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Antibody-drug conjugates (ADC) are monoclonal antibodies (man-made immune proteins) linked to chemotherapy drugs. The antibody acts like a homing device. It brings the chemo drug to the leukemia cells, where it enters the cells and kills them when they try to divide into new cells. An example of this type of targeted therapy for AML is gemtuzumab ozogamicin (Mylotarg).

This drug can be used to treat some children with AML in different situations:

• It can be used as part of the initial treatment of AML, along with chemo.
• It can be given if the leukemia comes back or is no longer responding to treatment.

This drug is given as an infusion into a vein (IV).

Possible side effects

The most common side effects are fever, nausea and vomiting, low levels of blood cells (with increased risks of infection, bleeding, and fatigue), swelling and sores in the mouth, constipation, rash, and headaches.

Less common but more serious side effects can include:

• Severe liver damage, including veno-occlusive disease (blockage of veins in the liver)
• Reactions during the infusion (similar to an allergic reaction). Your child will likely be given medicines before each infusion to help prevent this.
• Serious or life-threatening infections, especially in people who have already had a stem cell transplant
• Changes in heart rhythm

These drugs attack BCL-2, a protein that can help leukemia cells live longer. An example is venetoclax (Venclexta). In children with AML, this may be used when the cancer comes back or does not respond well to initial treatment.

These drugs are given as a tablet, by mouth, usually once daily.

Possible side effects

The most common side effects are fever, feeling tired or weak, low appetite, nausea, diarrhea, low blood counts, swelling, skin rash, and changes to some blood tests which may need to be watched while getting the drug.

In some children with ALL, leukemia cells have a type of change known as a translocation in the KMT2A gene. This causes the cells to make an abnormal KMT2A protein, which can combine with another protein called menin to help the cells grow.

Revumenib (Revuforj) is a menin inhibitor. It stops menin from combining with the KMT2A protein. This drug can be used to treat children with ALL whose leukemia cells have a KMT2A translocation, if the leukemia is no longer responding to treatment or has come back (relapsed) after other treatments. The child’s blood or bone marrow can be tested to see if the leukemia cells have this gene change.

This drug is taken by mouth as pills, typically twice a day.

Possible side effects

Common side effects of this drug can include nausea, vomiting, bleeding, diarrhea or constipation, fatigue, muscle pain, increased risk of infection, swelling in the arms and legs, and loss of appetite.

This drug may cause serious heart rhythm problems. This might lead to an irregular heartbeat, which could be life threatening. Your child’s doctor will check blood mineral levels and get electrocardiograms (EKGs) to test your child’s heart rhythm before and during treatment with this drug.

An important possible side effect of this drug is differentiation syndrome. This occurs when the leukemia cells release certain chemicals into the blood. It most often occurs shortly after starting treatment, but sometimes it can happen weeks or months later. Symptoms can include fever, cough, breathing problems (from fluid buildup in the lungs and around the heart), dizziness or lightheadedness (from low blood pressure), urinating less often (from damage to the kidneys), and severe fluid buildup elsewhere in the body. It can often be treated by stopping the drug for a while and giving other medicines (such as dexamethasone or hydroxyurea).

TKIs work by blocking the signals cells need to grow and divide. Examples of TKIs include imatinib (Gleevec), dasatinib (Sprycel), nilotinib (Tasigna), bosutinib (Bosulif), ponatinib (Iclusig) and ruxolitinib (Jakafi).

A small number of children with acute lymphocytic leukemia (ALL) have the Philadelphia chromosome (Ph+ ALL). TKIs attack cells that have the BCR-ABL gene change. For these children, combining chemo and TKI drugs results in better outcomes. They may also be used in treating some children with Ph-like ALL, where leukemia cells have gene changes other than BCR-ABL that cause growth signals to be overactive.

These drugs are taken daily as pills or capsules. Imatinib is also available as a liquid, under the name Imkeldi.

Possible side effects

Possible side effects of these drugs include diarrhea, nausea, muscle pain, fatigue, and skin rashes. These are generally mild. A common side effect is swelling around the eyes or in the hands or feet, which may be caused by the drugs’ effects on the heart. Other possible side effects include lower blood counts when treatment starts. These drugs might also slow a child’s growth, especially if used before puberty.

Antibody-drug conjugates (ADC) are monoclonal antibodies (man-made immune proteins) linked to chemotherapy drugs. The antibody acts like a homing device. It brings the chemo drug to the leukemia cells, where it enters the cells and kills them when they try to divide into new cells. An example of this type of targeted therapy for AML is gemtuzumab ozogamicin (Mylotarg).

This drug can be used to treat some children with AML in different situations:

• It can be used as part of the initial treatment of AML, along with chemo.
• It can be given if the leukemia comes back or is no longer responding to treatment.

This drug is given as an infusion into a vein (IV).

Possible side effects

The most common side effects are fever, nausea and vomiting, low levels of blood cells (with increased risks of infection, bleeding, and fatigue), swelling and sores in the mouth, constipation, rash, and headaches.

Less common but more serious side effects can include:

• Severe liver damage, including veno-occlusive disease (blockage of veins in the liver)
• Reactions during the infusion (similar to an allergic reaction). Your child will likely be given medicines before each infusion to help prevent this.
• Serious or life-threatening infections, especially in people who have already had a stem cell transplant
• Changes in heart rhythm

These drugs attack BCL-2, a protein that can help leukemia cells live longer. An example is venetoclax (Venclexta). In children with AML, this may be used when the cancer comes back or does not respond well to initial treatment.

These drugs are given as a tablet, by mouth, usually once daily.

Possible side effects

The most common side effects are fever, feeling tired or weak, low appetite, nausea, diarrhea, low blood counts, swelling, skin rash, and changes to some blood tests which may need to be watched while getting the drug.

Differentiation agents can be used to treat a special type of AML, called acute promyelocytic leukemia (APL). The leukemia cells in APL (called blasts), have certain gene changes that stop them from maturing into normal white blood cells. These drugs help the blasts mature (differentiate) into normal white blood cells. Two of these drugs can be used to treat APL.

All-trans retinoic acid (ATRA, tretinoin) is a form of vitamin A that is typically part of the initial treatment of APL. It is given either along with chemo or along with ATO. It can also be used during later phases of treatment.

Arsenic trioxide (ATO) can act in a way similar to ATRA in patients with APL and kills the blast cells. It is often given with ATRA in the treatment of APL, but it is also helpful in treating APL that comes back after treatment with ATRA plus chemo.

Possible side effects

Side effects of ATRA can include fever, headache, dry skin, mouth sores, skin rashes, swollen feet, itching and irritated eyes. It can also raise blood lipid levels (like cholesterol and triglycerides). Often blood liver test results become abnormal. These side effects often go away when the drug is stopped.

Most side effects of ATO are mild and can include feeling tired, nausea, vomiting, diarrhea, belly pain and nerve damage, called neuropathy, which can lead to numbness or tingling in the hands and feet. ATO can also cause problems with heart rhythm, which can sometimes be serious.

Both drugs can cause a serious side effect known as differentiation syndrome (previously called retinoic acid syndrome). This occurs when the leukemia cells release certain chemicals into the blood. It is most often seen during the first couple of weeks of treatment and in patients with a high white blood cell count.  Symptoms can include fever, breathing problems, due to fluid buildup in the lungs and around the heart, low blood pressure, kidney damage, and severe fluid buildup elsewhere in the body. While differentiation syndrome can be serious, it can often be treated by stopping the drugs for a while and giving a steroid such as dexamethasone.

These drugs block proteins on leukemia cells that are important for cells to grow and divide, which causes leukemia cells to die. Examples of drugs that can block FLT3 include sorafenib (Nexavar), gilteritinib (Xospata) and midostaurin (Rydapt). These drugs can be used in the initial treatment of AML with FLT3 gene changes or if the cancer comes back or does not respond well to treatment.

These drugs are taken by mouth in tablet or capsule form.

Possible side effects

Common side effects of these drugs include fever, mouth sores, feeling tired or weak, low blood counts, headaches or muscle aches, weight gain, stomach upset or pain, skin changes, and trouble sleeping.

Sorafenib can cause unique side effects. Some people treated with sorafenib develop high blood pressure, so doctors will often monitor the blood pressure during treatment. A distinct skin rash can occur with sorafenib as well. This causes redness, pain and sometimes swelling in the palms and soles of the feet.

In some children with AML, the leukemia cells have a type of change known as a translocation in the KMT2A gene. This causes the cells to make an abnormal KMT2A protein, which can combine with another protein called menin to help the cells grow.

Revumenib (Revuforj) is a menin inhibitor. It stops menin from combining with the KMT2A protein. This drug can be used to treat children with AML whose leukemia cells have a KMT2A translocation if the leukemia is no longer responding to treatment or has come back (relapsed) after other treatments. The child’s blood or bone marrow can be tested to see if the leukemia cells have this gene change.

This drug is taken by mouth as pills, typically twice a day.

Possible side effects

Common side effects of this drug can include nausea, vomiting, bleeding, diarrhea or constipation, fatigue, muscle pain, increased risk of infection, swelling in the arms and legs, and loss of appetite.

This drug may cause serious heart rhythm problems. This might lead to an irregular heartbeat, which could be life threatening. Your child’s doctor will check blood mineral levels and get electrocardiograms (EKGs) to test your child’s heart rhythm before and during treatment with this drug.

An important possible side effect of this drug is differentiation syndrome. This occurs when the leukemia cells release certain chemicals into the blood. It most often occurs shortly after starting treatment, but sometimes it can happen weeks or months later. Symptoms can include fever, coughing or breathing problems (from fluid buildup in the lungs and around the heart), dizziness or lightheadedness (from low blood pressure), urinating less often (from damage to the kidneys), and severe fluid buildup elsewhere in the body. It can often be treated by stopping the drug for a while and giving other medicines (such as dexamethasone or hydroxyurea).

Nearly all children with chronic myeloid leukemia (CML) have an abnormal chromosome in their leukemia cells known as the Philadelphia chromosome. These chromosomes have a specific gene change known as BCR-ABL, which helps the leukemia cells multiply.

TKIs such as imatinib (Gleevec), dasatinib (Sprycel), nilotinib (Tasigna), and bosutinib (Bosulif), attack cells that have the BCR-ABL gene change. These drugs are very effective at controlling the leukemia for long periods of time in most children.

These drugs are taken daily as pills or capsules. Imatinib is available as a liquid, under the name Imkeldi.

Possible side effects

Possible side effects of these drugs include diarrhea, nausea, muscle pain, fatigue, and skin rashes. These are generally mild. A common side effect is swelling around the eyes or in the hands or feet, which may be caused by the drugs’ effects on the heart. Other possible side effects include lower blood counts when treatment starts. These drugs might also slow a child’s growth, especially if used before puberty.