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369 results on '"Miles, Rodney R."'

1. Rapid growth of acquired UBA1 mutations predisposes male patients to low-risk MDS: MYELODYSPLASTIC NEOPLASM

Author

Li, Peng, Alnoor, F. N. U., Xie, Wei, Williams, Margaret, Feusier, Julie, Ding, Yi, Zhao, Xiangrong, Zheng, Gang, Zhao, Chen, Zieske, Arthur W., Zu, Youli, Raess, Philipp W., Tantravahi, Srinivas, Osman, Afaf, Patel, Ami B., Tashi, Tsewang, Patel, Jay L., Matynia, Anna P., Menon, Madhu P., Miles, Rodney R., Jacobsen, Jeffrey R., George, Tracy I., Sborov, Douglas W., Szankasi, Philippe, Rindler, Paul, Close, Devin, and Ohgami, Robert S.

Published
2025
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2. Children's Oncology Group Trial AALL1231: A Phase III Clinical Trial Testing Bortezomib in Newly Diagnosed T-Cell Acute Lymphoblastic Leukemia and Lymphoma

Author

Teachey, David T, Devidas, Meenakshi, Wood, Brent L, Chen, Zhiguo, Hayashi, Robert J, Hermiston, Michelle L, Annett, Robert D, Archer, J Hunter, Asselin, Barbara L, August, Keith J, Cho, Steve Y, Dunsmore, Kimberly P, Fisher, Brian T, Freedman, Jason L, Galardy, Paul J, Harker-Murray, Paul, Horton, Terzah M, Jaju, Alok I, Lam, Allison, Messinger, Yoav H, Miles, Rodney R, Okada, Maki, Patel, Samir I, Schafer, Eric S, Schechter, Tal, Singh, Neelam, Steele, Amii C, Sulis, Maria Luisa, Vargas, Sarah L, Winter, Stuart S, Wood, Charlotte, Zweidler-McKay, Patrick, Bollard, Catherine M, Loh, Mignon L, Hunger, Stephen P, and Raetz, Elizabeth A

Subjects
Clinical Trials and Supportive Activities, Pediatric Cancer, Pediatric, Orphan Drug, Cancer, Rare Diseases, Clinical Research, Childhood Leukemia, Hematology, Pediatric Research Initiative, 6.1 Pharmaceuticals, Evaluation of treatments and therapeutic interventions, Antineoplastic Combined Chemotherapy Protocols, Bortezomib, Child, Disease-Free Survival, Humans, Infant, Lymphoma, Precursor Cell Lymphoblastic Leukemia-Lymphoma, Precursor T-Cell Lymphoblastic Leukemia-Lymphoma, T-Lymphocytes, Young Adult, Clinical Sciences, Oncology and Carcinogenesis, Oncology & Carcinogenesis
Abstract

PurposeTo improve the outcomes of patients with T-cell acute lymphoblastic leukemia (T-ALL) and lymphoblastic lymphoma (T-LL), the proteasome inhibitor bortezomib was examined in the Children's Oncology Group phase III clinical trial AALL1231, which also attempted to reduce the use of prophylactic cranial radiation (CRT) in newly diagnosed T-ALL.Patients and methodsChildren and young adults with T-ALL/T-LL were randomly assigned to a modified augmented Berlin-Frankfurt-Münster chemotherapy regimen with/without bortezomib during induction and delayed intensification. Multiple modifications were made to the augmented Berlin-Frankfurt-Münster backbone used in the predecessor trial, AALL0434, including using dexamethasone instead of prednisone and adding two extra doses of pegaspargase in an attempt to eliminate CRT in most patients.ResultsAALL1231 accrued 824 eligible and evaluable patients from 2014 to 2017. The 4-year event-free survival (EFS) and overall survival (OS) for arm A (no bortezomib) versus arm B (bortezomib) were 80.1% ± 2.3% versus 83.8% ± 2.1% (EFS, P = .131) and 85.7% ± 2.0% versus 88.3% ± 1.8% (OS, P = .085). Patients with T-LL had improved EFS and OS with bortezomib: 4-year EFS (76.5% ± 5.1% v 86.4% ± 4.0%; P = .041); and 4-year OS (78.3% ± 4.9% v 89.5% ± 3.6%; P = .009). No excess toxicity was seen with bortezomib. In AALL0434, 90.8% of patients with T-ALL received CRT. In AALL1231, 9.5% of patients were scheduled to receive CRT. Evaluation of comparable AALL0434 patients who received CRT and AALL1231 patients who did not receive CRT demonstrated no statistical differences in EFS (P = .412) and OS (P = .600).ConclusionPatients with T-LL had significantly improved EFS and OS with bortezomib on the AALL1231 backbone. Systemic therapy intensification allowed elimination of CRT in more than 90% of patients with T-ALL without excess relapse.

Published
2022

3. Successful Outcomes of Newly Diagnosed T Lymphoblastic Lymphoma: Results From Children's Oncology Group AALL0434.

Author

Hayashi, Robert J, Winter, Stuart S, Dunsmore, Kimberly P, Devidas, Meenakshi, Chen, Zhiguo, Wood, Brent L, Hermiston, Michelle L, Teachey, David T, Perkins, Sherrie L, Miles, Rodney R, Raetz, Elizabeth A, Loh, Mignon L, Winick, Naomi J, Carroll, William L, Hunger, Stephen P, Lim, Megan S, Gross, Thomas G, and Bollard, Catherine M

Subjects
Hematology, Clinical Trials and Supportive Activities, Rare Diseases, Patient Safety, Pediatric, Orphan Drug, Pediatric Cancer, Clinical Research, Childhood Leukemia, Cancer, Neurosciences, Pediatric Research Initiative, Evaluation of treatments and therapeutic interventions, 6.1 Pharmaceuticals, Adolescent, Adult, Age Factors, Antineoplastic Combined Chemotherapy Protocols, Arabinonucleosides, Asparaginase, Child, Child, Preschool, Female, Humans, Infant, Male, Methotrexate, Polyethylene Glycols, Precursor T-Cell Lymphoblastic Leukemia-Lymphoma, Progression-Free Survival, Prospective Studies, Time Factors, United States, Young Adult, Clinical Sciences, Oncology and Carcinogenesis, Oncology & Carcinogenesis
Abstract

PurposeThe Children's Oncology Group (COG) protocol AALL0434 evaluated the safety and efficacy of multi-agent chemotherapy with Capizzi-based methotrexate/pegaspargase (C-MTX) in patients with newly diagnosed pediatric T-cell lymphoblastic lymphoma (T-LL) and gained preliminary data using nelarabine in high-risk patients.Patients and methodsThe trial enrolled 299 patients, age 1-31 years. High-risk (HR) patients had ≥ 1% minimal detectable disease (MDD) in the bone marrow at diagnosis or received prior steroid treatment. Induction failure was defined as failure to achieve a partial response (PR) by the end of the 4-week induction. All patients received the augmented Berlin-Frankfurt-Muenster (ABFM) C-MTX regimen. HR patients were randomly assigned to receive or not receive 6 5-day courses of nelarabine incorporated into ABFM. Patients with induction failure were nonrandomly assigned to ABFM C-MTX plus nelarabine. No patients received prophylactic cranial radiation; however, patients with CNS3 disease (CSF WBC ≥ 5/μL with blasts or cranial nerve palsies, brain/eye involvement, or hypothalamic syndrome) were ineligible.ResultsAt end-induction, 98.8% of evaluable participants had at least a PR. The 4-year event-free survival (EFS) and overall survival (OS) were 84.7% ± 2.3% and 89.0% ± 2.0%. The 4-year disease-free survival (DFS) from end-induction was 85.9% ± 2.6%. There was no difference in DFS observed between the HR and standard-risk groups (P = .29) or by treatment regimen (P = .55). Disease stage, tumor response, and MDD at diagnosis did not demonstrate thresholds that resulted in differences in EFS. Nelarabine did not show an advantage for HR patients. CNS relapse occurred in only 4 patients.ConclusionCOG AALL0434 produced excellent outcomes in one of the largest trials ever conducted for patients with newly diagnosed T-LL. The COG ABFM regimen with C-MTX provided excellent EFS and OS without cranial radiation.

Published
2020

4. Ibrutinib significantly inhibited Bruton’s tyrosine kinase (BTK) phosphorylation,in-vitro proliferation and enhanced overall survival in a preclinical Burkitt lymphoma (BL) model

Author

Chu, Yaya, Lee, Sanghoon, Shah, Tishi, Yin, Changhong, Barth, Matthew, Miles, Rodney R, Ayello, Janet, Morris, Erin, Harrison, Lauren, Van de Ven, Carmella, Galardy, Paul, Goldman, Stanton C, Lim, Megan S, Hermiston, Michelle, McAllister-Lucas, Linda M, Giulino-Roth, Lisa, Perkins, Sherrie L, and Cairo, Mitchell S

Subjects
Biomedical and Clinical Sciences, Cardiovascular Medicine and Haematology, Oncology and Carcinogenesis, Hematology, Orphan Drug, Lymphoma, Cancer, Rare Diseases, Clinical Research, Pediatric, Ibrutinib, Bruton's Tyrosine Kinase, Burkitt Lymphoma, Xenografted NSG Mice, Cell Proliferation, Bruton’s Tyrosine Kinase, Immunology, Oncology and carcinogenesis
Abstract

Pediatric and adult patients with recurrent/refractory Burkitt lymphoma (BL) continue to have poor outcomes, emphasizing the need for newer therapeutic agents. Bruton's tyrosine kinase (BTK) is activated following B-cell receptor stimulation and in part regulates normal B-cell development. Ibrutinib, a selective and irreversible BTK inhibitor, has been efficacious in chronic lymphocytic leukemia (CLL), mantle cell lymphoma (MCL), Waldenström's macroglobulinemia, and marginal zone lymphoma. In this study, we investigated the efficacy of ibrutinib alone and in selective adjuvant combinations against BL in-vitro and in a human BL xenografted immune-deficient NOD.Cg-PrkdcscidIl2rgtm1Wjl/SzJ (NSG) mouse model. Our data demonstrated that phospho-BTK level was significantly reduced in BL cells treated with ibrutinib (p

Published
2019

6. Rapid growth of acquired UBA1mutations predisposes male patients to low-risk MDS

Author

Li, Peng, Alnoor, F. N. U., Xie, Wei, Williams, Margaret, Feusier, Julie, Ding, Yi, Zhao, Xiangrong, Zheng, Gang, Zhao, Chen, Zieske, Arthur W., Zu, Youli, Raess, Philipp W., Tantravahi, Srinivas, Osman, Afaf, Patel, Ami B., Tashi, Tsewang, Patel, Jay L., Matynia, Anna P., Menon, Madhu P., Miles, Rodney R., Jacobsen, Jeffrey R., George, Tracy I., Sborov, Douglas W., Szankasi, Philippe, Rindler, Paul, Close, Devin, and Ohgami, Robert S.

Published
2025
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7. miR-155 promotes FLT3-ITD-induced myeloproliferative disease through inhibition of the interferon response.

Author

Wallace, Jared A, Kagele, Dominique A, Eiring, Anna M, Kim, Carissa N, Hu, Ruozhen, Runtsch, Marah C, Alexander, Margaret, Huffaker, Thomas B, Lee, Soh-Hyun, Patel, Ami B, Mosbruger, Timothy L, Voth, Warren P, Rao, Dinesh S, Miles, Rodney R, Round, June L, Deininger, Michael W, and O'Connell, Ryan M

Subjects
Pediatric Research Initiative, Cancer, Hematology, Stem Cell Research, Childhood Leukemia, Genetics, Rare Diseases, Pediatric Cancer, Pediatric, 2.1 Biological and endogenous factors, Aetiology, Animals, CRISPR-Cas Systems, Cell Line, Tumor, Disease Models, Animal, Female, Humans, Interferons, Leukemia, Myeloid, Acute, Male, Mice, Mice, Inbred C57BL, Mice, Knockout, Mice, Mutant Strains, MicroRNAs, Mutation, Myeloid Progenitor Cells, Myelopoiesis, Myeloproliferative Disorders, Tumor Stem Cell Assay, fms-Like Tyrosine Kinase 3, Cardiorespiratory Medicine and Haematology, Clinical Sciences, Paediatrics and Reproductive Medicine, Immunology
Abstract

FLT3-ITD+ acute myeloid leukemia (AML) accounts for ∼25% of all AML cases and is a subtype that carries a poor prognosis. microRNA-155 (miR-155) is specifically overexpressed in FLT3-ITD+ AML compared with FLT3 wild-type (FLT3-WT) AML and is critical for the growth of FLT3-ITD+ AML cells in vitro. However, miR-155's role in regulating FLT3-ITD-mediated disease in vivo remains unclear. In this study, we used a genetic mouse model to determine whether miR-155 influences the development of FLT3-ITD-induced myeloproliferative disease. Results indicate that miR-155 promotes FLT3-ITD-induced myeloid expansion in the bone marrow, spleen, and peripheral blood. Mechanistically, miR-155 increases proliferation of the hematopoietic stem and progenitor cell compartments by reducing the growth-inhibitory effects of the interferon (IFN) response, and this involves targeting of Cebpb. Consistent with our observations in mice, primary FLT3-ITD+ AML clinical samples have significantly higher miR-155 levels and a lower IFN response compared with FLT3-WT AML samples. Further, inhibition of miR-155 in FLT3-ITD+ AML cell lines using CRISPR/Cas9, or primary FLT3-ITD+ AML samples using locked nucleic acid antisense inhibitors, results in an elevated IFN response and reduces colony formation. Altogether, our data reveal that miR-155 collaborates with FLT3-ITD to promote myeloid cell expansion in vivo and that this involves a multitarget mechanism that includes repression of IFN signaling.

Published
2017

8. A dose substitution of anthracycline intensity with dose-dense rituximab in children and adolescents with good-risk mature B-cell lymphoma

Author

Goldman, Stan, Barth, Matthew, Shiramizu, Bruce, Shi, Qiuhu, Hochberg, Jessica, Klejmont, Liana, Harrison, Lauren, Basso, Jackie, Chu, Yaya, Islam, Humayun, Gerard, Perry, Agsalda-Garcia, Melissa, Shieh, Tiffany, Oesterheld, Javier, Heym, Kenneth, Kirov, Ivan, Drachtman, Richard, Harker-Murray, Paul, Perkins, Sherrie, Miles, Rodney R., and Cairo, Mitchell

Published
2021
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9. Daratumumab induces mechanisms of immune activation through CD38+ NK cell targeting

Author

Viola, Domenico, Dona, Ada, Caserta, Enrico, Troadec, Estelle, Besi, Francesca, McDonald, Tinisha, Ghoda, Lucy, Gunes, Emine Gulsen, Sanchez, James F., Khalife, Jihane, Martella, Marianna, Karanes, Chatchada, Htut, Myo, Wang, Xiuli, Rosenzweig, Michael, Chowdhury, Arnab, Sborov, Douglas, Miles, Rodney R., Yazaki, Paul J., Ebner, Todd, Hofmeister, Craig C., Forman, Stephen J., Rosen, Steven T., Marcucci, Guido, Shively, John, Keats, Jonathan J., Krishnan, Amrita, and Pichiorri, Flavia

Published
2021
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11. The whole-genome landscape of Burkitt lymphoma subtypes

Author

Panea, Razvan I., Love, Cassandra L., Shingleton, Jennifer R., Reddy, Anupama, Bailey, Jeffrey A., Moormann, Ann M., Otieno, Juliana A., Ong'echa, John Michael, Oduor, Cliff I., Schroeder, Kristin M.S., Masalu, Nestory, Chao, Nelson J., Agajanian, Megan, Major, Michael B., Fedoriw, Yuri, Richards, Kristy L., Rymkiewicz, Grzegorz, Miles, Rodney R., Alobeid, Bachir, Bhagat, Govind, Flowers, Christopher R., Ondrejka, Sarah L., Hsi, Eric D., Choi, William W.L., Au-Yeung, Rex K.H., Hartmann, Wolfgang, Lenz, Georg, Meyerson, Howard, Lin, Yen-Yu, Zhuang, Yuan, Luftig, Micah A., Waldrop, Alexander, Dave, Tushar, Thakkar, Devang, Sahay, Harshit, Li, Guojie, Palus, Brooke C., Seshadri, Vidya, Kim, So Young, Gascoyne, Randy D., Levy, Shawn, Mukhopadyay, Minerva, Dunson, David B., and Dave, Sandeep S.

Published
2019
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13. ACCELERATE and European Medicine Agency Paediatric Strategy Forum for medicinal product development for mature B-cell malignancies in children

Author

Pearson, Andrew D.J., Scobie, Nicole, Norga, Koenraad, Ligas, Franca, Chiodin, Davy, Burke, Amos, Minard-Colin, Veronique, Adamson, Peter, Marshall, Lynley V., Balakumaran, Arun, Benettaib, Bouchra, Bhargava, Pankaj, Bollard, Catherine M., Bolotin, Ellen, Bomken, Simon, Buechner, Jochen, Burkhardt, Birgit, Caron, Hubert, Copland, Christopher, Demolis, Pierre, Egorov, Anton, Farhan, Mahdi, Zugmaier, Gerhard, Gross, Thomas, Horton-Taylor, Danielle, Klapper, Wolfram, Lesa, Giovanni, Marcus, Robert, Miles, Rodney R., Nottage, Kerri, Pacaud, Lida, Ricafort, Rosanna, Schrappe, Martin, Sterba, Jaroslav, Vezan, Remus, Weiner, Susan, Kim, Su Young, Reaman, Gregory, and Vassal, Gilles

Published
2019
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15. MRD at the end of induction and EFS in T-cell lymphoblastic lymphoma: Children’s Oncology Group trial AALL1231

Author

Hayashi, Robert J., Hermiston, Michelle L., Wood, Brent L., Teachey, David T., Devidas, Meenakshi, Chen, Zhiguo, Annett, Robert D., Asselin, Barbara L., August, Keith, Cho, Steve, Dunsmore, Kimberly P., Freedman, Jason Lawrence, Galardy, Paul J., Harker-Murray, Paul, Horton, Terzah M., Jaju, Alok, Lam, Allison, Messinger, Yoav H., Miles, Rodney R., Okada, Maki, Patel, Samir, Schafer, Eric S., Schechter, Tal, Shimano, Kristin A., Singh, Neelam, Steele, Amii, Sulis, Maria L., Vargas, Sarah L., Winter, Stuart S., Wood, Charlotte, Zweidler-McKay, Patrick A., Loh, Mignon L., Hunger, Stephen P., Raetz, Elizabeth A., Bollard, Catherine M., and Allen, Carl E.

Published
2024
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16. Adult high-grade B-cell lymphoma with Burkitt lymphoma signature: genomic features and potential therapeutic targets

Author

Bouska, Alyssa, Bi, Chengfeng, Lone, Waseem, Zhang, Weiwei, Kedwaii, Ambreen, Heavican, Tayla, Lachel, Cynthia M., Yu, Jiayu, Ferro, Roberto, Eldorghamy, Nanees, Greiner, Timothy C., Vose, Julie, Weisenburger, Dennis D., Gascoyne, Randy D., Rosenwald, Andreas, Ott, German, Campo, Elias, Rimsza, Lisa M., Jaffe, Elaine S., Braziel, Rita M., Siebert, Reiner, Miles, Rodney R., Dave, Sandeep, Reddy, Anupama, Delabie, Jan, Staudt, Louis M., Song, Joo Y., McKeithan, Timothy W., Fu, Kai, Green, Michael, Chan, Wing C., and Iqbal, Javeed

Published
2017
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20. Supplementary Table 1 from Nuclear–Cytoplasmic Transport Is a Therapeutic Target in Myelofibrosis

Author

Yan, Dongqing, primary, Pomicter, Anthony D., primary, Tantravahi, Srinivas, primary, Mason, Clinton C., primary, Senina, Anna V., primary, Ahmann, Jonathan M., primary, Wang, Qiang, primary, Than, Hein, primary, Patel, Ami B., primary, Heaton, William L., primary, Eiring, Anna M., primary, Clair, Phillip M., primary, Gantz, Kevin C., primary, Redwine, Hannah M., primary, Swierczek, Sabina I., primary, Halverson, Brayden J., primary, Baloglu, Erkan, primary, Shacham, Sharon, primary, Khorashad, Jamshid S., primary, Kelley, Todd W., primary, Salama, Mohamed E., primary, Miles, Rodney R., primary, Boucher, Kenneth M., primary, Prchal, Josef T., primary, O'Hare, Thomas, primary, and Deininger, Michael W., primary

Published
2023
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21. Supplementary Figure 6 from Nuclear–Cytoplasmic Transport Is a Therapeutic Target in Myelofibrosis

Author

Yan, Dongqing, primary, Pomicter, Anthony D., primary, Tantravahi, Srinivas, primary, Mason, Clinton C., primary, Senina, Anna V., primary, Ahmann, Jonathan M., primary, Wang, Qiang, primary, Than, Hein, primary, Patel, Ami B., primary, Heaton, William L., primary, Eiring, Anna M., primary, Clair, Phillip M., primary, Gantz, Kevin C., primary, Redwine, Hannah M., primary, Swierczek, Sabina I., primary, Halverson, Brayden J., primary, Baloglu, Erkan, primary, Shacham, Sharon, primary, Khorashad, Jamshid S., primary, Kelley, Todd W., primary, Salama, Mohamed E., primary, Miles, Rodney R., primary, Boucher, Kenneth M., primary, Prchal, Josef T., primary, O'Hare, Thomas, primary, and Deininger, Michael W., primary

Published
2023
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22. Supplementary Table 2 from Nuclear–Cytoplasmic Transport Is a Therapeutic Target in Myelofibrosis

Author

Yan, Dongqing, primary, Pomicter, Anthony D., primary, Tantravahi, Srinivas, primary, Mason, Clinton C., primary, Senina, Anna V., primary, Ahmann, Jonathan M., primary, Wang, Qiang, primary, Than, Hein, primary, Patel, Ami B., primary, Heaton, William L., primary, Eiring, Anna M., primary, Clair, Phillip M., primary, Gantz, Kevin C., primary, Redwine, Hannah M., primary, Swierczek, Sabina I., primary, Halverson, Brayden J., primary, Baloglu, Erkan, primary, Shacham, Sharon, primary, Khorashad, Jamshid S., primary, Kelley, Todd W., primary, Salama, Mohamed E., primary, Miles, Rodney R., primary, Boucher, Kenneth M., primary, Prchal, Josef T., primary, O'Hare, Thomas, primary, and Deininger, Michael W., primary

Published
2023
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23. Supplementary Table 3-8 from Nuclear–Cytoplasmic Transport Is a Therapeutic Target in Myelofibrosis

Author

Yan, Dongqing, primary, Pomicter, Anthony D., primary, Tantravahi, Srinivas, primary, Mason, Clinton C., primary, Senina, Anna V., primary, Ahmann, Jonathan M., primary, Wang, Qiang, primary, Than, Hein, primary, Patel, Ami B., primary, Heaton, William L., primary, Eiring, Anna M., primary, Clair, Phillip M., primary, Gantz, Kevin C., primary, Redwine, Hannah M., primary, Swierczek, Sabina I., primary, Halverson, Brayden J., primary, Baloglu, Erkan, primary, Shacham, Sharon, primary, Khorashad, Jamshid S., primary, Kelley, Todd W., primary, Salama, Mohamed E., primary, Miles, Rodney R., primary, Boucher, Kenneth M., primary, Prchal, Josef T., primary, O'Hare, Thomas, primary, and Deininger, Michael W., primary

Published
2023
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24. Supplementary Figure 3 from Nuclear–Cytoplasmic Transport Is a Therapeutic Target in Myelofibrosis

Author

Yan, Dongqing, primary, Pomicter, Anthony D., primary, Tantravahi, Srinivas, primary, Mason, Clinton C., primary, Senina, Anna V., primary, Ahmann, Jonathan M., primary, Wang, Qiang, primary, Than, Hein, primary, Patel, Ami B., primary, Heaton, William L., primary, Eiring, Anna M., primary, Clair, Phillip M., primary, Gantz, Kevin C., primary, Redwine, Hannah M., primary, Swierczek, Sabina I., primary, Halverson, Brayden J., primary, Baloglu, Erkan, primary, Shacham, Sharon, primary, Khorashad, Jamshid S., primary, Kelley, Todd W., primary, Salama, Mohamed E., primary, Miles, Rodney R., primary, Boucher, Kenneth M., primary, Prchal, Josef T., primary, O'Hare, Thomas, primary, and Deininger, Michael W., primary

Published
2023
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26. Correlation of Minimal Residual Disease (MRD) at the End of Induction (EOI) and Event Free Survival (EFS) in T-Cell Lymphoblastic Lymphoma (T-LL), a Report from the Children's Oncology Group (COG) Trial AALL1231

Author

Hayashi, Robert J., primary, Hermiston, Michelle L., additional, Teachey, David T., additional, Devidas, Meenakshi, additional, Wood, Brent L., additional, Chen, Zhiguo, additional, Annett, Robert D, additional, Asselin, Barbara L, additional, August, Keith J, additional, Cho, Steve Y, additional, Dunsmore, Kimberly P., additional, Freedman, Jason L., additional, Galardy, Paul J., additional, Harker-Murray, Paul, additional, Horton, Terzah M., additional, Jaju, Alok I, additional, Lam, Allison, additional, Messinger, Yoav H., additional, Miles, Rodney R., additional, Okada, Maki, additional, Patel, Samir I, additional, Schafer, Eric S, additional, Schechter-Finkelstein, Tal, additional, Shimano, Kristin A., additional, Singh, Neelam, additional, Steele, Amii C., additional, Sulis, Maria Luisa, additional, Vargas, Sarah L, additional, Winter, Stuart S., additional, Wood, Charlotte, additional, Zweidler-McKay, Patrick A, additional, Loh, Mignon L., additional, Hunger, Stephen P., additional, Raetz, Elizabeth A., additional, Bollard, Catherine M, additional, and Allen, Carl, additional

Published
2022
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27. A Pilot Study of Azacitidine As Epigenetic Priming for Chemotherapy in Infants Less Than 1 Year of Age with KMT2A-Rearranged Acute Lymphoblastic Leukemia (ALL); Results from the Children's Oncology Group (COG) Trial AALL15P1

Author

Guest, Erin, primary, Kairalla, John, additional, Devidas, Meenakshi, additional, Hibbitts, Emily, additional, Carroll, Andrew J., additional, Heerema, Nyla A., additional, Kubaney, Holly, additional, August, Amanda, additional, Pauly, Melinda, additional, Wechsler, Daniel, additional, Miles, Rodney R., additional, Reid, Joel M., additional, Kihei, Cynthia, additional, Gore, Lia, additional, Raetz, Elizabeth A., additional, Hunger, Stephen P., additional, Loh, Mignon L., additional, and Brown, Patrick A., additional

Published
2022
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30. Zebrafish screen identifies novel compound with selective toxicity against leukemia

Author

Ridges, Suzanne, Heaton, Will L., Joshi, Deepa, Choi, Henry, Eiring, Anna, Batchelor, Lance, Choudhry, Priya, Manos, Elizabeth J., Sofla, Hossein, Sanati, Ali, Welborn, Seth, Agarwal, Archana, Spangrude, Gerald J., Miles, Rodney R., Cox, James E., Frazer, J. Kimble, Deininger, Michael, Balan, Kaveri, Sigman, Matthew, Müschen, Markus, Perova, Tatiana, Johnson, Radia, Montpellier, Bertrand, Guidos, Cynthia J., Jones, David A., and Trede, Nikolaus S.

Published
2012
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31. Reply to R. Lakhotia et al

Author

Burke, G.A. Amos, primary, Minard-Colin, Veronique, additional, Aupérin, Anne, additional, Alexander, Sarah, additional, Pillon, Marta, additional, Delgado, Rafael, additional, Zsíros, József, additional, Uyttebroeck, Anne, additional, Dartigues, Peggy, additional, Miles, Rodney R., additional, Kazanowska, Bernarda, additional, Chiang, Alan K., additional, Haouy, Stéphanie, additional, Bollard, Catherine M., additional, Csoka, Monika, additional, Wheatley, Keith, additional, Barkauskas, Donald A., additional, Adamson, Peter C., additional, Vassal, Gilles, additional, Patte, Catherine, additional, and Gross, Thomas G., additional

Published
2022
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32. Proteasome function is required for platelet production

Author

Shi, Dallas S., Smith, Matthew C.P., Campbel, Robert A., Zimmerman, Patrick W., Franks, Zechariah B., Kraemer, Bjorn F., Machlus, Kellie R., Ling, Jing, Kamba, Patrick, Schwertz, Hansjorg, Rowley, Jesse W., Miles, Rodney R., Liu, Zhi-Jian, Sola-Visner, Martha, Italiano, Jr., Joseph E., Christensen, Hilary, Kahr, Walter H.A., Li, Dean Y., and Weyrich, Andrew S.

Subjects
Ubiquitin-proteasome system -- Research, Bone marrow cells -- Physiological aspects, Bortezomib -- Complications and side effects, Thrombocytopenia -- Causes of, Health care industry
Abstract

The proteasome inhibiter bortezomib has been successfully used to treat patients with relapsed multiple myeloma; however, many of these patients become thrombocytopenic, and it is not clear how the proteasome influences platelet production. Here we determined that pharmacologic inhibition of proteasome activity blocks proplatelet formation in human and mouse megakaryocytes. We also found that megakaryocytes isolated from mice deficient for PSMC1, an essential subunit of the 26S proteasome, fail to produce proplatelets. Consistent with decreased proplatelet formation, mice lacking PSMC1 in platelets ([Psmc1.sup.fl/fl] Pf4-Cre mice) exhibited severe thrombocytopenia and died shortly after birth. The failure to produce proplatelets in proteasome-inhibited megakaryocytes was due to upregulation and hyperactivation of the small GTPase, RhoA, rather than NF-κB, as has been previously suggested. Inhibition of RhoA or its downstream target, Rho-associated protein kinase (ROCK), restored megakaryocyte proplatelet formation in the setting of proteasome inhibition in vitro. Similarly, fasudil, a ROCK inhibitor used clinically to treat cerebral vasospasm, restored platelet counts in adult mice that were made thrombocytopenic by tamoxifen-induced suppression of proteasome activity in megakaryocytes and platelets ([Psmc1.sup.fl/fl] PdgfCre-ER mice). These results indicate that proteasome function is critical for thrombopoiesis, and suggest inhibition of RhoA signaling as a potential strategy to treat thrombocytopenia in bortezomib-treated multiple myeloma patients., Introduction Thrombocytopenia (low platelet count) is observed in numerous diseases and can be life threatening due to bleeding complications. Bortezomib, a reversible chemotherapeutic inhibitor used to treat patients with relapsed [...]

Published
2014
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37. Dose-Adjusted Etoposide, Doxorubicin, and Cyclophosphamide With Vincristine and Prednisone Plus Rituximab Therapy in Children and Adolescents With Primary Mediastinal B-Cell Lymphoma: A Multicenter Phase II Trial

Author

Burke, G. A. Amos, primary, Minard-Colin, Veronique, additional, Aupérin, Anne, additional, Alexander, Sarah, additional, Pillon, Marta, additional, Delgado, Rafael, additional, Zsíros, József, additional, Uyttebroeck, Anne, additional, Dartigues, Peggy, additional, Miles, Rodney R., additional, Kazanowska, Bernarda, additional, Chiang, Alan K., additional, Haouy, Stéphanie, additional, Bollard, Catherine M., additional, Csoka, Monika, additional, Wheatley, Keith, additional, Barkauskas, Donald A., additional, Adamson, Peter C., additional, Vassal, Gilles, additional, Patte, Catherine, additional, and Gross, Thomas G., additional

Published
2021
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39. Association of Combined Focal 22q11.22 Deletion and IKZF1 Alterations With Outcomes in Childhood Acute Lymphoblastic Leukemia

Author

Mangum, David Spencer, primary, Meyer, Julia A., additional, Mason, Clinton C., additional, Shams, Soheil, additional, Maese, Luke D., additional, Gardiner, Jamie D., additional, Downie, Jonathan M., additional, Pei, Deqing, additional, Cheng, Cheng, additional, Gleason, Adam, additional, Luo, Minjie, additional, Pui, Ching-Hon, additional, Aplenc, Richard, additional, Hunger, Stephen P., additional, Loh, Mignon, additional, Greaves, Mel, additional, Trede, Nikolaus, additional, Raetz, Elizabeth, additional, Frazer, J. Kimble, additional, Mullighan, Charles G., additional, Engel, Michael E., additional, Miles, Rodney R., additional, Rabin, Karen R., additional, and Schiffman, Joshua D., additional

Published
2021
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40. Targeted Immunotherapy and Checkpoint Blockade in Children, Adolescents, and Young Adults with Lymphoma: Radical Hodgkin Cohort

Author

Klose, Kayleigh, Hochberg, Jessica, Basso, Jaclyn, Xavier, Ana C., Barth, Matthew J., Miles, Rodney R., Kahwash, Samir, Voss, Stephan D., Braniecki, Suzanne, Moorthy, Chitti, Armenian, Saro, Ehrhardt, Matthew J., Lim, Megan, Harrison, Lauren, Budhram, Arvind, Stonberg, Ariel, Goldman, Stanton, and Cairo, Mitchell S.

Published
2023
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41. Multicenter Pilot Trial of Intrathecal Liposomal Cytarabine Combined with FAB Chemoimmunotherapy with Reduced Doxorubicin in CAYA with Mature De-Novo B-NHL

Author

Audino, Anthony N., Goldman, Stanton, Barth, Matthew J., Hochberg, Jessica, Xavier, Ana C., Shiramizu, Bruce, Shi, Qiuhu, Harrison, Lauren, Basso, Jackie, Oesterheld, Javier, Heym, Kenneth Matthew, Drachtman, Richard A., Harker-Murray, Paul, Perkins, Sherrie L., Miles, Rodney R., and Cairo, Mitchell S.

Published
2023
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43. Rituximab for High-Risk, Mature B-Cell Non-Hodgkin's Lymphoma in Children

Author

Minard-Colin, Véronique, Aupérin, Anne, Pillon, Marta, Burke, G A Amos, Barkauskas, Donald A, Wheatley, Keith, Delgado, Rafael F, Alexander, Sarah, Uyttebroeck, Anne, Bollard, Catherine M, Zsiros, József, Csoka, Monika, Kazanowska, Bernarda, Chiang, Alan K, Miles, Rodney R, Wotherspoon, Andrew, Adamson, Peter C, Vassal, Gilles, Patte, Catherine, Gross, Thomas G, European Intergroup for Childhood Non-Hodgkin Lymphoma, Children’s Oncology Group, Brichard, Bénédicte, Clinical sciences, Growth and Development, Pediatrics, UCL - SSS/IREC/PEDI - Pôle de Pédiatrie, and UCL - (SLuc) Service d'hématologie et d'oncologie pédiatrique

Subjects
Oncology, Male, Pediatrics, medicine.medical_treatment, Lymphoma, B-Cell/drug therapy, Kaplan-Meier Estimate, 030204 cardiovascular system & hematology, Rituximab/administration & dosage, Antineoplastic Agents, Immunological, 0302 clinical medicine, Neutropenia/chemically induced, immune system diseases, hemic and lymphatic diseases, Antineoplastic Combined Chemotherapy Protocols, 030212 general & internal medicine, Child, Infusions, Intravenous, Medicine(all), General Medicine, Progression-Free Survival, Mature B-Cell Non-Hodgkin's Lymphoma, Rituximab, Female, Lymph, medicine.drug, medicine.medical_specialty, Lymphoma, B-Cell, Neutropenia, Adolescent, Child, preschool, Infections/etiology, MEDLINE, Infections, Article, 03 medical and health sciences, Antineoplastic Agents, Immunological/administration & dosage, Internal medicine, medicine, Humans, Progression-free survival, Chemotherapy, business.industry, Antineoplastic Combined Chemotherapy Protocols/adverse effects, Cancer, medicine.disease, Lymphoma, Clinical trial, business, Follow-Up Studies
Abstract

BACKGROUND: Rituximab added to chemotherapy prolongs survival among adults with B-cell cancer. Data on its efficacy and safety in children with high-grade, mature B-cell non-Hodgkin's lymphoma are limited. METHODS: We conducted an open-label, international, randomized, phase 3 trial involving patients younger than 18 years of age with high-risk, mature B-cell non-Hodgkin's lymphoma (stage III with an elevated lactate dehydrogenase level or stage IV) or acute leukemia to compare the addition of six doses of rituximab to standard lymphomes malins B (LMB) chemotherapy with standard LMB chemotherapy alone. The primary end point was event-free survival. Overall survival and toxic effects were also assessed. RESULTS: Analyses were based on 328 patients who underwent randomization (164 patients per group); 85.7% of the patients had Burkitt's lymphoma. The median follow-up was 39.9 months. Events were observed in 10 patients in the rituximab-chemotherapy group and in 28 in the chemotherapy group. Event-free survival at 3 years was 93.9% (95% confidence interval [CI], 89.1 to 96.7) in the rituximab-chemotherapy group and 82.3% (95% CI, 75.7 to 87.5) in the chemotherapy group (hazard ratio for primary refractory disease or first occurrence of progression, relapse after response, death from any cause, or second cancer, 0.32; 95% CI, 0.15 to 0.66; one-sided P = 0.00096, which reached the significance level required for this analysis). Eight patients in the rituximab-chemotherapy group died (4 deaths were disease-related, 3 were treatment-related, and 1 was from a second cancer), as did 20 in the chemotherapy group (17 deaths were disease-related, and 3 were treatment-related) (hazard ratio, 0.36; 95% CI, 0.16 to 0.82). The incidence of acute adverse events of grade 4 or higher after prephase treatment was 33.3% in the rituximab-chemotherapy group and 24.2% in the chemotherapy group (P = 0.07); events were related mainly to febrile neutropenia and infection. Approximately twice as many patients in the rituximab-chemotherapy group as in the chemotherapy group had a low IgG level 1 year after trial inclusion. CONCLUSIONS: Rituximab added to standard LMB chemotherapy markedly prolonged event-free survival and overall survival among children and adolescents with high-grade, high-risk, mature B-cell non-Hodgkin's lymphoma and was associated with a higher incidence of hypogammaglobulinemia and, potentially, more episodes of infection. (Funded by the Clinical Research Hospital Program of the French Ministry of Health and others; ClinicalTrials.gov number, NCT01516580.).

Published
2020
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49. Final Report of Reduced Anthracycline Dose Intensity with the Addition of Dose Dense Rituximab in Children, Adolescents and Young Adults with De Novo Good Risk Mature B-Cell Non Hodgkin Lymphoma (B-NHL)

Author

Hochberg, Jessica C., primary, Goldman, Stanton, additional, Barth, Matthew J., additional, Shi, Qiuhu, additional, Klejmont, Liana, additional, Harrison, Lauren, additional, Basso, Jackie, additional, Chu, Yaya, additional, Islam, Humayun, additional, Gerard, Perry, additional, Oesterheld, Javier, additional, Heym, Kenneth Matthew, additional, Kirov, Ivan I., additional, Drachtman, Richard A., additional, Harker-Murray, Paul, additional, Perkins, Sherrie L., additional, Miles, Rodney R., additional, Shiramizu, Bruce, additional, and Cairo, Mitchell S, additional

Published
2020
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50. Cranial Radiation Can be Eliminated in Most Children with T-Cell Acute Lymphoblastic Leukemia (T-ALL) and Bortezomib Potentially Improves Survival in Children with T-Cell Lymphoblastic Lymphoma (T-LL): Results of Children's Oncology Group (COG) Trial AALL1231

Author

Teachey, David T., primary, Devidas, Meenakshi, additional, Wood, Brent L, additional, Chen, Zhiguo, additional, Hayashi, Robert James, additional, Annett, Robert D, additional, Asselin, Barbara L, additional, August, Keith J, additional, Cho, Steve Y, additional, Dunsmore, Kimberly P., additional, Fisher, Brian T., additional, Freedman, Jason L., additional, Galardy, Paul J., additional, Harker-Murray, Paul, additional, Hermiston, Michelle L., additional, Horton, Terzah M, additional, Jaju, Alok I, additional, Lam, Allison, additional, Messinger, Yoav H., additional, Miles, Rodney R., additional, Okada, Maki, additional, Patel, Samir I, additional, Schafer, Eric S., additional, Schechter-Finkelstein, Tal, additional, Shimano, Kristin A., additional, Singh, Neelam, additional, Steele, Amii C., additional, Sulis, Maria L., additional, Vargas, Sarah L, additional, Winter, Stuart S., additional, Wood, Charlotte, additional, McKay, Patrick Zweider, additional, Bollard, Catherine M., additional, Loh, Mignon L., additional, Hunger, Stephen P., additional, and Raetz, Elizabeth A., additional

Published
2020
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