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Leukemia is a cancer that originates in the blood-forming tissues, primarily the bone marrow and lymphatic system. The cancer causes an overproduction of abnormal white blood cells, which can crowd out healthy blood cells and impair the body’s ability to fight off infection, carry oxygen to organs and tissues, and control bleeding. Leukemia is broadly classified as acute or chronic and by the type of white blood cell affected, such as lymphocytic or myeloid. Early diagnosis and treatment are essential to achieve the best possible outcome and quality of life.

Quick Facts: Leukemia Treatment Options

  • Surgical limitations: Because leukemia does not form solid tumors, it is generally not treated with surgery for cancer removal.
  • Systemic treatment: Chemotherapy is a primary treatment, utilizing powerful drugs to target and destroy rapidly dividing cells.
  • Immunotherapy: This rapidly advancing treatment boosts the body’s own immune system to recognize and attack leukemia cells.  
  • Cellular replacement: Bone marrow transplantation can replace diseased bone marrow with healthy stem cells for patients who have relapsed or not responded to other treatments.

Is Surgery Used to Treat Leukemia?

While surgery is a primary treatment for many solid tumors, leukemia is a cancer of the blood and bone marrow, meaning there is no localized mass to surgically remove. Therefore, it is rarely used as a direct treatment. However, minor surgical procedures often play a vital supportive role in your overall care plan. For instance, during the diagnostic phase, a doctor may perform a bone marrow biopsy to extract a sample for microscopic analysis. Later, your surgical team might place a central venous catheter (such as a port or Hickman line) to make receiving long-term chemotherapy, medications, and blood transfusions much easier and more comfortable. In rare cases where leukemia causes the spleen to become painfully enlarged, a splenectomy may be performed to alleviate discomfort and improve your blood cell counts.

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How Does Chemotherapy Work for Leukemia?

Chemotherapy remains a primary and highly effective treatment for many types of leukemia. Because leukemia cells circulate freely throughout the bloodstream and lymphatic system, localized treatments aren't enough. Chemotherapy acts as a systemic treatment, traveling throughout the entire body to seek out and destroy rapidly dividing cancer cells.

Depending on your specific diagnosis, chemotherapy may be administered orally in pill form, injected directly into a vein, or delivered into the cerebrospinal fluid to target cancer cells that have reached the central nervous system. Treatment is typically administered in carefully timed cycles, a period of intensive treatment followed by a period of rest. This gives your healthy cells time to recover and rebuild. Chemotherapy for leukemia is generally broken down into distinct phases:

  • Induction therapy: The initial, intensive phase designed to clear the blood and bone marrow of leukemia cells and achieve remission.
  • Consolidation (intensification) therapy: Administered after remission is achieved to eliminate any lingering, microscopic cancer cells and prevent a relapse.
  • Maintenance therapy: A long-term, lower-dose phase that can last for months or years to sustain remission and keep the cancer from returning.

When is Radiation Therapy Recommended?

Radiation therapy utilizes high-energy rays or particles that are precisely aimed at specific areas of the body to destroy cancer cells or inhibit their growth. While less frequently used than chemotherapy for leukemia, it can be effective for specific purposes, such as:

  • Total body irradiation (TBI): Often used as part of the conditioning regimen before bone marrow transplantation, TBI can help eliminate leukemia cells throughout the body and suppress the immune system to help prevent transplant rejection.
  • Targeted radiation: High-energy beams can be directed at specific areas where leukemia cells have accumulated, such as the spleen, brain, or spinal cord, to reduce symptoms or prevent cancer spread.
  • Supportive care: Radiation therapy can be used to alleviate symptoms caused by enlarged organs or masses of leukemia cells.

What Role Does Immunotherapy Play in Treating Leukemia?

Immunotherapy works by boosting the power of the body’s own immune system, helping it to recognize and attack cancer cells. Several approaches can be used for leukemia treatment, including:

  • Monoclonal antibodies: Laboratory-made molecules can bind to specific proteins found on the surface of leukemia cells, marking the cells for destruction by the immune system.
  • CAR T-cell therapy: This treatment involves extracting and modifying a patient’s own T-cells to express chimeric antigen receptors (CARs) that specifically target and destroy leukemia cells, which are then infused back into the patient’s bloodstream.  
  • Immune checkpoint inhibitors: Drugs such as pembrolizumab (Keytruda) and nivolumab (Opdivo) can help the immune system recognize and attack leukemia cells by blocking proteins that inhibit the body’s immune response.
  • Cytokines: Substances such as interleukins and interferons can boost the immune system’s activity against leukemia cells.

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How Does Bone Marrow Transplantation Work?

Bone marrow transplantation involves replacing diseased bone marrow with healthy stem cells. This procedure may be considered for a patient who has relapsed or not responded to other treatments. The process involves several steps:

  • Conditioning therapy: High-dose chemotherapy and sometimes radiation therapy are used to destroy the diseased bone marrow and leukemia cells.
  • Stem cell infusion: Healthy stem cells obtained from a donor (allogeneic transplant) or the patient’s own previously collected cells (autologous transplant) are infused into the patient’s bloodstream.
  • Engraftment: The transplanted stem cells circulate in the bloodstream and travel throughout the body to reach the bone marrow, where they begin to produce healthy new blood cells.

How Does Targeted Therapy Differ from Traditional Treatments?

Targeted therapy is a cornerstone of treatment for several types of leukemia, particularly chronic myeloid leukemia (CML) and some forms of acute lymphoblastic leukemia (ALL). Unlike standard chemotherapy, which attacks all rapidly dividing cells in the body, targeted therapies are designed to identify and attack specific genetic mutations or proteins that leukemia cells need to survive and multiply.

Because these drugs specifically zero in on cancer cells while largely sparing healthy tissues, they often cause fewer widespread side effects than traditional chemotherapy. Tyrosine kinase inhibitors (TKIs), for example, are oral targeted medications that have revolutionized the treatment of CML by blocking the specific enzyme that tells the cancer cells to grow, allowing many patients to manage their leukemia like a chronic illness.

What are the Most Likely Side Effects of Leukemia Treatment?

Leukemia treatment can potentially produce side effects, the most common of which include mouth ulcers, nausea, vomiting, diarrhea, constipation, skin rashes, loss of appetite, and fatigue. Other possible side effects include:

  • Low blood cell counts: Treatment can sometimes affect healthy blood cells along with abnormal ones, resulting in an overall decline in blood cell production.
  • Infections: A reduced white blood cell count can increase susceptibility to infections, as these cells help the immune system fight off germs.
  • Graft versus host disease (GVHD): After undergoing an allogeneic stem cell transplantation, a patient may develop GVHD, which occurs when donor immune cells mistakenly target healthy cells.
  • Tumor lysis syndrome (TLS): Sometimes induced by chemotherapy, this metabolic abnormality results from a sudden release of dying cancer cells into the bloodstream; if left untreated, TLS can lead to heart arrhythmias, seizures, loss of muscle control, and kidney failure.
  • Hyperglycemia and steroid-induced diabetes: Certain corticosteroids can produce high blood sugar levels, which can lead to the development of health complications in diabetic patients.

Frequently Asked Questions About Leukemia Treatment

  • How long does leukemia treatment typically last?

    The timeline for leukemia treatment varies widely based on the specific type of leukemia (acute or chronic), the treatment methods used, and how well the cancer responds. Acute leukemia often requires intensive treatment over several months, followed by up to two years of maintenance therapy. Chronic leukemia may involve long-term, ongoing targeted therapies to manage the disease over many years.

  • Is it possible to completely cure leukemia?

    While the term "cure" is used carefully in oncology, many types of leukemia can be successfully treated to achieve long-term remission. In remission, there are no detectable signs of cancer in the body. Some treatments, such as a successful bone marrow transplant, can offer the potential for a permanent cure, particularly for certain types of acute leukemia.

  • Will I need to stay in the hospital during my treatment?

    Hospital stays depend heavily on your specific treatment plan. Intensive induction chemotherapy for acute leukemia often requires an inpatient stay of several weeks so your care team can closely monitor your blood counts and prevent infection. Conversely, many maintenance therapies and treatments for chronic leukemia can be administered on an outpatient basis.

  • How can I prepare for leukemia treatment?

    Preparing for leukemia treatment involves both medical and practical steps to ensure you are as comfortable and supported as possible. Your care team may recommend completing a dental checkup (as chemotherapy can affect oral health) and baseline heart or lung tests. You may also want to discuss fertility preservation options before starting therapy. On a practical level, it is highly recommended to arrange for a trusted caregiver to assist with transportation, stock your home with easy-to-digest meals, and coordinate with your employer regarding time off or flexible work arrangements.

The following FAQs-related articles provide additional information about leukemia treatment:

Benefit from World-Class Care at Moffitt Cancer Center

Moffitt is recognized as a Comprehensive Cancer Center by the National Cancer Institute, a designation that recognizes our commitment to continually advancing cancer care through groundbreaking research and clinical trials. At Moffitt, we understand that each patient and diagnosis is different, and the leukemia team in our Malignant Hematology Program works collaboratively with the team in our Bone and Marrow Transplant and Cellular Immunotherapy Program to determine the optimal treatment approach in every case.

If you would like to learn more about leukemia treatment, you can request an appointment with a specialist in Moffitt’s Malignant Hematology Program by calling 1-888-663-3488 or submitting a new patient registration form online.

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