Over the last two decades, medical science has revolutionized the treatment of blood cancers leukemia, lymphoma, and myeloma. Still, a leukemia diagnosis can be unsettling; the prognosis is not always good and the treatment is difficult. However, leukemia is a condition many people get through. In fact, depending on the type of leukemia, most people respond very well to leukemia treatment. The first step is to understand the diagnosis and educate yourself on the available treatment options.
Leukemia is a type of cancer that primarily affects the white blood cells. Formed from hematopoietic cells, or “blood-making cells”, white blood cells are mainly produced in the bone marrow. Changes to the genetic material in these cells can cause them to grow out of control. Unlike many other cancers, however, leukemia does not normally produce tumors. Instead, the cancer cells spill into the bloodstream and circulate throughout the body.
Primitive stem cells in the bone marrow produce all the body’s blood cells. The process by which primitive all-purpose cells, like stem cells, turn into specialized cells, like blood cells, is called differentiation.
Bone marrow stem cells first differentiate into myeloid stem cells or lymphoid stem cells. Myeloid stem cells produce most of the body’s blood cells, including red blood cells (oxygen-carrying cells) and platelets (blood-clotting cells). Myeloblasts are also produced from myeloid stem cells and give rise to infection-fighting cells called granulocytes. Lymphoid stem cells give rise to lymphoblasts, which produce lymphatic white blood cells, such asT cells and B cells. These immune system cells spend most of their time outside the bloodstream fighting off invaders. If either myeloblasts or lymphoblasts become cancerous, they go into overdrive and start dumping undifferentiated blood cells (blast cells) or immature blood cells into the bloodstream.
The two major types of leukemia, then, are classified according to the type of blood-producing cell that has become cancerous. Cancers of the myeloblasts are myeloid (or myelogenous) leukemias. Cancers of the lymphoblasts are lymphocytic leukemias.
Additionally, leukemia can be slow-growing (chronic) or fast-growing (acute). Acute forms of leukemia produce an excess of blasts, cells too immature to carry out their proper function in the body. Chronic forms of leukemia overproduce cells that are only partly mature, so at least some of their functions can be carried out.
With these distinctions in mind, the different types of leukemia include:
Doctors measure the severity of a cancer diagnosis by assigning a stage to the condition. For tumor-producing cancers, the stage is defined by how extensively tumors have spread in the body. Since leukemia does not produce tumors, leukemia stages are determined by the type of cancer cells that are found in the bloodstream and bone marrow.
As leukemia progresses, the cancerous cells in the bone marrow crowd out the healthy bone marrow cells. The bone marrow produces fewer and fewer healthy blood cells until it fails altogether. As a result, most patients with leukemia will not have enough fully-functioning immune cells, which increases the risk of severe infections, the most common cause of death in leukemia patients. Other serious complications that can arise from different types of leukemia include:
While leukemia risk factors include a family history of leukemia, carcinogen exposure, radiation exposure (including X-rays), a previous history of leukemia-like cancers called myelodysplastic syndrome, or rare genetic conditions such as Down syndrome or Bloom syndrome, the majority of leukemia patients do not have any known risk factor.
Leukemia will strike around 60,000 people in the United States this year. 20,000 will perish from the condition. Leukemia can be cured, particularly in children. When treated, most patients will be cured or go into remission for several years, but this will depend on the type of leukemia and its stage.
Symptoms and a medical history will point a healthcare provider towards a leukemia diagnosis, but the final diagnosis is made from blood tests. Chronic (slow-growing) leukemia is often first caught through routine blood tests, but symptoms are usually the first sign of acute (fast-growing) leukemia.
Although symptoms vary depending on the type of leukemia, the most common symptoms of leukemia are:
Chronic leukemia, however, frequently has no or only mild symptoms.
A definitive diagnosis will be made by a hematologist, a specialist in blood diseases and disorders, or an oncologist, a specialist in cancer. Blood tests are used to count the number of blood cells, immature blood cells, and blasts in the bloodstream. These blood counts will help determine the type of leukemia and whether or not the cancer is chronic (slow-growing) or acute (fast-growing).
In some cases, a sample of the bone marrow will be removed from the hip bone using a needle, a procedure called aspiration. A pathologist will study the sample to identify the type and amount of cancerous cells in the bone marrow. Once treatment begins, blood tests and bone marrow biopsies will be regular parts of the treatment to test how effectively medications and other therapies are working.
Treatment will depend on the type of leukemia, whether it’s chronic or acute, and the stage the cancer has advanced to. For some patients with chronic leukemia, the only treatment will be observation and monitoring. For faster-growing leukemia, treatment will largely focus on controlling cancer with medications. A bone marrow transplant may be necessary to treat advanced stages of the condition.
The first-line therapy for leukemia depends on the type of leukemia. For some leukemias, first-line therapy is chemotherapy, drugs designed to kill cancer cells. For others, the first-line medication will be targeted therapy drugs that home in on a unique genetic characteristic of the cancer cells. Other medications include antibodies to amplify the immune system’s response to cancer cells, differentiation agents to help cancerous cells in the bloodstream mature, and growth factors that provide an alternative to blood transfusions by stimulating red blood cell production in the bone marrow.
Surgery is rarely used for leukemia. Unlike other cancers, leukemia does not form tumors. Instead, the cancer cells are distributed throughout the body in the bloodstream. Leukemia usually causes the spleen and liver to enlarge, so, on rare occasions, an enlarged spleen may need to be surgically removed, a procedure called a splenectomy.
Radiation is not normally used for leukemia except in cases where a tumor has formed or the cancer has spread to the central nervous system. Radiation can also be used to kill bone marrow cells in preparation for a stem cell transplant.
When leukemia medications fail to rein in the cancer, a stem cell transplantation is used to replace cancerous bone marrow stem cells with healthy stem cells. The existing bone marrow is first destroyed with radiation or chemotherapy. Healthy stem cells are then taken from a donor (called an allogeneic transplant) or from the patient’s blood or bone marrow (called an autologous transplant). The preferred source will depend on the type of leukemia being treated. For autologous transplants, the bone marrow will often be prepared in the lab to remove any cancerous cells. An autologous stem cell transplant can also be made from umbilical cord stem cells if these were banked at birth. These healthy bone marrow cells are then put into the patient’s bones.
The immune system fights off cancerous leukemia cells both in the bloodstream and the bone marrow. CAR T-cell therapy involves removing T-cells from the bloodstream. The T-cells are then genetically altered so that they specifically go after leukemia cells. These altered T-cells are grown in the lab and returned to the bloodstream to fight off leukemia cells throughout the body.
The spread of leukemia to the central nervous system can be a significant life-threatening complication of leukemia. Acute forms of leukemia may involve therapy to prevent cancer cells from spreading to the brain or spine using intrathecal chemotherapy injections, that is, injections made directly into the spinal fluid in the lower back or directly into the brain using a surgically-implanted Ommaya reservoir. The reservoir sits beneath the skin on the scalp and delivers drugs through a catheter passing through the skull. Chemo drugs are inserted directly into the reservoir using a needle. Intrathecal therapy is very commonly used in children with acute lymphocytic leukemia but is less commonly used with adults.
Medications for bladder cancer are prescribed based on the cancer’s stage and risk for spreading.
Chemotherapy is the primary treatment for nearly all cases of acute lymphocytic leukemia and the main treatment for acute myeloid leukemia and chronic lymphocytic leukemia. Chemotherapy drugs circulate throughout the body, so they are effective at targeting cancers like leukemia which proliferate in the bloodstream and lymphatic system.
Leukemia chemotherapy follows three phases:
When chemotherapy is used for chronic leukemia, it will be given in 12 four-week cycles. Chemotherapy drugs will be taken for one or two weeks followed by a rest period.
Chemotherapy drugs for leukemia come in several types:
Targeted therapy is used as the first-line treatment in chronic myeloid leukemia. For acute leukemia, targeted drugs are used when chemotherapy hasn’t succeeded. Targeted drugs work specifically against defective proteins in the cancer cell that cause cancer cells to grow out of control. They are often identified by the protein they affect, such as tyrosine kinase inhibitors or FLT3 inhibitors.
Antibodies are proteins produced by the body that attach to foreign cells such as bacteria or tumor cells. One part glues to the foreign cell and another part glues to an attacking immune system cell. Monoclonal antibodies are synthetic cells that specifically attach to leukemia cancer cells and either provoke immune system cells to attack the cell, such as Blincyto (blinatumomab), or act as a delivery system for a chemotherapy drug, such as Besponsa (inotuzumab ozogamicin).
For acute promyelocytic leukemia, differentiation agents are used to help immature white blood cells mature into fully functioning white blood cells. Unlike chemotherapy drugs, differentiation agents such as all-trans-retinoic acid (ATRA) or arsenic trioxide (ATO) do not kill the cancer cells. For this reason, these drugs can help lower the risk of complications such as excessive blood clotting or severe bleeding.
Leukemia will require other medications to treat complications of the disease or treatment. Supportive care treatments include antibiotics, antiviral medications, and vaccines to help prevent infection. Transfusions, growth factors, or corticosteroids may be used to help restore low red blood cell or platelet counts.
The most appropriate medications are determined by the type of leukemia being treated, the stage the cancer has progressed to, how the cancer has responded to other therapy, and the patient’s tolerance of side effects. There is no “best” medication for leukemia, just the most appropriate drug or combination of drugs for the medical situation.
Best medications for leukemia | ||||
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Drug Name | Drug Class | Dosage form | Standard Dosage | Common Side Effects |
Dexamethasone | Corticosteroid | Oral tablet, intravenous (IV) injection or intramuscular (IM) injection | 0.05 to 0.9 mg IV or IM initially once daily. Dosage may be individualized based on the condition and drug response of the patient. | Fluid retention, difficulty sleeping, mood, behavior changes |
Adriamycin (doxorubicin) | Anthracycline antibiotic | Intravenous (IV) infusion | 60 to 75 mg/m2 IV every 21 days. Then, in combination with other chemotherapy drugs, 40 to 75 mg/m2 every 21 to 28 days | Hair loss, nausea, vomiting |
Methotrexate | Antimetabolite | Oral tablet, intramuscular (IM) injection, or intrathecal injection | Dosage depends on the condition being treated, how the drug is administered, and other drugs it is administered with | Nausea, diarrhea, hair loss, photosensitivity |
Purinethol (6-mercaptopurine) | Purine analog | Oral tablet | 1.5 mg to 2.5 mg/kg once daily. Dosage varies depending on the condition being treated. | Bone marrow suppression, abdominal pain, nausea, malaise |
Vincristine | Alkaloid | Intravenous (IV) injection or intravenous (IV) infusion | 1.4 mg/m2 IV once weekly in combination with other chemotherapy drugs. Dosage may vary based on weight or body surface area. | Hair loss, low white blood cell counts, nerve pain |
Gleevec (imatinib) | Tyrosine kinase inhibitor | Oral tablet | 600 mg once daily. Dosage may vary depending on the type of cancer being treated. | Water retention, nausea, muscle cramps |
Sprycel (dasatinib) | Tyrosine kinase inhibitor | Oral tablet | 140 mg once daily. Dosage depends on type of leukemia or weight (in children) | Low white blood cell counts, anemia, diarrhea |
Rituxan (rituximab) | Monoclonal antibody | Intravenous (IV) infusion | Dosage depends on body surface area, chemotherapy cycle, and other drugs being administered in combination | Infusion reactions, low white blood cell counts, fever |
Mylotarg (gemtuzumab ozogamicin) | Antibody drug conjugate | Intravenous (IV) infusion | Dosage depends on body surface area, chemotherapy stage, and individual response to therapy | Hemorrhage, infection, fever |
Venclexta (venetoclax) | BCL-2 inhibitor | Oral tablet | 100 mg on day 1, 200 mg on day 2, 400 mg on day 3, and 400 mg once daily on day 4 and beyond. Dosage may vary depending on the type of cancer being treated. | Low white blood cell counts, anemia, diarrhea |
Many of the standard dosages above are from the U.S. Food and Drug Administration (FDA). Dosage is determined by your doctor based on your medical condition, response to treatment, age, and weight. Other possible side effects exist. This is not a complete list.
Side effects of leukemia medications will vary depending on the medication, the dosage prescribed, and any coexisting medical conditions. This is not a complete list.
All leukemia drugs reduce the ability of bone marrow to produce white blood cells. As a result, infections are a common side effect and could be serious enough to require hospitalization. Many leukemia drugs also suppress red blood cell and platelet production, so anemia, bleeding, and bruising are common.
One of the most serious side effects of leukemia treatments is tumor lysis syndrome. Chemotherapy and other treatments often kill off many cancer cells which then break up and dump their cellular contents into the bloodstream. This causes a rapid build-up of calcium, phosphate, and uric acid that overwhelms the body. Tumor lysis syndrome is a life-threatening medical emergency.
Most people will experience side effects of chemotherapy drugs including nausea, vomiting, flu-like symptoms, skin rash, and hair loss. Chemotherapy can have severe side effects such as liver damage, kidney damage, nerve damage, and heart damage.
The most commonly experienced side effects from monoclonal antibodies are infusion reactions, fever, headache, nausea, and an increased risk of infections. Severe side effects include potentially lethal allergic reactions, heart arrhythmias, and liver disease.
Side effects of targeted therapy are often mild, including diarrhea, muscle pain, and fatigue.
There are no effective home, diet, or natural remedies for acute or chronic leukemia. As with all cancers, self-care is an important part of minimizing the effects of cancer treatment.
You will be given a detailed set of instructions after chemotherapy, drug infusions, or any other type of leukemia treatment or surgery. Follow these instructions to the letter, particularly those related to avoiding infections, taking medications, and taking proper care of a catheter, PICC line, or other device that may have been implanted for administering chemotherapy.
Leukemia treatments significantly lower the body’s ability to fight off infections. The primary goal of home care is to avoid infections. Wash your hands regularly and practice good hygiene.
Chemotherapy may cause gastrointestinal problems, loss of appetite, and involuntary weight loss. Eat small meals spaced out several times a day rather than large meals. Bland foods, soups, and liquid foods are easier to keep down after treatments. Eat foods rich in nutrients and avoid junk food.
If cancer treatments cause diarrhea, which many do, drink fluids to avoid dehydration. Make sure to consume electrolytes in broths, electrolyte drinks, or foods like bananas. Consult your doctor if you experience persistent or severe diarrhea.
The drugs used to treat leukemia depend on the type of leukemia being treated and whether it is fast-growing (acute leukemia) or slow-growing (chronic leukemia). Drugs used to treat leukemia include chemotherapy drugs, targeted therapy drugs, immunotherapy drugs, differentiation agents, and growth factors.
No food or dietary supplement can cure leukemia. However, a balanced diet can keep the body strong so it can fight leukemia and recover more easily after treatments. Make sure to eat a consistent diet of fruits, vegetables, whole grains, and protein. Avoid raw or undercooked foods to prevent the risk of food poisoning.
Depending on the type of leukemia a person has, treatment can often be successful at curing cancer or inducing remission. The five-year survival rate for some types of leukemia can be as high as 89%. Relapse rates, unfortunately, are high.
Leukemia results in the overproduction of immature blood cells, particularly white blood cells. The excess of blood cells puts a great strain on the liver, which clears out excess blood cells, and the spleen, which collects and stores white blood cells. Therefore, swollen liver (hepatomegaly) and swollen spleen (splenomegaly) are two symptoms used to diagnose leukemia.
Also, the excess of white blood cells in the bloodstream can slow down or block blood circulation, a condition called leukostasis. When white blood cells clog up smaller blood vessels, the body’s organs—the heart, liver, kidneys, and brain—can be starved of needed oxygen. Organ failure due to leukostasis is one of the reasons people can die from leukemia.
Because leukemia attacks the body’s ability to fight off infections, the major cause of death among leukemia patients involves infections. Other life-threatening complications of leukemia include organ failure due to white blood cells clogging up the blood vessels, bleeding (hemorrhage), leukemia spreading to the brain or spinal cord, and lethal side effects from leukemia treatments.
Leukemia is caused by changes in the genetic material of the bone marrow cells that produce blood cells, causing the overproduction of immature blood cells.
According to the Leukemia and Lymphoma Society, the average three-year cost to treat chronic leukemia is $200,000, but the average three-year cost to treat acute leukemia is $800,000.
Most cases of leukemia will be treated by a combination of oral medications and injections.
Acute leukemia can be cured. Most cases of leukemia, including all cases of chronic leukemia, can only be managed, but not cured. A normal life is possible, but even people who have been cured must be checked regularly for relapse.
Natural, dietary, or alternative treatments will not cure leukemia.
Gerardo Sison, Pharm.D., graduated from the University of Florida. He has worked in both community and hospital settings, providing drug information and medication therapy management services. As a medical writer, he hopes to educate and empower patients to better manage their health and navigate their treatment plans.
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