Precision Medicine in Children and Young Adults with Hematologic Malignancies and Blood Disorders: The Columbia University Experience

Frontiers in Pediatrics
Lianna J MarksMaria Luisa Sulis

Abstract

The advent of comprehensive genomic profiling has markedly advanced the understanding of the biology of pediatric hematological malignancies, however, its application to clinical care is still unclear. We present our experience integrating genomic data into the clinical management of children with high-risk hematologic malignancies and blood disorders and describe the broad impact that genomic profiling has in multiple aspects of patient care. The Precision in Pediatric Sequencing Program at Columbia University Medical Center instituted prospective clinical next-generation sequencing (NGS) for high-risk malignancies and blood disorders. Testing included cancer whole exome sequencing (WES) of matched tumor-normal samples or targeted sequencing of 467 cancer-associated genes, when sample adequacy was a concern, and tumor transcriptome (RNA-seq). A multidisciplinary molecular tumor board conducted interpretation of results and final tiered reports were transmitted to the electronic medical record according to patient preferences. Sixty-nine samples from 56 patients with high-risk hematologic malignancies and blood disorders were sequenced. Patients carried diagnoses of myeloid malignancy (n = 25), lymphoid malignancy (n = 25), or ...Continue Reading

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Citations

Jul 28, 2018·Blood·Yana Pikman, Kimberly Stegmaier
Apr 26, 2020·International Journal of Laboratory Hematology·Alba MoraCarol Moreno
May 15, 2019·Annual Review of Genomics and Human Genetics·Sharon E Plon, Philip J Lupo
Jan 1, 2019·JCO Precision Oncology·Kelly M BaileyRajen J Mody

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Methods Mentioned

BETA
exome sequencing
PIPseq
RNA-seq
PCR
biopsy

Software Mentioned

TARGET
cBioPortal for Genomics
PIPseq

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