The types of cancers that develop in children are different from the types that are found in adults. In most cases, childhood cancers tend to respond better to chemotherapy. That’s because these cancers are growing quickly, and most forms of chemotherapy target cells that are growing fast. But because chemotherapy can have some long-term side effects, children who survive their cancer need careful attention for the rest of their lives.
Children with cancer and their families have special needs that are best met by children's cancer centers. Treatment in these centers offers them the advantage of a team of experts with experience in treating children. The team can include (besides doctors and nurses) psychologists, social workers, child life specialists, educators, and others.
Since the 1960s most children with cancer have been treated at these special centers. In the United States, most children with cancer are treated at a pediatric cancer center that is a member of the Children’s Oncology Group (COG). All of these centers are part of a university or a children’s hospital. As we have learned more about treating childhood cancer, it has become even more important that treatment be given by experienced experts.
What Is Childhood Leukemia?
Leukemia is a cancer of the blood-forming cells. Most of time, it involves the white blood cells, but it can involve other blood cell types as well. Leukemia starts in the bone marrow and then spreads to the blood. From there it can go to the lymph nodes, spleen, liver, central nervous system, and other organs.
In contrast, some other types of cancer can start in these organs and then spread to the bone marrow (or elsewhere). But those cancers are not leukemia.
What Causes Childhood Leukemia?
The exact cause of most cases of leukemia is not known. But doctors have found that this cancer is linked to a number of risk factors.
A risk factor is something that increases a person's chance of getting a disease. Different cancers have different risk factors. Some risk factors, such as smoking, can be controlled. Others, like a person's age or family history, can't be changed. But risk factors don't tell us everything. Having a risk factor, or even several risk factors, does not mean that you will get the disease. And many people who get the disease may not have had any known risk factors
Genetic Risk Factors
Certain genetic diseases that cause children to be born with an abnormal immune system increase their risk of getting leukemia. Other genetic conditions such as Li-Fraumeni syndrome, Down syndrome, Klinefelter syndrome, and others also carry an increased risk of leukemia. While some genetic factors increase the risk of childhood leukemia, most cases of leukemia are not linked to any known genetic causes.
Brothers and sisters of children with leukemia have a slightly increased chance of getting leukemia, although the overall risk is still low. The risk is much higher among identical twins. If an identical twin develops childhood leukemia, the other twin has about a 20% chance of getting leukemia as well. This risk is even higher if the leukemia starts in the first year of life.
Environmental Risk Factors
Environmental risk factors are things around us, such as radiation and certain chemicals, which increase the risk of getting diseases such as leukemias.
Radiation
Exposure to high levels of radiation is a risk factor for childhood leukemia. Japanese atomic bomb survivors had an increased risk of getting AML. There are also risks after exposure from a nuclear accident. There may also be some risk if a fetus is exposed to radiation within the first months of pregnancy, although the extent of the risk is not clear.
It is not known how much risk their might be when children are exposed to lower levels of radiation, such as from x-rays or CT scans. Any risk increase is likely to be small, but to be safe, most doctors do not order these tests for fetuses or children unless they are really needed.
Children and adults who were treated with radiation therapy and chemotherapy for other cancers have a higher risk of getting a second cancer such as AML.
Patients who are taking drugs to suppress their immune systems (mainly organ transplant patients) have a higher risk of getting certain cancers such as lymphoma and ALL.
Lifestyle Risk Factors
For the most part, lifestyle risk factors such as diet and exercise, while important in adult cancers, are not linked to childhood cancers. Some studies have suggested that a mother drinking a lot of alcohol during pregnancy may increase the risk of leukemia in her child, but not all studies have found such a link.
It is important to remember that most children with leukemia do not have any known risk factors, and the cause of their cancer is not known at this time.
While we don’t know the exact cause of most cases of leukemia, during the past few years, scientists have made great progress in understanding how certain changes in DNA can cause bone marrow stem cells to develop into leukemia. DNA is the substance that carries the instructions for nearly everything our cells do.
A few studies have suggested that some childhood leukemias may be caused by a combination of genetic and environmental factors. For example, certain genes normally control how our bodies break down and get rid of harmful chemicals. Some people have different versions of these genes that make them less effective. Children who inherit these genes may not be as able to break down harmful chemicals if they are exposed to them. The combination of genetics and exposure might increase their risk for leukemia.
How Is Childhood Leukemia Found?
At this time, there are no special tests to help find leukemia early. The best way to find the disease early is for the parents to call the doctor promptly if they notice that their child has any of the symptoms listed below.
Close follow-up is important for children who have been treated with chemotherapy or radiation therapy for an earlier cancer, children who have certain genetic conditions, and children who have received organ transplants. These children are at greater risk for certain types of leukemia.
The exams and tests below are used to find the disease, measure how advanced it may be, and help decide what type of leukemia it is. That way, treatment can be tailored to provide the best chance of success.
Signs and Symptoms of Childhood Leukemia
Most of the signs and symptoms of leukemia result from a lack of normal blood cells. This happens because the cancer cells crowd out the normal cells that make blood in the bone marrow. The leukemia cells may also invade other areas of the body, which can also cause symptoms. Keep in mind that many of the symptoms below also have causes other than leukemia.
Fatigue, Paleness
A child may complain of being very tired or skin may be pale because of a shortage of red blood cells.
Infections and Fever
A child with leukemia may get an infection that doesn’t get better with antibiotics and have a high fever. This can happen because there are not enough normal white blood cells. Although children with leukemia may have very high white blood cell counts, the cells are not normal and may not protect against infection the way they should.
Easy Bleeding or Bruising
A child with leukemia may bruise easily or have increased bleeding from small cuts and nosebleeds. There may be small red spots on the skin from bleeding from tiny blood vessels. The bleeding is caused by a lack of blood platelets, which are needed for plugging holes in blood vessels.
Bone Pain
Some children will have bone pain, and a smaller number will have joint pain. Leukemia cells can collect underneath the covering of the surface of the bone or inside the joint causing pain.
Swelling of the Belly (abdomen)
Leukemia can cause the liver or spleen to get larger. The doctor can feel this swelling.
Loss of Appetite, Weight Loss
If the spleen or the liver become large enough, they may press against other organs like the stomach. This can limit the amount of food that can be eaten, leading to a loss of appetite and weight loss over time.
Swollen Lymph Nodes
Leukemia can spread to lymph nodes causing them to swell. The child, a parent, or a doctor or nurse might notice swollen nodes on the sides of the neck, in the groin, in the underarm area, or above the collarbone. Swelling of the lymph nodes inside the chest or abdomen is usually found by tests such as CT or MRI scans. (An enlarged lymph node in a child is more often a sign of an infection than leukemia, but it should be checked by a doctor and followed closely.)
Coughing or Trouble Breathing
The T-cell type of acute lymphocytic leukemia (ALL) often involves the thymus gland which is found in the chest near the heart. Enlargement of the thymus or of lymph nodes inside the chest can press on the nearby windpipe. This can lead to coughing, shortness of breath, or even suffocation.
Swelling of the Face and Arms
A large vein that carries blood from the head and arms back to the heart, passes next to the thymus. Growth of the leukemia cells may press on this vein, causing the blood to "back up" in the veins. This can lead to swelling and bluish-red discoloration of the head, arms, and upper chest. This can affect the brain and threaten the child's life. Patients with this problem need immediate treatment.
Headache, Seizures, Vomiting
Leukemia cells can spread outside the bone marrow into the central nervous system, causing headaches, trouble concentrating, weakness, seizures, vomiting, problems with balance, and blurred vision..
Rashes, Gum Problems
Cancer cells can spread to the gums, causing swelling, pain, and bleeding. Spread to the skin can cause spots that look like rashes.
Extreme Tiredness, Weakness
One rare but very serious symptom of AML is extreme tiredness, weakness, and slurring of speech. This can happen when very high numbers of leukemia cells make the blood too thick and reduce the flow of blood to the brain.
Tests Used for Leukemia
Most of the symptoms of leukemia are common and can be caused by something other than cancer. The doctor will want to conduct certain tests to find out if cancer is present and, if so, what type of leukemia it is.
Medical History and Physical Exam
The doctor will want to get a thorough medical history, including how long your child has had symptoms and whether or not your child has any risk factors. A family history of cancer, especially leukemia, may also be important.
The physical exam will likely focus on any swollen lymph nodes, areas of bleeding or bruising, or signs of infection. The child's eyes and mouth will likely be looked at carefully. The doctor will feel the abdomen for signs of an swollen spleen or liver.
If there is any reason to think there might be problems caused by abnormal numbers of blood cells (anemia, infections, bleeding or bruising, etc.), the doctor will likely test your child's blood counts. If these are not normal, the doctor may refer you to a childhood cancer doctor, who may run one or more of the tests described below.
Types of Samples Used to Test for Leukemia in Children
Blood cell counts: Blood tests will be done on blood drawn from a vein or from a “finger stick.” Most children with leukemia will have too many white cells in their blood and not enough red blood cells or platelets. Also, the white blood cells will be "young" cells (called blasts) normally found in the bone marrow but not in the blood. Changes in the numbers of different cell types and how these cells look under the microscope often make the doctor suspect leukemia. But usually a sample of bone marrow will need to be taken to be certain.
Bone marrow samples: Bone marrow samples are obtained from a bone marrow aspiration and biopsy -- two tests that are usually done at the same time. The samples are usually taken from the back of the pelvic (hip) bone, although in some cases they may be taken from the breastbone or other bones.
This test involves removing a small amount of bone marrow from the back of the hip bone with a thin needle. The sample is sent to the lab to see if cancer cells are present. This test is also used later to see if the leukemia is responding to treatment.
For this test, the area around the bone is numbed and the child may be given medicine to reduce pain or to cause sleep during the test. . Once the biopsy is done, pressure will be applied to the site to help prevent any bleeding
Spinal tap (lumbar puncture): This test is done to look for leukemia cells in the liquid around the brain and spinal cord (cerebrospinal fluid or CSF). It can also be used to give drugs into the CSF to keep the cancer from spreading there.
After numbing the skin, a small needle is placed between the bones in the child’s spine in the lower back to draw out some cerebrospinal fluid (CSF). The fluid is looked at for leukemia cells The doctor may recommend that the child be given anesthesia to put them to sleep so he or she won’t squirm during the test. Squirming may keep the spinal tap from being done cleanly. It is important that someone who is an expert in this procedure do it. Doctors have found that if the spinal tap isn’t done right and some blood leaks into the spinal fluid, leukemia cells could escape into the spinal fluid and grow there.
Lymph node biopsy: For this test, an entire lymph node is removed. If the node is near the skin's surface, this is a simple operation that can be done by numbing just the area around the node. But if the node is inside the chest or abdomen, the child will need general anesthesia (to be put into a deep asleep). This test is only rarely needed for children with leukemia.
Lab Tests for Leukemia
A doctor with special training in blood and lymphoid tissue disease will examine all of the biopsy samples (bone marrow, lymph node tissue, blood, and cerebrospinal fluid) under a microscope. The doctor looks at the size and shape of the cells as well as other features to classify the cells into specific types.
An important factor is whether or not the cells look mature. Some leukemic cells do not have the features of normal blood cells and do not work well in fighting infections. The most immature cells are called "blasts".
There are also a number of very precise lab tests the doctor might use to diagnose and classify leukemia. You might hear some of the following terms used: cytochemistry, cytogenetics, flow cytometry, FISH, and immunocytochemistry. These tests are explained in the American Cancer Society's document Childhood Leukemia.
Other blood tests: If the child does have leukemia, other blood tests will be done to measure certain chemicals in the blood to tell how well the liver and kidneys are working. Tests may also be done to check for infections so they can be treated right away.
Imaging Tests
Imaging studies are ways of taking pictures of the inside of the body. There are several kinds of these studies that might be done in children with leukemia to get a better idea of the extent of the disease.
Chest x-rays: A chest x-ray can tell if the thymus or lymph nodes are enlarged. In can also help to find pneumonia if the child appears to have an infection.
CT (computed tomography) scans: CT scans (sometimes called "CAT" scans) are special kinds of x-rays in which a beam moves around the body, taking pictures from different angles. This test can help tell if the leukemia has spread into lymph nodes in the chest or in organs like the spleen or liver.
Instead of taking one picture, like a regular x-ray, a CT scanner takes many pictures as it rotates around your child. A computer then combines these pictures into detailed images of the part of your body that is being studied.
Often after the first set of pictures is taken, your child will have an injection of a contrast dye or be asked to drink a solution of contrast material. This helps better outline blood vessels and internal organs. A second set of pictures is then taken. CT scans take longer than regular x-rays. Your child will need to lie still on a table while they are being done.
MRI (magnetic resonance imaging): An MRI is like a CT scan except that magnets and radio waves are used instead of x-rays. MRI scans are helpful in looking at the brain and spinal cord.
MRI scans take longer than CT scans -- often up to an hour. Your child may have to lie inside a narrow tube, which can be hard for some children. Sedation is sometimes needed. Newer, "open" MRI machines may be another option. The MRI machine makes loud buzzing noises that your child may find disturbing. Some places provide headphones to block this out.
Ultrasound: The ultrasound uses sound waves to produce a picture of the inside of the body. Ultrasound can be used to look for enlarged organs inside the belly. This is an easy test to have done. Your child simply lies on a table, and a technician moves a sort of wand over the part of the body being looked at.
Gallium scan and bone scan: These can be useful if the child has bone pain that might be caused by infection or cancer in the bones. If the child has already been found to have leukemia, there is no need for these scans.
How Is Childhood Leukemia Treated?
This information represents the views of the doctors and nurses serving on the American Cancer Society's Cancer Information Database Editorial Board. These views are based on their interpretation of studies published in medical journals, as well as their own professional experience.
The treatment information in this document is not official policy of the Society and is not intended as medical advice to replace the expertise and judgment of your cancer care team. It is intended to help you and your family make informed decisions, together with your doctor.
Your doctor may have reasons for suggesting a treatment plan different from these general treatment options. Don't hesitate to ask him or her questions about your treatment options.
Your doctor should make sure that your child's treatment reflects his or her risk group. Your child should be treated according to a set of instructions called a protocol from the National Cancer Institute or a cooperative study group. This will ensure the most up-to-date treatment for your child. And be sure to ask your doctor about any side effects your child might have from treatment. Many parents find it helpful to bring a note pad or a tape recorder when they talk to the doctor.
Be sure to tell your child’s doctor about any drugs, herbal remedies, or other things you might be giving your child. These could affect how well the chemotherapy works.
This section contains general comments about types of treatments used for childhood leukemia, followed by a discussion of treatment options based on the type of leukemia.
Immediate Treatment
Some children are very ill when they are found to have leukemia, or they become ill during treatment. Low blood counts can result in serious problems such as infections, bleeding, and even heart failure. Antibiotics or other medicines and transfusions may be needed to treat or prevent some of these problems before the leukemia is treated.
Surgery
Unlike most other cancers, surgery rarely has any role in the treatment of leukemia. This is because leukemia is a disease of blood and bone marrow and cannot be cured with surgery.
But surgery may be used to help give treatment. A plastic tube may be placed into a large vein. The tube, called a venous access device (VAD), allows medicines to be given and blood samples to be removed without the need for repeated needle sticks. The end of the tube is just under the skin or sticks out in the chest area or upper arm. It is important for parents to learn how to care for the VAD.
Radiation Therapy
Radiation therapy is the use of high-energy radiation to kill cancer cells. For children with acute leukemia, radiation might be used to treat cancer in the membranes that cover the brain or in the testicles. It can also be used, though rarely, in an emergency to help relieve pressure on the windpipe.
Chemotherapy
Chemotherapy refers to the use of drugs to kill cancer cells. Usually the drugs are given either into a vein, into the cerebrospinal fluid, or as a pill. Once the drugs enter the bloodstream, they spread throughout the body. Chemotherapy is the main treatment for nearly all types of leukemia. Children might receive several drugs at different times during the course of treatment. In general, AML treatment will involve higher doses of chemotherapy over a shorter period of time while ALL will involve the use of lower doses over a longer period of time.
Immediate Side Effects
While chemotherapy drugs kill cancer cells, they can also damage normal cells. This happens because they target cells that are growing quickly such as cancer cells. But in the process they also damage other fast-growing cells. The lining of the mouth and intestines, hair, and blood cells all grow quickly and are likely to be damaged by chemotherapy, which can lead to the following side effects:
hair loss
mouth sores
loss of appetite
diarrhea
nausea and vomiting
lowered resistance to infection (because of low white blood cell counts)
bruising and bleeding easily (due to low platelet counts)
fatigue (due to low red blood cell counts)
These side effects usually go away shortly after treatment ends. And there are often ways to manage these side effects during treatment. For example, there are drugs that can be taken to prevent or reduce nausea and vomiting. Drugs known as growth factors are sometimes given to keep blood counts higher and reduce the chance of infection.
Tumor lysis syndrome can be a side effect of chemotherapy. When leukemia cells are killed, they break open and release their contents into the bloodstream. This can affect the kidneys, heart, and nervous system. Giving the child extra fluids or certain drugs that help rid the body of these toxins can prevent this problem.
Long-term Side Effects
Possible long-term effects of chemotherapy are described in the section, "Moving on After Treatment?"
Bone Marrow or Peripheral Blood Stem Cell Transplant (SCT)
This treatment can be used for children whose chances of survival are very poor with standard or even intense chemo. SCT allows doctors to use very high doses of chemotherapy. The high doses of these drugs destroy the bone marrow, which keeps new blood cells from being made. This could be a threat to the child’s life. Doctors try to avoid this problem by giving the child blood-forming stem cells. Stem cells are very early cells that are able to make new blood cells. These cells can be taken either from your child (and stored before treatment) or the cells can come from another person.
Allogeneic SCT
For childhood leukemia, the blood-forming stem cells are generally donated from another person. This is called an allogeneic stem cell transplant -- meaning the cells come from someone else. Usually the donor is a brother or sister but, rarely, the donor could be an unrelated volunteer. Stored cells from umbilical cord blood have also been used for unrelated donor transplants. This source has become more common although the supply is limited.
Autologous SCT
In an autologous stem cell transplant, the patient's own stem cells are removed from his or her bone marrow or bloodstream. They are frozen until after treatment with chemo or radiation and then given back to the patient.
This procedure is rarely used for childhood leukemia, for a couple of reasons. One concern is that there may be a risk that some of these cells have cancer. To avoid this, the doctors treat the stored cells with drugs to kill any cancer cells. And leukemia has more of a chance to relapse when the child's own stem cells are used. So stem cells from a donor are more often used.
When SCT May Be Used
SCT may be used for children with ALL whose leukemia comes back early after going into remission. Doctors are not as sure about using it if more than 6 months has passed since the first chemo was given. These children will often do well with another round of standard chemo.
Because AML comes back more often, some doctors will recommend SCT for some children with AML right after they have gone into remission if the child has a brother or sister who can donate cells. If the cancer comes back after the first round of chemo, then most doctors will suggest SCT as soon as the child goes into remission again.
In any case, it is important that the patient is in remission. Otherwise, the leukemia is more likely to return.
What It Involves
Here is how the SCT process works: Stem cells are collected from either the bone marrow or from the bloodstream in a process called apheresis. The cells are frozen and stored. The child is then given very high doses of chemo (and sometimes radiation) to kill the cancer cells. Normal cells in the bone marrow are killed as well. After treatment, the stored stem cells are thawed and given to the child as a blood transfusion. Then the waiting period begins as the stem cells settle in the child's bone marrow and start to grow and produce new blood cells. For the next 3 to 4 weeks the patient is a high risk for serious infections (because of low white blood cell counts), as well as bleeding (because of low platelet counts). Transfusions and antibiotics are often used to prevent or treat these problems.
Stem cell transplant is a complex treatment. If the doctors think that a child with leukemia might be helped by this treatment, it is important that it be done at a hospital where the staff has experience with the procedure.
Stem cell transplant is very expensive (more than $100,000) and requires a lengthy hospital stay. Because it is so costly, parents should be sure to get a written approval from their insurer if their child is to have this treatment.
Side Effects of SCT
Side effects from a stem cell transplant can be divided into early and long-term effects. Early side effects are basically the same as those of any other type of high-dose chemotherapy (low blood cell counts, nausea, vomiting, hair loss, etc.). They are caused by damage to the bone marrow and other rapidly reproducing tissues of the body. One of the most common and serious short-term effects is the increased risk for infection. Antibiotics are often given to try to prevent this from happening. Other side effects, like low red blood cell and platelet counts, may mean that the patient will need transfusions or other treatments.
Long-Term Side Effects
Some side effects can go on for a long time. Sometimes they don't show up until years after the transplant. Long-term side effects could include the following:
graft-versus-host disease (see below)
radiation damage to the lungs
problems with the thyroid or other hormone-making glands
problems with fertility
problems with bone growth
Graft-versus-host disease is the main problem of a donor stem cell transplant. It happens when the immune system of the patient is taken over by that of the donor. The donor immune system then begins reacting against the patient’s other tissues and organs.
Symptoms can include severe skin rashes with itching and severe diarrhea. The liver and lungs may also be damaged. The patient may also become tired and have aching muscles. If severe enough, the disease can be fatal. Drugs that weaken the immune system are often given to try to keep GVHD under control.
To learn more about stem cell transplants, see the American Cancer Society article Bone Marrow & Peripheral Blood Stem Cell Transplants.
Treatment of Children with ALL
Treatment of children with ALL is divided into 3 phases: induction, consolidation (intensification), and maintenance.
Induction
Children with ALL are divided into risk groups to make sure that the correct types and doses of drugs are given. In general, the purpose of the first phase, called induction, is to destroy as many cancer cells as quickly as possible. The child is in remission when cancer cells are no longer found in the bone marrow and the blood counts are normal. Until normal blood counts return, the child's condition is considered critical. More than 95% of children with ALL will enter remission after 1 month of treatment.
During induction, frequent trips to the doctor will be needed. Your child may spend some or much of this time in the hospital. All children will need to have spinal taps to inject chemo into the cerebrospinal fluid to keep cancer from spreading to the central nervous system. And some (those with T-cell leukemia, high white blood cell counts, or cancer cells in the cerebrospinal fluid) may need radiation to the head as well. Doctors try to avoid radiation because, no matter how low the dose, it may cause some slight problems in thinking and growth.
Remission means that more than 99.9% of the cancer cells have been killed; but there are still some leukemia cells in the body. That's why further phases of treatment are needed.
Consolidation
The goal of this phase (also called intensification) is to get rid of leukemia cells from places where they can "hide." This phase lasts 4 to 8 months. Several drugs are used in combination, depending on the child's risk category. Some children (such as those with Philadelphia chromosome-positive ALL) may benefit from a stem cell transplant at this time.
Maintenance
Once the number of leukemia cells has been reduced by the first 2 phases of treatment, this last phase, maintenance chemo, can begin. The total length of therapy for all three phases is 2 to 3 years for most children with ALL.
Treatment of Residual Disease
All these treatment plans may change if the leukemia hasn’t completely disappeared. A few days after treatment has begun the doctor may check the child’s bone marrow to see if the leukemia is going away. If not, treatment may be increased or given for a longer time. When the leukemia seems to have disappeared, the doctor may do a special chemical test to look for leukemia cells. If any are found, then once again, chemotherapy may be increased or prolonged.
Treatment of Recurrent ALL
If a child with ALL relapses, he or she will be treated again with chemo. Different drugs are added to the standard ones. A stem cell transplant may be considered for children whose leukemia comes back during treatment or within 6 months after treatment, especially if there is a brother or sister who is a good match. Stem cell transplant may also be used for other children who relapse after a second course of chemotherapy.
Some children have a relapse in which leukemia cells are found in one part of the body (such as the CSF or the testicles) but are not found in the bone marrow. These children may have radiation therapy, along with intense chemo, to the affected area.
Cure Rates for ALL
Cure rates for ALL are as follows:
low risk: about 85% to 95%
standard risk: about 65% to 85%
high-risk: about 60% to 65%.
Philadelphia-chromosome type ALL: For children with at a type of high-risk leukemia, (Philadelphia-chromosome type ALL) a stem cell transplant may be advised if induction treatment brings about a remission. New targeted drugs such as Gleevec and Sprycel are now being studied for patients whose cancer cells contain this chromosome. The drug has few side effects and is being given, along with chemotherapy, for those patients.
Treatment of Children with AML
Treatment for most children with AML has 2 phases: induction and intensification. Because treatment is very intense and there is a risk of serious complications, children with AML should be treated in cancer centers or hospitals that have experience with this disease.
Induction
The combinations of drugs used to treat AML are different from those used for ALL. Treatment is given in cycles that usually last several days. The schedule of treatment may be repeated in 10 days or 2 weeks, depending on how intense the doctor wants the treatment to be. Treatment with these drugs is repeated until the bone marrow shows no more leukemia. This usually happens after 2 or 3 treatments. Often chemotherapy is also given into the spinal column. Usually radiation therapy to the brain is not needed.
Intensification
This phase begins after a remission when no more leukemia cells are seen in the bone marrow. About 1 out of 5 children has a brother or sister who would be a good stem cell donor. For these children, a stem cell transplant is often recommended. Most studies have found this improves the chance for long-term survival over chemo alone. But it is also more likely to cause serious complications. For children with good prognostic factors, some doctors may recommend just giving high-dose chemo and holding off on the stem cell transplant in case the AML relapses.
For most other children, high-dose chemo is given for at least several months. Chemo into the CSF is usually given at the start and every 1 to 2 months for as long as this phase lasts.
Most children with AML (except those with APL) do not need maintenance chemo.
An important aspect of treatment for AML is supportive care (nursing care, nutrition, antibiotic use, blood transfusions, etc.). With this care, a 75% to 85% rate of remission at the end of induction can be achieved.
Refractory or recurrent AML: The outlook for the child who doesn’t go into remission (refractory AML) or who relapses is often poor. The doctor may recommend some type of stem cell transplant if it can be done. In a recent clinical trial, children who were not in complete remission after induction were given a drug called Mylotarg. Early results suggest this treatment may help improve survival rates for some children with AML.
As a rule, the outlook for a child whose AML comes back after treatment is slightly better. But this depends on how long the first remission lasted. More than half of these children will go into a second remission with more chemo. The chance of a second remission is better if the first one lasted for at least a year, but long-term second remissions are rare without a stem cell transplant.
Most of these children will be offered a clinical trial in the hope that if a remission is brought about, a stem cell transplant can be done. Sometimes the doctor may suggest a stem cell transplant even without a remission.
Cure Rates for AML
The cure rate for AML with standard treatment is 40% to 50%. Treatment with a stem cell transplant from a brother or sister with a good tissue match brings about a 55% to 60% cure rate.
Treatment of Children With APL (Acute Promyelocytic Leukemia)
Treatment of APL differs from usual AML treatment. Most cases respond well to this treatment.
Induction
Along with chemo, children with APL get a drug something like vitamin A called ATRA (all-trans retinoic acid). Although ATRA alone often brings about a remission, combining it with chemo gives better long-term results.
The side effects of this treatment differ from those of standard AML chemo because of a possible problem called that can cause breathing problems due to fluid buildup in the lungs, low blood pressure, kidney damage, and severe fluid buildup elsewhere in the body. It can often be treated by stopping the ATRA for a while and giving a steroid.
During treatment, some patients with APL may also have blood-clotting problems. They may be given an "blood thinner" to help prevent or treat this.
Consolidation (Intensification)
This is usually much like induction, involving both ATRA and. Because of the success of this treatment, stem cell transplant is not usually advised if the child stays in remission.
Maintenance
Children with APL may get maintenance therapy with ATRA, often with chemo for about one year.
Relapsed APL
If the leukemia comes back after treatment, most cases can be put into a second remission. Arsenic trioxide is a drug that is very effective in this setting, although it can sometimes cause problems with heart rhythms. Children getting this drug need to have their blood mineral levels watched closely. A stem cell transplant may be considered once a second remission is achieved.
Cure Rates for APL
Most studies suggest that the cure rate for APL is now higher than 80%.
Treatment of Children With JMML (Juvenile Myelomonocytic Leukemia)
JMML is fairly rare, and there is no standard treatment for this leukemia. An stem cell transplant offers the best chance to cure JMML and is the treatment of choice when possible. Because it is hard to treat with current chemo drugs, some studies are now looking at other options.
Childhood Leukemia Survival
The 5-year survival rate refers to the percentage of children who live at least 5 years after their cancer is found. Of course, many children live much longer than 5 years. The 5-year survival rate for ALL has greatly increased over time and is now more than 80% thanks to advances in treatment. Five-year survival rates of children with AML have also increased over time to more than 50%.
Of course, the outlook for each child is different, depending mostly on the prognostic factors mentioned above. Talk with your cancer care team if you have questions about your child’s chances of a cure. They know the situation best.
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