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Marks LJ, Oberg JA, Pendrick D, et al. Precision Medicine in Children and Young Adults with Hematologic Malignancies and Blood Disorders: The Columbia University Experience. Front Pediatr. 2017;5:265doi: 10.3389/fped.2017.00265.
Marks, L. J., Oberg, J. A., Pendrick, D., Sireci, A. N., Glasser, C., Coval, C., Zylber, R. J., Chung, W. K., Pang, J., Turk, A. T., Hsiao, S. J., Mansukhani, M. M., Glade Bender, J. L., Kung, A. L., & Sulis, M. L. (2017). Precision Medicine in Children and Young Adults with Hematologic Malignancies and Blood Disorders: The Columbia University Experience. Frontiers in pediatrics, 5265. https://doi.org/10.3389/fped.2017.00265
Marks, Lianna J, et al. "Precision Medicine in Children and Young Adults with Hematologic Malignancies and Blood Disorders: The Columbia University Experience." Frontiers in pediatrics vol. 5 (2017): 265. doi: https://doi.org/10.3389/fped.2017.00265
Marks LJ, Oberg JA, Pendrick D, Sireci AN, Glasser C, Coval C, Zylber RJ, Chung WK, Pang J, Turk AT, Hsiao SJ, Mansukhani MM, Glade Bender JL, Kung AL, Sulis ML. Precision Medicine in Children and Young Adults with Hematologic Malignancies and Blood Disorders: The Columbia University Experience. Front Pediatr. 2017 Dec 12;5:265. doi: 10.3389/fped.2017.00265. eCollection 2017. PMID: 29312904; PMCID: PMC5732960.
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Front Pediatr. 2017 Dec 12;5:265. doi: 10.3389/fped.2017.00265. eCollection 2017.

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

Frontiers in pediatrics

Lianna J Marks, Jennifer A Oberg, Danielle Pendrick, Anthony N Sireci, Chana Glasser, Carrie Coval, Rebecca J Zylber, Wendy K Chung, Jiuhong Pang, Andrew T Turk, Susan J Hsiao, Mahesh M Mansukhani, Julia L Glade Bender, Andrew L Kung, Maria Luisa Sulis

Affiliations

  1. Department of Pediatric Oncology, Memorial Sloan Kettering Cancer Center, New York, NY, United States.
  2. Division of Pediatric Hematology, Oncology and Stem Cell Transplant, Columbia University Medical Center, New York, NY, United States.
  3. Department of Pathology and Cell Biology, Columbia University Medical Center, New York, NY, United States.
  4. Department of Pediatric Hematology/Oncology, NYU Winthrop University Medical Center, Mineola, NY, United States.
  5. Department of Pediatrics, Columbia University Medical Center, New York, NY, United States.
  6. Herbert Irving Comprehensive Cancer Center, Columbia University Medical Center, New York, NY, United States.

PMID: 29312904 PMCID: PMC5732960 DOI: 10.3389/fped.2017.00265

Abstract

BACKGROUND: 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.

METHODS: 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.

RESULTS: Sixty-nine samples from 56 patients with high-risk hematologic malignancies and blood disorders were sequenced. Patients carried diagnoses of myeloid malignancy (

CONCLUSION: Incorporating whole exome and transcriptome profiling of tumor and normal tissue into clinical practice is feasible, and the value that comprehensive testing provides extends beyond the ability to target-specific mutations.

Keywords: genomic; hematologic malignancies; next-generation sequencing; pediatric leukemia; targeted therapy

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