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1. Decitabine and vorinostat with FLAG chemotherapy in pediatric relapsed/refractory AML: Report from the therapeutic advances in childhood leukemia and lymphoma (TACL) consortium

Author

Pommert, Lauren, Schafer, Eric S, Malvar, Jemily, Gossai, Nathan, Florendo, Ellynore, Pulakanti, Kirthi, Heimbruch, Katelyn, Stelloh, Cary, Chi, Yueh‐Yun, Sposto, Richard, Rao, Sridhar, Huynh, Van Thu, Brown, Patrick, Chang, Bill H, Colace, Susan I, Hermiston, Michelle L, Heym, Kenneth, Hutchinson, Raymond J, Kaplan, Joel A, Mody, Rajen, O'Brien, Tracey A, Place, Andrew E, Shaw, Peter H, Ziegler, David S, Wayne, Alan, Bhojwani, Deepa, and Burke, Michael J

Subjects
Biomedical and Clinical Sciences, Cardiovascular Medicine and Haematology, Oncology and Carcinogenesis, Stem Cell Research, Clinical Trials and Supportive Activities, Stem Cell Research - Nonembryonic - Human, Cancer, Pediatric, Minority Health, Orphan Drug, Hematology, Childhood Leukemia, Clinical Research, Rare Diseases, Transplantation, Genetics, Pediatric Cancer, 6.1 Pharmaceuticals, 6.2 Cellular and gene therapies, Antineoplastic Combined Chemotherapy Protocols, Child, Cytarabine, Decitabine, Humans, Leukemia, Myeloid, Acute, Lymphoma, Vorinostat, Cardiorespiratory Medicine and Haematology, Immunology, Cardiovascular medicine and haematology
Abstract

Survival outcomes for relapsed/refractory pediatric acute myeloid leukemia (R/R AML) remain dismal. Epigenetic changes can result in gene expression alterations which are thought to contribute to both leukemogenesis and chemotherapy resistance. We report results from a phase I trial with a dose expansion cohort investigating decitabine and vorinostat in combination with fludarabine, cytarabine, and G-CSF (FLAG) in pediatric patients with R/R AML [NCT02412475]. Thirty-seven patients enrolled with a median age at enrollment of 8.4 (range, 1-20) years. There were no dose limiting toxicities among the enrolled patients, including two patients with Down syndrome. The recommended phase 2 dose of decitabine in combination with vorinostat and FLAG was 10 mg/m2 . The expanded cohort design allowed for an efficacy evaluation and the overall response rate among 35 evaluable patients was 54% (16 complete response (CR) and 3 complete response with incomplete hematologic recovery (CRi)). Ninety percent of responders achieved minimal residual disease (MRD) negativity (

Published
2022

2. Matched Targeted Therapy for Pediatric Patients with Relapsed, Refractory, or High-Risk Leukemias: A Report from the LEAP Consortium

Author

Pikman, Yana, Tasian, Sarah K, Sulis, Maria Luisa, Stevenson, Kristen, Blonquist, Traci M, Apsel Winger, Beth, Cooper, Todd M, Pauly, Melinda, Maloney, Kelly W, Burke, Michael J, Brown, Patrick A, Gossai, Nathan, McNeer, Jennifer L, Shukla, Neerav N, Cole, Peter D, Kahn, Justine M, Chen, Jing, Barth, Matthew J, Magee, Jeffrey A, Gennarini, Lisa, Adhav, Asmani A, Clinton, Catherine M, Ocasio-Martinez, Nicole, Gotti, Giacomo, Li, Yuting, Lin, Shan, Imamovic, Alma, Tognon, Cristina E, Patel, Tasleema, Faust, Haley L, Contreras, Cristina F, Cremer, Anjali, Cortopassi, Wilian A, Garrido Ruiz, Diego, Jacobson, Matthew P, Dharia, Neekesh V, Su, Angela, Robichaud, Amanda L, Saur Conway, Amy, Tarlock, Katherine, Stieglitz, Elliot, Place, Andrew E, Puissant, Alexandre, Hunger, Stephen P, Kim, Annette S, Lindeman, Neal I, Gore, Lia, Janeway, Katherine A, Silverman, Lewis B, Tyner, Jeffrey W, Harris, Marian H, Loh, Mignon L, and Stegmaier, Kimberly

Subjects
Cancer, Rare Diseases, Human Genome, Genetics, Clinical Trials and Supportive Activities, Orphan Drug, Biotechnology, Hematology, Clinical Research, Pediatric Cancer, Childhood Leukemia, Pediatric, Development of treatments and therapeutic interventions, Aetiology, 5.1 Pharmaceuticals, 2.1 Biological and endogenous factors, Good Health and Well Being, Biomarkers, Tumor, Child, Cohort Studies, Disease Progression, Feasibility Studies, Female, Humans, Leukemia, Male, Molecular Targeted Therapy, Neoplasm Recurrence, Local, Prospective Studies, United States, Oncology and Carcinogenesis
Abstract

Despite a remarkable increase in the genomic profiling of cancer, integration of genomic discoveries into clinical care has lagged behind. We report the feasibility of rapid identification of targetable mutations in 153 pediatric patients with relapsed/refractory or high-risk leukemias enrolled on a prospective clinical trial conducted by the LEAP Consortium. Eighteen percent of patients had a high confidence Tier 1 or 2 recommendation. We describe clinical responses in the 14% of patients with relapsed/refractory leukemia who received the matched targeted therapy. Further, in order to inform future targeted therapy for patients, we validated variants of uncertain significance, performed ex vivo drug-sensitivity testing in patient leukemia samples, and identified new combinations of targeted therapies in cell lines and patient-derived xenograft models. These data and our collaborative approach should inform the design of future precision medicine trials. SIGNIFICANCE: Patients with relapsed/refractory leukemias face limited treatment options. Systematic integration of precision medicine efforts can inform therapy. We report the feasibility of identifying targetable mutations in children with leukemia and describe correlative biology studies validating therapeutic hypotheses and novel mutations.See related commentary by Bornhauser and Bourquin, p. 1322.This article is highlighted in the In This Issue feature, p. 1307.

Published
2021

4. On orbit performance of the GRACE Follow-On Laser Ranging Interferometer

Author

Abich, Klaus, Braxmaier, Claus, Gohlke, Martin, Sanjuan, Josep, Abramovici, Alexander, Okihiro, Brian Bachman, Barr, David C., Bize, Maxime P., Burke, Michael J., Clark, Ken C., de Vine, Glenn, Dickson, Jeffrey A., Dubovitsky, Serge, Folkner, William M., Francis, Samuel, Gilbert, Martin S., Katsumura, Mark, Klipstein, William, Larsen, Kameron, Liebe, Carl Christian, Liu, Jehhal, McKenzie, Kirk, Morton, Phillip R., Murray, Alexander T., Nguyen, Don J., Ravich, Joshua A., Shaddock, Daniel, Spero, Robert, Spiers, Gary, Sutton, Andrew, Trinh, Joseph, Wang, Duo, Wang, Rabi T., Ware, Brent, Woodruff, Christopher, Amparan, Bengie, Davis, Mike A., Howell, James, Kruger, Micah, Lobmeyer, Lynette, Pierce, Robert, Reavis, Gretchen, Sileo, Michael, Stephens, Michelle, Baatzsch, Andreas, Dahl, Christian, Dahl, Katrin, Gilles, Frank, Hager, Philipp, Herding, Mark, Kaufer, Marina, Nicklaus, Kolja, Voss, Kai, Bogan, Christina, Danzmann, Karsten, Barranco, Germán Fernández, Heinzel, Gerhard, Koch, Alexander, Mahrdt, Christoph, Misfeldt, Malte, Müller, Vitali, Reiche, Jens, Schütze, Daniel, Sheard, Benjamin, Stede, Gunnar, Wegener, Henry, Eckardt, Andreas, Guenther, Burghardt, Mangoldt, Thomas, Zender, Bernd, Ester, Thomas, Heine, Frank, Seiter, Christoph, Windisch, Steve, Flatscher, Reinhold, Flechtner, Frank, Grossard, Nicolas, Hauden, Jerome, Hinz, Martin, Leikert, Thomas, Zimmermann, Marcus, Lebeda, Anton, and Lebeda, Arnold

Subjects
Astrophysics - Instrumentation and Methods for Astrophysics, Physics - Geophysics
Abstract

The Laser Ranging Interferometer (LRI) instrument on the Gravity Recovery and Climate Experiment (GRACE) Follow-On mission has provided the first laser interferometric range measurements between remote spacecraft, separated by approximately 220 km. Autonomous controls that lock the laser frequency to a cavity reference and establish the 5 degree of freedom two-way laser link between remote spacecraft succeeded on the first attempt. Active beam pointing based on differential wavefront sensing compensates spacecraft attitude fluctuations. The LRI has operated continuously without breaks in phase tracking for more than 50 days, and has shown biased range measurements similar to the primary ranging instrument based on microwaves, but with much less noise at a level of $1\,{\rm nm}/\sqrt{\rm Hz}$ at Fourier frequencies above 100 mHz.

Published
2019
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5. Decitabine and Vorinostat with Chemotherapy in Relapsed Pediatric Acute Lymphoblastic Leukemia: A TACL Pilot Study

Author

Burke, Michael J, Kostadinov, Rumen, Sposto, Richard, Gore, Lia, Kelley, Shannon M, Rabik, Cara, Trepel, Jane B, Lee, Min-Jung, Yuno, Akira, Lee, Sunmin, Bhojwani, Deepa, Jeha, Sima, Chang, Bill H, Sulis, Maria Luisa, Hermiston, Michelle L, Gaynon, Paul, Huynh, Van, Verma, Anupam, Gardner, Rebecca, Heym, Kenneth M, Dennis, Robyn M, Ziegler, David S, Laetsch, Theodore W, Oesterheld, Javier E, Dubois, Steven G, Pollard, Jessica A, Glade-Bender, Julia, Cooper, Todd M, Kaplan, Joel A, Farooqi, Midhat S, Yoo, Byunggil, Guest, Erin, Wayne, Alan S, and Brown, Patrick A

Subjects
Biomedical and Clinical Sciences, Clinical Sciences, Hematology, Pediatric Cancer, Genetics, Rare Diseases, Cancer, Pediatric, Orphan Drug, Childhood Leukemia, Clinical Research, Good Health and Well Being, Adolescent, Adult, Antineoplastic Combined Chemotherapy Protocols, Asparaginase, Bortezomib, Child, Child, Preschool, Decitabine, Dexamethasone, Doxorubicin, Female, Follow-Up Studies, Humans, Infant, Male, Mitoxantrone, Neoplasm Recurrence, Local, Pilot Projects, Polyethylene Glycols, Precursor Cell Lymphoblastic Leukemia-Lymphoma, Prognosis, Salvage Therapy, Survival Rate, Vincristine, Vorinostat, Young Adult, Oncology and Carcinogenesis, Oncology & Carcinogenesis, Clinical sciences, Oncology and carcinogenesis
Abstract

PurposeTreatment failure from drug resistance is the primary reason for relapse in acute lymphoblastic leukemia (ALL). Improving outcomes by targeting mechanisms of drug resistance is a potential solution.Patients and methodsWe report results investigating the epigenetic modulators decitabine and vorinostat with vincristine, dexamethasone, mitoxantrone, and PEG-asparaginase for pediatric patients with relapsed or refractory B-cell ALL (B-ALL). Twenty-three patients, median age 12 years (range, 1-21) were treated in this trial.ResultsThe most common grade 3-4 toxicities included hypokalemia (65%), anemia (78%), febrile neutropenia (57%), hypophosphatemia (43%), leukopenia (61%), hyperbilirubinemia (39%), thrombocytopenia (87%), neutropenia (91%), and hypocalcemia (39%). Three subjects experienced dose-limiting toxicities, which included cholestasis, steatosis, and hyperbilirubinemia (n = 1); seizure, somnolence, and delirium (n = 1); and pneumonitis, hypoxia, and hyperbilirubinemia (n = 1). Infectious complications were common with 17 of 23 (74%) subjects experiencing grade ≥3 infections including invasive fungal infections in 35% (8/23). Nine subjects (39%) achieved a complete response (CR + CR without platelet recovery + CR without neutrophil recovery) and five had stable disease (22%). Nine (39%) subjects were not evaluable for response, primarily due to treatment-related toxicities. Correlative pharmacodynamics demonstrated potent in vivo modulation of epigenetic marks, and modulation of biologic pathways associated with functional antileukemic effects.ConclusionsDespite encouraging response rates and pharmacodynamics, the combination of decitabine and vorinostat on this intensive chemotherapy backbone was determined not feasible in B-ALL due to the high incidence of significant infectious toxicities. This study is registered at http://www.clinicaltrials.gov as NCT01483690.

Published
2020

6. Remediation at Robins AFB

Author

Burke, Michael J.

Subjects
AIR BASES - United States - Robins AFB, GA, ENVIRONMENT - Cleanup, DECONTAMINATION (FROM GASES, CHEMICALS, ETC), POLLUTION
Abstract

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Published
1998

7. Central nervous system status is prognostic in T-cell acute lymphoblastic leukemia: a Children’s Oncology Group report

Author

Gossai, Nathan P., Devidas, Meenakshi, Chen, Zhiguo, Wood, Brent L., Zweidler-McKay, Patrick A., Rabin, Karen R., Loh, Mignon L., Raetz, Elizabeth A., Winick, Naomi J., Burke, Michael J., Carroll, Andrew J., Esiashvili, Natia, Heerema, Nyla A., Carroll, William L., Hunger, Stephen P., Dunsmore, Kimberly P., Winter, Stuart S., and Teachey, David T.

Published
2023
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9. Racial and ethnic disparities in childhood and young adult acute lymphocytic leukaemia: secondary analyses of eight Children's Oncology Group cohort trials

Author

Gupta, Sumit, Dai, Yunfeng, Chen, Zhiguo, Winestone, Lena E, Teachey, David T, Bona, Kira, Aplenc, Richard, Rabin, Karen R, Zweidler-McKay, Patrick, Carroll, Andrew J, Heerema, Nyla A, Gastier-Foster, Julie, Borowitz, Michael J, Wood, Brent L, Maloney, Kelly W, Mattano, Leonard A, Jr, Larsen, Eric C, Angiolillo, Anne L, Burke, Michael J, Salzer, Wanda L, Winter, Stuart S, Brown, Patrick A, Guest, Erin M, Dunsmore, Kimberley P, Kairalla, John A, Winick, Naomi J, Carroll, William L, Raetz, Elizabeth A, Hunger, Stephen P, Loh, Mignon L, and Devidas, Meenakshi

Published
2023
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11. The genomic landscape of pediatric acute lymphoblastic leukemia

Author

Brady, Samuel W., Roberts, Kathryn G., Gu, Zhaohui, Shi, Lei, Pounds, Stanley, Pei, Deqing, Cheng, Cheng, Dai, Yunfeng, Devidas, Meenakshi, Qu, Chunxu, Hill, Ashley N., Payne-Turner, Debbie, Ma, Xiaotu, Iacobucci, Ilaria, Baviskar, Pradyuamna, Wei, Lei, Arunachalam, Sasi, Hagiwara, Kohei, Liu, Yanling, Flasch, Diane A., Liu, Yu, Parker, Matthew, Chen, Xiaolong, Elsayed, Abdelrahman H., Pathak, Omkar, Li, Yongjin, Fan, Yiping, Michael, J. Robert, Rusch, Michael, Wilkinson, Mark R., Foy, Scott, Hedges, Dale J., Newman, Scott, Zhou, Xin, Wang, Jian, Reilly, Colleen, Sioson, Edgar, Rice, Stephen V., Pastor Loyola, Victor, Wu, Gang, Rampersaud, Evadnie, Reshmi, Shalini C., Gastier-Foster, Julie, Guidry Auvil, Jaime M., Gesuwan, Patee, Smith, Malcolm A., Winick, Naomi, Carroll, Andrew J., Heerema, Nyla A., Harvey, Richard C., Willman, Cheryl L., Larsen, Eric, Raetz, Elizabeth A., Borowitz, Michael J., Wood, Brent L., Carroll, William L., Zweidler-McKay, Patrick A., Rabin, Karen R., Mattano, Leonard A., Maloney, Kelly W., Winter, Stuart S., Burke, Michael J., Salzer, Wanda, Dunsmore, Kimberly P., Angiolillo, Anne L., Crews, Kristine R., Downing, James R., Jeha, Sima, Pui, Ching-Hon, Evans, William E., Yang, Jun J., Relling, Mary V., Gerhard, Daniela S., Loh, Mignon L., Hunger, Stephen P., Zhang, Jinghui, and Mullighan, Charles G.

Published
2022
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12. Replacing cyclophosphamide/cytarabine/mercaptopurine with cyclophosphamide/etoposide during consolidation/delayed intensification does not improve outcome for pediatric B-cell acute lymphoblastic leukemia: a report from the COG

Author

Burke, Michael J, Salzer, Wanda L, Devidas, Meenakshi, Dai, Yunfeng, Gore, Lia, Hilden, Joanne M, Larsen, Eric, Rabin, Karen R, Zweidler-McKay, Patrick A, Borowitz, Michael J, Wood, Brent, Heerema, Nyla A, Carroll, Andrew J, Winick, Naomi, Carroll, William L, Raetz, Elizabeth A, Loh, Mignon L, and Hunger, Stephen P

Subjects
Biomedical and Clinical Sciences, Cardiovascular Medicine and Haematology, Oncology and Carcinogenesis, Orphan Drug, Clinical Trials and Supportive Activities, Hematology, Rare Diseases, Cancer, Clinical Research, Patient Safety, Pediatric Cancer, Pediatric, Childhood Leukemia, 6.1 Pharmaceuticals, Evaluation of treatments and therapeutic interventions, Good Health and Well Being, Adolescent, Adult, Antineoplastic Combined Chemotherapy Protocols, Case-Control Studies, Child, Child, Preschool, Cyclophosphamide, Cytarabine, Etoposide, Female, Follow-Up Studies, Humans, Infant, Male, Mercaptopurine, Precursor B-Cell Lymphoblastic Leukemia-Lymphoma, Prognosis, Prospective Studies, Survival Rate, Young Adult, Cardiorespiratory Medicine and Haematology, Immunology, Cardiovascular medicine and haematology
Abstract

With modern chemotherapy, approximately 90% of patients with pediatric acute lymphoblastic leukemia are now cured. However, subsets of patients can be identified who remain at very high risk of relapse with expected 4-year disease-free survival rates

Published
2019

13. Outcomes in adolescent and young adult patients (16 to 30 years) compared to younger patients treated for high-risk B-lymphoblastic leukemia: report from Children’s Oncology Group Study AALL0232

Author

Burke, Michael J., Devidas, Meenakshi, Chen, Zhiguo, Salzer, Wanda L., Raetz, Elizabeth A., Rabin, Karen R., Heerema, Nyla A., Carroll, Andrew J., Gastier-Foster, Julie M., Borowitz, Michael J., Wood, Brent L., Winick, Naomi J., Carroll, William L., Hunger, Stephen P., Loh, Mignon L., and Larsen, Eric C.

Published
2022
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15. Genomic Determinants of Outcome in Acute Lymphoblastic Leukemia.

Author

Chang, Ti-Cheng, Chen, Wenan, Qu, Chunxu, Cheng, Zhongshan, Hedges, Dale, Elsayed, Abdelrahman, Pounds, Stanley B., Shago, Mary, Rabin, Karen R., Raetz, Elizabeth A., Devidas, Meenakshi, Cheng, Cheng, Angiolillo, Anne, Baviskar, Pradyuamna, Borowitz, Michael, Burke, Michael J., Carroll, Andrew, Carroll, William L., Chen, I-Ming, and Harvey, Richard

Published
2024
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17. With great power comes great hunger: Marvel Zombies as parody of the superhero trope.

Author

Burke, Michael J.

Subjects
*ZOMBIES, *SUPERHEROES, *HEROES, *CANNIBALS, *CANNIBALISM
Abstract

There has always been something of the monster baked into the comic book superhero's DNA. Whether it pertains to the reality-bending powers of superhero soaring through the air, seeing through walls, setting her body aflame or altering her size, or by the more Gothic literary antecedents and 'science gone amok' themes constitutive of their origin stories, the comic book superhero treads a fine line between saviour and monster, as often evinced in the oscillating reactions of approbation and fear displayed by the diegetic public's reaction to them. Marvel Zombies, Marvel Comics' commercially successful depiction of its beloved superheroes as ravenous, flesh-eating cannibals, expresses one of the more recent and unsettling throwbacks to the monstrous origins and nature of the Marvel superhero. Employing Jeffrey Jerome Cohen's methodology of reading culture from the figure of the monster, this article proposes to explore Marvel Zombies as an indictment and parodic criticism of the Marvel Superhero. Just as the double is, per Sigmund Freud's psychoanalytic approach, an uncanny reminder of repressed truths and desires, so too does the decaying Marvel Zombie function as the uncanny doppelganger of the Marvel superhero, revealing the stagnant character development at its rotten core. [ABSTRACT FROM AUTHOR]

Published
2024
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21. Genomic analyses identify recurrent MEF2D fusions in acute lymphoblastic leukaemia.

Author

Gu, Zhaohui, Churchman, Michelle, Roberts, Kathryn, Li, Yongjin, Liu, Yu, Harvey, Richard C, McCastlain, Kelly, Reshmi, Shalini C, Payne-Turner, Debbie, Iacobucci, Ilaria, Shao, Ying, Chen, I-Ming, Valentine, Marcus, Pei, Deqing, Mungall, Karen L, Mungall, Andrew J, Ma, Yussanne, Moore, Richard, Marra, Marco, Stonerock, Eileen, Gastier-Foster, Julie M, Devidas, Meenakshi, Dai, Yunfeng, Wood, Brent, Borowitz, Michael, Larsen, Eric E, Maloney, Kelly, Mattano, Leonard A, Angiolillo, Anne, Salzer, Wanda L, Burke, Michael J, Gianni, Francesca, Spinelli, Orietta, Radich, Jerald P, Minden, Mark D, Moorman, Anthony V, Patel, Bella, Fielding, Adele K, Rowe, Jacob M, Luger, Selina M, Bhatia, Ravi, Aldoss, Ibrahim, Forman, Stephen J, Kohlschmidt, Jessica, Mrózek, Krzysztof, Marcucci, Guido, Bloomfield, Clara D, Stock, Wendy, Kornblau, Steven, Kantarjian, Hagop M, Konopleva, Marina, Paietta, Elisabeth, Willman, Cheryl L, Loh, Mignon L, Hunger, Stephen P, and Mullighan, Charles G

Subjects
NIH 3T3 Cells, Animals, Humans, Mice, Luciferases, Oncogene Proteins, Fusion, Treatment Outcome, Sequence Analysis, RNA, Genomics, Gene Expression Regulation, Leukemic, Gene Rearrangement, Base Sequence, Precursor Cell Lymphoblastic Leukemia-Lymphoma, Histone Deacetylase Inhibitors, Transcriptome, MEF2 Transcription Factors, Genetics, Pediatric Cancer, Cancer, Pediatric, 2.1 Biological and endogenous factors
Abstract

Chromosomal rearrangements are initiating events in acute lymphoblastic leukaemia (ALL). Here using RNA sequencing of 560 ALL cases, we identify rearrangements between MEF2D (myocyte enhancer factor 2D) and five genes (BCL9, CSF1R, DAZAP1, HNRNPUL1 and SS18) in 22 B progenitor ALL (B-ALL) cases with a distinct gene expression profile, the most common of which is MEF2D-BCL9. Examination of an extended cohort of 1,164 B-ALL cases identified 30 cases with MEF2D rearrangements, which include an additional fusion partner, FOXJ2; thus, MEF2D-rearranged cases comprise 5.3% of cases lacking recurring alterations. MEF2D-rearranged ALL is characterized by a distinct immunophenotype, DNA copy number alterations at the rearrangement sites, older diagnosis age and poor outcome. The rearrangements result in enhanced MEF2D transcriptional activity, lymphoid transformation, activation of HDAC9 expression and sensitive to histone deacetylase inhibitor treatment. Thus, MEF2D-rearranged ALL represents a distinct form of high-risk leukaemia, for which new therapeutic approaches should be considered.

Published
2016

22. Transplant Outcomes for Children with T Cell Acute Lymphoblastic Leukemia in Second Remission: A Report from the Center for International Blood and Marrow Transplant Research

Author

Burke, Michael J, Verneris, Michael R, Le Rademacher, Jennifer, He, Wensheng, Abdel-Azim, Hisham, Abraham, Allistair A, Auletta, Jeffery J, Ayas, Mouhab, Brown, Valerie I, Cairo, Mitchell S, Chan, Ka Wah, Diaz Perez, Miguel A, Dvorak, Christopher C, Egeler, R Maarten, Eldjerou, Lamis, Frangoul, Haydar, Guilcher, Gregory MT, Hayashi, Robert J, Ibrahim, Ahmed, Kasow, Kimberly A, Leung, Wing H, Olsson, Richard F, Pulsipher, Michael A, Shah, Niketa, Shah, Nirali N, Thiel, Elizabeth, Talano, Julie-An, and Kitko, Carrie L

Subjects
Biomedical and Clinical Sciences, Cardiovascular Medicine and Haematology, Immunology, Stem Cell Research, Pediatric, Pediatric Cancer, Transplantation, Hematology, Clinical Research, Rare Diseases, Stem Cell Research - Nonembryonic - Human, Cancer, Childhood Leukemia, Regenerative Medicine, Genetics, Academic Medical Centers, Acute Disease, Adolescent, Bone Marrow Transplantation, Child, Child, Preschool, Chronic Disease, Female, Graft vs Host Disease, Humans, Immunosuppressive Agents, International Cooperation, Male, Myeloablative Agonists, Precursor T-Cell Lymphoblastic Leukemia-Lymphoma, Prospective Studies, Recurrence, Remission Induction, Severity of Illness Index, Survival Analysis, Transplantation Conditioning, Transplantation, Homologous, Treatment Outcome, T-cell ALL, Relapse, Acute lymphoblastic leukemia, Clinical Sciences, Cardiovascular medicine and haematology
Abstract

Survival for children with relapsed T cell acute lymphoblastic leukemia (T-ALL) is poor when treated with chemotherapy alone, and outcomes after allogeneic hematopoietic cell transplantation (HCT) is not well described. Two hundred twenty-nine children with T-ALL in second complete remission (CR2) received an HCT after myeloablative conditioning between 2000 and 2011 and were reported to the Center for International Blood and Marrow Transplant Research. Median age was 10 years (range, 2 to 18). Donor source was umbilical cord blood (26%), matched sibling bone marrow (38%), or unrelated bone marrow/peripheral blood (36%). Acute (grades II to IV) and chronic graft-versus-host disease occurred in, respectively, 35% (95% confidence interval [CI], 27% to 45%) and 26% (95% CI, 20% to 33%) of patients. Transplant-related mortality at day 100 and 3-year relapse rates were 13% (95% CI, 9% to 18%) and 30% (95% CI, 24% to 37%), respectively. Three-year overall survival and disease-free survival rates were 48% (95% CI, 41% to 55%) and 46% (95% CI, 39% to 52%), respectively. In multivariate analysis, patients with bone marrow relapse, with or without concurrent extramedullary relapse before HCT, were most likely to relapse (hazard ratio, 3.94; P = .005) as compared with isolated extramedullary disease. In conclusion, HCT for pediatric T-ALL in CR2 demonstrates reasonable and durable outcomes, and consideration for HCT is warranted.

Published
2015

26. Similarity Metrics for Subcellular Analysis of FRET Microscopy Videos

Author

Burke, Michael J., Batista, Victor S., and Davis, Caitlin M.

Abstract

Understanding the heterogeneity of molecular environments within cells is an outstanding challenge of great fundamental and technological interest. Cells are organized into specialized compartments, each with distinct functions. These compartments exhibit dynamic heterogeneity under high-resolution microscopy, which reflects fluctuations in molecular populations, concentrations, and spatial distributions. To enhance our comprehension of the spatial relationships among molecules within cells, it is crucial to analyze images of high-resolution microscopy by clustering individual pixels according to their visible spatial properties and their temporal evolution. Here, we evaluate the effectiveness of similarity metrics based on their ability to facilitate fast and accurate data analysis in time and space. We discuss the capability of these metrics to differentiate subcellular localization, kinetics, and structures of protein-RNA interactions in Forster resonance energy transfer (FRET) microscopy videos, illustrated by a practical example from recent literature. Our results suggest that using the correlation similarity metric to cluster pixels of high-resolution microscopy data should improve the analysis of high-dimensional microscopy data in a wide range of applications.

Published
2024
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29. Data from Integration of Genomic Sequencing Drives Therapeutic Targeting of PDGFRA in T-Cell Acute Lymphoblastic Leukemia/Lymphoblastic Lymphoma

Author

Paolino, Jonathan, primary, Dimitrov, Boris, primary, Apsel Winger, Beth, primary, Sandoval-Perez, Angelica, primary, Rangarajan, Amith Vikram, primary, Ocasio-Martinez, Nicole, primary, Tsai, Harrison K., primary, Li, Yuting, primary, Robichaud, Amanda L., primary, Khalid, Delan, primary, Hatton, Charlie, primary, Gillani, Riaz, primary, Polonen, Petri, primary, Dilig, Anthony, primary, Gotti, Giacomo, primary, Kavanagh, Julia, primary, Adhav, Asmani A., primary, Gow, Sean, primary, Tsai, Jonathan, primary, Li, Yen Der, primary, Ebert, Benjamin L., primary, Van Allen, Eliezer M., primary, Bledsoe, Jacob, primary, Kim, Annette S., primary, Tasian, Sarah K., primary, Cooper, Stacy L., primary, Cooper, Todd M., primary, Hijiya, Nobuko, primary, Sulis, Maria Luisa, primary, Shukla, Neerav N., primary, Magee, Jeffrey A., primary, Mullighan, Charles G., primary, Burke, Michael J., primary, Luskin, Marlise R., primary, Mar, Brenton G., primary, Jacobson, Matthew P., primary, Harris, Marian H., primary, Stegmaier, Kimberly, primary, Place, Andrew E., primary, and Pikman, Yana, primary

Published
2023
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30. Supplementary Table 4 from Integration of Genomic Sequencing Drives Therapeutic Targeting of PDGFRA in T-Cell Acute Lymphoblastic Leukemia/Lymphoblastic Lymphoma

Author

Paolino, Jonathan, primary, Dimitrov, Boris, primary, Apsel Winger, Beth, primary, Sandoval-Perez, Angelica, primary, Rangarajan, Amith Vikram, primary, Ocasio-Martinez, Nicole, primary, Tsai, Harrison K., primary, Li, Yuting, primary, Robichaud, Amanda L., primary, Khalid, Delan, primary, Hatton, Charlie, primary, Gillani, Riaz, primary, Polonen, Petri, primary, Dilig, Anthony, primary, Gotti, Giacomo, primary, Kavanagh, Julia, primary, Adhav, Asmani A., primary, Gow, Sean, primary, Tsai, Jonathan, primary, Li, Yen Der, primary, Ebert, Benjamin L., primary, Van Allen, Eliezer M., primary, Bledsoe, Jacob, primary, Kim, Annette S., primary, Tasian, Sarah K., primary, Cooper, Stacy L., primary, Cooper, Todd M., primary, Hijiya, Nobuko, primary, Sulis, Maria Luisa, primary, Shukla, Neerav N., primary, Magee, Jeffrey A., primary, Mullighan, Charles G., primary, Burke, Michael J., primary, Luskin, Marlise R., primary, Mar, Brenton G., primary, Jacobson, Matthew P., primary, Harris, Marian H., primary, Stegmaier, Kimberly, primary, Place, Andrew E., primary, and Pikman, Yana, primary

Published
2023
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31. Supplementary Figure 2 from Integration of Genomic Sequencing Drives Therapeutic Targeting of PDGFRA in T-Cell Acute Lymphoblastic Leukemia/Lymphoblastic Lymphoma

Author

Paolino, Jonathan, primary, Dimitrov, Boris, primary, Apsel Winger, Beth, primary, Sandoval-Perez, Angelica, primary, Rangarajan, Amith Vikram, primary, Ocasio-Martinez, Nicole, primary, Tsai, Harrison K., primary, Li, Yuting, primary, Robichaud, Amanda L., primary, Khalid, Delan, primary, Hatton, Charlie, primary, Gillani, Riaz, primary, Polonen, Petri, primary, Dilig, Anthony, primary, Gotti, Giacomo, primary, Kavanagh, Julia, primary, Adhav, Asmani A., primary, Gow, Sean, primary, Tsai, Jonathan, primary, Li, Yen Der, primary, Ebert, Benjamin L., primary, Van Allen, Eliezer M., primary, Bledsoe, Jacob, primary, Kim, Annette S., primary, Tasian, Sarah K., primary, Cooper, Stacy L., primary, Cooper, Todd M., primary, Hijiya, Nobuko, primary, Sulis, Maria Luisa, primary, Shukla, Neerav N., primary, Magee, Jeffrey A., primary, Mullighan, Charles G., primary, Burke, Michael J., primary, Luskin, Marlise R., primary, Mar, Brenton G., primary, Jacobson, Matthew P., primary, Harris, Marian H., primary, Stegmaier, Kimberly, primary, Place, Andrew E., primary, and Pikman, Yana, primary

Published
2023
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32. Supplementary Table 3 from Integration of Genomic Sequencing Drives Therapeutic Targeting of PDGFRA in T-Cell Acute Lymphoblastic Leukemia/Lymphoblastic Lymphoma

Author

Paolino, Jonathan, primary, Dimitrov, Boris, primary, Apsel Winger, Beth, primary, Sandoval-Perez, Angelica, primary, Rangarajan, Amith Vikram, primary, Ocasio-Martinez, Nicole, primary, Tsai, Harrison K., primary, Li, Yuting, primary, Robichaud, Amanda L., primary, Khalid, Delan, primary, Hatton, Charlie, primary, Gillani, Riaz, primary, Polonen, Petri, primary, Dilig, Anthony, primary, Gotti, Giacomo, primary, Kavanagh, Julia, primary, Adhav, Asmani A., primary, Gow, Sean, primary, Tsai, Jonathan, primary, Li, Yen Der, primary, Ebert, Benjamin L., primary, Van Allen, Eliezer M., primary, Bledsoe, Jacob, primary, Kim, Annette S., primary, Tasian, Sarah K., primary, Cooper, Stacy L., primary, Cooper, Todd M., primary, Hijiya, Nobuko, primary, Sulis, Maria Luisa, primary, Shukla, Neerav N., primary, Magee, Jeffrey A., primary, Mullighan, Charles G., primary, Burke, Michael J., primary, Luskin, Marlise R., primary, Mar, Brenton G., primary, Jacobson, Matthew P., primary, Harris, Marian H., primary, Stegmaier, Kimberly, primary, Place, Andrew E., primary, and Pikman, Yana, primary

Published
2023
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33. Supplementary Table 1 from Integration of Genomic Sequencing Drives Therapeutic Targeting of PDGFRA in T-Cell Acute Lymphoblastic Leukemia/Lymphoblastic Lymphoma

Author

Paolino, Jonathan, primary, Dimitrov, Boris, primary, Apsel Winger, Beth, primary, Sandoval-Perez, Angelica, primary, Rangarajan, Amith Vikram, primary, Ocasio-Martinez, Nicole, primary, Tsai, Harrison K., primary, Li, Yuting, primary, Robichaud, Amanda L., primary, Khalid, Delan, primary, Hatton, Charlie, primary, Gillani, Riaz, primary, Polonen, Petri, primary, Dilig, Anthony, primary, Gotti, Giacomo, primary, Kavanagh, Julia, primary, Adhav, Asmani A., primary, Gow, Sean, primary, Tsai, Jonathan, primary, Li, Yen Der, primary, Ebert, Benjamin L., primary, Van Allen, Eliezer M., primary, Bledsoe, Jacob, primary, Kim, Annette S., primary, Tasian, Sarah K., primary, Cooper, Stacy L., primary, Cooper, Todd M., primary, Hijiya, Nobuko, primary, Sulis, Maria Luisa, primary, Shukla, Neerav N., primary, Magee, Jeffrey A., primary, Mullighan, Charles G., primary, Burke, Michael J., primary, Luskin, Marlise R., primary, Mar, Brenton G., primary, Jacobson, Matthew P., primary, Harris, Marian H., primary, Stegmaier, Kimberly, primary, Place, Andrew E., primary, and Pikman, Yana, primary

Published
2023
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34. Supplementary Table 5 from Integration of Genomic Sequencing Drives Therapeutic Targeting of PDGFRA in T-Cell Acute Lymphoblastic Leukemia/Lymphoblastic Lymphoma

Author

Paolino, Jonathan, primary, Dimitrov, Boris, primary, Apsel Winger, Beth, primary, Sandoval-Perez, Angelica, primary, Rangarajan, Amith Vikram, primary, Ocasio-Martinez, Nicole, primary, Tsai, Harrison K., primary, Li, Yuting, primary, Robichaud, Amanda L., primary, Khalid, Delan, primary, Hatton, Charlie, primary, Gillani, Riaz, primary, Polonen, Petri, primary, Dilig, Anthony, primary, Gotti, Giacomo, primary, Kavanagh, Julia, primary, Adhav, Asmani A., primary, Gow, Sean, primary, Tsai, Jonathan, primary, Li, Yen Der, primary, Ebert, Benjamin L., primary, Van Allen, Eliezer M., primary, Bledsoe, Jacob, primary, Kim, Annette S., primary, Tasian, Sarah K., primary, Cooper, Stacy L., primary, Cooper, Todd M., primary, Hijiya, Nobuko, primary, Sulis, Maria Luisa, primary, Shukla, Neerav N., primary, Magee, Jeffrey A., primary, Mullighan, Charles G., primary, Burke, Michael J., primary, Luskin, Marlise R., primary, Mar, Brenton G., primary, Jacobson, Matthew P., primary, Harris, Marian H., primary, Stegmaier, Kimberly, primary, Place, Andrew E., primary, and Pikman, Yana, primary

Published
2023
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35. Supplementary Table 6 from Integration of Genomic Sequencing Drives Therapeutic Targeting of PDGFRA in T-Cell Acute Lymphoblastic Leukemia/Lymphoblastic Lymphoma

Author

Paolino, Jonathan, primary, Dimitrov, Boris, primary, Apsel Winger, Beth, primary, Sandoval-Perez, Angelica, primary, Rangarajan, Amith Vikram, primary, Ocasio-Martinez, Nicole, primary, Tsai, Harrison K., primary, Li, Yuting, primary, Robichaud, Amanda L., primary, Khalid, Delan, primary, Hatton, Charlie, primary, Gillani, Riaz, primary, Polonen, Petri, primary, Dilig, Anthony, primary, Gotti, Giacomo, primary, Kavanagh, Julia, primary, Adhav, Asmani A., primary, Gow, Sean, primary, Tsai, Jonathan, primary, Li, Yen Der, primary, Ebert, Benjamin L., primary, Van Allen, Eliezer M., primary, Bledsoe, Jacob, primary, Kim, Annette S., primary, Tasian, Sarah K., primary, Cooper, Stacy L., primary, Cooper, Todd M., primary, Hijiya, Nobuko, primary, Sulis, Maria Luisa, primary, Shukla, Neerav N., primary, Magee, Jeffrey A., primary, Mullighan, Charles G., primary, Burke, Michael J., primary, Luskin, Marlise R., primary, Mar, Brenton G., primary, Jacobson, Matthew P., primary, Harris, Marian H., primary, Stegmaier, Kimberly, primary, Place, Andrew E., primary, and Pikman, Yana, primary

Published
2023
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36. Supplementary Table 2 from Integration of Genomic Sequencing Drives Therapeutic Targeting of PDGFRA in T-Cell Acute Lymphoblastic Leukemia/Lymphoblastic Lymphoma

Author

Paolino, Jonathan, primary, Dimitrov, Boris, primary, Apsel Winger, Beth, primary, Sandoval-Perez, Angelica, primary, Rangarajan, Amith Vikram, primary, Ocasio-Martinez, Nicole, primary, Tsai, Harrison K., primary, Li, Yuting, primary, Robichaud, Amanda L., primary, Khalid, Delan, primary, Hatton, Charlie, primary, Gillani, Riaz, primary, Polonen, Petri, primary, Dilig, Anthony, primary, Gotti, Giacomo, primary, Kavanagh, Julia, primary, Adhav, Asmani A., primary, Gow, Sean, primary, Tsai, Jonathan, primary, Li, Yen Der, primary, Ebert, Benjamin L., primary, Van Allen, Eliezer M., primary, Bledsoe, Jacob, primary, Kim, Annette S., primary, Tasian, Sarah K., primary, Cooper, Stacy L., primary, Cooper, Todd M., primary, Hijiya, Nobuko, primary, Sulis, Maria Luisa, primary, Shukla, Neerav N., primary, Magee, Jeffrey A., primary, Mullighan, Charles G., primary, Burke, Michael J., primary, Luskin, Marlise R., primary, Mar, Brenton G., primary, Jacobson, Matthew P., primary, Harris, Marian H., primary, Stegmaier, Kimberly, primary, Place, Andrew E., primary, and Pikman, Yana, primary

Published
2023
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37. Supplementary Figure 1 from Integration of Genomic Sequencing Drives Therapeutic Targeting of PDGFRA in T-Cell Acute Lymphoblastic Leukemia/Lymphoblastic Lymphoma

Author

Paolino, Jonathan, primary, Dimitrov, Boris, primary, Apsel Winger, Beth, primary, Sandoval-Perez, Angelica, primary, Rangarajan, Amith Vikram, primary, Ocasio-Martinez, Nicole, primary, Tsai, Harrison K., primary, Li, Yuting, primary, Robichaud, Amanda L., primary, Khalid, Delan, primary, Hatton, Charlie, primary, Gillani, Riaz, primary, Polonen, Petri, primary, Dilig, Anthony, primary, Gotti, Giacomo, primary, Kavanagh, Julia, primary, Adhav, Asmani A., primary, Gow, Sean, primary, Tsai, Jonathan, primary, Li, Yen Der, primary, Ebert, Benjamin L., primary, Van Allen, Eliezer M., primary, Bledsoe, Jacob, primary, Kim, Annette S., primary, Tasian, Sarah K., primary, Cooper, Stacy L., primary, Cooper, Todd M., primary, Hijiya, Nobuko, primary, Sulis, Maria Luisa, primary, Shukla, Neerav N., primary, Magee, Jeffrey A., primary, Mullighan, Charles G., primary, Burke, Michael J., primary, Luskin, Marlise R., primary, Mar, Brenton G., primary, Jacobson, Matthew P., primary, Harris, Marian H., primary, Stegmaier, Kimberly, primary, Place, Andrew E., primary, and Pikman, Yana, primary

Published
2023
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38. Reply to C.T. Matava et al.

Author

Alexander, Sarah, Kairalla, John A., Gupta, Sumit, Hibbitts, Emily, Weisman, Hannah, Anghelescu, Doralina, Winick, Naomi J., Krull, Kevin R., Salzer, Wanda L., Burke, Michael J., Gore, Lia, Devidas, Meenakshi, Embry, Leanne, Raetz, Elizabeth A., Hunger, Stephen P., Loh, Mignon L., and Hardy, Kristina K.

Published
2024
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40. Outcomes in Children, Adolescents, and Young Adults With Down Syndrome and ALL: A Report From the Children's Oncology Group

Author

Rabin, Karen R., primary, Devidas, Meenakshi, additional, Chen, Zhiguo, additional, Ji, Lingyun, additional, Kairalla, John, additional, Hitzler, Johann K., additional, Yang, Jun J., additional, Carroll, Andrew J., additional, Heerema, Nyla A., additional, Borowitz, Michael J., additional, Wood, Brent L., additional, Roberts, Kathryn G., additional, Mullighan, Charles G., additional, Harvey, Richard C., additional, Chen, I-Ming, additional, Willman, Cheryl L., additional, Reshmi, Shalini C., additional, Gastier-Foster, Julie M., additional, Bhojwani, Deepa, additional, Rheingold, Susan R., additional, Maloney, Kelly W., additional, Mattano, Leonard A., additional, Larsen, Eric C., additional, Schore, Reuven J., additional, Burke, Michael J., additional, Salzer, Wanda L., additional, Winick, Naomi J., additional, Carroll, William L., additional, Raetz, Elizabeth A., additional, Loh, Mignon L., additional, Hunger, Stephen P., additional, and Angiolillo, Anne L., additional

Published
2023
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41. Integration of genomic sequencing drives therapeutic targeting of PDGFRA in T-cell acute lymphoblastic leukemia/lymphoblastic lymphoma.

Author

Paolino, Jonathan, primary, Dimitrov, Boris, additional, Apsel Winger, Beth, additional, Sandoval-Perez, Angelica, additional, Rangarajan, Amith Vikram., additional, Ocasio-Martinez, Nicole, additional, Tsai, Harrison K., additional, Li, Yuting, additional, Robichaud, Amanda L., additional, Khalid, Delan, additional, Hatton, Charlie, additional, Gillani, Riaz, additional, Polonen, Petri, additional, Dilig, Anthony, additional, Gotti, Giacomo, additional, Kavanagh, Julia, additional, Adhav, Asmani A., additional, Gow, Sean, additional, Tsai, Jonathan, additional, Li, Yen Der, additional, Ebert, Benjamin L., additional, Van Allen, Eliezer M., additional, Bledsoe, Jacob, additional, Kim, Annette S., additional, Tasian, Sarah K., additional, Cooper, Stacy L., additional, Cooper, Todd M., additional, Hijiya, Nobuko, additional, Sulis, Maria Luisa, additional, Shukla, Neerav N., additional, Magee, Jeffrey A., additional, Mullighan, Charles G., additional, Burke, Michael J., additional, Luskin, Marlise R., additional, Mar, Brenton G., additional, Jacobson, Matthew P., additional, Harris, Marian H., additional, Stegmaier, Kimberly, additional, Place, Andrew E., additional, and Pikman, Yana, additional

Published
2023
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43. Targetable kinase gene fusions in high-risk B-ALL: a study from the Children's Oncology Group

Author

Reshmi, Shalini C., Harvey, Richard C., Roberts, Kathryn G., Stonerock, Eileen, Smith, Amy, Jenkins, Heather, Chen, I-Ming, Valentine, Marc, Liu, Yu, Li, Yongjin, Shao, Ying, Easton, John, Payne-Turner, Debbie, Gu, Zhaohui, Tran, Thai Hoa, Nguyen, Jonathan V., Devidas, Meenakshi, Dai, Yunfeng, Heerema, Nyla A., Carroll, Andrew J., III, Raetz, Elizabeth A., Borowitz, Michael J., Wood, Brent L., Angiolillo, Anne L., Burke, Michael J., Salzer, Wanda L., Zweidler-McKay, Patrick A., Rabin, Karen R., Carroll, William L., Zhang, Jinghui, Loh, Mignon L., Mullighan, Charles G., Willman, Cheryl L., Gastier-Foster, Julie M., and Hunger, Stephen P.

Published
2017
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47. Impaired neurocognitive functioning 3 months following diagnosis of high‐risk acute lymphoblastic leukemia: A report from the Children's Oncology Group

Author

Hardy, Kristina K., primary, Kairalla, John A., additional, Gioia, Anthony R., additional, Weisman, Hannah S., additional, Gurung, Meera, additional, Noll, Robert B., additional, Hinds, Pamela S., additional, Hibbitts, Emily, additional, Salzer, Wanda L., additional, Burke, Michael J., additional, Winick, Naomi J., additional, and Embry, Leanne, additional

Published
2023
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