Saturday, June 22, 2024

Revolutionizing Pediatric Oncology with DNA-Informed Prescribing: A New Multicenter Trial

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The integration of pharmacogenomics (PGx) into pediatric oncology represents a promising leap forward in personalized medicine. Despite the availability of international guidelines, its practical application in this field remains limited. A new national prospective, multicenter, randomized controlled trial aims to address this gap by assessing the impact of pre-emptive PGx testing on adverse drug reactions (ADRs) in children diagnosed with cancer or undergoing hematopoietic stem cell transplants.

Study Methodology

The Minimising Adverse Drug Reactions and Verifying Economic Legitimacy-Pharmacogenomics Implementation in Children trial will meticulously collect ADR data using surveys filled out by patients and their parents. These surveys will employ the National Cancer Institute Pediatric Patient Reported Common Terminology Criteria for Adverse Events (Ped-PRO-CTCAE) at weeks 1, 6, and 12. Pharmacists will employ semi-structured interviews and tools like the CTCAE and Liverpool Causality Assessment Tool to assess the causality and severity of ADRs.

The primary focus is to observe a reduction in ADRs among patients with actionable PGx variants, defined as any CTCAE grade 2 and above for non-hematological toxicities, and any CTCAE grade 3 and above for hematological toxicities. This initiative is expected to yield significant insights into the practical benefits of PGx in pediatric oncology.

Cost-Effectiveness Analysis

A secondary objective of the trial is to evaluate the cost-effectiveness of pre-emptive PGx testing compared to standard care. This will be achieved by analyzing hospital inpatient and outpatient data alongside the validated Childhood Health Utility 9D Instrument. The trial will include a sample size of 440 patients, with 220 patients in each arm, ensuring an 80% power to detect a 24% relative risk reduction in the primary endpoint of ADRs, allowing for a 10% dropout rate.

Key Inferences for Stakeholders

Key inferences from the trial include:

  • Pre-emptive PGx testing can significantly reduce ADR incidence in pediatric oncology patients.
  • Integrating PGx into standard care may enhance patient safety and treatment efficacy.
  • Economic analyses will provide crucial data on the cost-benefit ratio of implementing PGx on a wide scale.

The trial’s ethical approval has been secured from the Royal Children’s Hospital Ethics Committee, and each participating center nationally has also conducted an assessment of the protocol and governance submission. This rigor ensures that the study adheres to the highest ethical standards, protecting patient well-being while advancing medical knowledge.

The trial’s outcomes could pave the way for broader implementation of PGx in pediatric oncology, potentially setting new standards in personalized medicine. Such advancements underscore the importance of integrating genetic insights into clinical practice to optimize therapeutic outcomes and minimize adverse effects.

Original Article: BMJ Open. 2024 May 16;14(5):e085115. doi: 10.1136/bmjopen-2024-085115.

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