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1. The children's brain tumor network (CBTN) - Accelerating research in pediatric central nervous system tumors through collaboration and open science

Author

Lilly, Jena V, Rokita, Jo Lynne, Mason, Jennifer L, Patton, Tatiana, Stefankiewiz, Stephanie, Higgins, David, Trooskin, Gerri, Larouci, Carina A, Arya, Kamnaa, Appert, Elizabeth, Heath, Allison P, Zhu, Yuankun, Brown, Miguel A, Zhang, Bo, Farrow, Bailey K, Robins, Shannon, Morgan, Allison M, Nguyen, Thinh Q, Frenkel, Elizabeth, Lehmann, Kaitlin, Drake, Emily, Sullivan, Catherine, Plisiewicz, Alexa, Coleman, Noel, Patterson, Luke, Koptyra, Mateusz, Helili, Zeinab, Van Kuren, Nicholas, Young, Nathan, Kim, Meen Chul, Friedman, Christopher, Lubneuski, Alex, Blackden, Christopher, Williams, Marti, Baubet, Valerie, Tauhid, Lamiya, Galanaugh, Jamie, Boucher, Katie, Ijaz, Heba, Cole, Kristina A, Choudhari, Namrata, Santi, Mariarita, Moulder, Robert W, Waller, Jonathan, Rife, Whitney, Diskin, Sharon J, Mateos, Marion, Parsons, Donald W, Pollack, Ian F, Goldman, Stewart, Leary, Sarah, Caporalini, Chiara, Buccoliero, Anna Maria, Scagnet, Mirko, Haussler, David, Hanson, Derek, Firestein, Ron, Cain, Jason, Phillips, Joanna J, Gupta, Nalin, Mueller, Sabine, Grant, Gerald, Monje-Deisseroth, Michelle, Partap, Sonia, Greenfield, Jeffrey P, Hashizume, Rintaro, Smith, Amy, Zhu, Shida, Johnston, James M, Fangusaro, Jason R, Miller, Matthew, Wood, Matthew D, Gardner, Sharon, Carter, Claire L, Prolo, Laura M, Pisapia, Jared, Pehlivan, Katherine, Franson, Andrea, Niazi, Toba, Rubin, Josh, Abdelbaki, Mohamed, Ziegler, David S, Lindsay, Holly B, Stucklin, Ana Guerreiro, Gerber, Nicolas, Vaske, Olena M, Quinsey, Carolyn, Rood, Brian R, Nazarian, Javad, Raabe, Eric, Jackson, Eric M, Stapleton, Stacie, Lober, Robert M, Kram, David E, Koschmann, Carl, Storm, Phillip B, Lulla, Rishi R, Prados, Michael, Resnick, Adam C, and Waanders, Angela J

Subjects
Neurosciences, Pediatric Cancer, Brain Disorders, Pediatric Research Initiative, Pediatric, Brain Cancer, Rare Diseases, Cancer, Good Health and Well Being, Adult, Humans, Child, Quality of Life, Brain Neoplasms, Collaborative international research infrastructure, Pediatric brain tumors, Multi-omic data, Longitudinal clinical data, Biospecimens, Molecular clinical trials, Clinical Sciences, Oncology & Carcinogenesis
Abstract

Pediatric brain tumors are the leading cause of cancer-related death in children in the United States and contribute a disproportionate number of potential years of life lost compared to adult cancers. Moreover, survivors frequently suffer long-term side effects, including secondary cancers. The Children's Brain Tumor Network (CBTN) is a multi-institutional international clinical research consortium created to advance therapeutic development through the collection and rapid distribution of biospecimens and data via open-science research platforms for real-time access and use by the global research community. The CBTN's 32 member institutions utilize a shared regulatory governance architecture at the Children's Hospital of Philadelphia to accelerate and maximize the use of biospecimens and data. As of August 2022, CBTN has enrolled over 4700 subjects, over 1500 parents, and collected over 65,000 biospecimen aliquots for research. Additionally, over 80 preclinical models have been developed from collected tumors. Multi-omic data for over 1000 tumors and germline material are currently available with data generation for > 5000 samples underway. To our knowledge, CBTN provides the largest open-access pediatric brain tumor multi-omic dataset annotated with longitudinal clinical and outcome data, imaging, associated biospecimens, child-parent genomic pedigrees, and in vivo and in vitro preclinical models. Empowered by NIH-supported platforms such as the Kids First Data Resource and the Childhood Cancer Data Initiative, the CBTN continues to expand the resources needed for scientists to accelerate translational impact for improved outcomes and quality of life for children with brain and spinal cord tumors.

Published
2023

2. The children's brain tumor network (CBTN) - Accelerating research in pediatric central nervous system tumors through collaboration and open science

Author

Lilly, Jena V., Rokita, Jo Lynne, Mason, Jennifer L., Patton, Tatiana, Stefankiewiz, Stephanie, Higgins, David, Trooskin, Gerri, Larouci, Carina A., Arya, Kamnaa, Appert, Elizabeth, Heath, Allison P., Zhu, Yuankun, Brown, Miguel A., Zhang, Bo, Farrow, Bailey K., Robins, Shannon, Morgan, Allison M., Nguyen, Thinh Q., Frenkel, Elizabeth, Lehmann, Kaitlin, Drake, Emily, Sullivan, Catherine, Plisiewicz, Alexa, Coleman, Noel, Patterson, Luke, Koptyra, Mateusz, Helili, Zeinab, Van Kuren, Nicholas, Young, Nathan, Kim, Meen Chul, Friedman, Christopher, Lubneuski, Alex, Blackden, Christopher, Williams, Marti, Baubet, Valerie, Tauhid, Lamiya, Galanaugh, Jamie, Boucher, Katie, Ijaz, Heba, Cole, Kristina A., Choudhari, Namrata, Santi, Mariarita, Moulder, Robert W., Waller, Jonathan, Rife, Whitney, Diskin, Sharon J., Mateos, Marion, Parsons, Donald W., Pollack, Ian F., Goldman, Stewart, Leary, Sarah, Caporalini, Chiara, Buccoliero, Anna Maria, Scagnet, Mirko, Haussler, David, Hanson, Derek, Firestein, Ron, Cain, Jason, Phillips, Joanna J., Gupta, Nalin, Mueller, Sabine, Grant, Gerald, Monje-Deisseroth, Michelle, Partap, Sonia, Greenfield, Jeffrey P., Hashizume, Rintaro, Smith, Amy, Zhu, Shida, Johnston, James M., Fangusaro, Jason R., Miller, Matthew, Wood, Matthew D., Gardner, Sharon, Carter, Claire L., Prolo, Laura M., Pisapia, Jared, Pehlivan, Katherine, Franson, Andrea, Niazi, Toba, Rubin, Josh, Abdelbaki, Mohamed, Ziegler, David S., Lindsay, Holly B., Stucklin, Ana Guerreiro, Gerber, Nicolas, Vaske, Olena M., Quinsey, Carolyn, Rood, Brian R., Nazarian, Javad, Raabe, Eric, Jackson, Eric M., Stapleton, Stacie, Lober, Robert M., Kram, David E., Koschmann, Carl, Storm, Phillip B., Lulla, Rishi R., Prados, Michael, Resnick, Adam C., and Waanders, Angela J.

Published
2023
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4. Contributors List

Author

Ambady, Prakash, primary, Bagley, Stephen J., additional, Blakeley, Jaishri, additional, Brooks, Taylor, additional, Bussière, Marc R., additional, Campian, Jian L., additional, Chan, Michael D., additional, Chukwueke, Ugonma N., additional, Cramer, Christina K., additional, Couture, Daniel E., additional, Cummings, Tiffany L., additional, Dahiya, Sonika, additional, Blank, Peter de, additional, Diaz-Arias, Luisa A., additional, Franchino, Federica, additional, Franke, Jennifer L., additional, Geer, Carol Parks, additional, Gerstner, Elizabeth R., additional, Grossman, Stuart, additional, Henderson, Jacob J., additional, Henke, Lauren L., additional, Holdhoff, Matthias, additional, Hsu, Wesley, additional, Huang, Jiayi, additional, Jackson, Christina, additional, Jordan, Justin T., additional, Kamson, David Olayinka, additional, Kassem, Ahmad N., additional, Kim, Albert E., additional, Kim, Teddy E., additional, Knox, Molly, additional, Kram, David E., additional, Kumthekar, Priya, additional, Langmead, Shannon, additional, Laxton, Adrian W., additional, Lebow, Emily S., additional, Lim, Michael, additional, Lim-Fat, Mary Jane, additional, Ly, K. Ina, additional, Mancone, Sarah E., additional, Mohile, Nimish, additional, Mrugala, Maciej M., additional, Nechtman, Carl M., additional, Pathak, Sapna, additional, Netto, Joao Prola, additional, Peereboom, David M., additional, Pellerino, Alessia, additional, Probasco, John C., additional, Pruitt, Amy, additional, Ramkissoon, Shakti, additional, Robinson, David Wayne, additional, Romo, Carlos G., additional, Rudà, Roberta, additional, Shen, Colette, additional, Shih, Helen A., additional, Silvia, Mary, additional, Sivakumar, Ananyaa, additional, Soffietti, Riccardo, additional, Soike, Michael H., additional, Strowd, Roy E., additional, Sud, Shivani, additional, Szidonya, Laszlo, additional, Tatter, Stephen B., additional, Thakkar, Jigisha, additional, Uluc, Kutluay, additional, Valencia-Sanchez, Cristina, additional, Vaughn, Courtney M., additional, Vu, Thuy M., additional, Wasilewski, Andrea, additional, Wen, Patrick Y., additional, and Williams, Michelle Marie, additional

Published
2021
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6. Data from Infant High-Grade Gliomas Comprise Multiple Subgroups Characterized by Novel Targetable Gene Fusions and Favorable Outcomes

Author

Clarke, Matthew, primary, Mackay, Alan, primary, Ismer, Britta, primary, Pickles, Jessica C., primary, Tatevossian, Ruth G., primary, Newman, Scott, primary, Bale, Tejus A., primary, Stoler, Iris, primary, Izquierdo, Elisa, primary, Temelso, Sara, primary, Carvalho, Diana M., primary, Molinari, Valeria, primary, Burford, Anna, primary, Howell, Louise, primary, Virasami, Alex, primary, Fairchild, Amy R., primary, Avery, Aimee, primary, Chalker, Jane, primary, Kristiansen, Mark, primary, Haupfear, Kelly, primary, Dalton, James D., primary, Orisme, Wilda, primary, Wen, Ji, primary, Hubank, Michael, primary, Kurian, Kathreena M., primary, Rowe, Catherine, primary, Maybury, Mellissa, primary, Crosier, Stephen, primary, Knipstein, Jeffrey, primary, Schüller, Ulrich, primary, Kordes, Uwe, primary, Kram, David E., primary, Snuderl, Matija, primary, Bridges, Leslie, primary, Martin, Andrew J., primary, Doey, Lawrence J., primary, Al-Sarraj, Safa, primary, Chandler, Christopher, primary, Zebian, Bassel, primary, Cairns, Claire, primary, Natrajan, Rachael, primary, Boult, Jessica K.R., primary, Robinson, Simon P., primary, Sill, Martin, primary, Dunkel, Ira J., primary, Gilheeney, Stephen W., primary, Rosenblum, Marc K., primary, Hughes, Debbie, primary, Proszek, Paula Z., primary, Macdonald, Tobey J., primary, Preusser, Matthias, primary, Haberler, Christine, primary, Slavc, Irene, primary, Packer, Roger, primary, Ng, Ho-Keung, primary, Caspi, Shani, primary, Popović, Mara, primary, Faganel Kotnik, Barbara, primary, Wood, Matthew D., primary, Baird, Lissa, primary, Davare, Monika Ashok, primary, Solomon, David A., primary, Olsen, Thale Kristin, primary, Brandal, Petter, primary, Farrell, Michael, primary, Cryan, Jane B., primary, Capra, Michael, primary, Karremann, Michael, primary, Schittenhelm, Jens, primary, Schuhmann, Martin U., primary, Ebinger, Martin, primary, Dinjens, Winand N.M., primary, Kerl, Kornelius, primary, Hettmer, Simone, primary, Pietsch, Torsten, primary, Andreiuolo, Felipe, primary, Driever, Pablo Hernáiz, primary, Korshunov, Andrey, primary, Hiddingh, Lotte, primary, Worst, Barbara C., primary, Sturm, Dominik, primary, Zuckermann, Marc, primary, Witt, Olaf, primary, Bloom, Tabitha, primary, Mitchell, Clare, primary, Miele, Evelina, primary, Colafati, Giovanna Stefania, primary, Diomedi-Camassei, Francesca, primary, Bailey, Simon, primary, Moore, Andrew S., primary, Hassall, Timothy E.G., primary, Lowis, Stephen P., primary, Tsoli, Maria, primary, Cowley, Mark J., primary, Ziegler, David S., primary, Karajannis, Matthias A., primary, Aquilina, Kristian, primary, Hargrave, Darren R., primary, Carceller, Fernando, primary, Marshall, Lynley V., primary, von Deimling, Andreas, primary, Kramm, Christof M., primary, Pfister, Stefan M., primary, Sahm, Felix, primary, Baker, Suzanne J., primary, Mastronuzzi, Angela, primary, Carai, Andrea, primary, Vinci, Maria, primary, Capper, David, primary, Popov, Sergey, primary, Ellison, David W., primary, Jacques, Thomas S., primary, Jones, David T.W., primary, and Jones, Chris, primary

Published
2023
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7. Supplementary Data from Infant High-Grade Gliomas Comprise Multiple Subgroups Characterized by Novel Targetable Gene Fusions and Favorable Outcomes

Author

Clarke, Matthew, primary, Mackay, Alan, primary, Ismer, Britta, primary, Pickles, Jessica C., primary, Tatevossian, Ruth G., primary, Newman, Scott, primary, Bale, Tejus A., primary, Stoler, Iris, primary, Izquierdo, Elisa, primary, Temelso, Sara, primary, Carvalho, Diana M., primary, Molinari, Valeria, primary, Burford, Anna, primary, Howell, Louise, primary, Virasami, Alex, primary, Fairchild, Amy R., primary, Avery, Aimee, primary, Chalker, Jane, primary, Kristiansen, Mark, primary, Haupfear, Kelly, primary, Dalton, James D., primary, Orisme, Wilda, primary, Wen, Ji, primary, Hubank, Michael, primary, Kurian, Kathreena M., primary, Rowe, Catherine, primary, Maybury, Mellissa, primary, Crosier, Stephen, primary, Knipstein, Jeffrey, primary, Schüller, Ulrich, primary, Kordes, Uwe, primary, Kram, David E., primary, Snuderl, Matija, primary, Bridges, Leslie, primary, Martin, Andrew J., primary, Doey, Lawrence J., primary, Al-Sarraj, Safa, primary, Chandler, Christopher, primary, Zebian, Bassel, primary, Cairns, Claire, primary, Natrajan, Rachael, primary, Boult, Jessica K.R., primary, Robinson, Simon P., primary, Sill, Martin, primary, Dunkel, Ira J., primary, Gilheeney, Stephen W., primary, Rosenblum, Marc K., primary, Hughes, Debbie, primary, Proszek, Paula Z., primary, Macdonald, Tobey J., primary, Preusser, Matthias, primary, Haberler, Christine, primary, Slavc, Irene, primary, Packer, Roger, primary, Ng, Ho-Keung, primary, Caspi, Shani, primary, Popović, Mara, primary, Faganel Kotnik, Barbara, primary, Wood, Matthew D., primary, Baird, Lissa, primary, Davare, Monika Ashok, primary, Solomon, David A., primary, Olsen, Thale Kristin, primary, Brandal, Petter, primary, Farrell, Michael, primary, Cryan, Jane B., primary, Capra, Michael, primary, Karremann, Michael, primary, Schittenhelm, Jens, primary, Schuhmann, Martin U., primary, Ebinger, Martin, primary, Dinjens, Winand N.M., primary, Kerl, Kornelius, primary, Hettmer, Simone, primary, Pietsch, Torsten, primary, Andreiuolo, Felipe, primary, Driever, Pablo Hernáiz, primary, Korshunov, Andrey, primary, Hiddingh, Lotte, primary, Worst, Barbara C., primary, Sturm, Dominik, primary, Zuckermann, Marc, primary, Witt, Olaf, primary, Bloom, Tabitha, primary, Mitchell, Clare, primary, Miele, Evelina, primary, Colafati, Giovanna Stefania, primary, Diomedi-Camassei, Francesca, primary, Bailey, Simon, primary, Moore, Andrew S., primary, Hassall, Timothy E.G., primary, Lowis, Stephen P., primary, Tsoli, Maria, primary, Cowley, Mark J., primary, Ziegler, David S., primary, Karajannis, Matthias A., primary, Aquilina, Kristian, primary, Hargrave, Darren R., primary, Carceller, Fernando, primary, Marshall, Lynley V., primary, von Deimling, Andreas, primary, Kramm, Christof M., primary, Pfister, Stefan M., primary, Sahm, Felix, primary, Baker, Suzanne J., primary, Mastronuzzi, Angela, primary, Carai, Andrea, primary, Vinci, Maria, primary, Capper, David, primary, Popov, Sergey, primary, Ellison, David W., primary, Jacques, Thomas S., primary, Jones, David T.W., primary, and Jones, Chris, primary

Published
2023
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8. Supplementary Table S3 from Infant High-Grade Gliomas Comprise Multiple Subgroups Characterized by Novel Targetable Gene Fusions and Favorable Outcomes

Author

Clarke, Matthew, primary, Mackay, Alan, primary, Ismer, Britta, primary, Pickles, Jessica C., primary, Tatevossian, Ruth G., primary, Newman, Scott, primary, Bale, Tejus A., primary, Stoler, Iris, primary, Izquierdo, Elisa, primary, Temelso, Sara, primary, Carvalho, Diana M., primary, Molinari, Valeria, primary, Burford, Anna, primary, Howell, Louise, primary, Virasami, Alex, primary, Fairchild, Amy R., primary, Avery, Aimee, primary, Chalker, Jane, primary, Kristiansen, Mark, primary, Haupfear, Kelly, primary, Dalton, James D., primary, Orisme, Wilda, primary, Wen, Ji, primary, Hubank, Michael, primary, Kurian, Kathreena M., primary, Rowe, Catherine, primary, Maybury, Mellissa, primary, Crosier, Stephen, primary, Knipstein, Jeffrey, primary, Schüller, Ulrich, primary, Kordes, Uwe, primary, Kram, David E., primary, Snuderl, Matija, primary, Bridges, Leslie, primary, Martin, Andrew J., primary, Doey, Lawrence J., primary, Al-Sarraj, Safa, primary, Chandler, Christopher, primary, Zebian, Bassel, primary, Cairns, Claire, primary, Natrajan, Rachael, primary, Boult, Jessica K.R., primary, Robinson, Simon P., primary, Sill, Martin, primary, Dunkel, Ira J., primary, Gilheeney, Stephen W., primary, Rosenblum, Marc K., primary, Hughes, Debbie, primary, Proszek, Paula Z., primary, Macdonald, Tobey J., primary, Preusser, Matthias, primary, Haberler, Christine, primary, Slavc, Irene, primary, Packer, Roger, primary, Ng, Ho-Keung, primary, Caspi, Shani, primary, Popović, Mara, primary, Faganel Kotnik, Barbara, primary, Wood, Matthew D., primary, Baird, Lissa, primary, Davare, Monika Ashok, primary, Solomon, David A., primary, Olsen, Thale Kristin, primary, Brandal, Petter, primary, Farrell, Michael, primary, Cryan, Jane B., primary, Capra, Michael, primary, Karremann, Michael, primary, Schittenhelm, Jens, primary, Schuhmann, Martin U., primary, Ebinger, Martin, primary, Dinjens, Winand N.M., primary, Kerl, Kornelius, primary, Hettmer, Simone, primary, Pietsch, Torsten, primary, Andreiuolo, Felipe, primary, Driever, Pablo Hernáiz, primary, Korshunov, Andrey, primary, Hiddingh, Lotte, primary, Worst, Barbara C., primary, Sturm, Dominik, primary, Zuckermann, Marc, primary, Witt, Olaf, primary, Bloom, Tabitha, primary, Mitchell, Clare, primary, Miele, Evelina, primary, Colafati, Giovanna Stefania, primary, Diomedi-Camassei, Francesca, primary, Bailey, Simon, primary, Moore, Andrew S., primary, Hassall, Timothy E.G., primary, Lowis, Stephen P., primary, Tsoli, Maria, primary, Cowley, Mark J., primary, Ziegler, David S., primary, Karajannis, Matthias A., primary, Aquilina, Kristian, primary, Hargrave, Darren R., primary, Carceller, Fernando, primary, Marshall, Lynley V., primary, von Deimling, Andreas, primary, Kramm, Christof M., primary, Pfister, Stefan M., primary, Sahm, Felix, primary, Baker, Suzanne J., primary, Mastronuzzi, Angela, primary, Carai, Andrea, primary, Vinci, Maria, primary, Capper, David, primary, Popov, Sergey, primary, Ellison, David W., primary, Jacques, Thomas S., primary, Jones, David T.W., primary, and Jones, Chris, primary

Published
2023
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10. The Children’s Brain Tumor Network (CBTN) - Accelerating Research in Pediatric Central Nervous System Tumors through Collaboration and Open Science

Author

Lilly, Jena V., primary, Rokita, Jo Lynne, additional, Mason, Jennifer L., additional, Patton, Tatiana, additional, Stefankiewiz, Stephanie, additional, Higgins, David, additional, Trooskin, Gerri, additional, Larouci, Carina A., additional, Arya, Kamnaa, additional, Appert, Elizabeth, additional, Heath, Allison P., additional, Zhu, Yuankun, additional, Brown, Miguel A., additional, Zhang, Bo, additional, Farrow, Bailey K., additional, Robins, Shannon, additional, Morgan, Allison M., additional, Nguyen, Thinh Q., additional, Frenkel, Elizabeth, additional, Lehmann, Kaitlin, additional, Drake, Emily, additional, Sullivan, Catherine, additional, Plisiewicz, Alexa, additional, Coleman, Noel, additional, Patterson, Luke, additional, Koptyra, Mateusz, additional, Helili, Zeinab, additional, Van Kuren, Nicholas, additional, Young, Nathan, additional, Kim, Meen Chul, additional, Friedman, Christopher, additional, Lubneuski, Alex, additional, Blackden, Christopher, additional, Williams, Marti, additional, Baubet, Valerie, additional, Tauhid, Lamiya, additional, Galanaugh, Jamie, additional, Boucher, Katie, additional, Ijaz, Heba, additional, Cole, Kristina A., additional, Choudhari, Namrata, additional, Santi, Mariarita, additional, Moulder, Robert W., additional, Waller, Jonathan, additional, Rife, Whitney, additional, Diskin, Sharon J., additional, Mateos, Marion, additional, Parsons, Donald W., additional, Pollack, Ian F., additional, Goldman, Stewart, additional, Leary, Sarah, additional, Caporalini, Chiara, additional, Buccoliero, Anna Maria, additional, Scagnet, Mirko, additional, Haussler, David, additional, Hanson, Derek, additional, Firestein, Ron, additional, Cain, Jason, additional, Phillips, Joanna J., additional, Gupta, Nalin, additional, Mueller, Sabine, additional, Grant, Gerald, additional, Monje-Deisseroth, Michelle, additional, Partap, Sonia, additional, Greenfield, Jeffrey P., additional, Hashizume, Rintaro, additional, Smith, Amy, additional, Zhu, Shida, additional, Johnston, James M., additional, Fangusaro, Jason R, additional, Miller, Matthew, additional, Wood, Matthew D., additional, Gardner, Sharon, additional, Carter, Claire L., additional, Prolo, Laura M., additional, Pisapia, Jared, additional, Pehlivan, Katherine, additional, Franson, Andrea, additional, Niazi, Toba, additional, Rubin, Josh, additional, Abdelbaki, Mohamed, additional, Ziegler, David S., additional, Lindsay, Holly B., additional, Stucklin, Ana Guerreiro, additional, Gerber, Nicolas, additional, Vaske, Olena M., additional, Quinsey, Carolyn, additional, Rood, Brian R., additional, Nazarian, Javad, additional, Raabe, Eric, additional, Jackson, Eric M., additional, Stapleton, Stacie, additional, Lober, Robert M., additional, Kram, David E., additional, Koschmann, Carl, additional, Storm, Phillip B., additional, Lulla, Rishi R., additional, Prados, Michael, additional, Resnick, Adam C., additional, and Waanders, Angela J., additional

Published
2022
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13. Integrated Proteogenomic Characterization across Major Histological Types of Pediatric Brain Cancer

Author

Petralia, Francesca, primary, Tignor, Nicole, additional, Reva, Boris, additional, Koptyra, Mateusz, additional, Chowdhury, Shrabanti, additional, Rykunov, Dmitry, additional, Krek, Azra, additional, Ma, Weiping, additional, Zhu, Yuankun, additional, Ji, Jiayi, additional, Calinawan, Anna, additional, Whiteaker, Jeffrey R., additional, Colaprico, Antonio, additional, Stathias, Vasileios, additional, Omelchenko, Tatiana, additional, Song, Xiaoyu, additional, Raman, Pichai, additional, Guo, Yiran, additional, Brown, Miguel A., additional, Ivey, Richard G., additional, Szpyt, John, additional, Guha Thakurta, Sanjukta, additional, Gritsenko, Marina A., additional, Weitz, Karl K., additional, Lopez, Gonzalo, additional, Kalayci, Selim, additional, Gümüş, Zeynep H., additional, Yoo, Seungyeul, additional, da Veiga Leprevost, Felipe, additional, Chang, Hui-Yin, additional, Krug, Karsten, additional, Katsnelson, Lizabeth, additional, Wang, Ying, additional, Kennedy, Jacob J., additional, Voytovich, Uliana J., additional, Zhao, Lei, additional, Gaonkar, Krutika S., additional, Ennis, Brian M., additional, Zhang, Bo, additional, Baubet, Valerie, additional, Tauhid, Lamiya, additional, Lilly, Jena V., additional, Mason, Jennifer L., additional, Farrow, Bailey, additional, Young, Nathan, additional, Leary, Sarah, additional, Moon, Jamie, additional, Petyuk, Vladislav A., additional, Nazarian, Javad, additional, Adappa, Nithin D., additional, Palmer, James N., additional, Lober, Robert M., additional, Rivero-Hinojosa, Samuel, additional, Wang, Liang-Bo, additional, Wang, Joshua M., additional, Broberg, Matilda, additional, Chu, Rosalie K., additional, Moore, Ronald J., additional, Monroe, Matthew E., additional, Zhao, Rui, additional, Smith, Richard D., additional, Zhu, Jun, additional, Robles, Ana I., additional, Mesri, Mehdi, additional, Boja, Emily, additional, Hiltke, Tara, additional, Rodriguez, Henry, additional, Zhang, Bing, additional, Schadt, Eric E., additional, Mani, D.R., additional, Ding, Li, additional, Iavarone, Antonio, additional, Wiznerowicz, Maciej, additional, Schürer, Stephan, additional, Chen, Xi S., additional, Heath, Allison P., additional, Rokita, Jo Lynne, additional, Nesvizhskii, Alexey I., additional, Fenyö, David, additional, Rodland, Karin D., additional, Liu, Tao, additional, Gygi, Steven P., additional, Paulovich, Amanda G., additional, Resnick, Adam C., additional, Storm, Phillip B., additional, Rood, Brian R., additional, Wang, Pei, additional, Francis, Alicia, additional, Morgan, Allison M., additional, Waanders, Angela J., additional, Viaene, Angela N., additional, Buccoliero, Anna Maria, additional, Chinnaiyan, Arul M., additional, Leonard, Carina A., additional, Kline, Cassie N., additional, Caporalini, Chiara, additional, Kinsinger, Christopher R., additional, Li, Chunde, additional, Kram, David E., additional, Hanson, Derek, additional, Appert, Elizabeth, additional, Kawaler, Emily A., additional, Raabe, Eric H., additional, Jackson, Eric M., additional, Greenfield, Jeffrey P., additional, Stone, Gabrielle S., additional, Getz, Gad, additional, Grant, Gerald, additional, Teo, Guo Ci, additional, Pollack, Ian F., additional, Cain, Jason E., additional, Foster, Jessica B., additional, Phillips, Joanna J., additional, Palma, July E., additional, Ketchum, Karen A., additional, Ruggles, Kelly V., additional, Blumenberg, Lili, additional, Cornwell, Macintosh, additional, Sarmady, Mahdi, additional, Domagalski, Marcin J., additional, Cieślik, Marcin P., additional, Santi, Mariarita, additional, Li, Marilyn M., additional, Ellis, Matthew J., additional, Wyczalkowski, Matthew A., additional, Connors, Meghan, additional, Scagnet, Mirko, additional, Gupta, Nalin, additional, Edwards, Nathan J., additional, Vitanza, Nicholas A., additional, Vaske, Olena M., additional, Becher, Oren, additional, McGarvey, Peter B., additional, Firestein, Ron, additional, Mueller, Sabine, additional, Winebrake, Samuel G., additional, Dhanasekaran, Saravana Mohan, additional, Cai, Shuang, additional, Partap, Sonia, additional, Patton, Tatiana, additional, Le, Toan, additional, Lorentzen, Travis D., additional, Liu, Wenke, additional, and Bocik, William E., additional

Published
2020
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17. Infant High-Grade Gliomas Comprise Multiple Subgroups Characterized by Novel Targetable Gene Fusions and Favorable Outcomes

Author

Clarke, Matthew, primary, Mackay, Alan, additional, Ismer, Britta, additional, Pickles, Jessica C., additional, Tatevossian, Ruth G., additional, Newman, Scott, additional, Bale, Tejus A., additional, Stoler, Iris, additional, Izquierdo, Elisa, additional, Temelso, Sara, additional, Carvalho, Diana M., additional, Molinari, Valeria, additional, Burford, Anna, additional, Howell, Louise, additional, Virasami, Alex, additional, Fairchild, Amy R., additional, Avery, Aimee, additional, Chalker, Jane, additional, Kristiansen, Mark, additional, Haupfear, Kelly, additional, Dalton, James D., additional, Orisme, Wilda, additional, Wen, Ji, additional, Hubank, Michael, additional, Kurian, Kathreena M., additional, Rowe, Catherine, additional, Maybury, Mellissa, additional, Crosier, Stephen, additional, Knipstein, Jeffrey, additional, Schüller, Ulrich, additional, Kordes, Uwe, additional, Kram, David E., additional, Snuderl, Matija, additional, Bridges, Leslie, additional, Martin, Andrew J., additional, Doey, Lawrence J., additional, Al-Sarraj, Safa, additional, Chandler, Christopher, additional, Zebian, Bassel, additional, Cairns, Claire, additional, Natrajan, Rachael, additional, Boult, Jessica K.R., additional, Robinson, Simon P., additional, Sill, Martin, additional, Dunkel, Ira J., additional, Gilheeney, Stephen W., additional, Rosenblum, Marc K., additional, Hughes, Debbie, additional, Proszek, Paula Z., additional, Macdonald, Tobey J., additional, Preusser, Matthias, additional, Haberler, Christine, additional, Slavc, Irene, additional, Packer, Roger, additional, Ng, Ho-Keung, additional, Caspi, Shani, additional, Popović, Mara, additional, Faganel Kotnik, Barbara, additional, Wood, Matthew D., additional, Baird, Lissa, additional, Davare, Monika Ashok, additional, Solomon, David A., additional, Olsen, Thale Kristin, additional, Brandal, Petter, additional, Farrell, Michael, additional, Cryan, Jane B., additional, Capra, Michael, additional, Karremann, Michael, additional, Schittenhelm, Jens, additional, Schuhmann, Martin U., additional, Ebinger, Martin, additional, Dinjens, Winand N.M., additional, Kerl, Kornelius, additional, Hettmer, Simone, additional, Pietsch, Torsten, additional, Andreiuolo, Felipe, additional, Driever, Pablo Hernáiz, additional, Korshunov, Andrey, additional, Hiddingh, Lotte, additional, Worst, Barbara C., additional, Sturm, Dominik, additional, Zuckermann, Marc, additional, Witt, Olaf, additional, Bloom, Tabitha, additional, Mitchell, Clare, additional, Miele, Evelina, additional, Colafati, Giovanna Stefania, additional, Diomedi-Camassei, Francesca, additional, Bailey, Simon, additional, Moore, Andrew S., additional, Hassall, Timothy E.G., additional, Lowis, Stephen P., additional, Tsoli, Maria, additional, Cowley, Mark J., additional, Ziegler, David S., additional, Karajannis, Matthias A., additional, Aquilina, Kristian, additional, Hargrave, Darren R., additional, Carceller, Fernando, additional, Marshall, Lynley V., additional, von Deimling, Andreas, additional, Kramm, Christof M., additional, Pfister, Stefan M., additional, Sahm, Felix, additional, Baker, Suzanne J., additional, Mastronuzzi, Angela, additional, Carai, Andrea, additional, Vinci, Maria, additional, Capper, David, additional, Popov, Sergey, additional, Ellison, David W., additional, Jacques, Thomas S., additional, Jones, David T.W., additional, and Jones, Chris, additional

Published
2020
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20. dic(7;9)(p11-13;p11)

Author

Underdown, Mary J, primary, Russell, Thomas B, additional, Pettenati, Mark J, additional, and Kram, David E, additional

Published
2019
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21. Treatment of Pediatric Glioblastoma with Combination Olaparib and Temozolomide Demonstrates 2-Year Durable Response

Author

Valiakhmetova, Andge, primary, Gorelyshev, Sergey, additional, Konovalov, Alexander, additional, Trunin, Yuri, additional, Savateev, Alexander, additional, Kram, David E., additional, Severson, Eric, additional, Hemmerich, Amanda, additional, Edgerly, Claire, additional, Duncan, Daniel, additional, Britt, Nicholas, additional, Huang, Richard S.P., additional, Elvin, Julia, additional, Miller, Vincent, additional, Ross, Jeffrey S., additional, Gay, Laurie, additional, McCorkle, Joshua, additional, Rankin, Andrew, additional, Erlich, Rachel L., additional, Chudnovsky, Yakov, additional, and Ramkissoon, Shakti H., additional

Published
2019
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23. Treatment of Pediatric Glioblastoma with Combination Olaparib and Temozolomide Demonstrates 2‐Year Durable Response.

Author

Valiakhmetova, Andge, Gorelyshev, Sergey, Konovalov, Alexander, Trunin, Yuri, Savateev, Alexander, Kram, David E., Severson, Eric, Hemmerich, Amanda, Edgerly, Claire, Duncan, Daniel, Britt, Nicholas, Huang, Richard S.P., Elvin, Julia, Miller, Vincent, Ross, Jeffrey S., Gay, Laurie, McCorkle, Joshua, Rankin, Andrew, Erlich, Rachel L., and Chudnovsky, Yakov

Subjects
ANTINEOPLASTIC agents, COMBINED modality therapy, SEIZURES (Medicine), GLIOMAS, HETEROCYCLIC compounds, GENETIC mutation, SPASMS, TREATMENT effectiveness, TEMOZOLOMIDE, CYTOREDUCTIVE surgery, CHILDREN
Abstract

For pediatric patients with high‐grade gliomas, standard‐of‐care treatment includes surgery, chemotherapy, and radiation therapy; however, most patients ultimately succumb to their disease. With advances in genomic characterization of pediatric high‐grade gliomas, the use of targeted therapies in combination with current treatment modalities offer the potential to improve survival in this patient population. In this report, we present the case of a 3‐year‐old girl with glioblastoma who continues to experience an exceptional and durable response (>2 years) to the poly (ADP‐ribose) polymerase (PARP) inhibitor olaparib. Our patient presented with persistent and progressive seizure activity that upon workup was the result of a large heterogeneously enhancing, mixed cystic and solid mass in the left frontal‐parietal‐temporal region. Histopathologic analysis of resected tumor tissue confirmed the diagnosis of glioblastoma, and comprehensive genomic profiling demonstrated absence of any BRAF or H3F3A mutations. Genomic profiling, however, did reveal a probable germline heterozygous BRCA2 Lys3326Ter (K3226*) nonsense variant. After debulking surgery, the patient received standard‐of‐care treatment with radiation and temozolomide. Nine months later the PARP inhibitor olaparib was administered in combination with temozolomide for 16 cycles. This regimen was well tolerated by the patient and serial imaging showed reduction in tumor size. Since completion of the regimen, the patient remains neurologically intact with no evidence of tumor recurrence. To our knowledge, this represents the first case of a pediatric glioblastoma that maintains a durable response to a therapeutic strategy that included the PARP inhibitor olaparib and more generally highlights the potential clinical utility of incorporating these agents into the treatment of pediatric high‐grade gliomas. Key Points: Germline mutations detected in pediatric gliomas may represent a cancer predisposition syndrome.Integrating molecular testing into routine clinical care for pediatric patients with glioma is critical to identify therapeutic targets and patients with a cancer predisposition syndrome.Patients with glioma with defects in DNA repair pathway components (e.g., BRCA1/2) may show increased responsiveness to poly (ADP‐ribose) polymerase (PARP) inhibitors.Combining PARP inhibitors with temozolomide (standard‐of‐care treatment) revealed no adverse events or toxicities over the course of 18 months. This article reports the first case of a pediatric glioblastoma that maintained a durable response to a therapeutic strategy that included the PARP inhibitor olaparib, highlighting the potential clinical utility of incorporating these agents into the treatment of pediatric high‐grade gliomas. [ABSTRACT FROM AUTHOR]

Published
2020
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25. Embryonal Tumors of the Central Nervous System in Children: The Era of Targeted Therapeutics.

Author

Kram, David E., Henderson, Jacob J., Baig, Muhammad, Chakraborty, Diya, Gardner, Morgan A., Biswas, Subhasree, and Khatua, Soumen

Subjects
*EMBRYONAL tumors, *CENTRAL nervous system physiology, *TUMOR treatment, *TUMORS in children, *MEDULLOBLASTOMA
Abstract

Embryonal tumors (ET) of the central nervous system (CNS) in children encompass a wide clinical spectrum of aggressive malignancies. Until recently, the overlapping morphological features of these lesions posed a diagnostic challenge and undermined discovery of optimal treatment strategies. However, with the advances in genomic technology and the outpouring of biological data over the last decade, clear insights into the molecular heterogeneity of these tumors are now well delineated. The major subtypes of ETs of the CNS in children include medulloblastoma, atypical teratoid rhabdoid tumor (ATRT), and embryonal tumors with multilayered rosettes (ETMR), which are now biologically and clinically characterized as different entities. These important developments have paved the way for treatments guided by risk stratification as well as novel targeted therapies in efforts to improve survival and reduce treatment burden. [ABSTRACT FROM AUTHOR]

Published
2018
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27. dic(7;9)(p11-13;p11).

Author

Underdown MJ, Russell TB, Pettenati MJ, and Kram DE

Abstract

Dicentric (7;9)(p11-13;p11) is a rare but recurrent abnormality in pediatric and adult precursor B acute lymphoblastic leukemia (B-ALL). The rarity precludes a deep understanding of its biology and associated prognosis. However, recent findings have correlated dic(7;9) and PAX5 mutations, highlighting this cytogenetic event's involvement in leukemogenesis and may also shed light on the overall prognosis of dic(7;9) B-ALL.

Published
2006
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