Pilot Study Doubles Leukemia Cure Rates in Infants

A pilot study that has more than doubled cure rates for an often-fatal form of leukemia is the basis of an ongoing national study.

Coordinated by researchers at Texas Children's Cancer Center at Baylor College of Medicine and Texas Children's Hospital, the pilot study demonstrated cure rates of nearly 50 percent for infants with Acute Lymphoid Leukemia (ALL). ALL is the most common form of pediatric cancer and one of the most treatable in older children. In infants, however, the disease is often fatal.

"The cure rate for infants with ALL has been less than 20 percent," says Dr. ZoAnn Dreyer, principal investigator and director of the TCCC's Long-Term Survivor Program.

Traditionally, infants received two-and-a-half to three years of chemotherapy and, oftentimes, radiation to the brain. Because of the very poor survivor rates, some infants also received bone marrow transplantation.

"Despite the many weeks of traditional treatment, cure rates remained poor with nearly 80 percent of the infants relapsing within a year of diagnosis," Dr. Dreyer says. "The pilot study involved an intense but shortened 46-week chemotherapy program."

High doses of intensive chemotherapy were given at close intervals during the first 16 weeks of treatment and then reduced, Dr. Dreyer says. Side effects were minimal.

The national study, also headed by Dr. Dreyer, will attempt to replicate or improve upon the rates experienced by TCCC researchers. The study will be done by medical institutions that are members of the Pediatric Oncology Group, one of the two largest pediatric cancer collaborative groups in the U.S.

"Our hope is not to just improve survival rates but to eliminate the need for toxic radiation and bone marrow transplantation," she says. "We continue to monitor the patients in the initial study for late effects from their therapy. They appear to have few side effects, and it's exciting to watch them continue to do so well. Some of our patients are even entering kindergarten."

The pilot study followed 24 children from 1994 to 1997 through the TCCC Long-Term Survivor Clinic.

"The outcome of the pilot study is very encouraging and suggests that neither radiation therapy or bone marrow transplantation are required for a cure," Dr. Dreyer says.

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©2006 Texas Medical Center

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