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Implementation of a Pediatric Oncology Precision Medicine Clinic to Personalize Approaches for Diagnosing and
Madeline Keane1, Natalia Wojciechowska2, Lindsay Zumwalt1
1Anne Burnett Marion School of Medicine at Texas Christian University, Fort Worth, TX 76104, USA.
Background:
Precision medicine is an emerging approach for treating pediatric cancer due to its ability to target tumor-specific genetic drivers rather than provide broad and aggressive treatments. The study aimed to outline the establishment and impact of a Precision Medicine Clinic (PMC) in the setting of pediatric oncology, with the objective of offering targeted treatment options within the institution and creating a scalable model for adoption by other healthcare systems to achieve a wider impact.
Methods:
Recognizing this need for an individualized approach to treating patients, Cook Children's Medical Center (CCMC) established a multidisciplinary molecular tumor board in 2019, followed by the launch of an official PMC in 2021. Before this, there was no dedicated place to discuss and evaluate genetic sequencing results.
Results:
In 2022 and 2023, the PMC discussed 69 patients with a wide variety of oncologic diagnoses. Through the clinic's efforts, 133 genetic variants across 101 genes have been identified, spanning oncogenic pathways related to cell cycling, DNA processing, and cell signaling. Of the sequenced patients, four have received targeted therapy according to recommendations from the PMC.
Conclusion:
While the PMC continues to evaluate patients and their long-term outcomes, the continually growing PMC at CCMC represents the beginning of the advancement of treating pediatric oncology patients through the interpretation of genetic sequencing results, making actionable targeted treatment recommendations, and continuing to follow the patient's course of care over time. This additionally provides a framework for starting a PMC that can be adapted for specific clinical needs and implemented broadly.
Insights
A Precision Medicine Clinic (PMC) advances pediatric cancer care by targeting genetic drivers. This clinic successfully identified numerous genetic variants and facilitated targeted therapies for young patients.
Area of Science:
- Pediatric Oncology
- Genomics
- Precision Medicine
Background:
- Precision medicine offers targeted treatment for pediatric cancer by focusing on specific genetic mutations.
- Traditional treatments can be broad and aggressive, highlighting the need for individualized approaches.
Purpose of the Study:
- To establish and evaluate the impact of a Precision Medicine Clinic (PMC) in pediatric oncology.
- To create a scalable model for implementing PMCs in other healthcare systems.
Main Methods:
- Cook Children's Medical Center (CCMC) established a multidisciplinary molecular tumor board in 2019 and a PMC in 2021.
- The PMC was created to discuss and evaluate genetic sequencing results for pediatric cancer patients.
Main Results:
- The PMC evaluated 69 pediatric patients with diverse oncologic diagnoses between 2022 and 2023.
- 133 genetic variants across 101 genes were identified, impacting cell cycling, DNA processing, and cell signaling pathways.
- Four patients received targeted therapies based on PMC recommendations.
Conclusions:
- The CCMC PMC demonstrates progress in pediatric cancer treatment through genetic sequencing interpretation and targeted therapy recommendations.
- The clinic provides a framework for establishing similar PMCs adaptable to specific clinical needs and for broad implementation.
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