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2. Genomic predictors of response to PD-1 inhibition in children with germline DNA replication repair deficiency

Author

Das, Anirban, Sudhaman, Sumedha, Morgenstern, Daniel, Coblentz, Ailish, Chung, Jiil, Stone, Simone C, Alsafwani, Noor, Liu, Zhihui Amy, Karsaneh, Ola Abu Al, Soleimani, Shirin, Ladany, Hagay, Chen, David, Zatzman, Matthew, Cabric, Vanja, Nobre, Liana, Bianchi, Vanessa, Edwards, Melissa, Sambira Nahum, Lauren C, Ercan, Ayse B, Nabbi, Arash, Constantini, Shlomi, Dvir, Rina, Yalon-Oren, Michal, Campino, Gadi Abebe, Caspi, Shani, Larouche, Valerie, Reddy, Alyssa, Osborn, Michael, Mason, Gary, Lindhorst, Scott, Bronsema, Annika, Magimairajan, Vanan, Opocher, Enrico, De Mola, Rebecca Loret, Sabel, Magnus, Frojd, Charlotta, Sumerauer, David, Samuel, David, Cole, Kristina, Chiaravalli, Stefano, Massimino, Maura, Tomboc, Patrick, Ziegler, David S, George, Ben, Van Damme, An, Hijiya, Nobuko, Gass, David, McGee, Rose B, Mordechai, Oz, Bowers, Daniel C, Laetsch, Theodore W, Lossos, Alexander, Blumenthal, Deborah T, Sarosiek, Tomasz, Yen, Lee Yi, Knipstein, Jeffrey, Bendel, Anne, Hoffman, Lindsey M, Luna-Fineman, Sandra, Zimmermann, Stefanie, Scheers, Isabelle, Nichols, Kim E, Zapotocky, Michal, Hansford, Jordan R, Maris, John M, Dirks, Peter, Taylor, Michael D, Kulkarni, Abhaya V, Shroff, Manohar, Tsang, Derek S, Villani, Anita, Xu, Wei, Aronson, Melyssa, Durno, Carol, Shlien, Adam, Malkin, David, Getz, Gad, Maruvka, Yosef E, Ohashi, Pamela S, Hawkins, Cynthia, Pugh, Trevor J, Bouffet, Eric, and Tabori, Uri

Subjects
Rare Diseases, Pediatric, Cancer, Neurosciences, Pediatric Cancer, Genetics, 2.1 Biological and endogenous factors, Aetiology, Adolescent, Adult, B7-H1 Antigen, Biomarkers, Tumor, Child, DNA Repair, DNA Replication, Female, Germ-Line Mutation, Humans, Immune Checkpoint Inhibitors, Male, Neoplasms, Prospective Studies, Retrospective Studies, Survival Analysis, Tumor Microenvironment, Young Adult, Medical and Health Sciences, Immunology
Abstract

Cancers arising from germline DNA mismatch repair deficiency or polymerase proofreading deficiency (MMRD and PPD) in children harbour the highest mutational and microsatellite insertion-deletion (MS-indel) burden in humans. MMRD and PPD cancers are commonly lethal due to the inherent resistance to chemo-irradiation. Although immune checkpoint inhibitors (ICIs) have failed to benefit children in previous studies, we hypothesized that hypermutation caused by MMRD and PPD will improve outcomes following ICI treatment in these patients. Using an international consortium registry study, we report on the ICI treatment of 45 progressive or recurrent tumors from 38 patients. Durable objective responses were observed in most patients, culminating in a 3 year survival of 41.4%. High mutation burden predicted response for ultra-hypermutant cancers (>100 mutations per Mb) enriched for combined MMRD + PPD, while MS-indels predicted response in MMRD tumors with lower mutation burden (10-100 mutations per Mb). Furthermore, both mechanisms were associated with increased immune infiltration even in 'immunologically cold' tumors such as gliomas, contributing to the favorable response. Pseudo-progression (flare) was common and was associated with immune activation in the tumor microenvironment and systemically. Furthermore, patients with flare who continued ICI treatment achieved durable responses. This study demonstrates improved survival for patients with tumors not previously known to respond to ICI treatment, including central nervous system and synchronous cancers, and identifies the dual roles of mutation burden and MS-indels in predicting sustained response to immunotherapy.

Published
2022

3. Lorlatinib with or without chemotherapy in ALK-driven refractory/relapsed neuroblastoma: phase 1 trial results

Author

Goldsmith, Kelly C., Park, Julie R., Kayser, Kimberly, Malvar, Jemily, Chi, Yueh-Yun, Groshen, Susan G., Villablanca, Judith G., Krytska, Kateryna, Lai, Lillian M., Acharya, Patricia T., Goodarzian, Fariba, Pawel, Bruce, Shimada, Hiroyuki, Ghazarian, Susan, States, Lisa, Marshall, Lynley, Chesler, Louis, Granger, Meaghan, Desai, Ami V., Mody, Rajen, Morgenstern, Daniel A., Shusterman, Suzanne, Macy, Margaret E., Pinto, Navin, Schleiermacher, Gudrun, Vo, Kieuhoa, Thurm, Holger C., Chen, Joseph, Liyanage, Marlon, Peltz, Gerson, Matthay, Katherine K., Berko, Esther R., Maris, John M., Marachelian, Araz, and Mossé, Yael P.

Published
2023
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4. The clinical utility of integrative genomics in childhood cancer extends beyond targetable mutations

Author

Villani, Anita, Davidson, Scott, Kanwar, Nisha, Lo, Winnie W., Li, Yisu, Cohen-Gogo, Sarah, Fuligni, Fabio, Edward, Lisa-Monique, Light, Nicholas, Layeghifard, Mehdi, Harripaul, Ricardo, Waldman, Larissa, Gallinger, Bailey, Comitani, Federico, Brunga, Ledia, Hayes, Reid, Anderson, Nathaniel D., Ramani, Arun K., Yuki, Kyoko E., Blay, Sasha, Johnstone, Brittney, Inglese, Cara, Hammad, Rawan, Goudie, Catherine, Shuen, Andrew, Wasserman, Jonathan D., Venier, Rosemarie E., Eliou, Marianne, Lorenti, Miranda, Ryan, Carol Ann, Braga, Michael, Gloven-Brown, Meagan, Han, Jianan, Montero, Maria, Spatare, Famida, Whitlock, James A., Scherer, Stephen W., Chun, Kathy, Somerville, Martin J., Hawkins, Cynthia, Abdelhaleem, Mohamed, Ramaswamy, Vijay, Somers, Gino R., Kyriakopoulou, Lianna, Hitzler, Johann, Shago, Mary, Morgenstern, Daniel A., Tabori, Uri, Meyn, Stephen, Irwin, Meredith S., Malkin, David, and Shlien, Adam

Published
2023
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6. Characterisation of Paediatric Neuroblastic Tumours by Quantitative Structural and Diffusion-Weighted MRI.

Author

Tambasco, Domenica, Zlotnik, Margalit, Joshi, Sayali, Moineddin, Rahim, Harris, Shelley, Villani, Anita, Malkin, David, Morgenstern, Daniel A., and Doria, Andrea S.

Abstract

Purpose: To determine the diagnostic accuracy of quantitative diffusion-weighted (DW) MRI apparent diffusion coefficient (ADC) and tumour volumes to differentiate between malignant (neuroblastoma (NB)) and benign types of neuroblastic tumours (ganglioneuroma (GN) and ganglioneuroblastoma (GNB)) using different region-of-interest (ROI) sizes. Materials and Methods: This single-centre retrospective study included malignant and benign paediatric neuroblastic tumours that had undergone DW MRI at diagnosis. The outcome was diagnostic accuracy of the tumour volume from structural and ADC DW MRI, in comparison to histopathology (reference standard). Results: Data from 40 patients (NB, n = 24; GNB, n = 6; GN, n = 10), 18 (45%) females and 22 (55%) males, with a median age at diagnosis of 21 months (NB), 64 months (GNB), and 133 months (GN), respectively, ranging from 0 to 193 months, were evaluated. The area under the receiver operating characteristic (AUROC) curve for ADC for discriminating between neuroblastic tumours' histopathology for a small ROI was 0.86 (95% CI: 0.75–0.98), and for a large ROI, 0.83 (95% CI: 0.71–0.96). An ADC cut-off value of 1.06 × 10−3 mm2/s was able to distinguish malignant from benign tumours with 83% (68–98%) sensitivity and 75% (95% CI: 54–98%) specificity. Tumour volume was not indicative of malignant vs. benign tumour diagnosis. Conclusions: In this study, both small and large ROIs used to derive ADC DW MRI metrics demonstrated high accuracy to differentiate malignant from benign neuroblastic tumours, with the ADC AUROC for the averaged multiple small ROIs being slightly greater than that of large ROIs, but with overlapping 95% CIs. This should be taken into consideration for standardisation of ROI-related data analysis by international initiatives. [ABSTRACT FROM AUTHOR]

Published
2024
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11. Supplementary Figures S1-S5 from Combined Immunotherapy Improves Outcome for Replication-Repair-Deficient (RRD) High-Grade Glioma Failing Anti–PD-1 Monotherapy: A Report from the International RRD Consortium

Author

Das, Anirban, primary, Fernandez, Nicholas R., primary, Levine, Adrian, primary, Bianchi, Vanessa, primary, Stengs, Lucie K., primary, Chung, Jiil, primary, Negm, Logine, primary, Dimayacyac, Jose Rafael, primary, Chang, Yuan, primary, Nobre, Liana, primary, Ercan, Ayse B., primary, Sanchez-Ramirez, Santiago, primary, Sudhaman, Sumedha, primary, Edwards, Melissa, primary, Larouche, Valerie, primary, Samuel, David, primary, Van Damme, An, primary, Gass, David, primary, Ziegler, David S., primary, Bielack, Stefan S., primary, Koschmann, Carl, primary, Zelcer, Shayna, primary, Yalon-Oren, Michal, primary, Campino, Gadi Abede, primary, Sarosiek, Tomasz, primary, Nichols, Kim E., primary, Loret De Mola, Rebecca, primary, Bielamowicz, Kevin, primary, Sabel, Magnus, primary, Frojd, Charlotta A., primary, Wood, Matthew D., primary, Glover, Jason M., primary, Lee, Yi-Yen, primary, Vanan, Magimairajan, primary, Adamski, Jenny K., primary, Perreault, Sebastien, primary, Chamdine, Omar, primary, Hjort, Magnus Aasved, primary, Zapotocky, Michal, primary, Carceller, Fernando, primary, Wright, Erin, primary, Fedorakova, Ivana, primary, Lossos, Alexander, primary, Tanaka, Ryuma, primary, Osborn, Michael, primary, Blumenthal, Deborah T., primary, Aronson, Melyssa, primary, Bartels, Ute, primary, Huang, Annie, primary, Ramaswamy, Vijay, primary, Malkin, David, primary, Shlien, Adam, primary, Villani, Anita, primary, Dirks, Peter B., primary, Pugh, Trevor J., primary, Getz, Gad, primary, Maruvka, Yosef E., primary, Tsang, Derek S., primary, Ertl-Wagner, Birgit, primary, Hawkins, Cynthia, primary, Bouffet, Eric, primary, Morgenstern, Daniel A., primary, and Tabori, Uri, primary

Published
2024
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12. Access to innovative therapies in pediatric oncology: Report of the nationwide experience in Canada

Author

Judd, Sandra, primary, Revon‐Riviere, Gabriel, additional, Grover, Stephanie A., additional, Deyell, Rebecca J., additional, Vanan, Magimairajan Issai, additional, Lewis, Victor A., additional, Pecheux, Lucie, additional, Zorzi, Alexandra P., additional, Goudie, Catherine, additional, Santiago, Raoul, additional, Tran, Thai Hoa, additional, Abbott, Lesleigh S., additional, Brossard, Josee, additional, Moorehead, Paul, additional, Alvi, Saima, additional, Portwine, Carol, additional, Denburg, Avram, additional, Whitlock, James A., additional, Cohen‐Gogo, Sarah, additional, and Morgenstern, Daniel A., additional

Published
2024
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14. Combined Immunotherapy Improves Outcome for Replication-Repair-Deficient (RRD) High-Grade Glioma Failing Anti–PD-1 Monotherapy: A Report from the International RRD Consortium

Author

Das, Anirban, primary, Fernandez, Nicholas R., additional, Levine, Adrian, additional, Bianchi, Vanessa, additional, Stengs, Lucie K., additional, Chung, Jiil, additional, Negm, Logine, additional, Dimayacyac, Jose Rafael, additional, Chang, Yuan, additional, Nobre, Liana, additional, Ercan, Ayse B., additional, Sanchez-Ramirez, Santiago, additional, Sudhaman, Sumedha, additional, Edwards, Melissa, additional, Larouche, Valerie, additional, Samuel, David, additional, Van Damme, An, additional, Gass, David, additional, Ziegler, David S., additional, Bielack, Stefan S., additional, Koschmann, Carl, additional, Zelcer, Shayna, additional, Yalon-Oren, Michal, additional, Campino, Gadi Abede, additional, Sarosiek, Tomasz, additional, Nichols, Kim E., additional, Loret De Mola, Rebecca, additional, Bielamowicz, Kevin, additional, Sabel, Magnus, additional, Frojd, Charlotta A., additional, Wood, Matthew D., additional, Glover, Jason M., additional, Lee, Yi-Yen, additional, Vanan, Magimairajan, additional, Adamski, Jenny K., additional, Perreault, Sebastien, additional, Chamdine, Omar, additional, Hjort, Magnus Aasved, additional, Zapotocky, Michal, additional, Carceller, Fernando, additional, Wright, Erin, additional, Fedorakova, Ivana, additional, Lossos, Alexander, additional, Tanaka, Ryuma, additional, Osborn, Michael, additional, Blumenthal, Deborah T., additional, Aronson, Melyssa, additional, Bartels, Ute, additional, Huang, Annie, additional, Ramaswamy, Vijay, additional, Malkin, David, additional, Shlien, Adam, additional, Villani, Anita, additional, Dirks, Peter B., additional, Pugh, Trevor J., additional, Getz, Gad, additional, Maruvka, Yosef E., additional, Tsang, Derek S., additional, Ertl-Wagner, Birgit, additional, Hawkins, Cynthia, additional, Bouffet, Eric, additional, Morgenstern, Daniel A., additional, and Tabori, Uri, additional

Published
2024
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16. Metastatic Neuroblastoma Confined to Distant Lymph Nodes (stage 4N) Predicts Outcome in Patients With Stage 4 Disease: A Study From the International Neuroblastoma Risk Group Database

Author

Morgenstern, Daniel A, London, Wendy B, Stephens, Derek, Volchenboum, Samuel L, Hero, Barbara, Di Cataldo, Andrea, Nakagawara, Akira, Shimada, Hiroyuki, Ambros, Peter F, Matthay, Katherine K, Cohn, Susan L, Pearson, Andrew DJ, and Irwin, Meredith S

Subjects
Cancer, Pediatric, Rare Diseases, Neuroblastoma, Neurosciences, Clinical Research, Cohort Studies, Disease-Free Survival, Humans, Infant, Loss of Heterozygosity, Lymph Nodes, Lymphatic Metastasis, Neoplasm Metastasis, Neoplasm Staging, Prognosis, Retrospective Studies, Risk Factors, Treatment Outcome, Clinical Sciences, Oncology and Carcinogenesis, Oncology & Carcinogenesis
Abstract

PurposeThe presence of distant metastases is one of the most powerful predictors of outcome in patients with neuroblastoma. However, the pattern of metastatic spread is not incorporated into current risk stratification systems. Small case series have suggested that patients with neuroblastoma who have metastatic disease limited to distant lymph nodes (4N disease) may have improved outcomes.Patients and methodsWe analyzed retrospective data from the International Neuroblastoma Risk Group database for patients diagnosed from 1990 to 2002. 4N patients were compared with the remaining stage 4 patients (non-4N), excluding those with missing metastatic site data.ResultsIn all, 2,250 International Neuroblastoma Staging System stage 4 patients with complete data were identified, of whom 146 (6.5%) had 4N disease. For 4N patients, event-free survival (EFS; 5-year, 77% ± 4%) and overall survival (OS; 5-year, 85% ± 3%) were significantly better than EFS (5-year, 35% ± 1%) and OS (5-year, 42% ± 1%) for non-4N stage 4 patients (P < .001). 4N patients were more likely to be younger (P < .001) and have tumors with favorable characteristics, including absence of MYCN amplification (89% v 69%; P < .001). In a multivariable analysis, 4N disease remained a significant predictor of outcome (hazard ratio for non-4N v 4N: 3.40 for EFS and 3.69 for OS). Within subgroups defined by age at diagnosis and tumor MYCN status, 4N disease was significantly associated with improved outcomes.Conclusion4N represents a subgroup with better outcome than that of other patients with metastatic disease. These findings suggest that the biology and treatment response of 4N tumors differ from other stage 4 tumors, and less intensive therapy should be considered for this cohort. Future exploration of biologic factors determining the pattern of metastatic spread is warranted.

Published
2014

18. IMMU-24. SALVAGE IMMUNOTHERAPIES FOR REPLICATION REPAIR DEFICIENT (RRD) HIGH-GRADE GLIOMA FAILING ANTI-PD1 MONOTHERAPY: A REPORT FROM THE INTERNATIONAL RRD CONSORTIUM

Author

Das, Anirban, primary, Fernandez, Nicholas, additional, Levine, Adrian, additional, Bianchi, Vanessa, additional, Stengs, Lucie, additional, Edwards, Melissa, additional, Nobre, Liana, additional, Tsang, Derek, additional, Ertl-Wagner, Birgit, additional, Morgenstern, Daniel, additional, Shlien, Adam, additional, Hawkins, Cynthia, additional, Bouffet, Eric, additional, Tabori, Uri, additional, and Pugh, Trevor, additional

Published
2023
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19. 783 Combined immunotherapy improves outcome for replication repair deficient (RRD) high-grade glioma failing anti-PD1 monotherapy: a report from the International RRD consortium

Author

Das, Anirban, primary, Fernandez, Nicholas, additional, Levine, Adrian, additional, Bianchi, Vanessa, additional, Stengs, Lucie, additional, Edwards, Melissa, additional, Pugh, Trevor, additional, Tsang, Derek, additional, Ertl-Wagner, Birgit, additional, Morgenstern, Daniel, additional, Hawkins, Cynthia, additional, Bouffet, Eric, additional, and Tabori, Uri, additional

Published
2023
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20. Persisting inequalities in survival patterns of childhood neuroblastoma in Southern and Eastern Europe and the effect of socio-economic development compared with those of the US

Author

Panagopoulou, Paraskevi, Georgakis, Marios K., Baka, Margarita, Moschovi, Maria, Papadakis, Vassilios, Polychronopoulou, Sophia, Kourti, Maria, Hatzipantelis, Emmanuel, Stiakaki, Eftichia, Dana, Helen, Tragiannidis, Athanasios, Bouka, Evdoxia, Antunes, Luis, Bastos, Joana, Coza, Daniela, Demetriou, Anna, Agius, Domenic, Eser, Sultan, Gheorghiu, Raluca, Šekerija, Mario, Trojanowski, Maciej, Žagar, Tina, Zborovskaya, Anna, Ryzhov, Anton, Dessypris, Nick, Morgenstern, Daniel, and Petridou, Eleni Th

Published
2018
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21. Renal Implications of Long-Term Systemic Bevacizumab for Recurrent Respiratory Papillomatosis.

Author

Robinson, Cal H., Hart-Matyas, Michael, Morgenstern, Daniel A., Noone, Damien, and Campisi, Paolo

Subjects
VASCULAR endothelial growth factor antagonists, HYPERTENSION, BIOPSY, PAPILLOMA, CANCER relapse, ACE inhibitors, RESPIRATORY organ tumors, PROTEINURIA, BEVACIZUMAB, THERAPEUTIC complications, THROMBOCYTOPENIA, KIDNEY glomerulus
Abstract

Background: Bevacizumab is a vascular endothelial growth factor (VEGF) inhibitor that is used off-label for select cases of recurrent respiratory papillomatosis (RRP) that are severe, involve the distal airway or lung parenchyma, and refractory to other forms of adjuvant therapy. However, there is limited safety data for the use of bevacizumab in children and VEGF inhibitors are reported to have a range of adverse renal effects, including hypertension, proteinuria, and thrombotic microangiopathy (TMA). Case-Diagnosis/Treatment: This report describes a case of severe juvenile-onset RRP that had an exceptionally high operative burden that was refractory to several adjuvant treatment strategies (including intralesional cidofovir and subcutaneous pegylated interferon). Bevacizumab treatment resulted in a dramatic and sustained improvement in disease control over a 5-year period. However, after 3 years of treatment, the patient developed hypertension and proteinuria and was found to have evidence of a glomerular TMA on kidney biopsy. These complications were successfully managed with a reduction in bevacizumab frequency and angiotensin-converting enzyme inhibitor initiation. Conclusions: Clinicians caring for children treated with VEGF inhibitors should be aware of the potential renal complications and their management. [ABSTRACT FROM AUTHOR]

Published
2024
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22. List of Contributors

Author

Aminzadeh-Gohari, Sepideh, primary, Aveic, Sanja, additional, Aye, J., additional, Ayers, Duncan, additional, Beierle, E.A., additional, Croteau, Nicole J., additional, Dyer, Michael A., additional, Feichtinger, René Günther, additional, Grumati, Paolo, additional, Gupta, Ravi Kant, additional, Gustafson, William Clay, additional, Hung, Jung-Tung, additional, Ikegaki, Naohiko, additional, Inomistova, Mariia, additional, Irwin, Meredith S., additional, Khranovska, Natalia, additional, Kofler, Barbara, additional, Kudva, Anupa, additional, Kumar, Prerna, additional, Kumar, Rakesh, additional, La Quaglia, Michael P., additional, Matthay, Katherine K., additional, Modak, Shakeel, additional, Morgenstern, Daniel A., additional, Nguyen, Rosa, additional, Ray, Swapan K., additional, Ribatti, Domenico, additional, Saltsman, James A., additional, Schor, Nina F., additional, Shimada, Hiroyuki, additional, Skachkova, Oksana, additional, Stafman, L.L., additional, Tonini, Gian Paolo, additional, and Yu, Alice L., additional

Published
2019
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26. Trial protocol 1 from Efficacy of Nivolumab in Pediatric Cancers with High Mutation Burden and Mismatch Repair Deficiency

Author

Das, Anirban, primary, Tabori, Uri, primary, Sambira Nahum, Lauren C., primary, Collins, Natalie B., primary, Deyell, Rebecca, primary, Dvir, Rina, primary, Faure-Conter, Cecile, primary, Hassall, Timothy E., primary, Minturn, Jane E., primary, Edwards, Melissa, primary, Brookes, Elissa, primary, Bianchi, Vanessa, primary, Levine, Adrian, primary, Stone, Simone C., primary, Sudhaman, Sumedha, primary, Sanchez Ramirez, Santiago, primary, Ercan, Ayse B., primary, Stengs, Lucie, primary, Chung, Jill, primary, Negm, Logine, primary, Getz, Gad, primary, Maruvka, Yosef E., primary, Ertl-Wagner, Birgit, primary, Ohashi, Pamela S., primary, Pugh, Trevor, primary, Hawkins, Cynthia, primary, Bouffet, Eric, primary, and Morgenstern, Daniel A., primary

Published
2023
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27. Supplementary Table S1 from Efficacy of Nivolumab in Pediatric Cancers with High Mutation Burden and Mismatch Repair Deficiency

Author

Das, Anirban, primary, Tabori, Uri, primary, Sambira Nahum, Lauren C., primary, Collins, Natalie B., primary, Deyell, Rebecca, primary, Dvir, Rina, primary, Faure-Conter, Cecile, primary, Hassall, Timothy E., primary, Minturn, Jane E., primary, Edwards, Melissa, primary, Brookes, Elissa, primary, Bianchi, Vanessa, primary, Levine, Adrian, primary, Stone, Simone C., primary, Sudhaman, Sumedha, primary, Sanchez Ramirez, Santiago, primary, Ercan, Ayse B., primary, Stengs, Lucie, primary, Chung, Jill, primary, Negm, Logine, primary, Getz, Gad, primary, Maruvka, Yosef E., primary, Ertl-Wagner, Birgit, primary, Ohashi, Pamela S., primary, Pugh, Trevor, primary, Hawkins, Cynthia, primary, Bouffet, Eric, primary, and Morgenstern, Daniel A., primary

Published
2023
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28. Suppl Figure S1 from Efficacy of Nivolumab in Pediatric Cancers with High Mutation Burden and Mismatch Repair Deficiency

Author

Das, Anirban, primary, Tabori, Uri, primary, Sambira Nahum, Lauren C., primary, Collins, Natalie B., primary, Deyell, Rebecca, primary, Dvir, Rina, primary, Faure-Conter, Cecile, primary, Hassall, Timothy E., primary, Minturn, Jane E., primary, Edwards, Melissa, primary, Brookes, Elissa, primary, Bianchi, Vanessa, primary, Levine, Adrian, primary, Stone, Simone C., primary, Sudhaman, Sumedha, primary, Sanchez Ramirez, Santiago, primary, Ercan, Ayse B., primary, Stengs, Lucie, primary, Chung, Jill, primary, Negm, Logine, primary, Getz, Gad, primary, Maruvka, Yosef E., primary, Ertl-Wagner, Birgit, primary, Ohashi, Pamela S., primary, Pugh, Trevor, primary, Hawkins, Cynthia, primary, Bouffet, Eric, primary, and Morgenstern, Daniel A., primary

Published
2023
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29. Data from Efficacy of Nivolumab in Pediatric Cancers with High Mutation Burden and Mismatch Repair Deficiency

Author

Das, Anirban, primary, Tabori, Uri, primary, Sambira Nahum, Lauren C., primary, Collins, Natalie B., primary, Deyell, Rebecca, primary, Dvir, Rina, primary, Faure-Conter, Cecile, primary, Hassall, Timothy E., primary, Minturn, Jane E., primary, Edwards, Melissa, primary, Brookes, Elissa, primary, Bianchi, Vanessa, primary, Levine, Adrian, primary, Stone, Simone C., primary, Sudhaman, Sumedha, primary, Sanchez Ramirez, Santiago, primary, Ercan, Ayse B., primary, Stengs, Lucie, primary, Chung, Jill, primary, Negm, Logine, primary, Getz, Gad, primary, Maruvka, Yosef E., primary, Ertl-Wagner, Birgit, primary, Ohashi, Pamela S., primary, Pugh, Trevor, primary, Hawkins, Cynthia, primary, Bouffet, Eric, primary, and Morgenstern, Daniel A., primary

Published
2023
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30. Efficacy of Nivolumab in Pediatric Cancers with High Mutation Burden and Mismatch Repair Deficiency

Author

Das, Anirban, primary, Tabori, Uri, additional, Sambira Nahum, Lauren C., additional, Collins, Natalie B., additional, Deyell, Rebecca, additional, Dvir, Rina, additional, Faure-Conter, Cecile, additional, Hassall, Timothy E., additional, Minturn, Jane E., additional, Edwards, Melissa, additional, Brookes, Elissa, additional, Bianchi, Vanessa, additional, Levine, Adrian, additional, Stone, Simone C., additional, Sudhaman, Sumedha, additional, Sanchez Ramirez, Santiago, additional, Ercan, Ayse B., additional, Stengs, Lucie, additional, Chung, Jill, additional, Negm, Logine, additional, Getz, Gad, additional, Maruvka, Yosef E., additional, Ertl-Wagner, Birgit, additional, Ohashi, Pamela S., additional, Pugh, Trevor, additional, Hawkins, Cynthia, additional, Bouffet, Eric, additional, and Morgenstern, Daniel A., additional

Published
2023
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31. Ensuring access to innovative therapies for children, adolescents, and young adults across Canada: The single patient study experience

Author

Revon-Riviere, Gabriel, primary, Young, Leah C, additional, Stephenson, Elizabeth A, additional, Brodeur-Robb, Kathy, additional, Cohen-Gogo, Sarah, additional, Deyell, Rebecca, additional, Lacaze-Masmonteil, Thierry, additional, Palmer, Antonia, additional, Parekh, Rulan S, additional, Whitlock, James A, additional, and Morgenstern, Daniel A, additional

Published
2023
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32. Data from Mutations in the RAS/MAPK Pathway Drive Replication Repair–Deficient Hypermutated Tumors and Confer Sensitivity to MEK Inhibition

Author

Campbell, Brittany B., primary, Galati, Melissa A., primary, Stone, Simone C., primary, Riemenschneider, Alexandra N., primary, Edwards, Melissa, primary, Sudhaman, Sumedha, primary, Siddaway, Robert, primary, Komosa, Martin, primary, Nunes, Nuno M., primary, Nobre, Liana, primary, Morrissy, A. Sorana, primary, Zatzman, Matthew, primary, Zapotocky, Michal, primary, Joksimovic, Lazar, primary, Kalimuthu, Sangeetha N., primary, Samuel, David, primary, Mason, Gary, primary, Bouffet, Eric, primary, Morgenstern, Daniel A., primary, Aronson, Melyssa, primary, Durno, Carol, primary, Malkin, David, primary, Maris, John M., primary, Taylor, Michael D., primary, Shlien, Adam, primary, Pugh, Trevor J., primary, Ohashi, Pamela S., primary, Hawkins, Cynthia E., primary, and Tabori, Uri, primary

Published
2023
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33. SupplementalTables from Mutations in the RAS/MAPK Pathway Drive Replication Repair–Deficient Hypermutated Tumors and Confer Sensitivity to MEK Inhibition

Author

Campbell, Brittany B., primary, Galati, Melissa A., primary, Stone, Simone C., primary, Riemenschneider, Alexandra N., primary, Edwards, Melissa, primary, Sudhaman, Sumedha, primary, Siddaway, Robert, primary, Komosa, Martin, primary, Nunes, Nuno M., primary, Nobre, Liana, primary, Morrissy, A. Sorana, primary, Zatzman, Matthew, primary, Zapotocky, Michal, primary, Joksimovic, Lazar, primary, Kalimuthu, Sangeetha N., primary, Samuel, David, primary, Mason, Gary, primary, Bouffet, Eric, primary, Morgenstern, Daniel A., primary, Aronson, Melyssa, primary, Durno, Carol, primary, Malkin, David, primary, Maris, John M., primary, Taylor, Michael D., primary, Shlien, Adam, primary, Pugh, Trevor J., primary, Ohashi, Pamela S., primary, Hawkins, Cynthia E., primary, and Tabori, Uri, primary

Published
2023
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34. Supplemental-Figures-and-Legends from Mutations in the RAS/MAPK Pathway Drive Replication Repair–Deficient Hypermutated Tumors and Confer Sensitivity to MEK Inhibition

Author

Campbell, Brittany B., primary, Galati, Melissa A., primary, Stone, Simone C., primary, Riemenschneider, Alexandra N., primary, Edwards, Melissa, primary, Sudhaman, Sumedha, primary, Siddaway, Robert, primary, Komosa, Martin, primary, Nunes, Nuno M., primary, Nobre, Liana, primary, Morrissy, A. Sorana, primary, Zatzman, Matthew, primary, Zapotocky, Michal, primary, Joksimovic, Lazar, primary, Kalimuthu, Sangeetha N., primary, Samuel, David, primary, Mason, Gary, primary, Bouffet, Eric, primary, Morgenstern, Daniel A., primary, Aronson, Melyssa, primary, Durno, Carol, primary, Malkin, David, primary, Maris, John M., primary, Taylor, Michael D., primary, Shlien, Adam, primary, Pugh, Trevor J., primary, Ohashi, Pamela S., primary, Hawkins, Cynthia E., primary, and Tabori, Uri, primary

Published
2023
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37. Supplementary table 4 from A Promyelocytic Leukemia Protein–Thrombospondin-2 Axis and the Risk of Relapse in Neuroblastoma

Author

Dvorkina, Maria, primary, Nieddu, Valentina, primary, Chakelam, Shalini, primary, Pezzolo, Annalisa, primary, Cantilena, Sandra, primary, Leite, Ana Paula, primary, Chayka, Olesya, primary, Regad, Tarik, primary, Pistorio, Angela, primary, Sementa, Angela Rita, primary, Virasami, Alex, primary, Barton, Jack, primary, Montano, Ximena, primary, Lechertier, Tanguy, primary, Brindle, Nicola, primary, Morgenstern, Daniel, primary, Lebras, Morgane, primary, Burns, Alan J., primary, Saunders, Nigel J., primary, Hodivala-Dilke, Kairbaan, primary, Bagella, Luigi, primary, De The, Hugues, primary, Anderson, John, primary, Sebire, Neil, primary, Pistoia, Vito, primary, Sala, Arturo, primary, and Salomoni, Paolo, primary

Published
2023
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38. Data from A Promyelocytic Leukemia Protein–Thrombospondin-2 Axis and the Risk of Relapse in Neuroblastoma

Author

Dvorkina, Maria, primary, Nieddu, Valentina, primary, Chakelam, Shalini, primary, Pezzolo, Annalisa, primary, Cantilena, Sandra, primary, Leite, Ana Paula, primary, Chayka, Olesya, primary, Regad, Tarik, primary, Pistorio, Angela, primary, Sementa, Angela Rita, primary, Virasami, Alex, primary, Barton, Jack, primary, Montano, Ximena, primary, Lechertier, Tanguy, primary, Brindle, Nicola, primary, Morgenstern, Daniel, primary, Lebras, Morgane, primary, Burns, Alan J., primary, Saunders, Nigel J., primary, Hodivala-Dilke, Kairbaan, primary, Bagella, Luigi, primary, De The, Hugues, primary, Anderson, John, primary, Sebire, Neil, primary, Pistoia, Vito, primary, Sala, Arturo, primary, and Salomoni, Paolo, primary

Published
2023
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39. Supplementary table 2 from A Promyelocytic Leukemia Protein–Thrombospondin-2 Axis and the Risk of Relapse in Neuroblastoma

Author

Dvorkina, Maria, primary, Nieddu, Valentina, primary, Chakelam, Shalini, primary, Pezzolo, Annalisa, primary, Cantilena, Sandra, primary, Leite, Ana Paula, primary, Chayka, Olesya, primary, Regad, Tarik, primary, Pistorio, Angela, primary, Sementa, Angela Rita, primary, Virasami, Alex, primary, Barton, Jack, primary, Montano, Ximena, primary, Lechertier, Tanguy, primary, Brindle, Nicola, primary, Morgenstern, Daniel, primary, Lebras, Morgane, primary, Burns, Alan J., primary, Saunders, Nigel J., primary, Hodivala-Dilke, Kairbaan, primary, Bagella, Luigi, primary, De The, Hugues, primary, Anderson, John, primary, Sebire, Neil, primary, Pistoia, Vito, primary, Sala, Arturo, primary, and Salomoni, Paolo, primary

Published
2023
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40. Supplementary Table 1 from A Promyelocytic Leukemia Protein–Thrombospondin-2 Axis and the Risk of Relapse in Neuroblastoma

Author

Dvorkina, Maria, primary, Nieddu, Valentina, primary, Chakelam, Shalini, primary, Pezzolo, Annalisa, primary, Cantilena, Sandra, primary, Leite, Ana Paula, primary, Chayka, Olesya, primary, Regad, Tarik, primary, Pistorio, Angela, primary, Sementa, Angela Rita, primary, Virasami, Alex, primary, Barton, Jack, primary, Montano, Ximena, primary, Lechertier, Tanguy, primary, Brindle, Nicola, primary, Morgenstern, Daniel, primary, Lebras, Morgane, primary, Burns, Alan J., primary, Saunders, Nigel J., primary, Hodivala-Dilke, Kairbaan, primary, Bagella, Luigi, primary, De The, Hugues, primary, Anderson, John, primary, Sebire, Neil, primary, Pistoia, Vito, primary, Sala, Arturo, primary, and Salomoni, Paolo, primary

Published
2023
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47. Progression-Free Survival and Patterns of Response in Patients With Relapsed High-Risk Neuroblastoma Treated With Irinotecan/Temozolomide/Dinutuximab/Granulocyte-Macrophage Colony-Stimulating Factor

Author

Lerman, Benjamin J., primary, Li, Yimei, additional, Carlowicz, Cecilia, additional, Granger, Meaghan, additional, Cash, Thomas, additional, Sadanand, Arhanti, additional, Somers, Katherine, additional, Ranavaya, Aeesha, additional, Weiss, Brian D., additional, Choe, Michelle, additional, Foster, Jennifer H., additional, Pinto, Navin, additional, Morgenstern, Daniel A., additional, Rafael, Margarida Simão, additional, Streby, Keri A., additional, Zeno, Rachel N., additional, Mody, Rajen, additional, Yazdani, Sahr, additional, Desai, Ami V., additional, Macy, Margaret E., additional, Shusterman, Suzanne, additional, Federico, Sara M., additional, and Bagatell, Rochelle, additional

Published
2023
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48. sj-docx-1-aor-10.1177_00034894231184942 – Supplemental material for Renal Implications of Long-Term Systemic Bevacizumab for Recurrent Respiratory Papillomatosis

Author

Robinson, Cal H., Hart-Matyas, Michael, Morgenstern, Daniel A., Noone, Damien, and Campisi, Paolo

Subjects
Otorhinolaryngology
Abstract

Supplemental material, sj-docx-1-aor-10.1177_00034894231184942 for Renal Implications of Long-Term Systemic Bevacizumab for Recurrent Respiratory Papillomatosis by Cal H. Robinson, Michael Hart-Matyas, Daniel A. Morgenstern, Damien Noone and Paolo Campisi in Annals of Otology, Rhinology & Laryngology

Published
2023
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49. The clinical utility of integrative genomics in childhood cancer extends beyond targetable mutations

Author

Villani, Anita, primary, Davidson, Scott, additional, Kanwar, Nisha, additional, Lo, Winnie W., additional, Li, Yisu, additional, Cohen-Gogo, Sarah, additional, Fuligni, Fabio, additional, Edward, Lisa-Monique, additional, Light, Nicholas, additional, Layeghifard, Mehdi, additional, Harripaul, Ricardo, additional, Waldman, Larissa, additional, Gallinger, Bailey, additional, Comitani, Federico, additional, Brunga, Ledia, additional, Hayes, Reid, additional, Anderson, Nathaniel D., additional, Ramani, Arun K., additional, Yuki, Kyoko E., additional, Blay, Sasha, additional, Johnstone, Brittney, additional, Inglese, Cara, additional, Hammad, Rawan, additional, Goudie, Catherine, additional, Shuen, Andrew, additional, Wasserman, Jonathan D., additional, Venier, Rosemarie E., additional, Eliou, Marianne, additional, Lorenti, Miranda, additional, Ryan, Carol Ann, additional, Braga, Michael, additional, Gloven-Brown, Meagan, additional, Han, Jianan, additional, Montero, Maria, additional, Spatare, Famida, additional, Whitlock, James A., additional, Scherer, Stephen W., additional, Chun, Kathy, additional, Somerville, Martin J., additional, Hawkins, Cynthia, additional, Abdelhaleem, Mohamed, additional, Ramaswamy, Vijay, additional, Somers, Gino R., additional, Kyriakopoulou, Lianna, additional, Hitzler, Johann, additional, Shago, Mary, additional, Morgenstern, Daniel A., additional, Tabori, Uri, additional, Meyn, Stephen, additional, Irwin, Meredith S., additional, Malkin, David, additional, and Shlien, Adam, additional

Published
2022
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