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Feasibility of implementing molecular-guided therapy for the treatment of patients with relapsed or refractory
Giselle L Saulnier Sholler1,2, Jeffrey P Bond3, Genevieve Bergendahl1
1Helen DeVos Children's Hospital, Grand Rapids, Michigan.
Abstract:
The primary objective of the study was to evaluate the feasibility and safety of a process which would utilize genome-wide expression data from tumor biopsies to support individualized treatment decisions. Current treatment options for recurrent neuroblastoma are limited and ineffective, with a survival rate of <10%. Molecular profiling may provide data which will enable the practitioner to select the most appropriate therapeutic option for individual patients, thus improving outcomes. Sixteen patients with neuroblastoma were enrolled of which fourteen were eligible for this study. Feasibility was defined as completion of tumor biopsy, pathological evaluation, RNA quality control, gene expression profiling, bioinformatics analysis, generation of a drug prediction report, molecular tumor board yielding a treatment plan, independent medical monitor review, and treatment initiation within a 21 day period. All eligible biopsies passed histopathology and RNA quality control. Expression profiling by microarray and RNA sequencing were mutually validated. The average time from biopsy to report generation was 5.9 days and from biopsy to initiation of treatment was 12.4 days. No serious adverse events were observed and all adverse events were expected. Clinical benefit was seen in 64% of patients as stabilization of disease for at least one cycle of therapy or partial response. The overall response rate was 7% and the progression free survival was 59 days. This study demonstrates the feasibility and safety of performing real-time genomic profiling to guide treatment decision making for pediatric neuroblastoma patients.
Insights
This study shows that real-time genomic profiling of neuroblastoma tumors is feasible and safe. This approach helps guide personalized treatment decisions, potentially improving outcomes for pediatric patients.
Area of Science:
- Oncology
- Genomics
- Bioinformatics
Background:
- Recurrent neuroblastoma has limited treatment options and a poor survival rate (<10%).
- Molecular profiling can potentially identify optimal therapies for individual patients.
Purpose of the Study:
- To evaluate the feasibility and safety of using genome-wide expression data from tumor biopsies for individualized treatment decisions in neuroblastoma.
- To assess the turnaround time for genomic profiling and treatment plan generation.
Main Methods:
- Sixteen neuroblastoma patients were enrolled; fourteen were eligible.
- Feasibility included tumor biopsy, RNA quality control, gene expression profiling (microarray and RNA sequencing), bioinformatics analysis, and molecular tumor board review.
- Treatment initiation within 21 days of biopsy was a key feasibility metric.
Main Results:
- All eligible biopsies passed quality control, with validated results from microarray and RNA sequencing.
- The average time from biopsy to report generation was 5.9 days, and to treatment initiation was 12.4 days.
- Clinical benefit was observed in 64% of patients; overall response rate was 7%, with a 59-day progression-free survival.
Conclusions:
- Real-time genomic profiling is a feasible and safe method for guiding treatment decisions in pediatric neuroblastoma.
- This approach demonstrated timely generation of actionable data and showed clinical benefit in a majority of patients.
