Childhood Blood Cancers: New Treatments Bring Growing Hope

Few words frighten a parent more than hearing that their child has cancer. The diagnosis can turn an ordinary childhood—and an entire family’s life—upside down almost overnight.

“A pediatric cancer diagnosis is not easy for anyone involved,” acknowledges Doured Daghistani, M.D., a pediatric hematologist/oncologist and medical director of pediatric oncology at Baptist Health Herbert Wertheim Cancer Institute. Known affectionately as “Dr. D.,” Dr. Daghistani has spent decades helping families navigate treatment with expertise and hope.

Although childhood cancer is rare, leukemia is the most common cancer diagnosed in children younger than 15, and lymphomas are also among the most frequent, Dr. Daghistani says.

Treatment has advanced dramatically. U.S. childhood cancer death rates declined by approximately 70 percent between 1970 and 2020, according to the National Cancer Institute (NCI). Five-year survival for children younger than 15 with acute lymphoblastic leukemia, or ALL, is now approximately 90 percent.

What Is a Blood Cancer?

Blood cancers begin when abnormal blood-forming or immune-system cells grow uncontrollably. Unlike many solid tumors, they generally do not form a single mass that can be removed surgically, Dr. Daghistani says. 

The two principal categories in children are leukemia and lymphoma.

Leukemia
Leukemia begins in blood-forming cells in the bone marrow. Abnormal cells can crowd out healthy red blood cells, white blood cells and platelets, interfering with the body’s ability to carry oxygen, fight infection and control bleeding.

• Acute lymphoblastic leukemia: ALL affects immature lymphocytes and is the most common childhood cancer overall, accounting for approximately one-quarter of U.S. childhood cancers. It occurs most often between ages 1 and 4.

• Acute myeloid leukemia: AML begins in immature myeloid cells. About 20 percent of childhood leukemias are myeloid leukemias, most of them AML.

• Other leukemias: Chronic myeloid leukemia grows more slowly and is uncommon in children. Juvenile myelomonocytic leukemia is a rare cancer seen primarily in infants and young children.

Lymphoma
Lymphoma develops in lymphocytes, white blood cells that help fight infection. It may appear in lymph nodes, the chest, abdomen, bone marrow or other organs.

• Hodgkin lymphoma: This cancer is most common among adolescents ages 15 to 19.
• Non-Hodgkin lymphoma: Childhood NHL includes several fast-growing diseases, including Burkitt lymphoma, lymphoblastic lymphoma and anaplastic large cell lymphoma.Together, lymphomas account for about 15 percent of cancers diagnosed in children and teenagers.

Signs That Deserve Attention

Symptoms of childhood blood cancers often resemble common illnesses. A fever, fatigue or swollen lymph node usually does not mean cancer, but parents should contact their pediatrician when symptoms are unexplained, persistent, worsening or occurring together.

Warning signs may include:

• Persistent fever
• Fatigue or paleness
• Frequent infections
• Easy bruising or bleeding
• Bone, joint or abdominal pain
• Unexplained weight loss
• Painless swelling in the neck, underarm or groin
• Night sweats
• Breathing difficulties

Blood tests can identify abnormal numbers or types of cells, says Dr. Daghistani.

“When leukemia is suspected, we generally perform a bone marrow aspiration or biopsy to confirm the diagnosis and identify the cancer’s subtype. Lymphoma evaluation may include imaging studies and a biopsy of an enlarged lymph node or other abnormal tissue,” he says.

Genetic, molecular and chromosomal testing can further classify the disease, estimate how it may respond and help physicians select the most effective therapy.

Why Do Children Develop Blood Cancer?

For most children with leukemia or lymphoma, there is no identifiable cause—and nothing a parent did or failed to do caused the disease. Unlike many adult cancers, childhood cancers generally are not linked to smoking, diet, weight, alcohol use or other lifestyle factors.

A small number of cases are associated with inherited genetic conditions, changes in genes involved in blood-cell development, previous chemotherapy or radiation, high levels of ionizing radiation, a weakened immune system, Epstein-Barr virus in some lymphomas or a close family history of certain blood cancers.

“Having one of these risk factors does not mean a child will develop cancer,” Dr. Daghistani emphasizes. “Conversely, most children diagnosed with a blood cancer have none of them.”

Treatment Tailored to Each Child

Treatment depends on the exact diagnosis, the cancer’s molecular characteristics, the child’s age and health, whether the disease involves the brain or spinal cord, and how quickly it responds to initial therapy.

• Chemotherapy
Chemotherapy remains the foundation of treatment for most childhood leukemias and lymphomas. For ALL, treatment may extend over several years to eliminate remaining cancer cells and prevent relapse. Pediatric teams also anticipate and manage side effects such as nausea, fatigue, hair loss, mouth sores and infection risk. 

• Targeted Therapy
Targeted medicines interfere with proteins or genetic changes that help cancer cells survive. Children whose ALL or chronic myeloid leukemia contains certain abnormalities may receive a tyrosine kinase inhibitor with chemotherapy, improving effectiveness without necessarily increasing their chemotherapy burden.

• Immunotherapy
Immunotherapy helps the immune system recognize and attack cancer. Blinatumomab has become important for certain children with B-cell ALL. CAR T-cell therapy, which modifies a patient’s own immune cells to destroy leukemia cells, may be considered when B-cell ALL resists standard treatment or returns, Dr. Daghistani says.

• Stem Cell Transplantation
A blood or bone marrow stem cell transplant replaces diseased blood-forming cells with healthy cells. It may be recommended for selected children with high-risk, persistent or recurrent disease, although the decision requires careful consideration of short- and long-term risks.

• Radiation Therapy
Radiation is used selectively, including for some Hodgkin lymphomas or cancers involving certain organs or the central nervous system. Physicians limit exposure for young patients whenever possible to minimize possible late effects. When radiation is necessary, Wertheim Cancer Institute offers proton therapy, which directs treatment precisely at the target, reducing exposure to nearby healthy organs and tissues.

Treating the Child, Not Just the Cancer

Effective pediatric cancer care extends beyond medicines and procedures. Children may fear needles, unfamiliar equipment, changes in appearance or separation from school and friends. Parents may face emotional, financial and logistical strain.

At Wertheim Cancer Institute and Baptist Children’s Hospital, pediatric oncology care brings together physicians, nurses, pharmacists, psychologists, social workers, child life specialists, nutrition professionals, palliative care experts and spiritual care providers. 

They help children understand treatment, manage symptoms and anxiety, stay engaged with school and preserve normalcy. That support can be as important as sophisticated technology, says Dr. Daghistani. Follow-up care is equally important, he adds.

“Children require personalized follow-up after treatment so emerging issues can be identified and managed early.”

 

Why Specialized Pediatric Cancer Care Matters

Childhood blood cancers are biologically different from adult cancers and should be treated by teams with pediatric expertise. The NCI recommends care at centers where multidisciplinary professionals understand young patients’ distinct medical, developmental and emotional needs.

Wertheim Cancer Institute combines pediatric hematology-oncology expertise with genomic testing, precision medicine, proton therapy, clinical research and collaboration through the Memorial Sloan Kettering Cancer Alliance. Families have coordinated access to specialists and technologies, while supportive-care experts focus on quality of life.

Today, many children treated for leukemia or lymphoma go on to attend school, play sports, build careers and raise families.

“Each child’s prognosis is different, but improvements in chemotherapy, precision medicine, immunotherapy, transplantation and supportive care continue to create more reasons for families to hope—and more opportunities for children to reclaim their futures,” Dr. Daghistani says.

Click here for more information about leukemia and other blood cancers, and the treatments and specialists available at Baptist Health Herbert Wertheim Cancer Institute.