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Pediatric Chemotherapy

  • A cancer treatment approach that targets rapidly growing (cancerous) cells in the body
  • Used to treat childhood cancers
  • Immunotherapy may used together with chemotherapy for treatment
  • Involves Pediatric Hematology & Oncology Program and Yale Cancer Center

Pediatric Chemotherapy


Over the past half century, survival rates for children with cancer have risen dramatically. More than 80% of children diagnosed with cancer are cured, whereas in 1975, that was true for just a fraction of patients. This big increase is the result of major advances in cancer treatments and decades of clinical trials.

In both children and adults, cancer begins when abnormal cells in the body start to grow uncontrollably. Certain types of cancer are more likely to develop in children, including acute leukemias, neuroblastoma, retinoblastoma, bone sarcomas, brain (or central nervous system) tumors, and lymphomas.

For each child with cancer, the treatment plan depends on the type of cancer and where it’s located in the body. Perhaps the most common options for pediatric cancer patients (infancy to age 18) is chemotherapy. Surgery and radiation therapy also play a key role in treating certain types of pediatric cancers.   

“Previously our treatment for cancer would include a combination of chemotherapy plus either surgery or radiation, or sometimes a combination of both,” says Farzana Pashankar, MD, a hematologist-oncologist in Yale Medicine’s Pediatrics Department and a member of Yale Cancer Center’s Pediatric Hematology & Oncology Program. “However now we've expanded the arsenal to include immunotherapy as well.”

What is chemotherapy?

Chemotherapy (or “chemo”) refers to drugs that destroy cancer cells by interfering with their growth. It’s especially effective for cancers that are spread throughout the body, such as leukemia and lymphoma. Though chemotherapy can be the sole treatment, it’s often given in combination with other treatments, like surgery and radiation therapy.

Patients may receive chemotherapy before surgery (called neoadjuvant chemotherapy) to shrink the tumor and make the surgery less invasive. They may also receive it after surgery (adjuvant chemotherapy) to kill any cancer cells that couldn’t be removed during the procedure.

How do children receive chemotherapy?

Children with cancer are treated at the Yale New Haven Children’s Hospital and Smilow Cancer Hospital. Doses of chemotherapy are delivered in cycles, separated by periods of rest and recovery. Sometimes, multiple cycles, over several weeks or even months, are necessary.

Like other drugs, chemotherapy comes in many forms. Often, it’s given as pills or injections. When it’s given intravenously, one end of a tube (called an IV) is inserted directly into a vein, and the other end is attached to a bag that holds the medicine. 

What are the side effects of chemotherapy in children?

Though the goal of chemotherapy is to destroy fast-growing cancer cells, it ends up killing some fast-growing normal cells, too. These include cells in the hair, bloodstream, mouth, and digestive tract—which explains why chemotherapy patients often have hair loss, low blood counts (anemia), mouth sores and nausea, among other ailments. It’s also normal for children undergoing chemotherapy for cancer to feel very tired and even to have cognitive challenges during treatment. Fortunately, these effects tend to go away once chemotherapy is over.

Chemotherapy depletes white blood cells, which help the body to fight infection. For this reason, caregivers may be asked to limit the patient’s exposure to possible contaminants, such as animals and certain foods. It’s also important to stay away from sick family members and friends.

What are the long-term effects of chemotherapy in children?

Though they can be life-saving, it’s important to recognize that chemotherapy treatments in childhood can have lifelong effects. Among younger children, who are still developing, chemotherapy can slow down growth. Also, studies have shown that up to half of those who survive pediatric cancer develop some sort of chronic condition by age 50—some of these conditions (known as “late effects”) have been linked to chemotherapy.

In the long term, chemotherapy may lead to problems in any organ, including the brain, heart, lungs, and kidneys. Evidence suggests that, in both boys and girls, chemotherapy has the potential to cause sexual problems and infertility. Ultimately, doctors recommend lifelong follow-up survivorship care, including emotional support, for all childhood cancer patients who have received chemotherapy treatment. 

What is Yale Cancer Center’s approach to treating childhood cancers?

The Pediatric Hematology & Oncology Program at Smilow Cancer Hospital provides care for children with all forms of cancer, including leukemia, solid tumors, and lymphomas.

Our participation in the Children’s Oncology Group, a National Cancer Institute-supported clinical trials group, makes it possible to provide the most current and advanced treatment regimens to children with any form of cancer, common or rare.

Yale Medicine doctors take an individualized approach to caring for children with cancer. “We now are really looking at profiling the tumors,” says Dr. Pashankar. “We are using newer and different modalities such as immunotherapy to give more directed ‘focus therapy’ for that particular child's cancer, as opposed to more broad ‘just-chemotherapy’ regimens.”

“Adding immunotherapy to a child’s cancer treatment means there is significant improvement in not only long term survival for some cancers, but also event-free survival, which means not having the cancer come back at all,” she says.