Leukemias

Acute promyelocytic leukemia (APL)

Also called Acute promyelocytic leukemia with PML::RARA fusion

If you or someone you love has just heard this diagnosis, start here. This guide explains what the condition is, how it is usually treated and whether a transplant plays any part.

Acute promyelocytic leukemia (APL) is a distinct form of AML, usually driven by a PML::RARA gene fusion. It can cause dangerous bleeding, but prompt treatment with differentiation medicines allows many people to recover without a stem cell transplant.

Other names and abbreviations

APL, AML-M3, PML-RARA AML, Acute promyelocytic leukaemia, FAB M3

In short

  • APL is a type of acute myeloid leukemia (AML). It can cause dangerous bleeding, so it needs expert care right away.
  • Many people are treated with two medicines, ATRA and arsenic trioxide. Some people with higher-risk APL also get chemo.
  • A transplant is usually not needed. If APL comes back, some people may have one, using their own cells or sometimes a donor's cells.
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Where transplant fits

is generally unnecessary in first . Selected APL may use after molecular remission or an for persistent or further-relapsed disease; only the allogeneic procedure requires a donor.

Treatment depends on the exact diagnosis, disease stage, prior treatment and the person’s health.

Key facts

Who it affects
APL occurs in children and adults and is often diagnosed at a younger age than AML overall.
How common
About 5 to 8 in every 100 cases of AMLShare of acute myeloid leukemia (AML) cases that are APL, as summarized in the U.S. National Cancer Institute's adult AML treatment summary (PDQ, updated March 2025), which notes APL occurs mostly in adults in midlife Source: How common
Cells used in a transplant
The patient’s own collected cells for autologous rescue; donated blood-forming cells only when an allogeneic procedure is selected.
Where a donor fits
Limited transplant role

The condition

What it is

APL affects immature called promyelocytes. In the form described here, a PML::RARA fusion disrupts their normal development. This genetic finding distinguishes APL from other AML and explains why its main medicines work differently.

APL can develop in children and adults. Blood and testing identifies the abnormal cells, and molecular or chromosome testing confirms the fusion. Suspected APL requires immediate specialist care because bleeding complications can develop before all test results are available.

Where acute promyelocytic leukemia (APL) starts in the bloodAPL starts in promyelocytes, immature myeloid cells on their way to becoming granulocytes; a PML::RARA gene fusion disrupts their normal development.Simplified illustration.

Marked as affected: granulocytes.

  • Blood stem cell, In the bone marrow
    • Myeloid line
      • Red blood cells
      • Platelets
      • Granulocytes, Affected
      • Monocytes
    • Lymphoid line
      • B cells
        • Plasma cells, Develop from B cells
      • T cells
      • NK cells, Natural killer cells

What causes it

PML::RARA usually forms when parts of chromosomes 15 and 17 rearrange in a blood-forming cell. This is generally an acquired change, not something passed from a parent to a child.

Most people have no identifiable cause. APL can occasionally follow previous cancer treatment. It cannot be caught from another person. Other rare rearrangements can resemble APL, but their response to treatment may differ and requires precise diagnosis.

Symptoms and effects

APL can reduce normal red cells and , causing tiredness, breathlessness, bruising or bleeding. It can also disrupt the clotting system, so serious bleeding and abnormal clotting may occur together.

During treatment, differentiation syndrome is another possible emergency: maturing leukemia cells can trigger inflammation, fluid retention and breathing difficulties. The care team monitors for this and provides prompt treatment when needed.

Diagnosis and treatment

How APL is diagnosed

A blood count and a look at the blood under a microscope (a blood smear) can raise the first suspicion of APL. Clotting tests, such as fibrinogen and prothrombin time, show whether the blood's clotting system is disturbed.

Care does not wait for the final test result. European expert guidelines say a person with suspected APL is admitted to the hospital right away and treated as a medical emergency. A medicine called ATRA and to support clotting start at once. Clotting tests are then repeated at least daily until the clotting problem has cleared.

Specialist labs confirm APL by finding the PML::RARA gene change, ideally in a bone marrow sample. Fast tests, such as FISH or a stain for the PML protein, can give an early answer. A PCR test is run alongside them. It shows the exact form of the gene change, and it is later used to track whether any leukemia is left.

A standard chromosome test can miss the gene change. European guidelines say it is the test most likely to give a false-negative result, which is why faster, more sensitive tests are used as well.

Blood counts in acute promyelocytic leukemia (APL)APL can reduce normal red cells and platelets, causing tiredness, breathlessness, bruising or bleeding.Simplified illustration.

How blood counts can look in acute promyelocytic leukemia (APL): red blood cells low and platelets low.

  • Red blood cells: Low
  • Platelets: Low

Each lab has its own usual range, and it changes with age. The drawing shows only the direction a count often moves, not how far, and one person’s results can look different.

How it is treated

All-trans retinoic acid (ATRA) helps abnormal promyelocytes mature. Arsenic trioxide is also a central treatment. Many patients receive ATRA with arsenic; higher-risk disease may require additional treatment to control the leukemia-cell count, including chemotherapy in some protocols.

Treatment includes intensive support for bleeding and clotting abnormalities. After the initial response, consolidation and molecular testing help establish whether the PML::RARA signal has cleared and whether it returns.

Transplant is generally unnecessary in first remission. In relapse, treatment depends on what was given before and the molecular response. An autologous transplant, using the person’s own collected cells, may be considered after molecular remission. Allogeneic transplantation may be considered when disease persists or for selected further relapses.

How acute promyelocytic leukemia (APL) can be treatedA transplant is usually not needed for APL, but if it comes back some people may have one.Simplified illustration.

Kinds of treatment described for acute promyelocytic leukemia (APL): supportive care, medicines, a donor stem cell transplant (for a few people) and a transplant with the person’s own cells (for some people).

After diagnosis, the options described here

  • Supportive care

    Transfusions to support clotting start at once, as part of intensive support for bleeding and clotting problems.

  • Medicines

    ATRA helps the abnormal cells mature and arsenic trioxide is also a central treatment, with chemotherapy for some higher-risk APL.

  • Donor stem cell transplant, For a few people

    May be considered when APL persists or for selected further relapses.

  • Transplant with the person’s own cells, For some people

    If APL comes back, a transplant with the person’s own collected cells may be considered once tests no longer find the leukemia signal.

These are the kinds of treatment this page describes, not a plan. Which ones fit, in what order and whether they are combined differs from person to person.

When transplant specialists are usually consulted

A transplant is not part of first treatment for APL. If APL comes back, European expert guidelines say the main aim is a deep (molecular) remission as a bridge to transplant. They name a transplant of the person's own cells as the first choice when tests no longer find the PML::RARA signal. A donor transplant is recommended when that signal does not clear. The same guidelines note a UK report that questions whether every person in second remission needs a transplant.

Read the guidance

Daily life and the donor’s role

Living with the condition and treatment

The early days may involve hospitalization, frequent blood tests and transfusions even while medicines are beginning to work. Later treatment can include repeated clinic visits and molecular monitoring. A good early response does not make follow-up unnecessary.

ATRA and arsenic have side effects that need monitoring, including differentiation syndrome; arsenic can also affect heart rhythm and electrolytes. The treatment team explains which symptoms need an urgent call and how follow-up fits around home, school or work.

The role of a blood stem cell donor

A registry donor is not part of the usual first-line APL treatment. An autologous transplant also does not use a registry donor.

For a person who needs allogeneic transplantation after relapse, a relative or unrelated donor may be considered, with alternative options assessed by the transplant team. Most people with APL are treated without a donor transplant.

Looking ahead

Looking ahead

Outlook for APL

Researchers now call APL the most curable type of acute myeloid leukemia. In a large Italian-German trial, 99.2% of adults with lower-risk APL treated with ATRA and arsenic trioxide were alive about four years later. St. Jude, a children's cancer hospital, reports that more than 9 in 10 patients treated for APL are considered cured.

The hardest time is the start. Bleeding in the brain or lungs is the most common cause of death, both before and soon after treatment begins. Studies that count everyone diagnosed, not just people in , find more early deaths than trials do. Two things raise this early risk: older age and a white blood cell count above 10,000 per microliter at diagnosis. That high count is also what makes APL high-risk.

After the first weeks, care teams use PCR tests to check that the PML::RARA signal clears and stays gone. In lower-risk APL treated with ATRA and arsenic trioxide, relapse is uncommon. When APL does come back, arsenic trioxide brings many people into a second remission.

About these numbers. Each one says which group of people it comes from, and the place and years where the source gives them. It describes what happened across that group, not what will happen to any one person. And a figure measured among people who had a transplant is not the same as the number of people who need one.

Trial figures describe people who were well enough to enroll and start treatment. They cannot predict how any one person will do.

Common questions

Is acute promyelocytic leukemia curable?

Yes, most of the time. Researchers now call APL the most curable type of acute myeloid leukemia (AML). The main danger is bleeding in the first days and weeks, which is why APL is treated as an emergency. The outlook section on this page gives the figures, with the groups they describe. Group results cannot predict one person's outcome.

Why is APL treated as a medical emergency?

APL can disturb the blood's clotting system, so serious bleeding, and sometimes clotting, can happen suddenly. Bleeding in the brain or lungs is the most common cause of death before and soon after treatment starts. For this reason, European expert guidelines say a person with suspected APL should be admitted to the hospital right away. A medicine called ATRA and transfusions to support clotting start at once, before genetic tests confirm the diagnosis. Studies that count everyone diagnosed find more early deaths than clinical trials do.

How is APL different from other types of AML?

APL is a form of acute myeloid leukemia (AML) in which young white blood cells called promyelocytes build up. It is caused by a gene change called PML::RARA. This change sets APL apart from other AML and explains why its main medicines, ATRA and arsenic trioxide, work so well. Many people with lower-risk APL are treated with these two medicines without chemotherapy. APL makes up about 5 to 8 in every 100 cases of AML, and it commonly disturbs blood clotting.

Does APL need a bone marrow transplant?

Usually not. A transplant is not part of first treatment for APL, because ATRA and arsenic trioxide work so well. In a large trial, fewer than 2 in 100 adults with lower-risk APL treated this way had their leukemia come back within about four years. If APL does return, it is treated again. European guidelines say a transplant using the person's own cells is usually the first choice if tests then no longer find the PML::RARA signal. A donor transplant is recommended when the signal does not clear.

Can children get acute promyelocytic leukemia?

Yes. APL makes up about 7 in every 100 cases of AML in children. St. Jude says it is rare before age 3 and is most often diagnosed in children of Hispanic or Mediterranean descent. Children are treated with ATRA and arsenic trioxide, and those with high-risk APL may also get chemotherapy. In a Children's Oncology Group trial, 98% of children with standard-risk APL and 96% with high-risk APL were alive with no relapse or treatment failure two years after diagnosis.

How a transplant using your own cells works

For your next appointment

Acute promyelocytic leukemia (APL)

From the Jada Bascom Foundation disease library, jadabascomfoundation.org. Printed .

Questions to bring to your care team

  • Is this APL low risk or high risk, based on the white blood cell count at diagnosis, and how does that change the treatment plan?
  • Which clotting tests are you checking each day, and what signs of bleeding or clotting should we report right away?
  • Will treatment use ATRA and arsenic trioxide alone, or is chemotherapy also needed, and how long will treatment last?
  • How and when will you test for the PML::RARA signal after treatment, and what would happen if it came back?
  • What is the goal of each treatment you are suggesting?
  • What would make a transplant worth considering later on?
  • Are there clinical trials that might fit?
  • Where can our family find support during treatment?

A one-page list to take to the next appointment, with room for notes.

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Sources and further reading

  1. Acute Myeloid Leukemia Treatment (PDQ), Health Professional Version
    NCI, Accessed 2026-09-05
  2. AML in Adults
    EBMT Handbook / NCBI Bookshelf, Accessed 2026-09-05
  3. WHO fifth-edition classification: Myeloid and Histiocytic/Dendritic Neoplasms
    WHO classification authors / Leukemia, Accessed 2026-09-05
  4. Indications for haematopoietic cell transplantation and CAR-T: 2025 EBMT practice recommendations
    EBMT / Bone Marrow Transplantation, Accessed 2026-09-05
  5. Stem Cell and Bone Marrow Transplants for Cancer
    NCI, Accessed 2026-09-05
  6. Childhood Acute Myeloid Leukemia Treatment (PDQ), Health Professional Version
    National Cancer Institute, Updated 2025-04-15; accessed 2026-09-26
  7. Sanz MA et al. Management of acute promyelocytic leukemia: updated recommendations from an expert panel of the European LeukemiaNet
    Blood (American Society of Hematology) / European LeukemiaNet, 2019; accessed 2026-09-26
  8. Platzbecker U et al. Improved outcomes with retinoic acid and arsenic trioxide compared with retinoic acid and chemotherapy in non-high-risk APL: final results of the randomized Italian-German APL0406 trial
    Journal of Clinical Oncology (via Europe PMC), 2017; accessed 2026-09-26
  9. Kutny MA et al. Assessment of arsenic trioxide and all-trans retinoic acid for the treatment of pediatric APL: a report from the Children's Oncology Group AAML1331 trial
    JAMA Oncology (via PubMed Central), 2022; accessed 2026-09-26
  10. Zhu HH et al. Early death and survival of patients with acute promyelocytic leukemia in ATRA plus arsenic era: a population-based study
    Frontiers in Oncology (via PubMed Central), 2021; accessed 2026-09-26
  11. Park JH et al. Early death rate in acute promyelocytic leukemia remains high despite all-trans retinoic acid
    Blood (via PubMed Central), 2011; accessed 2026-09-26
  12. Acute Promyelocytic Leukemia (APL)
    St. Jude Children's Research Hospital, Accessed 2026-09-26

This information explains a condition and its treatments. It cannot diagnose an illness or recommend treatment for an individual. Your care team can explain how the evidence applies to you. Written and source-checked by the Jada Bascom Foundation. Each page lists the published sources it draws on.

Ways to help

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