salvage to molecular remission, then autologous transplant if PML::RARA negative, allogeneic transplant if not.
Subtypes & biomarkers
top- Low- and intermediate-risk APL (white count 10 x 10^9/L or below)
- High-risk APL (white count above 10 x 10^9/L)
- Variant APL with RARA fusions other than PML (ZBTB16, NPM1, STAT5B; some resist arsenic or ATRA)
- Therapy-related APL
- PML ::RARA fusion by PCR or FISH
- t (15;17) karyotype
- White cell count at presentation (Sanz risk)
- Fibrinogen, D-dimer and platelet count
- PML ::RARA transcript MRD after consolidation
- FLT3-ITD (common in the microgranular variant)
How often this target appears
- 1957Hillestad describes acute promyelocytic leukaemia
- 1977t(15;17) translocation identified
- 1988All-trans retinoic acid produces remissions in Shanghai: the first differentiation therapy
- 2000Arsenic trioxide approved for relapsed APL
- 2013APL0406: retinoic acid plus arsenic beats retinoic acid plus chemotherapy
- 2018Arsenic trioxide approved for first-line standard-risk APL in the United States
Dated changes read from the records linked to this cancer: approvals, regulatory steps, reported trials, guideline versions and milestones. Newest first; no date is inferred.
All 11 changes by month →- 2026-09-17This recordAcute promyelocytic leukaemiaFacts on this page last checked
When this page itself was last checked or edited.
- 2018ApprovalArsenic trioxideArsenic trioxide approved in US
Newly diagnosed low-risk APL with tretinoin
- 2018MilestoneArsenic trioxideArsenic trioxide approved for first-line standard-risk APL in the United States
A milestone in how this cancer is treated.
- 2013MilestoneTretinoin (all-trans retinoic acid, ATRA)APL0406: retinoic acid plus arsenic beats retinoic acid plus chemotherapy
A milestone in how this cancer is treated.
- 2002ApprovalArsenic trioxideArsenic trioxide approved in EU
APL
- 2000ApprovalArsenic trioxideArsenic trioxide approved in US
Relapsed/refractory APL
What is in development for Acute promyelocytic leukaemia, drawn from the whole corpus: 0 items. Drugs are grouped by the most advanced trial phase they have reached anywhere; approved treatments sit under standard of care. Technologies are the methods being tested for this cancer, trials are the studies recorded here, and ideas are proposals not yet in a trial.
Nothing recorded in development for this cancer yet.
Open problems and what is being done
Early haemorrhagic death before treatment starts, especially where diagnosis is slow.
Treatment of variant RARA fusions that do not respond to arsenic or retinoic acid.
Whether high-risk patients can also be spared chemotherapy.
Trials
topTrials recruiting now
Country and place are remembered in this browser only. A postcode is sent to OpenStreetMap's Nominatim service to find coordinates when you press the button; nothing else leaves your device.
Expert centres
topExpert centres
Indianapolis, IN · cancer center | United States | 0 | 159 | 4,238 | - | ||
Hamilton, ON · cancer center | Canada | none recorded | 0 | 103 | 1,158 | - | |
Baltimore, MD · cancer center | United States | 0 | 101 | 2,306 | - | ||
Omaha, NE · cancer center | United States | 0 | 37 | 4,159 | - | ||
Minneapolis, MN · cancer center | United States | 0 | not matched | - | - | ||
Shanghai · hospital | China | none recorded | 0 | not matched | - | - |
These are the things we can measure; they are not a ranking of quality. Each column is a field on the institution record or a count over what OnCo has linked; a centre that treats many patients with Acute promyelocytic leukaemia but is thinly recorded here will look small.
Not known: OnCo holds no case-volume or outcome figures for centres, so none are shown. Where a national audit or registry publishes them, the centre's page links to it. Default order: Newsweek rank, then trials for this cancer, then research output.
Questions to ask
topQuestions to ask your oncologist about Acute promyelocytic leukaemia
Newly diagnosed
- What is my exact diagnosis, stage, and grade, and which tests established them?Why: Everything else follows from an accurate stage and subtype.
- Which biomarkers have been tested on my tumour (for example PML::RARA fusion by PCR or FISH, tkaryotype, White cell count at presentation, Fibrinogen, D-dimer and platelet count, PML::RARA transcript MRD after consolidation), and what were the results?Why: These results decide eligibility for targeted therapy, immunotherapy, and trials.
- Which subtype is my cancer, and does that change the recommended treatment?Why: Recognised subtypes for this cancer include Low- and intermediate-risk APL, High-risk APL, Variant APL with RARA fusions other than PML.
- Is germline (inherited) genetic testing recommended for me or my family?Why: Inherited variants can change treatment and matter for relatives.
Suspected APL, first hours
- 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.
- 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.
Low- and intermediate-risk
- 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.
- 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.
High-risk
- 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.
- 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.
Molecular relapse
- 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.
- 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.
Any stage
- Are there clinical trials I could join, for example of Arsenic trioxide, Tretinoin (all-trans retinoic acid, ATRA)?Why: Trials are how the next standard of care is set; asking early keeps options open.
- Would a second opinion at a high-volume centre change anything, and can you help arrange it?Why: Rare or high-stakes decisions benefit from a centre that treats many similar patients.
- What supportive care (symptom control, nutrition, exercise, mental health, financial help) is available from the start?Why: Supportive care improves quality of life and helps patients complete treatment.
- I read that “Early haemorrhagic death before treatment starts, especially where diagnosis is slow”. How does that affect my plan?Why: Open problems are where trials and second opinions matter most.
- I read that “Treatment of variant RARA fusions that do not respond to arsenic or retinoic acid”. How does that affect my plan?Why: Open problems are where trials and second opinions matter most.
Newly diagnosed? Read the first 60 days with Acute promyelocytic leukaemia, then print the one-page appointment sheet with room for the answers.
Print this page for your appointment (your browser's print command). These prompts are for discussion; your clinical team knows your case.
Direct links plus the targets, companies, and technologies of this cancer's products.
technologies
9targets
2drugs
5companies
1terms
3Latest papers
topQuery for this cancer: (TITLE:"Acute promyelocytic leukaemia" OR ABSTRACT:"Acute promyelocytic leukaemia" OR TITLE:"APL" OR ABSTRACT:"APL" OR TITLE:"AML-M3" OR ABSTRACT:"AML-M3" OR TITLE:"PML::RARA acute leukaemia" OR ABSTRACT:"PML::RARA acute leukaemia" OR TITLE:"Promyelocytic leukaemia" OR ABSTRACT:"Promyelocytic leukaemia") AND (treatment OR therapy OR trial OR survival OR diagnosis). Results are unfiltered search hits about Acute promyelocytic leukaemia, not a curated reading list.
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