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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.
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.
Averages across everyone diagnosed, often years ago. A median is the middle of a group: half the people counted lived longer than the figure shown, and some lived far longer. Your stage, subtype, age, fitness and the treatment you receive matter more than the average, and the numbers are improving quickly.
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.
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.
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: High-grade B-cell lymphoma with MYC and BCL2 rearrangements (double-hit lymphoma), Mediastinal grey zone lymphoma, Primary effusion lymphoma, Plasmablastic lymphoma, T-cell/histiocyte-rich large B-cell lymphoma, EBV-positive diffuse large B-cell lymphoma, Primary large B-cell lymphoma of the testis, Gastric MALT lymphoma, Ocular adnexal MALT lymphoma, Extranodal NK/T-cell lymphoma, Adult T-cell leukaemia/lymphoma, ALK-positive anaplastic large cell lymphoma, ALK-negative anaplastic large cell lymphoma, Breast implant-associated anaplastic large cell lymphoma, Primary cutaneous anaplastic large cell lymphoma, Lymphomatoid papulosis, Mycosis fungoides, Enteropathy-associated T-cell lymphoma, Monomorphic epitheliotropic intestinal T-cell lymphoma, Extranodal marginal zone lymphoma of mucosa-associated lymphoid tissue (MALT lymphoma), Splenic marginal zone lymphoma, Nodal marginal zone lymphoma, Primary cutaneous marginal zone lymphoma, Primary cutaneous follicle centre lymphoma, Sezary syndrome, Nodal T-follicular helper cell lymphoma, angioimmunoblastic type (angioimmunoblastic T-cell lymphoma), Hepatosplenic T-cell lymphoma, Intravascular large B-cell lymphoma, Lymphomatoid granulomatosis, T-cell prolymphocytic leukaemia, Splenic B-cell lymphoma/leukaemia with prominent nucleoli (formerly B-cell prolymphocytic leukaemia and hairy cell leukaemia variant), T-cell large granular lymphocytic leukaemia, Mixed-phenotype acute leukaemia, Myeloid leukaemia of Down syndrome, Burkitt leukaemia, 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, Standard-risk B-cell acute lymphoblastic leukaemia in children, High-risk acute lymphoblastic leukaemia in children (high-risk B-ALL and T-ALL), Philadelphia chromosome-positive acute lymphoblastic leukaemia in children (Ph-positive ALL), Philadelphia chromosome-like acute lymphoblastic leukaemia (Ph-like or BCR::ABL1-like ALL), Infant acute lymphoblastic leukaemia (KMT2A-rearranged, under one year), Relapsed and refractory acute lymphoblastic leukaemia in children, Acute myeloid leukaemia in children, Erdheim-Chester disease, Rosai-Dorfman-Destombes disease, Single-system Langerhans cell histiocytosis (bone, skin or one other organ), Multisystem Langerhans cell histiocytosis (with or without risk-organ involvement), Indolent and smouldering systemic mastocytosis, Advanced systemic mastocytosis (aggressive SM, SM with an associated haematological neoplasm, mast cell leukaemia), Early-stage classical Hodgkin lymphoma (stage I to II), Advanced-stage classical Hodgkin lymphoma (stage III to IV), Nodular lymphocyte-predominant Hodgkin lymphoma (nodular lymphocyte-predominant B-cell lymphoma), Relapsed and refractory classical Hodgkin lymphoma
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.
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Starting retinoic acid on morphological suspicion, aggressive blood product support and arsenic-based induction for non-high-risk disease all follow from this document.
Low- and intermediate-risk APL is now treated without cytotoxic chemotherapy. The regimen has become the standard worldwide and made APL the most curable adult acute leukaemia.
Differentiation therapy, making leukaemic cells mature rather than killing them, became the first targeted treatment in leukaemia. Every modern APL regimen starts with retinoic acid on suspicion of the diagnosis.
Query 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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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.
Bleeding that does not stop by itself, bleeding from more than one site, or new bruising in several places or one large area.
Temperature of 38 C or higher, or feeling shivery and unwell even without a fever. Antibody-drug conjugates suppress the bone marrow, and several carry a boxed warning for severe neutropenia.
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.
When engineered T cells or a bispecific antibody switch on, the immune system can overshoot. The first sign is a fever, and the treatment is a drug that blocks the main signal, given early. A smaller number of people become confused or drowsy, which is graded and treated separately.
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 trioxideDexamethasoneGemtuzumab ozogamicinIdarubicinTretinoin (all-trans retinoic acid, ATRA)·Printable cards in the navigator
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