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Trial Aims to Match Pediatric Patients With More Precise Leukemia Therapy

Brandon Scalea
Published: Thursday, Dec 20, 2018

Yana Pikman, MD

Yana Pikman, MD

An advanced understanding of the biology of hematologic malignancies has significantly improved survival outcomes. However, in pediatric acute leukemias, said Yana Pikman, MD, the integration of real-time genomic sequencing data into clinical practice has fallen behind.

, Pikman further discussed the clinical implications of using this approach to match targeted therapy to pediatric patients with leukemia.

OncLive: Please provide some background to this study.

Pikman: Pediatric acute leukemia is the number 1 cause of pediatric cancer. In 2018, it still accounts for a significant number of cancer-related deaths; in fact, it is the number 2 cause of pediatric cancer deaths overall. We need to find a way to provide better treatment for these patients.

The study we are conducting is evaluating the feasibility of identifying targetable mutations in pediatric patients with relapsed or high-risk leukemia. [Our strategy is to] match these targets with available therapies and create a precision medicine approach in this space.

What is the design of the trial?

At the time of enrollment, patients have a sample of their leukemia sequenced; those data are then reviewed by a panel comprised of pediatric oncologists, experts in therapeutics, and pathologists. This [process] is to determine if we are able to identify any targetable mutations. If a targetable recommendation is made, we pass that information along. [It is important to remember that] this is not a therapeutic study; it is a feasibility study. Therefore, we are not treating any patient, but we hope this research paves the way for a therapeutic study.

We are discovering several novel genomic alterations that can be of interest and we are now evaluating the significance of these [alterations]. Ongoing work is happening to see if we can tailor our approach to these [alterations].


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View Conference Coverage
Online CME Activities
TitleExpiration DateCME Credits
Community Practice Connections™: 22nd Annual International Congress on Hematologic Malignancies®: Focus on Leukemias, Lymphomas and MyelomaMay 30, 20192.0
Online Medical Crossfire®: 5th Annual Miami Lung Cancer ConferenceMay 30, 20196.5
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