Leukemia is a cancer of blood-forming cells in the bone marrow. Unlike solid tumors, leukemia affects the blood itself, so it spreads throughout the body. There are several types with very different outlooks—some have excellent survival rates, especially in children, while others are more aggressive. Understanding your specific type is crucial for treatment planning.
What This Is
The bone marrow produces blood cells: red blood cells (for oxygen), white blood cells (for immunity), and platelets (for clotting). Leukemia occurs when bone marrow cells become malignant and multiply uncontrollably, crowding out healthy cells. This leads to anemia (low red blood cells), infections (low white blood cells), and bleeding problems (low platelets). About 60,000 new cases are diagnosed in the US annually.
Main Types of Leukemia
- Acute Lymphoblastic Leukemia (ALL): Develops rapidly in lymphocytes. Most common childhood leukemia; excellent survival rates in children (90%), variable in adults.
- Acute Myeloid Leukemia (AML): Develops rapidly in myeloid cells. Most common adult leukemia; median age at diagnosis ~68 years. 5-year survival ~32%.
- Chronic Lymphocytic Leukemia (CLL): Develops slowly in lymphocytes. Most common adult leukemia; many people live 10+ years with treatment.
- Chronic Myeloid Leukemia (CML): Develops slowly in myeloid cells. Strongly associated with the Philadelphia chromosome; excellent response to targeted therapy (tyrosine kinase inhibitors). 5-year survival ~70%+.
Key Terms to Know
- "Acute" vs "Chronic": Acute = rapid onset and progression; Chronic = slower progression (though still serious).
- Philadelphia chromosome: An abnormal chromosome found in CML; predicts excellent response to targeted therapy.
- Tyrosine kinase inhibitors (TKIs): Targeted drugs like imatinib (Gleevec) that block specific cancer signals; standard for CML.
- Cytochemistry/Cytogenetics: Lab tests that classify leukemia cells and identify mutations.
- Complete remission: Cancer is undetectable, but doesn't mean cured; continued monitoring essential.
Risk Factors
- Age: Some types (AML, CLL) more common in older adults; ALL more common in children.
- Genetic conditions: Down syndrome, Li-Fraumeni syndrome increase risk.
- Radiation exposure: Previous cancer treatment or occupational exposure.
- Chemical exposure: Benzene (solvent in industry).
- Previous chemotherapy: Secondary leukemia risk after other cancer treatment.
- Family history: Rare familial clustering.
Symptoms and Diagnosis
Symptoms (can overlap with many conditions):
- Fatigue and weakness (from anemia)
- Frequent infections (from low white blood cells)
- Easy bruising or bleeding (from low platelets)
- Bone or joint pain
- Enlarged lymph nodes, spleen, or liver
- Night sweats or fevers
These symptoms can be caused by many conditions. Blood tests are needed for diagnosis.
Diagnosis involves:
- Complete blood count (CBC): Counts different blood cell types.
- Peripheral blood smear: Microscopic examination of blood cells.
- Bone marrow biopsy: Sample of marrow examined for cancer cells.
- Cytogenetics and molecular testing: Identifies specific mutations (e.g., Philadelphia chromosome in CML).
Treatment Options
Chemotherapy
Standard treatment for acute leukemias (ALL, AML):
- Intensive induction chemotherapy aims for complete remission (undetectable cancer).
- Consolidation therapy follows remission to prevent relapse.
- Side effects can be significant but manageable with support care.
Targeted Therapy
For specific mutations:
- Tyrosine kinase inhibitors (TKIs) for CML: Imatinib (Gleevec), dasatinib, nilotinib. Often taken as oral daily pills; dramatically improved CML survival.
- FLT3 inhibitors, IDH inhibitors, BCL2 inhibitors: For specific AML subtypes with mutations.
Stem Cell Transplant (Bone Marrow/Hematopoietic Stem Cell Transplant)
Used for high-risk or relapsed leukemias:
- Replaces patient's diseased marrow with healthy donor cells (allogeneic transplant) or reinfuses patient's own previously harvested cells (autologous transplant).
- Intensive procedure with significant risks and benefits; candidacy depends on age, fitness, disease risk.
Immunotherapy
CAR-T cell therapy: Engineered immune cells targeting leukemia; FDA-approved for relapsed/refractory ALL in children and young adults.
Supportive Care
Critical for all leukemia patients:
- Blood transfusions (platelets and red blood cells)
- Antibiotics for infection prevention/treatment
- Growth factors to boost white blood cell production
- Pain and symptom management
Common Side Effects and Management
- Chemotherapy-induced nausea/vomiting: Anti-nausea medications are highly effective.
- Hair loss: Temporary; hair regrows after treatment ends.
- Infections: White blood cell counts drop; prophylactic antibiotics may be used.
- Bleeding/bruising: Platelet transfusions prevent serious bleeds.
- Mouth sores (mucositis): Pain management, special mouthwashes help.
- Fatigue: Common during and after treatment; activity pacing helps.
- Fertility impacts: Chemotherapy can affect fertility; discuss fertility preservation before treatment.
Questions to Ask Your Doctor
- What type and subtype of leukemia do I have?
- What are the risk factors in my specific case (cytogenetics, molecular markers)?
- What is the standard treatment for my type?
- What is my prognosis?
- Do I need a stem cell transplant? If so, what are the risks and benefits?
- What side effects should I expect, and how can we manage them?
- Will treatment affect my fertility?
- How often will I need monitoring after treatment?
- Are there clinical trials available?
When to Seek Urgent Care
- High fever (≥101.5°F / 38.6°C) during chemotherapy
- Severe bleeding or uncontrolled bleeding
- Severe shortness of breath
- Chest pain
- Altered mental status or severe headache
Sources and References
- National Cancer Institute. "Leukemia—Patient Version." PDQ. cancer.gov
- American Cancer Society. "Leukemia." cancer.org
- NCCN Clinical Practice Guidelines: "Acute Myeloid Leukemia," "Chronic Myeloid Leukemia," "Acute Lymphoblastic Leukemia." Version 3.2024.
- Döhner H, et al. "Acute Myeloid Leukemia." Nature Reviews Disease Primers. 2022;8:3.
- Jameson JL, et al. Harrison's Principles of Internal Medicine. 21st ed. McGraw-Hill; 2022.
Last reviewed: February 2026. This page is not medical advice. Always discuss diagnosis, treatment, and prognosis with your medical team.