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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Pediatric solid tumors have fewer hotspot somatic mutations suitable for traditional liquid biopsy methods than adult tumors.
“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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A method and server for predicting damaging missense mutations.
Adzhubei, I. A., Schmidt, S., Peshkin, L., Ramensky, V. E., Gerasimova, A., Bork, P., Kondrashov, A. S., & Sunyaev, S. R. (2010) · Nature Methods · doi:10.1038/nmeth0410-248 · Cited by 13,194 · see citing works
<i>EGFR</i> Mutations in Lung Cancer: Correlation with Clinical Response to Gefitinib Therapy
Paez, J. G., Jänne, P. A., Lee, J. C., Tracy, S., Greulich, H., Gabriel, S., Herman, P., Kaye, F. J., Lindeman, N., Boggon, T. J., et al. (2004) · Science · doi:10.1126/science.1099314 · Cited by 9,368 · see citing works
Self-citationVogelstein, B., Papadopoulos, N., Velculescu, V. E., Zhou, S., Diaz, L. A., & Kinzler, K. W. (2013) · Science · doi:10.1126/science.1235122 · Cited by 7,863 · see citing works
Kumar, P., Henikoff, S., & Ng, P. C. (2009) · Nature Protocols · doi:10.1038/nprot.2009.86 · Cited by 6,521 · see citing works
Trastuzumab plus Adjuvant Chemotherapy for Operable HER2-Positive Breast Cancer
Romond, E. H., Perez, E. A., Bryant, J., Suman, V. J., Geyer, C. E., Davidson, N. E., Tan-Chiu, E., Martino, S., Paik, S., Kaufman, P. A., et al. (2005) · New England Journal of Medicine · doi:10.1056/nejmoa052122 · Cited by 5,355 · see citing works
Discovery and saturation analysis of cancer genes across 21 tumour types
Lawrence, M. S., Stojanov, P., Mermel, C. H., Robinson, J. T., Garraway, L. A., Golub, T. R., Meyerson, M., Gabriel, S. B., Lander, E. S., & Getz, G. (2014) · Nature · doi:10.1038/nature12912 · Cited by 3,096 · see citing works
The genetic landscape of high-risk neuroblastoma
Pugh, T. J., Morozova, O., Attiyeh, E. F., Asgharzadeh, S., Wei, J. S., Auclair, D., Carter, S. L., Cibulskis, K., Hanna, M., Kieżun, A., et al. (2013) · Nature Genetics · doi:10.1038/ng.2529 · Cited by 1,170 · see citing works
Self-citationShern, J. F., Chen, L., Chmielecki, J., Wei, J. S., Patidar, R., Rosenberg, M. W., Ambrogio, L., Auclair, D., Wang, J., Song, Y. K., et al. (2014) · Cancer Discovery · doi:10.1158/2159-8290.cd-13-0639 · Cited by 742 · see citing works
The Genomic Landscape of Pediatric Ewing Sarcoma
Crompton, B. D., Stewart, C., Taylor-Weiner, A. N., Alexe, G., Kurek, K. C., Calicchio, M. L., Kieżun, A., Carter, S. L., Shukla, S. A., Mehta, S. S., et al. (2014) · Cancer Discovery · doi:10.1158/2159-8290.cd-13-1037 · Cited by 573 · see citing works
Self-citationWagle, N., Berger, M. F., Davis, M. J., Blumenstiel, B. S., DeFelice, M. T., Pochanard, P., Ducar, M. D., Hummelen, P. V., MacConaill, L. E., Hahn, W. C., et al. (2011) · Cancer Discovery · doi:10.1158/2159-8290.cd-11-0184 · Cited by 526 · see citing works
Self-citationPerry, J. A., Kiezun, A., Tonzi, P., Van Allen, E. M., Carter, S. L., Baca, S. C., Cowley, G. S., Bhatt, A. S., Rheinbay, E., Pedamallu, C. S., et al. (2014) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.1419260111 · Cited by 481 · see citing works
Self-citationProfiling Critical Cancer Gene Mutations in Clinical Tumor Samples
MacConaill, L. E., Campbell, C. D., Kehoe, S. M., Bass, A. J., Hatton, C., Niu, L., Davis, M., Yao, K., Hanna, M., Mondal, C., et al. (2009) · PLoS ONE · doi:10.1371/journal.pone.0007887 · Cited by 434 · see citing works
Self-citationPRADA: pipeline for RNA sequencing data analysis
Torres‐García, W., Zheng, S., Sivachenko, A., Vegesna, R., Wang, Q., Yao, R., Berger, M. F., Weinstein, J. N., Getz, G. A., & Verhaak, R. G. W. (2014) · Bioinformatics · doi:10.1093/bioinformatics/btu169 · Cited by 154 · see citing works
Primary Pulmonary Myxoid Sarcoma With EWSR1-CREB1 Fusion
Thway, K., Nicholson, A. G., Lawson, K., de Castro, D. G., Rice, A. J., Balzer, B. L., Swansbury, J. G., Min, T., Thompson, L., Kwame, A.-P., et al. (2011) · The American Journal of Surgical Pathology · doi:10.1097/pas.0b013e318227e4d2 · Cited by 142 · see citing works
Prospective Enterprise-Level Molecular Genotyping of a Cohort of Cancer Patients
MacConaill, L. E., Garcia, E., Shivdasani, P., Ducar, M. D., Adusumilli, R., Breneiser, M., Byrne, M. N., Chung, L. P., Conneely, J., Crosby, L., et al. (2014) · Journal of Molecular Diagnostics · doi:10.1016/j.jmoldx.2014.06.004 · Cited by 87 · see citing works
Self-citationFalconieri, G., Bacchi, C. E., & Luzar, B. (2012) · American Journal of Dermatopathology · doi:10.1097/dad.0b013e3182473190 · Cited by 50 · see citing works
Hawryluk, M. J., Wang, K., Chmielecki, J., Ali, S. M., Palmer, G. A., Garcia, L., White, E. T., Yelensky, R., Stephens, P. J., Ross, J. S., et al. (2014) · Cancer Research · doi:10.1158/1538-7445.pedcan-a23 · Cited by 1 · see citing works
Flagged first, then by citations
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Institutional implementation of clinical tumor profiling on an unselected cancer population
Sholl, L. M., Do, K., Shivdasani, P., Cerami, E., Dubuc, A. M., Kuo, F. C., Garcia, E. P., Jia, Y., Davineni, P., Abo, R. P., et al. (2016) · JCI Insight · doi:10.1172/jci.insight.87062 · Cited by 450 · see citing works
Self-citationNext-Generation Sequencing in Oncology: Genetic Diagnosis, Risk Prediction and Cancer Classification
Kamps, R., Brandão, R. D., van den Bosch, B. J. C., Paulussen, A. D. C., Xanthoulea, S., Blok, M. J., & Romano, A. (2017) · International Journal of Molecular Sciences · doi:10.3390/ijms18020308 · Cited by 454 · see citing works
Wong, M., Mayoh, C., Lau, L. M. S., Khuong-Quang, D.-A., Pinese, M., Kumar, A., Barahona, P., Wilkie, E. E., Sullivan, P. A., Bowen-James, R., et al. (2020) · Nature Medicine · doi:10.1038/s41591-020-1072-4 · Cited by 397 · see citing works
van Tilburg, C. M., Pfaff, E., Pajtler, K. W., Langenberg, K. P. S., Fiesel, P., Jones, B. C., Balasubramanian, G. P., Stark, S., Johann, P. D., Blattner-Johnson, M., et al. (2021) · Cancer Discovery · doi:10.1158/2159-8290.cd-21-0094 · Cited by 264 · see citing works
Rusch, M., Nakitandwe, J., Shurtleff, S. A., Newman, S., Zhang, Z., Edmonson, M. N., Parker, M. D., Jiao, Y., Ma, X., Liu, Y., et al. (2018) · Nature Communications · doi:10.1038/s41467-018-06485-7 · Cited by 212 · see citing works
Hargrave, D. R., Bouffet, É., Tabori, U. Y., Broniscer, A., Cohen, K. J., Hansford, J. R., Geoerger, B., Hingorani, P., Dunkel, I. J., Russo, M. W., et al. (2019) · Clinical Cancer Research · doi:10.1158/1078-0432.ccr-19-2177 · Cited by 215 · see citing works
Oberg, J. A., Bender, J. G., Sulis, M. L., Pendrick, D., Sireci, A. N., Hsiao, S. J., Turk, A. T., Cruz, F. S. D., Hibshoosh, H. H., Remotti, H. E., et al. (2016) · Genome Medicine · doi:10.1186/s13073-016-0389-6 · Cited by 184 · see citing works
Berlanga, P., Pierron, G., Lacroix, L., Chicard, M., de Beaumais, T. A., Marchais, A., Harttrampf, A. C., Iddir, Y., Larive, A., Soriano, A., et al. (2022) · Cancer Discovery · doi:10.1158/2159-8290.cd-21-1136 · Cited by 174 · see citing works
Fiala, E. M., Jayakumaran, G., Mauguen, A., Kennedy, J. A., Bouvier, N., Kemel, Y. M., Fleischut, M. H., Maio, A., Salo‐Mullen, E. E., Sheehan, M. R., et al. (2021) · Nature Cancer · doi:10.1038/s43018-021-00172-1 · Cited by 172 · see citing works
Molecular characteristics and therapeutic vulnerabilities across paediatric solid tumours
Jones, D., Banito, A., Grünewald, T. G. P., Haber, M., Jäger, N., Kool, M., Milde, T., Molenaar, J. J., Nabbi, A., Pugh, T. J., et al. (2019) · Nature reviews. Cancer · doi:10.1038/s41568-019-0169-x · Cited by 155 · see citing works
Stewart, E., McEvoy, J., Wang, H., Chen, X., Honnell, V., Ocarz, M., Gordon, B., Dapper, J., Blankenship, K., Yang, Y., et al. (2018) · Cancer Cell · doi:10.1016/j.ccell.2018.07.012 · Cited by 153 · see citing works
A first-generation pediatric cancer dependency map
Dharia, N. V., Kugener, G., Guenther, L. M., Malone, C. F., Durbin, A. D., Hong, A. L., Howard, T. P., Bandopadhayay, P., Wechsler, C. S., Fung, I., et al. (2021) · Nature Genetics · doi:10.1038/s41588-021-00819-w · Cited by 153 · see citing works
Self-citationPrecision medicine in pediatric oncology
Forrest, S. J., Geoerger, B., & Janeway, K. A. (2017) · Current Opinion in Pediatrics · doi:10.1097/mop.0000000000000570 · Cited by 148 · see citing works
Self-citationHarttrampf, A. C., Lacroix, L., Deloger, M., Deschamps, F., Puget, S., Auger, N., Vielh, P. R., Varlet, P., Balogh, Z., Abbou, S. D., et al. (2017) · Clinical Cancer Research · doi:10.1158/1078-0432.ccr-17-0381 · Cited by 146 · see citing works
Suehara, Y., Alex, D., Bowman, A., Middha, S., Zehir, A., Chakravarty, D., Wang, L., Jour, G., Nafa, K., Hayashi, T., et al. (2019) · Clinical Cancer Research · doi:10.1158/1078-0432.ccr-18-4032 · Cited by 135 · see citing works
Enrichment of Targetable Mutations in the Relapsed Neuroblastoma Genome
Padovan-Merhar, O. M., Raman, P., Ostrovnaya, I., Kalletla, K., Rubnitz, K. R., Sanford, E. M., Ali, S. M., Miller, V. A., Mossé, Y. P., Granger, M. P., et al. (2016) · PLoS Genetics · doi:10.1371/journal.pgen.1006501 · Cited by 134 · see citing works
Clinical utility of custom-designed NGS panel testing in pediatric tumors
Surrey, L. F., MacFarland, S. P., Chang, F., Cao, K., Rathi, K. S., Akgumus, G. T., Gallo, D., Lin, F., Gleason, A. G., Raman, P., et al. (2019) · Genome Medicine · doi:10.1186/s13073-019-0644-8 · Cited by 129 · see citing works
The clinical utility of integrative genomics in childhood cancer extends beyond targetable mutations
Villani, A., Davidson, S., Kanwar, N., Lo, W. W., Li, Y., Cohen‐Gogo, S., Fuligni, F., Edward, L.-M., Light, N. D., Layeghifard, M., et al. (2022) · Nature Cancer · doi:10.1038/s43018-022-00474-y · Cited by 121 · see citing works
Advances in sarcoma diagnostics and treatment
Dancsok, A. R., Asleh, K., & Nielsen, T. O. (2016) · Oncotarget · doi:10.18632/oncotarget.12548 · Cited by 118 · see citing works
Recent progress in the treatment of cancer in children
Butler, E. B., Ludwig, K. F., Pacenta, H. L., Klesse, L. J., Watt, T. C., & Laetsch, T. W. (2021) · CA A Cancer Journal for Clinicians · doi:10.3322/caac.21665 · Cited by 121 · see citing works
Nagasubramanian, R., Wei, J. L., Gordon, P., Rastatter, J. C., Cox, M. C., & Pappo, A. S. (2016) · Pediatric Blood & Cancer · doi:10.1002/pbc.26026 · Cited by 116 · see citing works
Identification of NTRK fusions in pediatric mesenchymal tumors
Pavlick, D. C., Schrock, A. B., Malicki, D. M., Stephens, P. J., Kuo, D. J., Ahn, H., Turpin, B. K., Allen, J. M., Rosenzweig, M. R., Badizadegan, K., et al. (2017) · Pediatric Blood & Cancer · doi:10.1002/pbc.26433 · Cited by 105 · see citing works
Zheng, P., Li, J., & Kros, J. M. (2017) · Medicinal Research Reviews · doi:10.1002/med.21463 · Cited by 97 · see citing works
Chmielecki, J., Bailey, M., He, J., Elvin, J. A., Vergilio, J., Ramkissoon, S. H., Suh, J. H., Frampton, G. M., Sun, J., Morley, S., et al. (2017) · Cancer Research · doi:10.1158/0008-5472.can-16-1106 · Cited by 94 · see citing works
Self-citationPrecision medicine in pediatric oncology: Lessons learned and next steps
Mody, R. J., Prensner, J. R., Everett, J. N., Parsons, D. W., & Chinnaiyan, A. M. (2016) · Pediatric Blood & Cancer · doi:10.1002/pbc.26288 · Cited by 92 · see citing works
Kieran, M. W., Goumnerova, L. C., Manley, P. E., Chi, S. N., Marcus, K. J., Manzanera, A. G., Polanco, M. L. S., Guzik, B. W., Aguilar-Córdova, E., Diaz-Montero, C. M., et al. (2019) · Neuro-Oncology · doi:10.1093/neuonc/noy202 · Cited by 91 · see citing works
George, S. L., Izquierdo, E., Campbell, J., Koutroumanidou, E., Proszek, P., Jamal, S., Hughes, D., Yuan, L., Marshall, L. V., Carceller, F., et al. (2019) · European Journal of Cancer · doi:10.1016/j.ejca.2019.07.027 · Cited by 86 · see citing works
Hiemenz, M., Ostrow, D. G., Busse, T., Buckley, J. D., Maglinte, D. T., Bootwalla, M., Done, J., Ji, J., Raca, G., Ryutov, A., et al. (2018) · Journal of Molecular Diagnostics · doi:10.1016/j.jmoldx.2018.06.009 · Cited by 80 · see citing works
Early phase clinical trials of anticancer agents in children and adolescents — an ITCC perspective
Moreno, L., Pearson, A. D. J., Paolettí, X., Jiménez, I., Geoerger, B., Kearns, P. R., Zwaan, C. M., Doz, F., Baruchel, A., Vormoor, J. H., et al. (2017) · Nature Reviews Clinical Oncology · doi:10.1038/nrclinonc.2017.59 · Cited by 80 · see citing works
The Future of Precision Oncology
Rulten, S. L., Grose, R. P., Gatz, S. A., Jones, J. L., & Cameron, A. J. M. (2023) · International Journal of Molecular Sciences · doi:10.3390/ijms241612613 · Cited by 86 · see citing works
Precision-guided treatment in high-risk pediatric cancers
Lau, L. M. S., Khuong-Quang, D.-A., Mayoh, C., Wong, M., Barahona, P., Ajuyah, P., Senapati, A., Nagabushan, S., Sherstyuk, A., Alteköester, A.-K., et al. (2024) · Nature Medicine · doi:10.1038/s41591-024-03044-0 · Cited by 77 · see citing works
Assessment of circulating tumor DNA in pediatric solid tumors: The promise of liquid biopsies
Abbou, S. D., Shulman, D. S., DuBois, S. G., & Crompton, B. D. (2019) · Pediatric Blood & Cancer · doi:10.1002/pbc.27595 · Cited by 72 · see citing works
Self-citationMedical guidelines for Li–Fraumeni syndrome 2019, version 1.1
Kumamoto, T., Yamazaki, F., Nakano, Y., Tamura, C., Tashiro, S., Hattori, H., Nakagawara, A., & Tsunematsu, Y. (2021) · International Journal of Clinical Oncology · doi:10.1007/s10147-021-02011-w · Cited by 71 · see citing works
The paradox of precision medicine
Kimmelman, J., & Tannock, I. F. (2018) · Nature Reviews Clinical Oncology · doi:10.1038/s41571-018-0016-0 · Cited by 67 · see citing works
Marron, J. M., DuBois, S. G., Bender, J. G., Kim, A., Crompton, B. D., Meyer, S. C., Janeway, K. A., & Mack, J. W. (2016) · Pediatric Blood & Cancer · doi:10.1002/pbc.26137 · Cited by 66 · see citing works
Self-citationPincez, T., Clément, N., Lapouble, E., Pierron, G., Kamal, M. T., Bièche, I., Bernard, V., Fréneaux, P., Michon, J. M., Orbach, D. H., et al. (2016) · Pediatric Blood & Cancer · doi:10.1002/pbc.26365 · Cited by 65 · see citing works
Walsh, M. F., Ritter, D., Kesserwan, C. A., Sonkin, D., Chakravarty, D., Chao, E., Ghosh, R., Kemel, Y. M., Wu, G., Lee, K., et al. (2018) · Human Mutation · doi:10.1002/humu.23640 · Cited by 65 · see citing works
Molecular profiling identifies targeted therapy opportunities in pediatric solid cancer
Church, A. J., Corson, L., Kao, P., Imamović, A., Reidy, D., Doan, D., Kang, W., Pinto, N. R., Maese, L. D., Laetsch, T. W., et al. (2022) · Nature Medicine · doi:10.1038/s41591-022-01856-6 · Cited by 64 · see citing works
Self-citationLangenberg, K. P. S., Meister, M. T., Bakhuizen, J. J., Boer, J. M., van Eijkelenburg, N. K. A., Hulleman, E., Ilan, U., Looze, E. J., Dierselhuis, M. P., van der Lugt, J., et al. (2022) · European Journal of Cancer · doi:10.1016/j.ejca.2022.09.001 · Cited by 65 · see citing works
Progress toward liquid biopsies in pediatric solid tumors
Weiser, D. A., West-Szymanski, D. C., Fraint, E., Weiner, S., Rivas, M. A., Zhao, C. W. T., He, C., & Applebaum, M. A. (2019) · Cancer and Metastasis Reviews · doi:10.1007/s10555-019-09825-1 · Cited by 60 · see citing works
Molecular Profiling of Hard-to-Treat Childhood and Adolescent Cancers
Khater, F., Vairy, S., Langlois, S., Dumoucel, S., Sontag, T., St-Onge, P., Bittencourt, H. N. D. S., Soglio, D. B. D., Champagne, J., Duval, M., et al. (2019) · JAMA Network Open · doi:10.1001/jamanetworkopen.2019.2906 · Cited by 60 · see citing works
Mayoh, C., Mao, J., Xie, J., Tax, G., Chow, S., Cadiz, R., Pazaky, K., Barahona, P., Ajuyah, P., Trebilcock, P., et al. (2023) · Cancer Research · doi:10.1158/0008-5472.can-22-3702 · Cited by 55 · see citing works
Langenberg, K. P. S., Looze, E. J., & Molenaar, J. J. (2021) · Cancers · doi:10.3390/cancers13174324 · Cited by 57 · see citing works
Functional Precision Medicine Identifies New Therapeutic Candidates for Medulloblastoma
Rusert, J. M., Juarez, E. F., Brabetz, S., Jensen, J., Garancher, A., Chau, L. Q., Tacheva-Grigorova, S. K., Wahab, S., Udaka, Y. T., Finlay, D., et al. (2020) · Cancer Research · doi:10.1158/0008-5472.can-20-1655 · Cited by 55 · see citing works
Basse, C., Morel, C., Alt, M., Sablin, M. P., Franck, C., Pierron, G., Callens, C., Melaabi, S., Masliah‐Planchon, J., Bataillon, G., et al. (2018) · ESMO Open · doi:10.1136/esmoopen-2018-000339 · Cited by 52 · see citing works
Ushering in the next generation of precision trials for pediatric cancer
DuBois, S. G., Corson, L., Stegmaier, K., & Janeway, K. A. (2019) · Science · doi:10.1126/science.aaw4153 · Cited by 52 · see citing works
Self-citationEmerging Opportunities for Target Discovery in Rare Cancers
Sharifnia, T., Hong, A. L., Painter, C., & Boehm, J. S. (2017) · Cell chemical biology · doi:10.1016/j.chembiol.2017.08.002 · Cited by 51 · see citing works
Self-citationPrecision medicine in pediatric oncology
Burdach, S. E. G., Westhoff, M., Steinhauser, M., & Debatin, K. (2018) · Molecular and Cellular Pediatrics · doi:10.1186/s40348-018-0084-3 · Cited by 51 · see citing works
Genomics of adult and pediatric solid tumors.
Rahal, Z., Abdulhai, F., Kadara, H. N., & Saab, R. H. (2018) · Europe PMC (PubMed Central) · Cited by 50 · see citing works
ACCELERATE – Five years accelerating cancer drug development for children and adolescents
Pearson, A. D. J., Weiner, S. L., Adamson, P. C., Karres, D., Reaman, G. H., Rousseau, R. F., Blanc, P., Norga, K., Skolnik, J. M., Kearns, P. R., et al. (2022) · European Journal of Cancer · doi:10.1016/j.ejca.2022.01.033 · Cited by 50 · see citing works
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Zage, P. E. (2018) · Children · doi:10.3390/children5110148 · Cited by 49 · see citing works
Pediatric oncology enters an era of precision medicine
Seibel, N. L., Janeway, K. A., Allen, C. E., Chi, S. N., Cho, Y., Bender, J. G., Kim, A., Laetsch, T. W., Irwin, M. S., Takebe, N., et al. (2017) · Current Problems in Cancer · doi:10.1016/j.currproblcancer.2017.01.002 · Cited by 47 · see citing works
Self-citationMarks, L. J., Oberg, J. A., Pendrick, D., Sireci, A. N., Glasser, C. L., Coval, C., Zylber, R. J., Chung, W. K., Pang, J., Turk, A. T., et al. (2017) · Frontiers in Pediatrics · doi:10.3389/fped.2017.00265 · Cited by 47 · see citing works
Dyer, M. A. (2016) · Trends in Molecular Medicine · doi:10.1016/j.molmed.2016.07.010 · Cited by 46 · see citing works
Precision Medicine in Pediatric Oncology
Vo, K. T., Parsons, D. W., & Seibel, N. L. (2019) · Surgical Oncology Clinics of North America · doi:10.1016/j.soc.2019.08.005 · Cited by 46 · see citing works
In vitro and in vivo drug screens of tumor cells identify novel therapies for high‐risk child cancer
Lau, L. M. S., Mayoh, C., Xie, J., Barahona, P., MacKenzie, K. L., Wong, M., Kamili, A., Tsoli, M., Failes, T. W., Kumar, A., et al. (2021) · EMBO Molecular Medicine · doi:10.15252/emmm.202114608 · Cited by 43 · see citing works
Subbiah, V., Holmes, O., Gowen, K. A., Spritz, D., Amini, B., Wang, W., Schrock, A. B., Meric‐Bernstam, F., Zinner, R., Piha-Paul, S. A., et al. (2016) · Oncology · doi:10.1159/000449204 · Cited by 42 · see citing works
Kytölä, V., Topaloĝlu, Ü., Miller, L. D., Bitting, R. L., Goodman, M. M., D`Agostino, R. B., Desnoyers, R. J., Albright, C. A., Yacoub, G., Qasem, S. A., et al. (2017) · Theranostics · doi:10.7150/thno.20355 · Cited by 42 · see citing works
Copy-number analysis identified new prognostic marker in acute myeloid leukemia
Nibourel, O., Guihard, S., Roumier, C., Pottier, N., Terré, C., Paquet, A. C., Peyrouze, P., Geffroy, S., Quentin, S., Alberdi, A., et al. (2016) · Leukemia · doi:10.1038/leu.2016.265 · Cited by 41 · see citing works
Samuel, G., Crow, J., Klein, J. B., Merchant, M. L., Nissen, E., Koestler, D. C., Laurence, K., Liang, X., Neville, K. A., Staggs, V. S., et al. (2020) · Oncotarget · doi:10.18632/oncotarget.27678 · Cited by 41 · see citing works
Selective Modulation of a Pan-Essential Protein as a Therapeutic Strategy in Cancer
Malone, C. F., Dharia, N. V., Kugener, G., Forman, A. B., Rothberg, M. V., Abdusamad, M., Gonzalez, A. E., Kuljanin, M., Robichaud, A. L., Conway, A. S., et al. (2021) · Cancer Discovery · doi:10.1158/2159-8290.cd-20-1213 · Cited by 41 · see citing works
Self-citationHuang, R. Q., Liao, Y., Zhou, X., Fu, Y., & Xing, D. (2017) · Sensors and Actuators B Chemical · doi:10.1016/j.snb.2017.03.055 · Cited by 40 · see citing works
Pikman, Y., Tasian, S. K., Sulis, M. L., Stevenson, K. E., Blonquist, T. M., Winger, B. A., Cooper, T. M., Pauly, M. G., Maloney, K. W., Burke, M. J., et al. (2021) · Cancer Discovery · doi:10.1158/2159-8290.cd-20-0564 · Cited by 38 · see citing works
Self-citationGenomics and Precision Medicine for Clinicians and Scientists in Hypertension
Dominiczak, A. F., Delles, C., & Padmanabhan, S. (2017) · Hypertension · doi:10.1161/hypertensionaha.116.08252 · Cited by 38 · see citing works
Emerging and investigational therapies for neuroblastoma
Applebaum, M. A., Desai, A. V., Bender, J. G., & Cohn, S. L. (2017) · Expert Opinion on Orphan Drugs · doi:10.1080/21678707.2017.1304212 · Cited by 38 · see citing works
Precision Oncology in Sarcomas: Divide and Conquer
Pestana, R. C., Groisberg, R., Roszik, J., & Subbiah, V. (2019) · JCO Precision Oncology · doi:10.1200/po.18.00247 · Cited by 37 · see citing works
Review: Ewing Sarcoma Predisposition
Gargallo, P., Yáñez, Y., Juan, A. D., Segura, V., Balaguer, J., Torres, B., Oltra, S., Castel, V., & Cañete, A. (2019) · Pathology & Oncology Research · doi:10.1007/s12253-019-00765-3 · Cited by 39 · see citing works
Schienda, J., Church, A. J., Corson, L. B., Decker, B. J., Clinton, C. M., Manning, D. K., Imamović, A., Reidy, D., Strand, G. R., Applebaum, M. A., et al. (2021) · JCO Precision Oncology · doi:10.1200/po.21.00281 · Cited by 34 · see citing works
Self-citationSequencing-based diagnostics for pediatric genetic diseases: progress and potential
Tayoun, A. N. A., Krock, B. L., & Spinner, N. B. (2016) · Expert Review of Molecular Diagnostics · doi:10.1080/14737159.2016.1209411 · Cited by 33 · see citing works
The Roles of Common Variation and Somatic Mutation in Cancer Pharmacogenomics
Chan, T., Chin, Y. M., & Low, S. (2019) · Oncology and Therapy · doi:10.1007/s40487-018-0090-6 · Cited by 35 · see citing works
Anakura, M., Nachankar, A., Kobayashi, D., Amornwichet, N., Hirota, Y., Shibata, A., Oike, T., & Nakano, T. (2019) · International Journal of Molecular Sciences · doi:10.3390/ijms20153635 · Cited by 33 · see citing works
Targetable BRAF and RAF1 Alterations in Advanced Pediatric Cancers
Rankin, A., Johnson, A., Roos, A., Kannan, G. S., Knipstein, J. A., Britt, N., Rosenzweig, M. R., Haberberger, J., Pavlick, D. C., Severson, E. A., et al. (2020) · The Oncologist · doi:10.1002/onco.13519 · Cited by 33 · see citing works
Clinical Utility of Precision Medicine in Pediatric Oncology: A Systematic Review
Lee, J., Gillam, L. H., Visvanathan, K., Hansford, J. R., & McCarthy, M. (2021) · JCO Precision Oncology · doi:10.1200/po.20.00405 · Cited by 32 · see citing works
Healthcare professionals’ attitudes toward cancer precision medicine: A systematic review
Vetsch, J., Wakefield, C. E., Techakesari, P., Warby, M., Ziegler, D. S., O'Brien, T. A., Drinkwater, C., Neeman, N., & Tucker, K. (2019) · Seminars in Oncology · doi:10.1053/j.seminoncol.2019.05.001 · Cited by 30 · see citing works
Stankunaite, R., George, S. L., Gallagher, L., Jamal, S., Shaikh, R., Yuan, L., Hughes, D., Proszek, P., Carter, P. M., Pietka, G., et al. (2021) · European Journal of Cancer · doi:10.1016/j.ejca.2021.09.042 · Cited by 28 · see citing works
Tsoli, M., Wadham, C., Pinese, M., Failes, T. W., Joshi, S. M., Mould, E. V. A., Yin, J. X. M., Gayevskiy, V., Kumar, A., Kaplan, W. H., et al. (2018) · Cancer Biology & Therapy · doi:10.1080/15384047.2018.1491498 · Cited by 28 · see citing works
ARID1B alterations identify aggressive tumors in neuroblastoma
Lee, S. H., Kim, J.-S., Zheng, S., Huse, J. T., Bae, J. S., Lee, J. W., Yoo, K. H., Koo, H. H., Kyung, S., Park, W., et al. (2017) · Oncotarget · doi:10.18632/oncotarget.17500 · Cited by 26 · see citing works
Precision Medicine in Pediatric Neurooncology: A Review
Mochizuki, A. Y., Frost, I. M., Mastrodimos, M. B., Plant, A., Wang, A., Moore, T. B., Prins, R. M., Weiss, P. S., & Jonas, S. J. (2017) · ACS Chemical Neuroscience · doi:10.1021/acschemneuro.7b00388 · Cited by 25 · see citing works
Innovations in Cancer Treatment of Children
Helms, L., Guimera, A. E., Janeway, K. A., & Bailey, K. M. (2023) · PEDIATRICS · doi:10.1542/peds.2023-061539 · Cited by 23 · see citing works
Self-citationPrecision oncology: lessons learned and challenges for the future
Yang, H., Shah, R. H., Tegay, D. H., & Onel, K. (2019) · Cancer Management and Research · doi:10.2147/cmar.s201326 · Cited by 24 · see citing works
Murumägi, A., Ungureanu, D., Khan, S. A., Arjama, M., Välimäki, K. E., Ianevski, A., Ianevski, P., Bergström, R., Dini, A., Kanerva, A., et al. (2022) · British Journal of Cancer · doi:10.1038/s41416-022-02067-z · Cited by 24 · see citing works
Østrup, O., Nysom, K., Scheie, D., Schmidt, A. Y., Mathiasen, R., Hjalgrim, L. L., Olsen, T. E., Skjøth‐Rasmussen, J., Henriksen, B. M., Nielsen, F. C., et al. (2018) · Frontiers in Pediatrics · doi:10.3389/fped.2018.00114 · Cited by 22 · see citing works
Coutinho, D. F., Mundi, P. S., Marks, L. J., Burke, C., Ortiz, M. V., Diolaiti, D., Bird, L., Vallance, K., Ibáñez, G., You, D., et al. (2022) · Med · doi:10.1016/j.medj.2022.09.002 · Cited by 22 · see citing works
Marron, J. M., Cronin, A. M., DuBois, S. G., Bender, J. G., Kim, A., Crompton, B. D., Meyer, S. C., Janeway, K. A., & Mack, J. W. (2019) · JCO Precision Oncology · doi:10.1200/po.18.00176 · Cited by 22 · see citing works
Self-citationIzquierdo, E., Yuan, L., George, S. L., Hubank, M. J. F., Jones, C., Proszek, P., SHIPLEY, J. M., Gatz, S. A., Stinson, C. L., Moore, A. S., et al. (2017) · Oncotarget · doi:10.18632/oncotarget.23000 · Cited by 21 · see citing works
Deyell, R. J., Shen, Y., Titmuss, E., Dixon, K., Williamson, L. M., Pleasance, E., Nelson, J., Abbasi, S., Krzywinski, M. I., Armstrong, L., et al. (2024) · Nature Communications · doi:10.1038/s41467-024-48363-5 · Cited by 20 · see citing works
Mutational analysis of uterine cervical cancer that survived multiple rounds of radiotherapy
Nuryadi, E., Sasaki, Y., Hagiwara, Y., Permata, T. B. M., Sato, H., Komatsu, S., Yoshimoto, Y., Murata, K., Ando, K., Kubo, N., et al. (2018) · Oncotarget · doi:10.18632/oncotarget.25982 · Cited by 20 · see citing works
Precision medicine in Ewing sarcoma: a translational point of view
Gargallo, P., Juan, A. D., Yáñez, Y., Dolz, S., Segura, V., Castel, V., & Cañete, A. (2020) · Clinical & Translational Oncology · doi:10.1007/s12094-020-02298-7 · Cited by 21 · see citing works
Waldman, L., Hancock, K., Gallinger, B., Johnstone, B., Brunga, L., Malkin, D. M., Barrera, M. E., & Villani, A. (2022) · JCO Precision Oncology · doi:10.1200/po.21.00444 · Cited by 20 · see citing works
McGee, R. B., Oak, N., Harrison, L. H., Xu, K., Nuccio, R., Blake, A. K., Mostafavi, R. M., Lewis, S., Taylor, L. M., Kubal, M., et al. (2023) · Clinical Cancer Research · doi:10.1158/1078-0432.ccr-22-2482 · Cited by 20 · see citing works
Kumamoto, T., Aoki, Y., Sonoda, T., Yamanishi, M., Arakawa, A., Sugiyama, M., Shirakawa, N., Ishimaru, S., Saito, Y., Maeshima, A. M., et al. (2018) · International Journal of Hematology · doi:10.1007/s12185-018-2553-9 · Cited by 19 · see citing works
Programmatic Precision Oncology Decision Support for Patients With Gastrointestinal Cancer
Keller, R. B., Mazor, T., Sholl, L. M., Aguirre, A. J., Singh, H., Sethi, N. S., Bass, A. J., Nagaraja, A. K., Brais, L. K., Hill, E. R., et al. (2023) · JCO Precision Oncology · doi:10.1200/po.22.00342 · Cited by 18 · see citing works
Self-citationGatz, S. A., Harttrampf, A. C., Brard, C., Bautista, F. J., André, N., Abbou, S. D., Rubino, J., Rondof, W., Deloger, M., Rübsam, M., et al. (2023) · Clinical Cancer Research · doi:10.1158/1078-0432.ccr-23-2959 · Cited by 18 · see citing works
Oberg, J. A., Ruiz, J., Ali-Shaw, T., Schlechtweg, K., Ricci, A. M., Kung, A. L., Chung, W. K., Appelbaum, P. S., Bender, J. G., & Levine, J. M. (2018) · JCO Precision Oncology · doi:10.1200/po.17.00104 · Cited by 18 · see citing works
Pediatric Cancer Predisposition and Surveillance Update: Summary Perspective and Future Directions
Brodeur, G. M., Diller, L., Nichols, K. E., Plon, S. E., Porter, C. C., & Malkin, D. M. (2025) · Clinical Cancer Research · doi:10.1158/1078-0432.ccr-25-0423 · Cited by 17 · see citing works
Self-citationLee, Y. J., Kim, D., Shim, J. E., Bae, S., Jung, Y., Kim, S., Lee, H., Kim, S. H., Jo, S. B., Lee, J., et al. (2019) · International Journal of Cancer · doi:10.1002/ijc.32729 · Cited by 17 · see citing works
Ethical Challenges in Pediatric Oncology Care and Clinical Trials
Benedetti, D. J., & Marron, J. M. (2021) · Recent results in cancer research/Recent Results in Cancer Research · doi:10.1007/978-3-030-63749-1_11 · Cited by 18 · see citing works
Poonnen, P. J., Duffy, J. E., Hintze, B. J., Shukla, M. P., Brettin, T. S., Conrad, N. R., Yoo, H., Guertin, C. M., Looney, J. A., Vashistha, V., et al. (2019) · JCO Precision Oncology · doi:10.1200/po.19.00075 · Cited by 16 · see citing works
Omic approaches to pediatric bone sarcomas
Tirtei, E., Cereda, M., Luna, E. D., Quarello, P., Asaftei, S. D., & Fagioli, F. (2019) · Pediatric Blood & Cancer · doi:10.1002/pbc.28072 · Cited by 15 · see citing works
Cullinan, N. M., Schiller, I., Giuseppe, G. D., Mamun, M. R. I., Reichman, L., Cacciotti, C., Wheaton, L., Caswell, K., DiMonte, B., Gibson, P. J., et al. (2021) · Journal of Clinical Oncology · doi:10.1200/jco.21.00018 · Cited by 16 · see citing works
- 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 · 2014: 1
- 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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