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Multicenter Feasibility Study of Tumor Molecular Profiling to Inform Therapeutic Decisions in Advanced Pediatric Solid Tumors: The Individualized Cancer Therapy (iCat) Study
Open accessAbstract
IMPORTANCE: Pediatric cancers represent a unique case with respect to cancer genomics and precision medicine, as the mutation frequency is low, and targeted therapies are less available. Consequently, it is unknown whether clinical sequencing can be of benefit. OBJECTIVE: To assess the feasibility of identifying actionable alterations and making individualized cancer therapy (iCat) recommendations in pediatric patients with extracranial solid tumors. DESIGN, SETTING, AND PARTICIPANTS: Clinical sequencing study at 4 academic medical centers enrolling patients between September 5, 2012, and November 19, 2013, with 1 year of clinical follow-up. Participants were 30 years or younger with high-risk, recurrent, or refractory extracranial solid tumors. The data analysis was performed October 28, 2014. INTERVENTIONS: Tumor profiling performed on archived clinically acquired specimens consisted of mutation detection by a Sequenom assay or targeted next-generation sequencing and copy number assessment by array comparative genomic hybridization. Results were reviewed by a multidisciplinary expert panel, and iCat recommendations were made if an actionable alteration was present, and an appropriate drug was available. MAIN OUTCOMES AND MEASURES: Feasibility was assessed using a 2-stage design based on the proportion of patients with recommendations. RESULTS: Of 100 participants (60 male; median [range] age, 13.4 [0.8-29.8] years), profiling was technically successful in 89 (89% [95% CI, 83%-95%]). Median (range) follow-up was 6.8 (2.0-23.6) months. Overall, 31 (31% [95% CI, 23%-41%]) patients received an iCat recommendation and 3 received matched therapy. The most common actionable alterations leading to an iCat recommendation were cancer-associated signaling pathway gene mutations (n = 10) and copy number alterations in MYC/MYCN (n = 6) and cell cycle genes (n = 11). Additional alterations with implications for clinical care but not resulting in iCat recommendations were identified, including mutations indicating the possible presence of a cancer predisposition syndrome and translocations suggesting a change in diagnosis. In total, 43 (43% [95% CI, 33%-53%]) participants had results with potential clinical significance. CONCLUSIONS AND RELEVANCE: A multi-institution clinical genomics study in pediatric oncology is feasible and a substantial proportion of relapsed or refractory pediatric solid tumors have actionable alterations. TRIAL REGISTRATION: clinicaltrials.gov Identifier: NCT01853345.
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“Compared with adult tumors, pediatric solid tumors have fewer hotspot somatic mutations that are amenable to traditional liquid biopsy methodology.13”
From Clinical Liquid Biopsy Testing for Detection of Actionable Genomic Alterations in Children and Young Adults With Advanced Solid Tumors on the GAIN Study (2026) · cites this paper as [13]
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Metadata
- Authors
- Harris, M. H., DuBois, S. G., Glade Bender, J. L., Kim, A., Crompton, B. D., Parker, E., Dumont, I. P., Hong, A. L., Guo, D., Church, A., Stegmaier, K., Roberts, C. W. M., Shusterman, S., London, W. B., MacConaill, L. E., Lindeman, N. I., Diller, L., Rodriguez-Galindo, C., & Janeway, K. A.
- Affiliations
- Boston Children's Hospital (US)
- University of California, San Francisco (US)
- Division of Pediatric Hematology/Oncology/Stem Cell Transplantation, NewYork–Presbyterian Morgan Stanley Children’s Hospital, New York
- Children's National (US)
- Dana-Farber/Boston Children's Cancer and Blood Disorders Center (US)
- Harvard University (US)
- Brigham and Women's Hospital (US)
- Dana-Farber/Boston Children’s Cancer and Blood Disorders Center, Boston, Massachusetts
- Corresponding author
- Janeway, Katherine A.
- ORCID
- Venue
- JAMA Oncology
- Publisher
- American Medical Association (AMA)
- Published
- 2016-05-01
- Volume
- 2
- Issue
- 5
- Pages
- 608
- ISSN
- 2374-2437 (ISSN-L)
- OpenAlex
- W2269136289
- Cited by
- 206
- References
- 17
- Citations by year
- 2026: 10 · 2025: 8 · 2024: 17 · 2023: 17 · 2022: 20 · 2021: 25 · 2020: 10 · 2019: 30 · 2018: 26 · 2017: 28 · 2016: 14
- FWCI
- 13.60
- Type
- journal article
- Language
- en
- Open access
- bronze
- doi.org
- Locations
- JAMA Oncology (publishedVersion)
- PubMed (publishedVersion)
- Funders
- National Cancer Institute (P30CA013696)
- Hyundai Hope On Wheels
- Topics
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Cancer Research › Cancer Genomics and Diagnostics
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Genetics › Genomics and Rare Diseases
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › Chromatin Remodeling and Cancer
- Keywords
- pediatric solid tumors, tumor molecular profiling, actionable alterations, precision oncology, copy number alterations, cell cycle genes, cancer predisposition syndromes, translocation, targeted therapy, extracranial solid tumors, array comparative genomic hybridization, targeted next-generation sequencing
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