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ASH 2014: Pediatric Leukemia Treatment Regimens Lead to Improved Outcomes in Adolescents, Young Adults

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Key Points

  • In this large prospective trial, adolescent and young adult patients with ALL received the standard pediatric ALL treatment protocol, including four intensive courses of chemotherapy.
  • Two-year overall survival was 79%, and event-free survival was 66%, a significant improvement over historical controls.
  • Patients with a Philadelphia chromosome–like gene-expression signature indicating aggressive disease experienced markedly worse outcomes.

Results from a large prospective study suggest that children and young adults with acute lymphocytic leukemia (ALL) may respond better to a chemotherapy regimen pioneered in pediatric patients. The findings were presented at the 56th American Society of Hematology (ASH) Annual Meeting and Exposition (Abstract 796).

Children and young adults ALL typically have better treatment outcomes than adults, due in part to differences in the disease, but also to higher-intensity pediatric treatment protocols. While research indicates that more intensive pediatric regimens may be more effective, adolescents and young adults aged 16 to 39 most often receive adult regimens.

Study Details

To determine whether adolescent and young adult ALL patients would have better outcomes with pediatric regimens, 296 adolescent and young adult ALL patients participating in a large, prospective U.S. Intergroup clinical trial received the standard pediatric ALL treatment protocol, including four intensive courses of chemotherapy. After 2 years of follow-up, the overall survival rate was 79%, and 66% of patients had maintained event-free survival.

Factors linked to poor outcomes included high initial white blood cell counts as well as presence of minimal residual disease following completion of the first month of therapy. The nearly 30% of patients enrolled in the trial with a Philadelphia chromosome–like gene-expression signature indicating aggressive disease experienced markedly worse outcomes (2-year event-free survival of 52% vs 81% among those without the genetic alteration). Five patients died due to treatment-related reasons during therapy.  

“Our results illustrate a clear opportunity to improve care by treating adolescents and young adults with an intensive pediatric regimen, which resulted in low treatment-related mortality and promising disease-free and overall survival,” said lead study author Wendy Stock, MD, of the University of Chicago Medical Center. “It is important to note that these results reflect a significant improvement from historical control studies, in which event-free survival was only 34%. With these new insights, we can now focus additional research on evaluating novel treatment combinations to reduce minimal residual disease in these patients to further improve their duration of response and long-term survival.”

Dr. Stock reported membership on the Board of Directors or advisory committees for Sigma-Tau and research funding from Sigma-Tau. For full disclosures of the study authors, view the study abstract.

The content in this post has not been reviewed by the American Society of Clinical Oncology, Inc. (ASCO®) and does not necessarily reflect the ideas and opinions of ASCO®.


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