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

Submission information
Submission Number: 36
Submission ID: 148216
Submission UUID: d3ee80c7-a1f4-4188-ab5e-b56ba0c20405

Created: Wed, 08/06/2025 - 11:51
Completed: Wed, 08/06/2025 - 12:07
Changed: Wed, 08/06/2025 - 12:07

Remote IP address: 10.208.24.36
Submitted by: Anonymous
Language: English

Is draft: No
Abstract Title: Real-World Molecularly Targeted Treatment Registry (MaTTeR): a Pilot Study to Enrich CCDI Data Utilizing Directed EMR Extraction
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:
Authors:
  1. First Name: Suzanne
    Middle Initial: J
    Last Name: Forrest
    Degree(s): M.D.
    Organization: Dana-Farber/Boston Children's Cancer and Blood Disorder Center
  2. First Name: Kee Kat (Aaron)
    Last Name: Yeo
    Degree(s): M.D.
    Organization: Dana-Farber/Boston Children's Cancer and Blood Disorder Center
  3. First Name: Evelina
    Last Name: Ceca
    Organization: Dana-Farber/Boston Children's Cancer and Blood Disorder Center
  4. First Name: Maeve
    Last Name: Smart
    Organization: Dana-Farber/Boston Children's Cancer and Blood Disorder Center
  5. First Name: Olivia
    Last Name: Puopolo
    Organization: Dana-Farber/Boston Children's Cancer and Blood Disorder Center
  6. First Name: Sabrina
    Last Name: Testa
    Organization: Dana-Farber/Boston Children's Cancer and Blood Disorder Center
  7. First Name: Elizabeth
    Middle Initial: F
    Last Name: Cohen-Sacks
    Organization: Dana-Farber Cancer Institute
  8. First Name: Ledia
    Middle Initial: A
    Last Name: Gebremedhin
    Organization: Dan-Farber Cancer Institute
  9. First Name: Katherine
    Middle Initial: A
    Last Name: Janeway
    Degree(s): M.D.
    Organization: Dana-Farber/Boston Children's Cancer and Blood Disorder Center
  10. First Name: Yana
    Last Name: Pikman
    Degree(s): M.D.
    Organization: Dana-Farber/Boston Children's Cancer and Blood Disorder Center
Presenting Author: Suzanne J. Forrest
Institution: Dana-Farber/Boston Children's Cancer and Blood Disorder Center
Email Address: Suzanne_forrest@dfci.harvard.edu