Childhood Cancer Data Initiative Annual Symposium (Abstract Registration): Submission #36

serial: '36'
sid: '148216'
uuid: d3ee80c7-a1f4-4188-ab5e-b56ba0c20405
uri: /nci/ccdisymposium/abstract
created: '1754495482'
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langcode: en
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data:
  authors_:
    - add_author_degree: M.D.
      add_author_first_name: Suzanne
      add_author_last_name: Forrest
      add_author_middle: J
      add_author_organization: "Dana-Farber/Boston Children's Cancer and Blood Disorder Center"
    - add_author_degree: M.D.
      add_author_first_name: 'Kee Kat (Aaron)'
      add_author_last_name: Yeo
      add_author_middle: ''
      add_author_organization: "Dana-Farber/Boston Children's Cancer and Blood Disorder Center"
    - add_author_degree: ''
      add_author_first_name: Evelina
      add_author_last_name: Ceca
      add_author_middle: ''
      add_author_organization: "Dana-Farber/Boston Children's Cancer and Blood Disorder Center"
    - add_author_degree: ''
      add_author_first_name: Maeve
      add_author_last_name: Smart
      add_author_middle: ''
      add_author_organization: "Dana-Farber/Boston Children's Cancer and Blood Disorder Center"
    - add_author_degree: ''
      add_author_first_name: Olivia
      add_author_last_name: Puopolo
      add_author_middle: ''
      add_author_organization: "Dana-Farber/Boston Children's Cancer and Blood Disorder Center"
    - add_author_degree: ''
      add_author_first_name: Sabrina
      add_author_last_name: Testa
      add_author_middle: ''
      add_author_organization: "Dana-Farber/Boston Children's Cancer and Blood Disorder Center"
    - add_author_degree: ''
      add_author_first_name: Elizabeth
      add_author_last_name: Cohen-Sacks
      add_author_middle: F
      add_author_organization: 'Dana-Farber Cancer Institute'
    - add_author_degree: ''
      add_author_first_name: Ledia
      add_author_last_name: Gebremedhin
      add_author_middle: A
      add_author_organization: 'Dan-Farber Cancer Institute'
    - add_author_degree: M.D.
      add_author_first_name: Katherine
      add_author_last_name: Janeway
      add_author_middle: A
      add_author_organization: "Dana-Farber/Boston Children's Cancer and Blood Disorder Center"
    - add_author_degree: M.D.
      add_author_first_name: Yana
      add_author_last_name: Pikman
      add_author_middle: ''
      add_author_organization: "Dana-Farber/Boston Children's Cancer and Blood Disorder Center"
  abstract: 'The incorporation of genomic profiling into the care of pediatric, adolescent and young adult (AYA) cancer patients has resulted in identification of targetable alterations and use of molecularly targeted therapy (MTT), necessitating collection and reporting of outcomes of real-world use of MTT. Using genomic data contributed to the CCDI and our institutional cohorts, we identified a cohort of patients who received MTT outside of clinical trials. We included patients with cancer seen at the Dana-Farber/Boston Children’s, who enrolled in a clinical sequencing/banking study and had next-generation sequencing (NGS) performed (OncoPanel, Rapid Heme and Fusion Panels). Sequencing results were used to identify patients with potentially targetable alterations in a list of 66 genes. Patients who received MTT were identified, and data including dosing, toxicity and response were extracted from the medical record. Between 2013 and 2024, 2163 patients had tumor profiling via targeted NGS. 35% (760/2163) had an actionable alteration detected for which MTT could have been administered. 114 patients received at least one regimen that included an MTT (72 patients with brain tumors, 28 with solid tumors, 14 with hematologic malignancies). Alterations in BRAF, NF1, ALK, FLT3, and PIK3CA led to the most MTT use. Of the 114 patients who received MTT, 79% (90/114) received at least one targeted therapy regimen outside of a clinical trial. This project will create a registry of MTT use within the CCDI that can be expanded to include additional patients and be a resource for MTT use for the pediatric and AYA cancer community.'
  abstract_title_: 'Real-World Molecularly Targeted Treatment Registry (MaTTeR): a Pilot Study to Enrich CCDI Data Utilizing Directed EMR Extraction'
  email_address_: Suzanne_forrest@dfci.harvard.edu
  institution_: "Dana-Farber/Boston Children's Cancer and Blood Disorder Center"
  presenting_author_: 'Suzanne J. Forrest'