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Acute lymphoblastic leukemia (ALL) is rare in infants. About 90 cases occur in children 1 or younger each year in the United States – about 3 percent of childhood ALL cases. Even the largest pediatric cancer centers may only see a few cases a year.
Diagnosis of ALL involves a physical exam, medical history, blood tests, bone marrow aspiration and biopsy, and lumbar puncture. Tests will be run to determine the specific kind of ALL and identify chromosome changes, genes, proteins, and other factors involved in the leukemia. This information will have an impact on treatment approaches and predicted treatment outcome (prognosis).
Infant ALL is biologically different from ALL in older children. It is typically very aggressive. Most infants – as many as 80% -- have a rearrangement in a gene called MLL (mixed lineage leukemia). Another name for MLL is KMT2A.
The main treatment for ALL in infants is chemotherapy with many different medicines. Treatment has different phases. It typically lasts about 2 years. Chemotherapy medicines may include cyclophosphamide, cytarabine, daunorubicin, dexamethasone, Erwinia asparaginase, etoposide, methotrexate, leucovorin, mercaptopurine, mitoxantrone, pegaspargase, prednisone, thioguanine, and vincristine.
Some patients may receive a hematopoietic cell transplant (also known as bone marrow transplant or stem cell transplant).
Physicians take a risk-stratified approach to treating infant ALL. In general, this approach means patients with a higher risk of relapse will receive more intensive treatment than those with lower risk.
When assigning a risk category, physicians consider:
The survival rate for infants with ALL is less than 50%.
Infants with the MLL (KMT2A) rearrangement have a high risk of relapse. About two-thirds of infants will relapse within a year of diagnosis. At present, there are no treatment protocols for relapsed ALL in children younger than 1.
Side effects vary among patients. The care team will work with families to try to prevent and/or lessen side effects as much as possible. The team will monitor side effects closely so that they can be treated.
Infants are especially vulnerable to:
Scientists and physicians continue to work closely in international cooperative groups to improve treatment options.
Clinical trials are underway to try new therapies designed to improve cure rates.
Reviewed: December 2018