Acute myeloid leukemia: Causes, treatment, and outlook

Hearing the words acute myeloid leukemia (AML) can feel like the world just tilted on its axis.
AML is a serious blood cancer, and there’s nothing fun about that. But understanding what’s going onwhy it happens,
how it’s treated, and what the future might look likecan make the journey feel a little less terrifying and a lot more manageable.

In this guide, we’ll walk through AML in clear, everyday language. We’ll cover how it develops, major causes and risk factors,
modern treatment options, and what doctors mean when they talk about prognosis and outlook.
You’ll also find practical tips, questions to ask your care team, and some real-world experiences at the end to help you feel less alone.

Important note: This article is for education only and cannot replace advice from your own healthcare team.

What is acute myeloid leukemia?

Acute myeloid leukemia is a cancer of the blood and bone marrowthe spongy tissue inside your bones where new blood cells are made.
In AML, immature cells called myeloid blasts grow out of control and crowd out healthy blood-forming cells.
The word “acute” means the disease tends to progress quickly if it isn’t treated.

Normally, bone marrow makes:

• Red blood cells to carry oxygen
• White blood cells to fight infections
• Platelets to help blood clot

In AML, abnormal blasts don’t mature properly and don’t do any useful work. Instead, they pile up in the marrow and spill into the blood.
As healthy cells are pushed out, people can develop anemia, frequent infections, and easy bruising or bleeding.

In the United States, AML is relatively uncommon compared with breast or lung cancer, but it’s the most common acute leukemia in adults.
Recent estimates suggest a little over 22,000 people each year are diagnosed with AML in the U.S., and most of them are adults over age 60.

How AML develops in the body

AML starts with changes (mutations) in the DNA of a single stem cell or early myeloid cell in the bone marrow.
These mutations can affect genes that control cell growth, division, and death. Once enough key genes are altered,
the cell stops following the rules and begins to:

• Divide too quickly
• Avoid normal “self-destruct” signals
• Block other cells from maturing properly

Over time, the abnormal blast cells swell in number. They don’t leave much room for healthy cells to grow,
which is why people with AML often feel exhausted, get sick more easily, and bruise or bleed with minimal trauma.

These abnormal cells can also circulate in the bloodstream and sometimes collect in places like the gums, skin, lymph nodes, or spleen.

Causes and risk factors for acute myeloid leukemia

One of the most frustrating truths about AML is that many people have no obvious cause.
You can do “everything right” and still develop it. That said, researchers have identified several important risk factors.

Age and sex

AML can occur at any age, including in children, but it’s much more common in older adults.
Many people are diagnosed in their late 60s or 70s. The disease is slightly more common in men than women.

Previous chemotherapy or radiation

Some people develop what’s called therapy-related AML years after receiving chemotherapy or radiation for another cancer.
Certain drugs, especially alkylating agents and topoisomerase II inhibitors, are associated with a higher risk of later developing AML.

Smoking

We usually think of smoking and lung cancer, but tobacco smoke doesn’t stay politely in the lungs.
Cancer-causing chemicals enter the bloodstream and travel throughout the body.
Smoking is the only clearly proven lifestyle-related risk factor for AML.

Chemical exposures

Long-term exposure to benzene, a chemical used in certain industries and found in some solvents and fuels,
is linked with a higher risk of AML. Workplace safety regulations help reduce this risk, but it hasn’t disappeared completely.

Pre-existing blood disorders

AML can sometimes evolve from other bone marrow diseases, such as:

• Myelodysplastic syndromes (MDS)
• Myeloproliferative neoplasms (like polycythemia vera or primary myelofibrosis)

In these conditions, the bone marrow is already “not quite right,” and additional mutations can push cells over the line into AML.

Genetic predisposition

Some inherited syndromes, such as Down syndrome and certain familial bone marrow failure syndromes,
increase the risk of AML, especially in children. There are also families in which multiple relatives have had AML or related blood cancers,
suggesting a hereditary component.

And then there’s the big catch: many people with AML have no known risk factors at all.
Having a risk factor doesn’t mean you’ll definitely get AML, and lacking one doesn’t guarantee protection.

Symptoms and early warning signs

AML symptoms often show up over days to weeks, not years. Because the disease hits the bone marrowyour blood cell factory
most signs come from low counts of normal cells. Common symptoms include:​

• Fatigue and weakness (from low red blood cells/anemia)
• Shortness of breath, especially with activity
• Pale skin
• Frequent infections or fevers that don’t go away (from low white blood cells)
• Easy bruising or bleeding, nosebleeds, or bleeding gums (from low platelets)
• Tiny red or purple spots on the skin (petechiae)
• Bone or joint pain
• Swollen gums, enlarged spleen, or swollen lymph nodes in some cases
• Unintentional weight loss and night sweats

All of these symptoms can be caused by many things that aren’t cancerfrom viral infections to iron deficiency.
But if they’re persistent, getting worse, or just feel “off,” it’s worth seeing a doctor and asking whether a complete blood count (CBC) is appropriate.

How acute myeloid leukemia is diagnosed

Diagnosis starts with listening to the story. A clinician will ask about your symptoms, medical history, medications, past cancer treatments,
and exposures (like smoking or chemicals). Then they’ll move on to testing. Common steps include:​

Blood tests

A complete blood count (CBC) measures numbers of red cells, white cells, and platelets. In AML, the CBC might show:

• Low red blood cells (anemia)
• Low platelets (thrombocytopenia)
• Very high or low white blood cell counts
• Immature blasts circulating in the blood smear

Bone marrow biopsy

To confirm AML, doctors perform a bone marrow aspiration and biopsy, usually from the hip bone.
Under the microscope, they look for:

• At least 20% blasts in the marrow (a typical diagnostic cutoff for AML)
• How the cells look and stain
• Specific genetic and molecular changes

These tests help classify the leukemia into subtypes and risk groups. For example, some chromosomal and gene changes
are considered “favorable,” some “intermediate,” and some “adverse,” and these categories guide treatment decisions and expected outcomes.

Additional tests

Depending on the situation, doctors may also order:

• Flow cytometry to identify what markers are present on the leukemia cells (helps confirm the exact type of leukemia)
• Cytogenetic and molecular testing (for FLT3, NPM1, IDH1/2, and other mutations that matter for prognosis and targeted therapy)
• Imaging tests if they’re concerned the leukemia is affecting organs outside the marrow
• Lumbar puncture in some cases to check if leukemia cells have reached the fluid around the brain and spinal cord

Treatment options for acute myeloid leukemia

Because AML can progress quickly, treatment often starts soon after diagnosis. There isn’t a one-size-fits-all plan.
Treatment is tailored to your age, overall health, AML subtype, and specific genetic changes in the leukemia cells.

Induction therapy: The first big push

The first phase of treatment is called induction therapy. The goal is to wipe out as many leukemia cells as possible and bring the disease into
remissionmeaning no signs of leukemia in the blood, fewer than 5% blasts in the marrow, and recovery of normal blood counts.

For many adults, standard induction involves combinations of chemotherapy drugs (for example, a “7 + 3” regimen:
seven days of one drug plus three days of another). Roughly two-thirds of people with AML who receive standard induction chemo go into remission,
especially those who are younger and otherwise healthy.

Induction therapy is intense, often requiring a hospital stay of several weeks. During this time, people are closely monitored and supported
through side effects such as infections, mouth sores, nausea, and hair loss.

Consolidation therapy and stem cell transplant

Even when remission is achieved, tiny numbers of leukemia cells can remain. That’s why remission is followed by
consolidation therapy, which may include:

• Additional cycles of high-dose chemotherapy
• Allogeneic stem cell transplant (also called bone marrow transplant) for some patients

In an allogeneic transplant, you receive stem cells from a donor whose tissue type closely matches yours.
Before the transplant, high-dose chemotherapy (sometimes with radiation) clears out your own marrow.
The donor cells then move in and rebuild a new blood-forming system, which can also mount an immune attack against remaining leukemia cells.

Transplant offers a chance at a long-term cure for some people, but it also carries significant risks, including infections
and graft-versus-host disease. Whether it’s recommended depends on your risk category, age, and overall health.

Targeted therapies and newer options

AML treatment has changed dramatically in the last decade with the introduction of targeted therapies that home in on specific mutations.
Examples include:​

• FLT3 inhibitors (such as midostaurin or gilteritinib) for AML with FLT3 mutations
• IDH1 and IDH2 inhibitors (like ivosidenib and enasidenib) for AML with IDH1 or IDH2 mutations
• BCL-2 inhibitor venetoclax combined with lower-intensity chemotherapy or hypomethylating agents in older adults
  or those who can’t tolerate intensive chemo
• Antibody-drug conjugates (such as gemtuzumab ozogamicin) targeting markers like CD33 on leukemia cells
• Specialized approaches for acute promyelocytic leukemia (APL), a unique subtype of AML that is often treated with
  all-trans retinoic acid (ATRA) and arsenic trioxide rather than standard chemo

For some people in remission, maintenance therapysuch as oral azacitidinemay be used to help keep the leukemia from coming back.

Supportive care (the unsung hero)

Alongside leukemia-specific therapy, supportive care plays a huge role in quality of life and overall outcomes. This can include:

• Red blood cell and platelet transfusions
• Antibiotics, antifungals, or antivirals to prevent or treat infections
• Medications for nausea, pain, or mouth sores
• Growth factors to help blood counts recover in some situations
• Emotional, nutritional, and financial support services

Clinical trials are also an important option at many stages of AML treatment, offering access to cutting-edge therapies
and helping shape the future standard of care.

AML in children and young adults

Children and adolescents with AML are usually treated with intensive combination chemotherapy and, in some cases, stem cell transplant.
Fortunately, childhood AML outcomes have improved substantially, with 5-year survival now around 65–70% in many settings,
though it varies by subtype and other risk factors.

Outlook and survival: What to expect

Prognosis in AML is not one numberit’s a blend of many factors, including:

• Age at diagnosis
• Overall health and other medical conditions
• AML subtype and genetic/molecular changes
• Response to initial treatment (how quickly blasts clear)
• Whether stem cell transplant is possible or appropriate

For adults overall, the 5-year survival rate for AML is roughly in the 25–35% range, depending on the data source and population.
For example, some large datasets report a 5-year survival of about 30–32% for AML in general.
That number hides a lot of variation: younger adults with favorable genetic features can do much better, while older adults with high-risk disease
may have more limited options.

Meanwhile, ongoing research continues to improve outcomes. New targeted drugs, refined transplant techniques, maintenance strategies,
and better infection prevention are all gradually moving the needle in the right direction.

If you or someone you love has AML, it’s completely reasonable to ask your care team:

• “What specific subtype and risk group do I have?”
• “Which genetic mutations are present in my leukemia?”
• “What are my treatment options, and what are the goals of each?”
• “Is a stem cell transplant recommended in my case?”
• “Are there clinical trials I should know about?”

Living with AML: Day-to-day realities

AML treatment is more of a marathon with sprints built in than a quick 100-meter dash.
Hospital stays, clinic visits, transfusions, and scans can dominate life for months. A few strategies can help:

• Build your support squad. Friends and family can help with rides, meals, childcare, and emotional backup.
  Don’t be shy about askingyou’d do it for them.
• Track your health. Keep a notebook or app with lab results, medications, side effects, and questions for your care team.
• Protect yourself from infection. Follow your team’s advice on handwashing, crowds, masks, food safety, and vaccinations.
• Move when you can. Gentle walking or stretching, when approved by your care team, can help with fatigue and mood.
• Care for your mental health. Psychologists, social workers, support groups, and online communities can make a big difference.

It’s okay to have days when you feel determined and days when you just want to binge-watch something and not think about white blood cells at all.
Both are normal parts of adjusting to life with AML.

Real-world experiences with acute myeloid leukemia

Every AML story is unique, but certain themes show up again and again. The following are composite experiences based on many patient and caregiver reports,
not descriptions of any one individual.

“I thought I just needed more sleep” – A younger adult’s story

Alex was 38, a parent of two, juggling work, school events, and a half-hearted attempt at marathon training.
When the exhaustion hit, it felt like regular “too many tabs open in life” tirednessuntil walking up one flight of stairs left them breathless.

The bruises came next. At first, Alex blamed a clumsy bump into the coffee table. Then there were bruises with no remembered impact at all,
plus a nosebleed that took forever to stop. A routine visit turned into blood work “just to be safe,” then a call from the doctor asking Alex to
come back today, not “next week.”

The diagnosisacute myeloid leukemiawas a shock. Alex remembers hearing only fragments: “high white count,” “blast cells,” “we need to admit you.”
The hospital stay for induction therapy felt like stepping into an alternate universe where time was measured in lab draws and IV bags.
Hair fell out, food tasted weird, and there were days when getting out of bed felt like summiting a mountain.

But there were also bright spots: a nurse who explained every medication in plain language, a friend who coordinated meal deliveries,
and another patient on the floor who cracked jokes about hospital gowns and chemo “fashion.” When Alex heard the words “you’re in remission,”
it didn’t mean everything was suddenly fine, but it did mean hope had more room at the table.

“We had to balance treatment with real life” – An older adult’s experience

Maria was 72 and already managing heart disease and diabetes when AML entered the picture. Intensive chemo and transplant were technically possible,
but came with serious risks. The oncology team sat with her and her daughter, walking through each path in detail.

Instead of the most aggressive route, Maria chose a combination of lower-intensity chemotherapy and a targeted drug that fit better with her goals:
more time with reasonable quality of life, fewer long hospital stays, and the ability to remain at home as much as possible.

The treatment still wasn’t easy. There were transfusions, clinic visits, and days when fatigue made simple tasks feel like marathons.
But there were also afternoons in the garden, video calls with grandkids, and a running joke with her doctor about who had the better hat collection
(Maria did, by a mile).

Her story highlights an important truth: “best treatment” isn’t only what looks good on a survival curve. It’s also about what matters most to the person
actually living the story.

“We never treat a lab result in isolation” – A clinician’s perspective

Hematologists who treat AML often say they practice “science plus storytelling.” The science is in the lab values, bone marrow biopsy reports,
and genetic test results. The storytelling is in understanding who the patient is, what their life looks like, and what they hope for.

One clinician might see two patients with very similar AML biology on paper, but totally different realities in person.
One is a 30-year-old hoping to return to a physically demanding job and to someday run around with young kids.
The other is an 80-year-old who values staying at home, reading on the porch, and minimizing hospital time.
The “right” plan is different in each case.

From the clinician’s side, progress in AML feels both exciting and humbling. Targeted therapies and better transplant strategies are giving some patients
options that didn’t exist a decade ago. At the same time, AML remains a tough disease, especially for older adults.
Many providers describe the job as holding onto realism with one hand and hope with the other.

Finding your own path forward

Living with acute myeloid leukemia is not something anyone signs up for, but you do have power in how you navigate it.
Asking questions, understanding your options, leaning on your support system, and giving yourself permission to have hard days are all part of the process.

As treatments continue to improve, so does the outlook for many people with AML. The statistics can provide context,
but they don’t tell your entire storyand you are absolutely allowed to be more than a number on a chart.