Unclassified acute myeloid leukemia

Last reviewed

🖨 Print for my doctorAdvocacy Hub →
ORPHA:167714OMIM:601626
Who is this for?
Show terms as
2FDA treatments8Treatment centers1Financial resources

Where are you in your journey?

UniteRare data is sourced from FDA.gov, ClinicalTrials.gov, Orphanet, OMIM, and NORD.
Report missing data

Overview

Unclassified acute myeloid leukemia (AML) refers to cases of acute myeloid leukemia that do not fit into any of the specifically defined categories under the World Health Organization (WHO) classification system. AML is a cancer of the myeloid line of blood cells, characterized by the rapid, uncontrolled growth of abnormal white blood cells (blasts) that accumulate in the bone marrow and interfere with the production of normal blood cells. This subtype designation is used when the leukemia lacks the specific genetic abnormalities, morphologic features, or clinical histories that would allow classification into recognized AML subtypes such as AML with recurrent genetic abnormalities, therapy-related AML, or AML with myelodysplasia-related changes. Patients with unclassified AML typically present with symptoms related to bone marrow failure, including fatigue and weakness due to anemia, increased susceptibility to infections from neutropenia (low white blood cell counts), and easy bruising or bleeding caused by thrombocytopenia (low platelet counts). Other symptoms may include fever, weight loss, bone pain, and enlargement of the spleen or liver. The disease primarily affects the hematopoietic (blood-forming) system but can potentially involve other organs if leukemic cells spread. Treatment for unclassified AML generally follows standard AML treatment protocols, which typically include intensive induction chemotherapy aimed at achieving complete remission, followed by consolidation therapy. Depending on the patient's risk profile, age, and overall health, treatment options may include additional chemotherapy cycles, allogeneic hematopoietic stem cell transplantation, or targeted therapies where applicable molecular targets are identified. Prognosis varies considerably depending on individual patient factors and response to initial therapy. Because these cases lack the defining features of better-characterized AML subtypes, risk stratification and treatment decisions may be more challenging and are often guided by additional molecular and cytogenetic testing.

Also known as:

Inheritance

Sporadic

Usually appears on its own, not inherited from a parent

Age of Onset

Adult

Begins in adulthood (age 18 or older)

Orphanet ↗OMIM ↗NORD ↗

Treatments

2 available

Mylotarg

Gemtuzumab ozogamicin· Wyeth Pharmaceuticals, Inc., a Pfizer Company■ Boxed Warning

treatment of relapsed or refractory CD33-positive AML in adults and pediatric patients 2 years and older

Tibsovo

IVOSIDENIB· Servier Pharmaceutical LLC■ Boxed Warning

In combination with azacitidine or as monotherapy for the treatment of newly diagnosed AML in adults 75 years or older, or who have comorbidities that preclude use of intensive induction chemotherapy

No actively recruiting trials found for Unclassified acute myeloid leukemia at this time.

New trials open frequently. Follow this disease to get notified.

Search ClinicalTrials.gov ↗Join the Unclassified acute myeloid leukemia community →

No specialists are currently listed for Unclassified acute myeloid leukemia.

View NORD Rare Disease Centers ↗Undiagnosed Disease Network ↗

Treatment Centers

8 centers
🏥 NORD

Baylor College of Medicine Rare Disease Center

Baylor College of Medicine

📍 Houston, TX

🏥 NORD

Stanford Medicine Rare Disease Center

Stanford Medicine

📍 Stanford, CA

🔬 UDN

NIH Clinical Center Undiagnosed Diseases Program

National Institutes of Health

📍 Bethesda, MD

🔬 UDN

UCLA UDN Clinical Site

UCLA Health

📍 Los Angeles, CA

🔬 UDN

Baylor College of Medicine UDN Clinical Site

Baylor College of Medicine

📍 Houston, TX

🔬 UDN

Harvard/MGH UDN Clinical Site

Massachusetts General Hospital

📍 Boston, MA

🏥 NORD

Mayo Clinic Center for Individualized Medicine

Mayo Clinic

📍 Rochester, MN

👤 Mayo Clinic Center for Individualized Medicine

🏥 NORD

UCLA Rare Disease Day Program

UCLA Health

📍 Los Angeles, CA

Financial Resources

1 resources
Mylotarg(Gemtuzumab ozogamicin)Wyeth Pharmaceuticals, Inc., a Pfizer Company

Travel Grants

No travel grants are currently matched to Unclassified acute myeloid leukemia.

Search all travel grants →NORD Financial Assistance ↗

Community

Open Unclassified acute myeloid leukemiaForum →

No community posts yet. Be the first to share your experience with Unclassified acute myeloid leukemia.

Start the conversation →

Latest news about Unclassified acute myeloid leukemia

No recent news articles for Unclassified acute myeloid leukemia.

Follow this condition to be notified when news becomes available.

Caregiver Resources

NORD Caregiver Resources

Support, advocacy, and financial assistance for caregivers of rare disease patients.

Mental Health Support

Rare disease caregiving can be isolating. Connect with counseling and peer support.

Family & Caregiver Grants

Financial assistance programs specifically for caregivers of rare disease patients.

Social Security Disability

Learn how rare disease patients may qualify for SSDI/SSI benefits.

Common questions about Unclassified acute myeloid leukemia

What is Unclassified acute myeloid leukemia?

Unclassified acute myeloid leukemia (AML) refers to cases of acute myeloid leukemia that do not fit into any of the specifically defined categories under the World Health Organization (WHO) classification system. AML is a cancer of the myeloid line of blood cells, characterized by the rapid, uncontrolled growth of abnormal white blood cells (blasts) that accumulate in the bone marrow and interfere with the production of normal blood cells. This subtype designation is used when the leukemia lacks the specific genetic abnormalities, morphologic features, or clinical histories that would allow cl

How is Unclassified acute myeloid leukemia inherited?

Unclassified acute myeloid leukemia follows a sporadic inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.

At what age does Unclassified acute myeloid leukemia typically begin?

Typical onset of Unclassified acute myeloid leukemia is adult. Age of onset can vary across affected individuals.

What treatment and support options exist for Unclassified acute myeloid leukemia?

1 patient support program are currently tracked on UniteRare for Unclassified acute myeloid leukemia. See the treatments and support programs sections for copay assistance, eligibility, and contact details.