Bone marrow transplantation does not improve outcome in babies with high-risk acute lymphoblastic leukemia

St. Jude Children's Research Hospital (ALSAC)
Friday, 31 May 2002

St. Jude research proves physicians should rely on chemotherapy

Babies treated for acute lymphoblastic leukemia (ALL) whose leukemic cells have a specific chromosomal abnormality do not benefit from bone marrow transplantation, a new study indicates. In fact, these babies have worse outcomes and increased long-term complications from the procedure.

The research indicates that physicians treating these patients should rely on improved chemotherapy.

These results were published in the June issue of the journal Lancet by an international team led by Ching-Hon Pui, M.D., and William Evans, Pharm.D., at St. Jude Children's Research Hospital. The study casts new light on treatment for childhood ALL patients with chromosomal 11q23 abnormalities in their leukemic cells.

This type of leukemia is most common in babies less than one year old.

Study data was derived from childhood ALL patients treated in Western Europe and North America over a 10-year period. About 2 percent of ALL patients, or about 60 patients annually, are diagnosed with this type of leukemia in the U.S.

"This study shows for the first time that a baby with this type of leukemia does not benefit from transplant," Pui said. "At St. Jude, we've already discouraged transplantation for these patients."

Physicians should rely on intensive chemotherapy to treat these patients, not transplantation. These findings should reverse treatment approach at many medical centers. According to Pui and Evans, published results citing successful transplantation involved very small number of patients and were biased because most investigators generally do not report treatment failures.

"Recent chemotherapy results seem to improve outcome for these patients," Pui said, noting that several months, and at times a year, lapsed between diagnosis and transplantation. Some of these patients might have already been cured by pre-transplant chemotherapy by the time they received their bone marrow transplant. "Transplantation yields a worse outcome overall for these patients. Even in the best circumstances, transplantation with a matched-sibling donor, the outcome did not improve."

Additionally, all of the patients who received transplantation will suffer from long-term side effects.

St. Jude Children's Research Hospital, in Memphis, Tennessee, was founded by the late entertainer Danny Thomas. The hospital is an internationally recognized biomedical research center dedicated to finding cures for childhood catastrophic diseases. The hospital's work is supported through funds raised by ALSAC, St. Jude's fundraising arm. ALSAC covers all costs not covered by insurance for medical treatment rendered at St. Jude Children's Research Hospital. Families without insurance are never asked to pay.

For more information, or to contact St. Jude Children's Research Hospital (ALSAC), see their website at: www.stjude.org