Precision medicine in pediatric oncology: Lessons learned and next steps

Michaela Kotrova, Katerina Muzikova, Ester Mejstrikova, Michaela Novakova, Violeta Bakardjieva-Mihaylova, Karel Fiser, Jan Stuchly, Mathieu Giraud, Mikaël Salson, Christiane Pott, Monika Brüggemann, Marc Füllgrabe, Jan Stary, Jan Trka, Eva Fronkova, The predictive strength of next-generation sequencing MRD detection for relapse compared with current methods in childhood ALL Blood. ,vol. 126, pp. 1045- 1047 ,(2015) , 10.1182/BLOOD-2015-07-655159

Sho Kurihara, Yuka Ueda, Yoshiyuki Onitake, Taijiro Sueda, Emi Ohta, Nagisa Morihara, Shoko Hirano, Fumiko Irisuna, Eiso Hiyama, Circulating Free DNA as Non-invasive Diagnostic Biomarker for Childhood solid Tumors Journal of Pediatric Surgery. ,vol. 50, pp. 2094- 2097 ,(2015) , 10.1016/J.JPEDSURG.2015.08.033

Malcolm A Smith, Oliver A Hampton, C Patrick Reynolds, Min H Kang, John M Maris, Richard Gorlick, E Anders Kolb, Richard Lock, Hernan Carol, Stephen T Keir, Jianrong Wu, Raushan T Kurmasheva, David A Wheeler, Peter J Houghton, None, Initial testing (stage 1) of the PARP inhibitor BMN 673 by the pediatric preclinical testing program: PALB2 mutation predicts exceptional in vivo response to BMN 673 Pediatric Blood & Cancer. ,vol. 62, pp. 91- 98 ,(2015) , 10.1002/PBC.25201

Matthew M. Gubin, Maxim N. Artyomov, Elaine R. Mardis, Robert D. Schreiber, Tumor neoantigens: building a framework for personalized cancer immunotherapy Journal of Clinical Investigation. ,vol. 125, pp. 3413- 3421 ,(2015) , 10.1172/JCI80008

Heidi L. Rehm, Jonathan S. Berg, Lisa D. Brooks, Carlos D. Bustamante, James P. Evans, Melissa J. Landrum, David H. Ledbetter, Donna R. Maglott, Christa Lese Martin, Robert L. Nussbaum, Sharon E. Plon, Erin M. Ramos, Stephen T. Sherry, Michael S. Watson, ClinGen, None, ClinGen — The Clinical Genome Resource The New England Journal of Medicine. ,vol. 372, pp. 2235- 2242 ,(2015) , 10.1056/NEJMSR1406261

David M. Hyman, Igor Puzanov, Vivek Subbiah, Jason E. Faris, Ian Chau, Jean-Yves Blay, Jürgen Wolf, Noopur S. Raje, Eli L. Diamond, Antoine Hollebecque, Radj Gervais, Maria Elena Elez-Fernandez, Antoine Italiano, Ralf-Dieter Hofheinz, Manuel Hidalgo, Emily Chan, Martin Schuler, Susan Frances Lasserre, Martina Makrutzki, Florin Sirzen, Maria Luisa Veronese, Josep Tabernero, José Baselga, Vemurafenib in Multiple Nonmelanoma Cancers with BRAF V600 Mutations The New England Journal of Medicine. ,vol. 373, pp. 726- 736 ,(2015) , 10.1056/NEJMOA1502309

Laurence B. McCullough, Melody J. Slashinski, Amy L. McGuire, Richard L. Street, Christine M. Eng, Richard A. Gibbs, D. William Parsons, Sharon E. Plon, Is Whole‐Exome Sequencing an Ethically Disruptive Technology? Perspectives of Pediatric Oncologists and Parents of Pediatric Patients With Solid Tumors Pediatric Blood & Cancer. ,vol. 63, pp. 511- 515 ,(2016) , 10.1002/PBC.25815

Angela Bellini, Virginie Bernard, Quentin Leroy, Thomas Rio Frio, Gaelle Pierron, Valérie Combaret, Eve Lapouble, Nathalie Clement, Herve Rubie, Estelle Thebaud, Pascal Chastagner, Anne Sophie Defachelles, Christophe Bergeron, Nimrod Buchbinder, Sophie Taque, Anne Auvrignon, Dominique Valteau-Couanet, Jean Michon, Isabelle Janoueix-Lerosey, Olivier Delattre, Gudrun Schleiermacher, Deep Sequencing Reveals Occurrence of Subclonal ALK Mutations in Neuroblastoma at Diagnosis. Clinical Cancer Research. ,vol. 21, pp. 4913- 4921 ,(2015) , 10.1158/1078-0432.CCR-15-0423

Rajen J Mody, Yi-Mi Wu, Robert J Lonigro, Xuhong Cao, Sameek Roychowdhury, Pankaj Vats, Kevin M Frank, John R Prensner, Irfan Asangani, Nallasivam Palanisamy, Jonathan R Dillman, Raja M Rabah, Laxmi Priya Kunju, Jessica Everett, Victoria M Raymond, Yu Ning, Fengyun Su, Rui Wang, Elena M Stoffel, Jeffrey W Innis, J Scott Roberts, Patricia L Robertson, Gregory Yanik, Aghiad Chamdin, James A Connelly, Sung Choi, Andrew C Harris, Carrie Kitko, Rama Jasty Rao, John E Levine, Valerie P Castle, Raymond J Hutchinson, Moshe Talpaz, Dan R Robinson, Arul M Chinnaiyan, None, Integrative Clinical Sequencing in the Management of Refractory or Relapsed Cancer in Youth JAMA. ,vol. 314, pp. 913- 925 ,(2015) , 10.1001/JAMA.2015.10080

Katherine Tarlock, Bill Chang, Todd Cooper, Thomas Gross, Sumit Gupta, Steven Neudorf, Kathleen Adlard, Phoenix A. Ho, Suzanne McGoldrick, Tanya Watt, Tina Templeman, India Sisler, Amy Garee, Blythe Thomson, Ann Woolfrey, Elihu Estey, Soheil Meshinchi, Jessica A. Pollard, Sorafenib treatment following hematopoietic stem cell transplant in pediatric FLT3/ITD acute myeloid leukemia Pediatric Blood & Cancer. ,vol. 62, pp. 1048- 1054 ,(2015) , 10.1002/PBC.25437