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2. Olutasidenib (FT-2102) induces durable complete remissions in patients with relapsed or refractory IDH1-mutated AML.

Author

de Botton, Stéphane, Fenaux, Pierre, Yee, Karen, Récher, Christian, Wei, Andrew, Montesinos, Pau, Taussig, David, Pigneux, Arnaud, Braun, Thorsten, Curti, Antonio, Grove, Carolyn, Khwaja, Asim, Legrand, Ollivier, Peterlin, Pierre, Arnan, Montserrat, Blum, William, Cilloni, Daniela, Hiwase, Devendra, Jurcic, Joseph, Krauter, Jürgen, Thomas, Xavier, Watts, Justin, Yang, Jay, Polyanskaya, Olga, Brevard, Julie, Sweeney, Jennifer, Barrett, Emma, Cortes, Jorge, and Jonas, Brian

Subjects
Humans, Adult, Middle Aged, Aged, Aged, 80 and over, Pyridines, Quinolines, Leukemia, Myeloid, Acute, Prognosis, Isocitrate Dehydrogenase
Abstract

Olutasidenib (FT-2102) is a potent, selective, oral, small-molecule inhibitor of mutant isocitrate dehydrogenase 1 (mIDH1). Overall, 153 IDH1 inhibitor-naive patients with mIDH1R132 relapsed/refractory (R/R) acute myeloid leukemia (AML) received olutasidenib monotherapy 150 mg twice daily in the pivotal cohort of this study. The median age of participants was 71 years (range, 32-87 years) and the median number of prior regimens received by patients was 2 (1-7). The rate of complete remission (CR) plus CR with partial hematologic recovery (CRh) was 35%, and the overall response rate was 48%. Response rates were similar in patients who had, and who had not, received prior venetoclax. With 55% of patients censored at the time of data cut-off, the median duration of CR/CRh was 25.9 months. The median duration of overall response was 11.7 months, and the median overall survival was 11.6 months. Of 86 patients who were transfusion dependent at baseline, a 56-day transfusion independence was achieved in 29 (34%), which included patients in all response groups. Grade 3 or 4 treatment-emergent adverse events (≥10%) were febrile neutropenia and anemia (n = 31; 20% each), thrombocytopenia (n = 25; 16%), and neutropenia (n = 20; 13%). Differentiation syndrome adverse events of special interest occurred in 22 (14%) patients, with 14 (9%) grade ≥3 and 1 fatal case reported. Overall, olutasidenib induced durable remissions and transfusion independence with a well-characterized and manageable side effect profile. The observed efficacy represents a therapeutic advance in this molecularly defined, poor-prognostic population of patients with mIDH1 R/R AML. This trial was registered at www.clinicaltrials.gov as #NCT02719574.

Published
2023

3. How comparable are patient outcomes in the “real-world” with populations studied in pivotal AML trials?

Author

Tiong, Ing Soo, Wall, Meaghan, Bajel, Ashish, Kalro, Akash, Fleming, Shaun, Roberts, Andrew W., Thiagarajah, Nisha, Chua, Chong Chyn, Latimer, Maya, Yeung, David, Marlton, Paula, Johnston, Amanda, Enjeti, Anoop, Fong, Chun Yew, Cull, Gavin, Larsen, Stephen, Kennedy, Glen, Schwarer, Anthony, Kipp, David, Ramanathan, Sundra, Verner, Emma, Tiley, Campbell, Morris, Edward, Hahn, Uwe, Moore, John, Taper, John, Purtill, Duncan, Warburton, Pauline, Stevenson, William, Murphy, Nicholas, Tan, Peter, Beligaswatte, Ashanka, Mutsando, Howard, Hertzberg, Mark, Shortt, Jake, Szabo, Ferenc, Dunne, Karin, and Wei, Andrew H.

Published
2024
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4. Clinical evaluation of complete remission (CR) with partial hematologic recovery (CRh) in acute myeloid leukemia: a report of 7235 patients from seven cohorts

Author

Appelbaum, Jacob S., Wei, Andrew H., Mandrekar, Sumithra J., Tiong, Ing S., Chua, Chong Chyn, Teh, Tse-Chieh, Fong, Chun Yew, Ting, Stephen B., Weber, Daniela, Benner, Axel, Hill, Hannah, Saadati, Maral, Yin, Jun, Stone, Richard M., Garcia-Manero, Guillermo, Erba, Harry P., Uy, Geoffrey L., Marcucci, Guido, Larson, Richard A., Thomas, Abin, Freeman, Sylvie D., Almuina, Nuria Marquez, Döhner, Konstanze, Thomas, Ian, Russel, Nigel H., Döhner, Hartmut, Othus, Megan, Estey, Elihu H., and Walter, Roland B.

Published
2024
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5. Efficient and Accurate Quantized Image Super-Resolution on Mobile NPUs, Mobile AI & AIM 2022 challenge: Report

Author

Ignatov, Andrey, Timofte, Radu, Denna, Maurizio, Younes, Abdel, Gankhuyag, Ganzorig, Huh, Jingang, Kim, Myeong Kyun, Yoon, Kihwan, Moon, Hyeon-Cheol, Lee, Seungho, Choe, Yoonsik, Jeong, Jinwoo, Kim, Sungjei, Smyl, Maciej, Latkowski, Tomasz, Kubik, Pawel, Sokolski, Michal, Ma, Yujie, Chao, Jiahao, Zhou, Zhou, Gao, Hongfan, Yang, Zhengfeng, Zeng, Zhenbing, Zhuge, Zhengyang, Li, Chenghua, Zhu, Dan, Sun, Mengdi, Duan, Ran, Gao, Yan, Kong, Lingshun, Sun, Long, Li, Xiang, Zhang, Xingdong, Zhang, Jiawei, Wu, Yaqi, Pan, Jinshan, Yu, Gaocheng, Zhang, Jin, Zhang, Feng, Ma, Zhe, Wang, Hongbin, Cho, Hojin, Kim, Steve, Li, Huaen, Ma, Yanbo, Luo, Ziwei, Li, Youwei, Yu, Lei, Wen, Zhihong, Wu, Qi, Fan, Haoqiang, Liu, Shuaicheng, Zhang, Lize, Zong, Zhikai, Kwon, Jeremy, Zhang, Junxi, Li, Mengyuan, Fu, Nianxiang, Ding, Guanchen, Zhu, Han, Chen, Zhenzhong, Li, Gen, Zhang, Yuanfan, Sun, Lei, Zhang, Dafeng, Yang, Neo, Liu, Fitz, Zhao, Jerry, Ayazoglu, Mustafa, Bilecen, Bahri Batuhan, Hirose, Shota, Arunruangsirilert, Kasidis, Ao, Luo, Leung, Ho Chun, Wei, Andrew, Liu, Jie, Liu, Qiang, Yu, Dahai, Li, Ao, Luo, Lei, Zhu, Ce, Hong, Seongmin, Park, Dongwon, Lee, Joonhee, Lee, Byeong Hyun, Lee, Seunggyu, Chun, Se Young, He, Ruiyuan, Jiang, Xuhao, Ruan, Haihang, Zhang, Xinjian, Liu, Jing, Gendy, Garas, Sabor, Nabil, Hou, Jingchao, and He, Guanghui

Subjects
Electrical Engineering and Systems Science - Image and Video Processing, Computer Science - Computer Vision and Pattern Recognition
Abstract

Image super-resolution is a common task on mobile and IoT devices, where one often needs to upscale and enhance low-resolution images and video frames. While numerous solutions have been proposed for this problem in the past, they are usually not compatible with low-power mobile NPUs having many computational and memory constraints. In this Mobile AI challenge, we address this problem and propose the participants to design an efficient quantized image super-resolution solution that can demonstrate a real-time performance on mobile NPUs. The participants were provided with the DIV2K dataset and trained INT8 models to do a high-quality 3X image upscaling. The runtime of all models was evaluated on the Synaptics VS680 Smart Home board with a dedicated edge NPU capable of accelerating quantized neural networks. All proposed solutions are fully compatible with the above NPU, demonstrating an up to 60 FPS rate when reconstructing Full HD resolution images. A detailed description of all models developed in the challenge is provided in this paper., Comment: arXiv admin note: text overlap with arXiv:2105.07825, arXiv:2105.08826, arXiv:2211.04470, arXiv:2211.03885, arXiv:2211.05256

Published
2022

6. Outcomes in Patients With Poor-risk Cytogenetics With or Without TP53 Mutations Treated With Venetoclax and Azacitidine

Author

Pollyea, Daniel A, Pratz, Keith W, Wei, Andrew H, Pullarkat, Vinod, Jonas, Brian A, Recher, Christian, Babu, Sunil, Schuh, Andre C, Dail, Monique, Sun, Yan, Potluri, Jalaja, Chyla, Brenda, and DiNardo, Courtney D

Subjects
Cancer, Hematology, Clinical Research, Clinical Trials and Supportive Activities, Evaluation of treatments and therapeutic interventions, 6.1 Pharmaceuticals, Humans, Antineoplastic Combined Chemotherapy Protocols, Azacitidine, Bridged Bicyclo Compounds, Heterocyclic, Cytogenetic Analysis, Leukemia, Myeloid, Acute, Mutation, Tumor Suppressor Protein p53, Treatment Outcome, Clinical Trials, Phase III as Topic, Clinical Trials, Phase I as Topic, Oncology and Carcinogenesis, Oncology & Carcinogenesis
Abstract

PurposeTo evaluate efficacy and safety of venetoclax + azacitidine in treatment-naïve patients with acute myeloid leukemia harboring poor-risk cytogenetics and TP53mut or TP53wt.Patients and methodsWe analyzed data from a phase III study (NCT02993523) comparing venetoclax (400 mg orally days 1-28) + azacitidine (75 mg/m2 days 1-7) or placebo + azacitidine, and from a phase Ib study (NCT02203773) of venetoclax + azacitidine. Patients were ineligible for intensive therapy. TP53 status was analyzed centrally; cytogenetic studies were performed locally.ResultsPatients (n = 127) with poor-risk cytogenetics receiving venetoclax + azacitidine (TP53wt = 50; TP53mut = 54) were compared with patients with poor-risk cytogenetics (n = 56) receiving azacitidine alone (TP53wt = 22; TP53mut = 18).For poor-risk cytogenetics + TP53wt patients, venetoclax + azacitidine versus azacitidine alone resulted in composite remission rates (CRc) of 70% versus 23%, median duration of remission (DoR) of 18.4 versus 8.5 months, and median overall survival (OS) of 23.4 versus 11.3 months, respectively. Outcomes with venetoclax + azacitidine were comparable with similarly treated patients with intermediate-risk cytogenetics and TP53wt.For poor-risk cytogenetics + TP53mut patients, venetoclax + azacitidine versus azacitidine alone resulted in CRc of 41% versus 17%, median DoR of 6.5 versus 6.7 months, and median OS of 5.2 versus 4.9 months, respectively.For poor-risk cytogenetics + TP53mut patients, predominant grade ≥3 adverse events (AE) for venetoclax + azacitidine versus azacitidine were febrile neutropenia (55%/39%), thrombocytopenia (28%/28%), neutropenia (26%/17%), anemia (13%/6%), and pneumonia (28%/33%). AEs were comparable between TP53mut and TP53wt patients.ConclusionsIn poor-risk cytogenetics + TP53mut patients, venetoclax + azacitidine improved remission rates but not DoR or OS compared with azacitidine alone. However, in poor-risk cytogenetics + TP53wt patients, venetoclax + azacitidine resulted in higher remission rates and longer DoR and OS than azacitidine alone, with outcomes comparable with similarly treated patients with intermediate-risk cytogenetics. Toxicities were similar in TP53mut and TP53wt patients. See related commentary by Green and Zeidner, p. 5235.

Published
2022

10. Venetoclax combinations delay the time to deterioration of HRQoL in unfit patients with acute myeloid leukemia

Author

Pratz, Keith W, Panayiotidis, Panayiotis, Recher, Christian, Wei, Xudong, Jonas, Brian A, Montesinos, Pau, Ivanov, Vladimir, Schuh, Andre C, DiNardo, Courtney D, Novak, Jan, Pejsa, Vlatko, Stevens, Don, Yeh, Su-Peng, Kim, Inho, Turgut, Mehmet, Fracchiolla, Nicola, Yamamoto, Kazuhito, Ofran, Yishai, Wei, Andrew H, Bui, Cat N, Benjamin, Katy, Kamalakar, Rajesh, Potluri, Jalaja, Mendes, Wellington, Devine, Jacob, and Fiedler, Walter

Subjects
Biomedical and Clinical Sciences, Cardiovascular Medicine and Haematology, Oncology and Carcinogenesis, Clinical Research, Rare Diseases, Cancer, Antineoplastic Combined Chemotherapy Protocols, Bridged Bicyclo Compounds, Heterocyclic, Cytarabine, Fatigue, Humans, Leukemia, Myeloid, Acute, Quality of Life, Sulfonamides, Cardiorespiratory Medicine and Haematology, Cardiovascular medicine and haematology, Oncology and carcinogenesis
Abstract

Phase 3 trials Viale-A and Viale-C evaluated health-related quality of life (HRQoL) in patients with AML unfit for intensive chemotherapy who received venetoclax (VEN) + (AZA) (Viale-A) or low-dose cytarabine (LDAC) (Viale-C) or placebo (PBO) + AZA or LDAC. Patient-reported outcomes included: EORTC QLQ-C30 global health status (GHS/QoL) and physical functioning (PF), PROMIS Cancer Fatigue Short Form 7a (Fatigue), and EQ-5D-5L health status visual analog scale (HS-VAS). Time to deterioration (TTD), defined as worsening from baseline in meaningful change thresholds (MCT) of ≥10, 5, or 7 points for GHS/QoL or PF, fatigue, and HS-VAS, respectively, was assessed; differences between groups were analyzed using Kaplan-Meier and unadjusted log-rank analyses. VEN + AZA vs PBO + AZA patients had longer TTD in GHS/QoL (P = 0.066) and fatigue (P = 0.189), and significantly longer TTD in PF (P = 0.028) and HS-VAS (P MCT in GHS/QoL. Overall, VEN may positively impact HRQoL in patients with AML ineligible for intensive chemotherapy, leading to longer preservation of functioning and overall health status.

Published
2022

11. Efficient and Accurate Quantized Image Super-Resolution on Mobile NPUs, Mobile AI & AIM 2022 Challenge: Report

Author

Ignatov, Andrey, Timofte, Radu, Denna, Maurizio, Younes, Abdel, Gankhuyag, Ganzorig, Huh, Jingang, Kim, Myeong Kyun, Yoon, Kihwan, Moon, Hyeon-Cheol, Lee, Seungho, Choe, Yoonsik, Jeong, Jinwoo, Kim, Sungjei, Smyl, Maciej, Latkowski, Tomasz, Kubik, Pawel, Sokolski, Michal, Ma, Yujie, Chao, Jiahao, Zhou, Zhou, Gao, Hongfan, Yang, Zhengfeng, Zeng, Zhenbing, Zhuge, Zhengyang, Li, Chenghua, Zhu, Dan, Sun, Mengdi, Duan, Ran, Gao, Yan, Kong, Lingshun, Sun, Long, Li, Xiang, Zhang, Xingdong, Zhang, Jiawei, Wu, Yaqi, Pan, Jinshan, Yu, Gaocheng, Zhang, Jin, Zhang, Feng, Ma, Zhe, Wang, Hongbin, Cho, Hojin, Kim, Steve, Li, Huaen, Ma, Yanbo, Luo, Ziwei, Li, Youwei, Yu, Lei, Wen, Zhihong, Wu, Qi, Fan, Haoqiang, Liu, Shuaicheng, Zhang, Lize, Zong, Zhikai, Kwon, Jeremy, Zhang, Junxi, Li, Mengyuan, Fu, Nianxiang, Ding, Guanchen, Zhu, Han, Chen, Zhenzhong, Li, Gen, Zhang, Yuanfan, Sun, Lei, Zhang, Dafeng, Yang, Neo, Liu, Fitz, Zhao, Jerry, Ayazoglu, Mustafa, Bilecen, Bahri Batuhan, Hirose, Shota, Arunruangsirilert, Kasidis, Ao, Luo, Leung, Ho Chun, Wei, Andrew, Liu, Jie, Liu, Qiang, Yu, Dahai, Li, Ao, Luo, Lei, Zhu, Ce, Hong, Seongmin, Park, Dongwon, Lee, Joonhee, Lee, Byeong Hyun, Lee, Seunggyu, Chun, Se Young, He, Ruiyuan, Jiang, Xuhao, Ruan, Haihang, Zhang, Xinjian, Liu, Jing, Gendy, Garas, Sabor, Nabil, Hou, Jingchao, He, Guanghui, Goos, Gerhard, Founding Editor, Hartmanis, Juris, Founding Editor, Bertino, Elisa, Editorial Board Member, Gao, Wen, Editorial Board Member, Steffen, Bernhard, Editorial Board Member, Yung, Moti, Editorial Board Member, Karlinsky, Leonid, editor, Michaeli, Tomer, editor, and Nishino, Ko, editor

Published
2023
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13. Venetoclax–based low intensity therapy in molecular failure of NPM1-mutated AML

Author

Jimenez-Chillon, Carlos, Othman, Jad, Taussig, David, Jimenez-Vicente, Carlos, Martinez-Roca, Alexandra, Tiong, Ing Soo, Jain, Manish, Aries, James, Cakmak, Seda, Knapper, Steven, Kristensen, Daniel Tuyet, Murthy, Vidhya, Galani, Joy Zacharoula, Kallmeyer, Charlotte, Ngu, Loretta, Veale, David, Bolam, Simon, Orfali, Nina, Parker, Anne, Manson, Cara, Parker, Jane, Erblich, Thomas, Richardson, Deborah, Mokretar, Katya, Potter, Nicola, Overgaard, Ulrik Malthe, Roug, Anne Stidsholt, Wei, Andrew H., Esteve, Jordi, Jädersten, Martin, Russell, Nigel, and Dillon, Richard

Published
2024
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16. Sorafenib plus intensive chemotherapy in newly diagnosed FLT3-ITD AML: a randomized, placebo-controlled study by the ALLG

Author

Loo, Sun, Roberts, Andrew W., Anstee, Natasha S., Kennedy, Glen A., He, Simon, Schwarer, Anthony P., Enjeti, Anoop K., D’Rozario, James, Marlton, Paula, Bilmon, Ian A., Taper, John, Cull, Gavin, Tiley, Campbell, Verner, Emma, Hahn, Uwe, Hiwase, Devendra K., Iland, Harry J., Murphy, Nick, Ramanathan, Sundra, Reynolds, John, Ong, Doen Ming, Tiong, Ing Soo, Wall, Meaghan, Murray, Michael, Rawling, Tristan, Leadbetter, Joanna, Rowley, Leesa, Latimer, Maya, Yuen, Sam, Ting, Stephen B., Fong, Chun Yew, Morris, Kirk, Bajel, Ashish, Seymour, John F., Levis, Mark J., and Wei, Andrew H.

Published
2023
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17. Cusatuzumab plus azacitidine in newly diagnosed acute myeloid leukaemia ineligible for intensive chemotherapy (CULMINATE): part one of a randomised, phase 2, dose optimisation study

Author

Pabst, Thomas, Papayannidis, Cristina, Demirkan, Fatih, Doronin, Vadim, Fogliatto, Laura M, Guttke, Christina, Gyan, Emmanuel, Hamad, Nada, Herrera, Pilar, Hultberg, Anna, Jacobs, Julie, Johnson, Amy J, Langlois, Angélique, Ma, Xuewen, Martinelli, Giovanni, Arnan, Montserrat, Müller, Rouven, Nottage, Kerri, Ofran, Yishai, Özcan, Muhit, Samoilova, Olga, Tolbert, Jaszianne A, Trudel, Géralyn C, Xiu, Liang, Vey, Norbert, and Wei, Andrew H

Published
2023
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18. Classification, risk stratification and response assessment in myelodysplastic syndromes/neoplasms (MDS): A state-of-the-art report on behalf of the International Consortium for MDS (icMDS)

Author

Stahl, Maximilian, Bewersdorf, Jan Philipp, Xie, Zhuoer, Porta, Matteo Giovanni Della, Komrokji, Rami, Xu, Mina L., Abdel-Wahab, Omar, Taylor, Justin, Steensma, David P., Starczynowski, Daniel T., Sekeres, Mikkael A., Sanz, Guillermo, Sallman, David A., Roboz, Gail J., Platzbecker, Uwe, Patnaik, Mrinal M., Padron, Eric, Odenike, Olatoyosi, Nimer, Stephen D., Nazha, Aziz, Majeti, Ravi, Loghavi, Sanam, Little, Richard F., List, Alan F., Kim, Tae Kon, Hourigan, Christopher S., Hasserjian, Robert P., Halene, Stephanie, Griffiths, Elizabeth A., Gore, Steven D., Greenberg, Peter, Figueroa, Maria E., Fenaux, Pierre, Efficace, Fabio, DeZern, Amy E., Daver, Naval G., Churpek, Jane E., Carraway, Hetty E., Buckstein, Rena, Brunner, Andrew M., Boultwood, Jacqueline, Borate, Uma, Bejar, Rafael, Bennett, John M., Wei, Andrew H., Santini, Valeria, Savona, Michael R., and Zeidan, Amer M.

Published
2023
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19. Somatic mutational landscape of hereditary hematopoietic malignancies caused by germline variants in RUNX1, GATA2, and DDX41

Author

Homan, Claire C., Drazer, Michael W., Yu, Kai, Lawrence, David M., Feng, Jinghua, Arriola-Martinez, Luis, Pozsgai, Matthew J., McNeely, Kelsey E., Ha, Thuong, Venugopal, Parvathy, Arts, Peer, King-Smith, Sarah L., Cheah, Jesse, Armstrong, Mark, Wang, Paul, Bödör, Csaba, Cantor, Alan B., Cazzola, Mario, Degelman, Erin, DiNardo, Courtney D., Duployez, Nicolas, Favier, Remi, Fröhling, Stefan, Rio-Machin, Ana, Klco, Jeffery M., Krämer, Alwin, Kurokawa, Mineo, Lee, Joanne, Malcovati, Luca, Morgan, Neil V., Natsoulis, Georges, Owen, Carolyn, Patel, Keyur P., Preudhomme, Claude, Raslova, Hana, Rienhoff, Hugh, Ripperger, Tim, Schulte, Rachael, Tawana, Kiran, Velloso, Elvira, Yan, Benedict, Kim, Erika, Sood, Raman, Hsu, Amy P., Holland, Steven M., Phillips, Kerry, Poplawski, Nicola K., Babic, Milena, Wei, Andrew H., Forsyth, Cecily, Mar Fan, Helen, Lewis, Ian D., Cooney, Julian, Susman, Rachel, Fox, Lucy C., Blombery, Piers, Singhal, Deepak, Hiwase, Devendra, Phipson, Belinda, Schreiber, Andreas W., Hahn, Christopher N., Scott, Hamish S., Liu, Paul, Godley, Lucy A., and Brown, Anna L.

Published
2023
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20. Current landscape of translational and clinical research in myelodysplastic syndromes/neoplasms (MDS): Proceedings from the 1st International Workshop on MDS (iwMDS) Of the International Consortium for MDS (icMDS)

Author

Bewersdorf, Jan Philipp, Xie, Zhuoer, Bejar, Rafael, Borate, Uma, Boultwood, Jacqueline, Brunner, Andrew M., Buckstein, Rena, Carraway, Hetty E., Churpek, Jane E., Daver, Naval G., Porta, Matteo Giovanni Della, DeZern, Amy E., Fenaux, Pierre, Figueroa, Maria E., Gore, Steven D., Griffiths, Elizabeth A., Halene, Stephanie, Hasserjian, Robert P., Hourigan, Christopher S., Kim, Tae Kon, Komrokji, Rami, Kuchroo, Vijay K., List, Alan F., Loghavi, Sanam, Majeti, Ravindra, Odenike, Olatoyosi, Patnaik, Mrinal M., Platzbecker, Uwe, Roboz, Gail J., Sallman, David A., Santini, Valeria, Sanz, Guillermo, Sekeres, Mikkael A., Stahl, Maximilian, Starczynowski, Daniel T., Steensma, David P., Taylor, Justin, Abdel-Wahab, Omar, Xu, Mina L., Savona, Michael R., Wei, Andrew H., and Zeidan, Amer M.

Published
2023
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21. Finding consistency in classifications of myeloid neoplasms: a perspective on behalf of the International Workshop for Myelodysplastic Syndromes

Author

Zeidan, Amer M., Bewersdorf, Jan Philipp, Buckstein, Rena, Sekeres, Mikkael A., Steensma, David P., Platzbecker, Uwe, Loghavi, Sanam, Boultwood, Jacqueline, Bejar, Rafael, Bennett, John M., Borate, Uma, Brunner, Andrew M., Carraway, Hetty, Churpek, Jane E., Daver, Naval G., Della Porta, Matteo, DeZern, Amy E., Efficace, Fabio, Fenaux, Pierre, Figueroa, Maria E., Greenberg, Peter, Griffiths, Elizabeth A., Halene, Stephanie, Hasserjian, Robert P., Hourigan, Christopher S., Kim, Nina, Kim, Tae Kon, Komrokji, Rami S., Kutchroo, Vijay, List, Alan F., Little, Richard F., Majeti, Ravi, Nazha, Aziz, Nimer, Stephen D., Odenike, Olatoyosi, Padron, Eric, Patnaik, Mrinal M., Roboz, Gail J., Sallman, David A., Sanz, Guillermo, Stahl, Maximilian, Starczynowski, Daniel T., Taylor, Justin, Xie, Zhuoer, Xu, Mina, Savona, Michael R., Wei, Andrew H., Abdel-Wahab, Omar, and Santini, Valeria

Published
2022
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24. Acquired mutations in BAX confer resistance to BH3-mimetic therapy in acute myeloid leukemia

Author

Moujalled, Donia M., Brown, Fiona C., Chua, Chong Chyn, Dengler, Michael A., Pomilio, Giovanna, Anstee, Natasha S., Litalien, Veronique, Thompson, Ella, Morley, Thomas, MacRaild, Sarah, Tiong, Ing S., Morris, Rhiannon, Dun, Karen, Zordan, Adrian, Shah, Jaynish, Banquet, Sebastien, Halilovic, Ensar, Morris, Erick, Herold, Marco J., Lessene, Guillaume, Adams, Jerry M., Huang, David C. S., Roberts, Andrew W., Blombery, Piers, and Wei, Andrew H.

Published
2023
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26. Enasidenib vs conventional care in older patients with late-stage mutant-IDH2 relapsed/refractory AML: a randomized phase 3 trial

Author

de Botton, Stéphane, Montesinos, Pau, Schuh, Andre C., Papayannidis, Cristina, Vyas, Paresh, Wei, Andrew H., Ommen, Hans, Semochkin, Sergey, Kim, Hee-Je, Larson, Richard A., Koprivnikar, Jaime, Frankfurt, Olga, Thol, Felicitas, Chromik, Jörg, Byrne, Jenny, Pigneux, Arnaud, Thomas, Xavier, Salamero, Olga, Vidriales, Maria Belen, Doronin, Vadim, Döhner, Hartmut, Fathi, Amir T., Laille, Eric, Yu, Xin, Hasan, Maroof, Martin-Regueira, Patricia, and DiNardo, Courtney D.

Published
2023
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27. Olutasidenib alone or with azacitidine in IDH1-mutated acute myeloid leukaemia and myelodysplastic syndrome: phase 1 results of a phase 1/2 trial

Author

Watts, Justin M, Baer, Maria R, Yang, Jay, Prebet, Thomas, Lee, Sangmin, Schiller, Gary J, Dinner, Shira N, Pigneux, Arnaud, Montesinos, Pau, Wang, Eunice S, Seiter, Karen P, Wei, Andrew H, De Botton, Stephane, Arnan, Montserrat, Donnellan, Will, Schwarer, Anthony P, Récher, Christian, Jonas, Brian A, Ferrell, P Brent, Jr, Marzac, Christophe, Kelly, Patrick, Sweeney, Jennifer, Forsyth, Sanjeev, Guichard, Sylvie M, Brevard, Julie, Henrick, Patrick, Mohamed, Hesham, and Cortes, Jorge E

Published
2023
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29. Genomic subtyping and therapeutic targeting of acute erythroleukemia

Author

Iacobucci, Ilaria, Wen, Ji, Meggendorfer, Manja, Choi, John K, Shi, Lei, Pounds, Stanley B, Carmichael, Catherine L, Masih, Katherine E, Morris, Sarah M, Lindsley, R Coleman, Janke, Laura J, Alexander, Thomas B, Song, Guangchun, Qu, Chunxu, Li, Yongjin, Payne-Turner, Debbie, Tomizawa, Daisuke, Kiyokawa, Nobutaka, Valentine, Marcus, Valentine, Virginia, Basso, Giuseppe, Locatelli, Franco, Enemark, Eric J, Kham, Shirley KY, Yeoh, Allen EJ, Ma, Xiaotu, Zhou, Xin, Sioson, Edgar, Rusch, Michael, Ries, Rhonda E, Stieglitz, Elliot, Hunger, Stephen P, Wei, Andrew H, To, L Bik, Lewis, Ian D, D’Andrea, Richard J, Kile, Benjamin T, Brown, Anna L, Scott, Hamish S, Hahn, Christopher N, Marlton, Paula, Pei, Deqing, Cheng, Cheng, Loh, Mignon L, Ebert, Benjamin L, Meshinchi, Soheil, Haferlach, Torsten, and Mullighan, Charles G

Subjects
Agricultural, Veterinary and Food Sciences, Biological Sciences, Bioinformatics and Computational Biology, Genetics, Agricultural Biotechnology, Human Genome, Pediatric, Clinical Research, Rare Diseases, Pediatric Cancer, Orphan Drug, Hematology, Childhood Leukemia, Precision Medicine, Cancer, Biotechnology, Cancer Genomics, 2.1 Biological and endogenous factors, 4.1 Discovery and preclinical testing of markers and technologies, Adolescent, Adult, Child, Child, Preschool, Female, Genomics, Homeodomain Proteins, Humans, Infant, Infant, Newborn, Leukemia, Erythroblastic, Acute, Male, Mutation, Myeloid-Lymphoid Leukemia Protein, Nuclear Proteins, Nucleophosmin, Prognosis, Tumor Suppressor Protein p53, Young Adult, fms-Like Tyrosine Kinase 3, Medical and Health Sciences, Developmental Biology, Agricultural biotechnology, Bioinformatics and computational biology
Abstract

Acute erythroid leukemia (AEL) is a high-risk leukemia of poorly understood genetic basis, with controversy regarding diagnosis in the spectrum of myelodysplasia and myeloid leukemia. We compared genomic features of 159 childhood and adult AEL cases with non-AEL myeloid disorders and defined five age-related subgroups with distinct transcriptional profiles: adult, TP53 mutated; NPM1 mutated; KMT2A mutated/rearranged; adult, DDX41 mutated; and pediatric, NUP98 rearranged. Genomic features influenced outcome, with NPM1 mutations and HOXB9 overexpression being associated with a favorable prognosis and TP53, FLT3 or RB1 alterations associated with poor survival. Targetable signaling mutations were present in 45% of cases and included recurrent mutations of ALK and NTRK1, the latter of which drives erythroid leukemogenesis sensitive to TRK inhibition. This genomic landscape of AEL provides the framework for accurate diagnosis and risk stratification of this disease, and the rationale for testing targeted therapies in this high-risk leukemia.

Published
2019

30. Diagnosis and management of AML in adults: 2022 recommendations from an international expert panel on behalf of the ELN

Author

Döhner, Hartmut, Wei, Andrew H., Appelbaum, Frederick R., Craddock, Charles, DiNardo, Courtney D., Dombret, Hervé, Ebert, Benjamin L., Fenaux, Pierre, Godley, Lucy A., Hasserjian, Robert P., Larson, Richard A., Levine, Ross L., Miyazaki, Yasushi, Niederwieser, Dietger, Ossenkoppele, Gert, Röllig, Christoph, Sierra, Jorge, Stein, Eytan M., Tallman, Martin S., Tien, Hwei-Fang, Wang, Jianxiang, Wierzbowska, Agnieszka, and Löwenberg, Bob

Published
2022
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31. International Consensus Classification of Myeloid Neoplasms and Acute Leukemias: integrating morphologic, clinical, and genomic data

Author

Arber, Daniel A., Orazi, Attilio, Hasserjian, Robert P., Borowitz, Michael J., Calvo, Katherine R., Kvasnicka, Hans-Michael, Wang, Sa A., Bagg, Adam, Barbui, Tiziano, Branford, Susan, Bueso-Ramos, Carlos E., Cortes, Jorge E., Dal Cin, Paola, DiNardo, Courtney D., Dombret, Hervé, Duncavage, Eric J., Ebert, Benjamin L., Estey, Elihu H., Facchetti, Fabio, Foucar, Kathryn, Gangat, Naseema, Gianelli, Umberto, Godley, Lucy A., Gökbuget, Nicola, Gotlib, Jason, Hellström-Lindberg, Eva, Hobbs, Gabriela S., Hoffman, Ronald, Jabbour, Elias J., Kiladjian, Jean-Jacques, Larson, Richard A., Le Beau, Michelle M., Loh, Mignon L.-C., Löwenberg, Bob, Macintyre, Elizabeth, Malcovati, Luca, Mullighan, Charles G., Niemeyer, Charlotte, Odenike, Olatoyosi M., Ogawa, Seishi, Orfao, Alberto, Papaemmanuil, Elli, Passamonti, Francesco, Porkka, Kimmo, Pui, Ching-Hon, Radich, Jerald P., Reiter, Andreas, Rozman, Maria, Rudelius, Martina, Savona, Michael R., Schiffer, Charles A., Schmitt-Graeff, Annette, Shimamura, Akiko, Sierra, Jorge, Stock, Wendy A., Stone, Richard M., Tallman, Martin S., Thiele, Jürgen, Tien, Hwei-Fang, Tzankov, Alexandar, Vannucchi, Alessandro M., Vyas, Paresh, Wei, Andrew H., Weinberg, Olga K., Wierzbowska, Agnieszka, Cazzola, Mario, Döhner, Hartmut, and Tefferi, Ayalew

Published
2022
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32. Idasanutlin plus cytarabine in relapsed or refractory acute myeloid leukemia: results of the MIRROS trial

Author

Konopleva, Marina Y., Röllig, Christoph, Cavenagh, Jamie, Deeren, Dries, Girshova, Larisa, Krauter, Jürgen, Martinelli, Giovanni, Montesinos, Pau, Schäfer, Jonas A., Ottmann, Oliver, Petrini, Mario, Pigneux, Arnaud, Rambaldi, Alessandro, Recher, Christian, Rodriguez-Veiga, Rebeca, Taussig, David, Vey, Norbert, Yoon, Sung-Soo, Ott, Marion, Muehlbauer, Susanne, Beckermann, Benjamin M., Catalani, Olivier, Genevray, Magali, Mundt, Kirsten, Jamois, Candice, Fenaux, Pierre, and Wei, Andrew H.

Published
2022
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36. Venetoclax combined with low dose cytarabine compared to standard of care intensive chemotherapy for the treatment of favourable risk adult acute myeloid leukaemia (VICTOR): Study protocol for an international, open-label, multicentre, molecularly-guided randomised, phase II trial

Author

Dillon, Richard, Maycock, Shanna, Jackson, Aimee, Fox, Sonia, Freeman, Sylvie, Craddock, Charles, Thomas, Catherine, Homer, Emma, Leahy, Jane, Mamwell, Anna, Potter, Nicola, Russell, Nigel, Wei, Andrew, Ommen, Hans Beier, Hemmaway, Claire, Knapper, Steve, and Billingham, Lucinda

Published
2022
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38. Clonal hematopoiesis, myeloid disorders and BAX-mutated myelopoiesis in patients receiving venetoclax for CLL

Author

Blombery, Piers, Lew, Thomas E., Dengler, Michael A., Thompson, Ella R., Lin, Victor S., Chen, Xiangting, Nguyen, Tamia, Panigrahi, Ashish, Handunnetti, Sasanka M., Carney, Dennis A., Westerman, David A., Tam, Constantine S., Adams, Jerry M., Wei, Andrew H., Huang, David C.S., Seymour, John F., Roberts, Andrew W., and Anderson, Mary Ann

Published
2022
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41. Olutasidenib in post-venetoclax patients with mutant isocitrate dehydrogenase 1 (mIDH1) acute myeloid leukemia (AML).

Author

Cortes, Jorge, Jonas, Brian A., Schiller, Gary, Mims, Alice, Roboz, Gail J., Wei, Andrew H., Montesinos, Pau, Ferrell, P. Brent, Yee, Karen WL., Fenaux, Pierre, Schwarer, Anthony, and Watts, Justin M.

Subjects
ACUTE myeloid leukemia, ISOCITRATE dehydrogenase, VENETOCLAX
Abstract

Olutasidenib, a potent, selective, oral, mutant isocitrate dehydrogenase 1 (mIDH1) inhibitor, is FDA-approved for relapsed/refractory (R/R) acute myeloid leukemia (AML). Here we report efficacy and safety of olutasidenib in 18 patients with mIDH1 AML who were relapsed (10), refractory (6) or had complete remission with incomplete hematologic recovery (CRi; 2) to a venetoclax combination. Of the 16 patients who were R/R, 4 (25%) achieved complete remission (CR), one (6.3%) achieved CR with partial hematologic recovery (CRh), and 7 (43.8%) achieved a composite complete remission (CRc). Median time to CRc was 1.9 months (range 1–2.8). As of data cutoff (18 June 2021), median duration of CRc was not reached (range, 1.2-NR, ongoing at 30.4+ months). Both patients with CRi at study entry achieved a CR. Safety was consistent with the overall profile of olutasidenib. Olutasidenib offers a valuable treatment option for patients with mIDH1 AML previously treated with venetoclax. [ABSTRACT FROM AUTHOR]

Published
2024
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42. Mitochondrial inhibitors circumvent adaptive resistance to venetoclax and cytarabine combination therapy in acute myeloid leukemia

Author

Bosc, Claudie, Saland, Estelle, Bousard, Aurélie, Gadaud, Noémie, Sabatier, Marie, Cognet, Guillaume, Farge, Thomas, Boet, Emeline, Gotanègre, Mathilde, Aroua, Nesrine, Mouchel, Pierre-Luc, Polley, Nathaniel, Larrue, Clément, Kaphan, Eléonore, Picard, Muriel, Sahal, Ambrine, Jarrou, Latifa, Tosolini, Marie, Rambow, Florian, Cabon, Florence, Nicot, Nathalie, Poillet-Perez, Laura, Wang, Yujue, Su, Xiaoyang, Fovez, Quentin, Kluza, Jérôme, Argüello, Rafael José, Mazzotti, Céline, Avet-Loiseau, Hervé, Vergez, François, Tamburini, Jérôme, Fournié, Jean-Jacques, Tiong, Ing S., Wei, Andrew H., Kaoma, Tony, Marine, Jean-Christophe, Récher, Christian, Stuani, Lucille, Joffre, Carine, and Sarry, Jean-Emmanuel

Published
2021
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43. An MRD-stratified pediatric protocol is as deliverable in adolescents and young adults as in children with ALL

Author

Greenwood, Matthew, Trahair, Toby, Sutton, Rosemary, Osborn, Michael, Kwan, John, Mapp, Sally, Howman, Rebecca, Anazodo, Antoinette, Wylie, Brenton, D'Rozario, James, Hertzberg, Mark, Irving, Ian, Yeung, David, Coyle, Luke, Jager, Amanda, Engeler, Dan, Venn, Nicola, Frampton, Chris, Wei, Andrew H., Bradstock, Kenneth, and Dalla-Pozza, Luciano

Published
2021
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45. Olutasidenib in post-venetoclax patients with mutant isocitrate dehydrogenase 1 (m IDH1 ) acute myeloid leukemia (AML)

Author

Cortes, Jorge, primary, Jonas, Brian A., additional, Schiller, Gary, additional, Mims, Alice, additional, Roboz, Gail J., additional, Wei, Andrew H., additional, Montesinos, Pau, additional, Ferrell, P. Brent, additional, Yee, Karen WL., additional, Fenaux, Pierre, additional, Schwarer, Anthony, additional, and Watts, Justin M., additional

Published
2024
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46. Targeting Molecular Measurable Residual Disease and Low-Blast Relapse in AML With Venetoclax and Low-Dose Cytarabine: A Prospective Phase II Study (VALDAC)

Author

Tiong, Ing Soo, primary, Hiwase, Devendra K., additional, Abro, Emad, additional, Bajel, Ashish, additional, Palfreyman, Emma, additional, Beligaswatte, Ashanka, additional, Reynolds, John, additional, Anstee, Natasha, additional, Nguyen, Tamia, additional, Loo, Sun, additional, Chua, Chong Chyn, additional, Ashby, Michael, additional, Wiltshire, Kaitlyn M., additional, Fleming, Shaun, additional, Fong, Chun Y., additional, Teh, Tse-Chieh, additional, Blombery, Piers, additional, Dillon, Richard, additional, Ivey, Adam, additional, and Wei, Andrew H., additional

Published
2024
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47. Enasidenib plus azacitidine versus azacitidine alone in patients with newly diagnosed, mutant-IDH2 acute myeloid leukaemia (AG221-AML-005): a single-arm, phase 1b and randomised, phase 2 trial

Author

DiNardo, Courtney D, Schuh, Andre C, Stein, Eytan M, Montesinos, Pau, Wei, Andrew H, de Botton, Stéphane, Zeidan, Amer M, Fathi, Amir T, Kantarjian, Hagop M, Bennett, John M, Frattini, Mark G, Martin-Regueira, Patricia, Lersch, Frederik, Gong, Jing, Hasan, Maroof, Vyas, Paresh, and Döhner, Hartmut

Published
2021
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48. Single-cell Rapid Capture Hybridization sequencing (scRaCH-seq) to reliably detect isoform usage and coding mutations in targeted genes at a single-cell level

Author

Peng, Hongke, primary, Jabbari, Jafar S., additional, Tian, Luyi, additional, Chua, Chong Chyn, additional, Anstee, Natasha S., additional, Amin, Noorul, additional, Wei, Andrew H., additional, Davidson, Nadia M., additional, Roberts, Andrew W., additional, Huang, David C. S., additional, Ritchie, Matthew E., additional, and Thijssen, Rachel, additional

Published
2024
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