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1,218 results on '"Montalban-Bravo, Guillermo"'

1. A phase II trial of ipilimumab, nivolumab, or ipilimumab and nivolumab with or without azacitidine in relapsed or refractory myelodysplastic neoplasms

Author

Bouligny, Ian M., Montalban-Bravo, Guillermo, Sasaki, Koji, Daver, Naval, Jabbour, Elias, Alvarado, Yesid, DiNardo, Courtney D., Ravandi, Farhad, Borthakur, Gautam, Pemmaraju, Naveen, Kadia, Tapan, Masarova, Lucia, Takahashi, Koichi, Andreeff, Michael, Bazinet, Alexandre, Yang, Hui, Kanagal, Rashmi, Pierce, Sherry, Meyer, Meghan, Huang, Xuelin, and Garcia-Manero, Guillermo

Published
2024
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2. Venetoclax in combination with hypomethylating agents in chronic myelomonocytic leukemia: a propensity score matched multicenter cohort study

Author

Tremblay, Douglas, Csizmar, Clifford, DiNardo, Courtney D., Ball, Somedeb, Rippel, Noa, Hammond, Danielle, Kadia, Tapan M., Ravandi, Farhad, Chien, Kelly, Van Hyfte, Grace, Mazumdar, Madhu, Saliba, Antoine, Mangaonkar, Abhishek, Lasho, Terra, Al-Kali, Aref, Kremyanskaya, Marina, Feld, Jonathan, Silverman, Lewis R., Komrokji, Rami, Mascarenhas, John, Padron, Eric, Garcia-Manero, Guillermo, Sallman, David A., Patnaik, Mrinal M., and Montalban-Bravo, Guillermo

Published
2024
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3. Outcomes of patients with acute myeloid leukemia and bone marrow fibrosis

Author

Urrutia, Samuel, Kantarjian, Hagop M., Ravandi-Kashani, Farhad, Bueso-Ramos, Carlos, Kanagal-Shamanna, Rashmi, Jabbour, Elias, Montalban-Bravo, Guillermo, Short, Nicholas J., Daver, Naval, Borthakur, Gautam, Dinardo, Courtney D., Kadia, Tapan M., Masarova, Lucia, Bose, Prithviraj, Pemmaraju, Naveen, Garcia-Manero, Guillermo, and Sasaki, Koji

Published
2024
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5. Liver elastography for risk-assessment of liver toxicity and risk factors for Sinusoidal obstruction syndrome in patients with acute lymphoblastic leukemia receiving inotuzumab ozogamicin

Author

Senapati, Jayastu, Jabbour, Elias, Short, Nicholas J., Jain, Nitin, Haddad, Fadi, Bathala, Tharakeswara, Kovalenko, Iuliia, Bidikian, Aram, Ravandi, Farhad, Khouri, Issa, Kadia, Tapan M., Garris, Rebecca, Montalban Bravo, Guillermo, Chien, Kelly, Shpall, Elizabeth, Kebriaei, Partow, and Kantarjian, Hagop M.

Published
2024
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6. Hematopoietic stem cells with granulo-monocytic differentiation state overcome venetoclax sensitivity in patients with myelodysplastic syndromes

Author

Rodriguez-Sevilla, Juan Jose, Ganan-Gomez, Irene, Ma, Feiyang, Chien, Kelly, Del Rey, Monica, Loghavi, Sanam, Montalban-Bravo, Guillermo, Adema, Vera, Wildeman, Bethany, Kanagal-Shamanna, Rashmi, Bazinet, Alexandre, Chifotides, Helen T., Thongon, Natthakan, Calvo, Xavier, Hernández-Rivas, Jesús María, Díez-Campelo, Maria, Garcia-Manero, Guillermo, and Colla, Simona

Published
2024
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7. Influence of co-mutational patterns in disease phenotype and clinical outcomes of chronic myelomonocytic leukemia

Author

Montalban-Bravo, Guillermo, Rodriguez-Sevilla, Juan Jose, Swanson, David Michael, Kanagal-Shamanna, Rashmi, Hammond, Danielle, Chien, Kelly, Sasaki, Koji, Jabbour, Elias, DiNardo, Courtney, Takahashi, Koichi, Short, Nicholas, Issa, Ghayas C., Pemmaraju, Naveen, Kadia, Tapan, Ravandi, Farhad, Daver, Naval, Borthakur, Gautam, Loghavi, Sanam, Pierce, Sherry, Bueso-Ramos, Carlos, Kantarjian, Hagop, and Garcia-Manero, Guillermo

Published
2024
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8. Targeting the EIF2AK1 signaling pathway rescues red blood cell production in SF3B1-mutant myelodysplastic syndromes with ringed sideroblasts

Author

Adema, Vera, Ma, Feiyang, Kanagal-Shamanna, Rashmi, Thongon, Natthakan, Montalban-Bravo, Guillermo, Yang, Hui, Peslak, Scott A, Wang, Feng, Acha, Pamela, Sole, Francesc, Lockyer, Pamela, Cassari, Margherita, Maciejewski, Jaroslaw P, Visconte, Valeria, Ganan-Gomez, Irene, Song, Yuanbin, Bueso-Ramos, Carlos, Pellegrini, Matteo, Tan, Tuyet M, Bejar, Rafael, Carew, Jennifer S, Halene, Stephanie, Santini, Valeria, Al-Atrash, Gheath, Clise-Dwyer, Karen, Garcia-Manero, Guillermo, Blobel, Gerd A, and Colla, Simona

Subjects
Biomedical and Clinical Sciences, Cardiovascular Medicine and Haematology, Rare Diseases, Hematology, Stem Cell Research - Nonembryonic - Human, Stem Cell Research, Blood, Humans, Aged, RNA Splicing Factors, Phosphoproteins, Myelodysplastic Syndromes, Erythroid Precursor Cells, Signal Transduction, eIF-2 Kinase
Abstract

SF3B1 mutations, which occur in 20% of patients with myelodysplastic syndromes (MDS), are the hallmarks of a specific MDS subtype, MDS with ringed sideroblasts (MDS-RS), which is characterized by the accumulation of erythroid precursors in the bone marrow and primarily affects the elderly population. Here, using single-cell technologies and functional validation studies of primary SF3B1-mutant MDS-RS samples, we show that SF3B1 mutations lead to the activation of the EIF2AK1 pathway in response to heme deficiency and that targeting this pathway rescues aberrant erythroid differentiation and enables the red blood cell maturation of MDS-RS erythroblasts. These data support the development of EIF2AK1 inhibitors to overcome transfusion dependency in patients with SF3B1-mutant MDS-RS with impaired red blood cell production.SignificanceMDS-RS are characterized by significant anemia. Patients with MDS-RS die from a shortage of red blood cells and the side effects of iron overload due to their constant need for transfusions. Our study has implications for the development of therapies to achieve long-lasting hematologic responses. This article is highlighted in the In This Issue feature, p. 476.

Published
2022

11. Targeting MCL1-driven anti-apoptotic pathways overcomes blast progression after hypomethylating agent failure in chronic myelomonocytic leukemia

Author

Montalban-Bravo, Guillermo, Thongon, Natthakan, Rodriguez-Sevilla, Juan Jose, Ma, Feiyang, Ganan-Gomez, Irene, Yang, Hui, Kim, Yi June, Adema, Vera, Wildeman, Bethany, Tanaka, Tomoyuki, Darbaniyan, Faezeh, Al-Atrash, Gheath, Dwyer, Karen, Loghavi, Sanam, Kanagal-Shamanna, Rashmi, Song, Xingzhi, Zhang, Jianhua, Takahashi, Koichi, Kantarjian, Hagop, Garcia-Manero, Guillermo, and Colla, Simona

Published
2024
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12. Therapy-related chronic myelomonocytic leukemia does not have the high-risk features of a therapy-related neoplasm

Author

Bataller, Alex, Gener-Ricos, Georgina, Almanza-Huante, Emmanuel, Chien, Kelly S., Urrutia, Samuel, Bazinet, Alexandre, Rodriguez-Sevilla, Juan Jose, Hammond, Danielle, Sasaki, Koji, Takahashi, Koichi, DiNardo, Courtney D., Ravandi, Farhad, Borthakur, Gautam, Kadia, Tapan M., Kanagal-Shamanna, Rashmi, Kantarjian, Hagop M., Garcia-Manero, Guillermo, and Montalban-Bravo, Guillermo

Published
2024
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13. Oral decitabine and cedazuridine plus venetoclax for older or unfit patients with acute myeloid leukaemia: a phase 2 study

Author

Bazinet, Alexandre, Garcia-Manero, Guillermo, Short, Nicholas, Alvarado, Yesid, Bataller, Alex, Abuasab, Tareq, Islam, Rabiul, Montalbano, Kathryn, Issa, Ghayas, Maiti, Abhishek, Yilmaz, Musa, Jain, Nitin, Masarova, Lucia, Kornblau, Steven, Jabbour, Elias, Montalban-Bravo, Guillermo, Rausch, Caitlin R, Pierce, Sherry, DiNardo, Courtney D, Kadia, Tapan, Daver, Naval, Konopleva, Marina, Huang, Xuelin, Kantarjian, Hagop, and Ravandi, Farhad

Published
2024
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14. Stem cell architecture drives myelodysplastic syndrome progression and predicts response to venetoclax-based therapy

Author

Ganan-Gomez, Irene, Yang, Hui, Ma, Feiyang, Montalban-Bravo, Guillermo, Thongon, Natthakan, Marchica, Valentina, Richard-Carpentier, Guillaume, Chien, Kelly, Manyam, Ganiraju, Wang, Feng, Alfonso, Ana, Chen, Shuaitong, Class, Caleb, Kanagal-Shamanna, Rashmi, Ingram, Justin P, Ogoti, Yamini, Rose, Ashley, Loghavi, Sanam, Lockyer, Pamela, Cambo, Benedetta, Muftuoglu, Muharrem, Schneider, Sarah, Adema, Vera, McLellan, Michael, Garza, John, Marchesini, Matteo, Giuliani, Nicola, Pellegrini, Matteo, Wang, Jing, Walker, Jason, Li, Ziyi, Takahashi, Koichi, Leverson, Joel D, Bueso-Ramos, Carlos, Andreeff, Michael, Clise-Dwyer, Karen, Garcia-Manero, Guillermo, and Colla, Simona

Subjects
Stem Cell Research, Rare Diseases, Stem Cell Research - Nonembryonic - Non-Human, Stem Cell Research - Nonembryonic - Human, Hematology, Clinical Research, Orphan Drug, Regenerative Medicine, Cancer, 2.1 Biological and endogenous factors, Aetiology, Blood, Bridged Bicyclo Compounds, Heterocyclic, Hematopoietic Stem Cells, Humans, Myelodysplastic Syndromes, Proto-Oncogene Proteins c-bcl-2, Sulfonamides, Medical and Health Sciences, Immunology
Abstract

Myelodysplastic syndromes (MDS) are heterogeneous neoplastic disorders of hematopoietic stem cells (HSCs). The current standard of care for patients with MDS is hypomethylating agent (HMA)-based therapy; however, almost 50% of MDS patients fail HMA therapy and progress to acute myeloid leukemia, facing a dismal prognosis due to lack of approved second-line treatment options. As cancer stem cells are the seeds of disease progression, we investigated the biological properties of the MDS HSCs that drive disease evolution, seeking to uncover vulnerabilities that could be therapeutically exploited. Through integrative molecular profiling of HSCs and progenitor cells in large patient cohorts, we found that MDS HSCs in two distinct differentiation states are maintained throughout the clinical course of the disease, and expand at progression, depending on recurrent activation of the anti-apoptotic regulator BCL-2 or nuclear factor-kappa B-mediated survival pathways. Pharmacologically inhibiting these pathways depleted MDS HSCs and reduced tumor burden in experimental systems. Further, patients with MDS who progressed after failure to frontline HMA therapy and whose HSCs upregulated BCL-2 achieved improved clinical responses to venetoclax-based therapy in the clinical setting. Overall, our study uncovers that HSC architectures in MDS are potential predictive biomarkers to guide second-line treatments after HMA failure. These findings warrant further investigation of HSC-specific survival pathways to identify new therapeutic targets of clinical potential in MDS.

Published
2022

15. Improving patient understanding and outcomes in myelodysplastic syndromes - An animated patient guide to MDS with visual formats of learning.

Author

Sallman, David A, Bejar, Rafael, Montalban-Bravo, Guillermo, Kurtin, Sandra E, List, Alan F, Garcia-Manero, Guillermo, Nimer, Stephen D, O'Connell, Casey L, Schaar, Dale, Butchko, Janice, Iraca, Tracey, and Searle, Stephanie

Subjects
Health literacy, Health outcomes, Myelodysplastic syndromes, Patient education, Visual formats of learning, Cancer, Clinical Research, Behavioral and Social Science, Hematology, Rare Diseases, Management of diseases and conditions, 7.1 Individual care needs, Quality Education
Abstract

ObjectivesPatient education resources that address barriers to health literacy to improve understanding and outcomes in myelodysplastic syndromes (MDS) are limited. The aim of this study was to evaluate the impact and outcomes benefits of An Animated Patient's Guide to Myelodysplastic Syndromes (MDS) cancer educational modules (which includes the 'You and MDS' website and YouTube hosted resources) related to MDS education, awareness, understanding and health outcomes.MethodsThis was a retrospective study of learner feedback, metrics, and utilization data from July 2018 to August 2021. We evaluated audience reach (number of visit sessions, unique visitors, page views) and calculated top views by media type (animation, expert video, patient video, and slide show) and top retention videos from the modules. We also assessed the educational impact and utilization through learner feedback surveys.ResultsDuring the study period, 'You and MDS' had 233,743 views worldwide of which 104,214 were unique visitors and 78,161 (or 76% unique visitors) were from the United States. Of these, 61% were patients; 29% family members or caregivers; 5% were healthcare providers and 5% represented other groups. Most popular topics viewed among the animations were "Understanding Myelodysplastic Syndromes (MDS)" (40,219 views), "Managing and Treating MDS" (19,240 views), "Understanding Erythropoiesis" (17,564 views.) The most popular expert videos viewed were "What is iron overload, and how it is treated?" (20,310 views), "How serious a cancer is MDS? What is the prognosis for MDS?" (8,327 views), "What is MDS?" (3,157 views). Of participants who completed the online feedback survey, ≥ 95% reported improved knowledge gains and commitments to change.ConclusionsMDS patients using 'You and MDS - An Animated Patient's Guide to MDS' and its visual formats of learning represented a wide U.S. and global learner audience. This MDS educational resource had a significant impact on improved understanding among patients, families, and caregivers. Continued efforts should be made to provide patient-effective resources that address health literacy, improve patient understanding, and address educational needs that respond to the concerns of patients to achieve better quality of life and improved health outcomes in MDS.

Published
2022

16. Oral decitabine plus cedazuridine and venetoclax in patients with higher-risk myelodysplastic syndromes or chronic myelomonocytic leukaemia: a single-centre, phase 1/2 study

Author

Bataller, Alex, Montalban-Bravo, Guillermo, Bazinet, Alexandre, Alvarado, Yesid, Chien, Kelly, Venugopal, Sangeetha, Ishizawa, Jo, Hammond, Danielle, Swaminathan, Mahesh, Sasaki, Koji, Issa, Ghayas C, Short, Nicholas J, Masarova, Lucia, Daver, Naval G, Kadia, Tapan M, Colla, Simona, Qiao, Wei, Huang, Xuelin, Kanagal-Shamanna, Rashmi, Hendrickson, Stephany, Ravandi, Farhad, Jabbour, Elias, Kantarjian, Hagop, and Garcia-Manero, Guillermo

Published
2024
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17. A phase 1/2 study of mini-hyper-CVD plus venetoclax in patients with relapsed/refractory acute lymphoblastic leukemia

Author

Short, Nicholas J., Jabbour, Elias, Jain, Nitin, Senapati, Jayastu, Nasr, Lewis, Haddad, Fadi G., Li, Zhenhua, Hsiao, Yu-Chih, Yang, Jun J., Pemmaraju, Naveen, Ohanian, Maro, Wierda, William G., Montalban-Bravo, Guillermo, Borthakur, Gautam, Han, Lina, Xiao, Lianchun, Huang, Xuelin, Abramova, Regina, Zhao, Min, Garris, Rebecca, Konopleva, Marina, Ravandi, Farhad, and Kantarjian, Hagop

Published
2024
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18. Prognostic risk signature in patients with acute myeloid leukemia treated with hypomethylating agents and venetoclax

Author

Bataller, Alex, Bazinet, Alexandre, DiNardo, Courtney D., Maiti, Abhishek, Borthakur, Gautam, Daver, Naval G., Short, Nicholas J., Jabbour, Elias J., Issa, Ghayas C., Pemmaraju, Naveen, Yilmaz, Musa, Montalban-Bravo, Guillermo, Takahashi, Koichi, Loghavi, Sanam, Garcia-Manero, Guillermo, Ravandi, Farhad, Kantarjian, Hagop M., and Kadia, Tapan M.

Published
2024
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19. Response patterns and impact of MRD in patients with IDH1/2-mutated AML treated with venetoclax and hypomethylating agents

Author

Hammond, Danielle, Loghavi, Sanam, Wang, Sa A., Konopleva, Marina Y., Kadia, Tapan M., Daver, Naval G., Ohanian, Maro, Issa, Ghayas C., Alvarado, Yesid, Short, Nicholas J., Sasaki, Koji, Pemmaraju, Naveen, Montalban-Bravo, Guillermo, Lachowiez, Curtis A., Maiti, Abhishek, Garcia-Manero, Guillermo, Jabbour, Elias J., Borthakur, Gautam, Ravandi, Farhad, Takahashi, Koichi, Pierce, Sherry R., Kantarjian, Hagop M., and DiNardo, Courtney D.

Published
2023
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20. Hematopoiesis under telomere attrition at the single-cell resolution

Author

Thongon, Natthakan, Ma, Feiyang, Santoni, Andrea, Marchesini, Matteo, Fiorini, Elena, Rose, Ashley, Adema, Vera, Ganan-Gomez, Irene, Groarke, Emma M, Gutierrez-Rodrigues, Fernanda, Chen, Shuaitong, Lockyer, Pamela, Schneider, Sarah, Bueso-Ramos, Carlos, Montalban-Bravo, Guillermo, Class, Caleb A, Soltysiak, Kelly A, Pellegrini, Matteo, Sahin, Ergun, Bertuch, Alison A, DiNardo, Courtney D, Garcia-Manero, Guillermo, Young, Neal S, Dwyer, Karen, and Colla, Simona

Subjects
Genetics, Stem Cell Research - Nonembryonic - Non-Human, Stem Cell Research, Stem Cell Research - Nonembryonic - Human, Regenerative Medicine, Underpinning research, 1.1 Normal biological development and functioning, Inflammatory and immune system, Animals, Bone Marrow Failure Disorders, Cell Self Renewal, Cellular Reprogramming, Hematopoiesis, Hematopoietic Stem Cells, Humans, Interferons, Megakaryocytes, Mice, Nuclear Proteins, Oligodeoxyribonucleotides, Phosphoproteins, Signal Transduction, Single-Cell Analysis, Telomere, Telomere Shortening
Abstract

The molecular mechanisms that drive hematopoietic stem cell functional decline under conditions of telomere shortening are not completely understood. In light of recent advances in single-cell technologies, we sought to redefine the transcriptional and epigenetic landscape of mouse and human hematopoietic stem cells under telomere attrition, as induced by pathogenic germline variants in telomerase complex genes. Here, we show that telomere attrition maintains hematopoietic stem cells under persistent metabolic activation and differentiation towards the megakaryocytic lineage through the cell-intrinsic upregulation of the innate immune signaling response, which directly compromises hematopoietic stem cells' self-renewal capabilities and eventually leads to their exhaustion. Mechanistically, we demonstrate that targeting members of the Ifi20x/IFI16 family of cytosolic DNA sensors using the oligodeoxynucleotide A151, which comprises four repeats of the TTAGGG motif of the telomeric DNA, overcomes interferon signaling activation in telomere-dysfunctional hematopoietic stem cells and these cells' skewed differentiation towards the megakaryocytic lineage. This study challenges the historical hypothesis that telomere attrition limits the proliferative potential of hematopoietic stem cells by inducing apoptosis, autophagy, or senescence, and suggests that targeting IFI16 signaling axis might prevent hematopoietic stem cell functional decline in conditions affecting telomere maintenance.

Published
2021

21. Targeted therapy with the mutant IDH2 inhibitor enasidenib for high-risk IDH2-mutant myelodysplastic syndrome

Author

DiNardo, Courtney D., Venugopal, Sangeetha, Lachowiez, Curtis, Takahashi, Koichi, Loghavi, Sanam, Montalban-Bravo, Guillermo, Wang, Xuemei, Carraway, Hetty, Sekeres, Mikkael, Sukkur, Ameenah, Hammond, Danielle, Chien, Kelly, Maiti, Abhishek, Masarova, Lucia, Sasaki, Koji, Alvarado, Yesid, Kadia, Tapan, Short, Nicholas J., Daver, Naval, Borthakur, Gautam, Ravandi, Farhad, Kantarjian, Hagop M., Patel, Bhumika, Dezern, Amy, Roboz, Gail, and Garcia-Manero, Guillermo

Published
2023
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22. Mini-hyper-CVD plus inotuzumab ozogamicin, with or without blinatumomab, in the subgroup of older patients with newly diagnosed Philadelphia chromosome-negative B-cell acute lymphocytic leukaemia: long-term results of an open-label phase 2 trial

Author

Jabbour, Elias, Short, Nicholas J, Senapati, Jayastu, Jain, Nitin, Huang, Xuelin, Daver, Naval, DiNardo, Courtney D, Pemmaraju, Naveen, Wierda, William, Garcia-Manero, Guillermo, Montalban Bravo, Guillermo, Sasaki, Koji, Kadia, Tapan M, Khoury, Joseph, Wang, Sa A, Haddad, Fadi G, Jacob, Jovitta, Garris, Rebecca, Ravandi, Farhad, and Kantarjian, Hagop M

Published
2023
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23. Ponatinib and blinatumomab for Philadelphia chromosome-positive acute lymphoblastic leukaemia: a US, single-centre, single-arm, phase 2 trial

Author

Jabbour, Elias, Short, Nicholas J, Jain, Nitin, Huang, Xuelin, Montalban-Bravo, Guillermo, Banerjee, Pinaki, Rezvani, Katayoun, Jiang, Xianli, Kim, Kun Hee, Kanagal-Shamanna, Rashmi, Khoury, Joseph D, Patel, Keyur, Kadia, Tapan M, Daver, Naval, Chien, Kelly, Alvarado, Yesid, Garcia-Manero, Guillermo, Issa, Ghayas C, Haddad, Fadi G, Kwari, Monica, Thankachan, Jennifer, Delumpa, Ricardo, Macaron, Walid, Garris, Rebecca, Konopleva, Marina, Ravandi, Farhad, and Kantarjian, Hagop

Published
2023
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24. High-resolution structural variant profiling of myelodysplastic syndromes by optical genome mapping uncovers cryptic aberrations of prognostic and therapeutic significance

Author

Yang, Hui, Garcia-Manero, Guillermo, Sasaki, Koji, Montalban-Bravo, Guillermo, Tang, Zhenya, Wei, Yue, Kadia, Tapan, Chien, Kelly, Rush, Diana, Nguyen, Ha, Kalia, Awdesh, Nimmakayalu, Manjunath, Bueso-Ramos, Carlos, Kantarjian, Hagop, Medeiros, L. Jeffrey, Luthra, Rajyalakshmi, and Kanagal-Shamanna, Rashmi

Published
2022
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25. Immunohistochemical loss of enhancer of Zeste Homolog 2 (EZH2) protein expression correlates with EZH2 alterations and portends a worse outcome in myelodysplastic syndromes

Author

Sakhdari, Ali, Class, Caleb, Montalban-Bravo, Guillermo, Sasaki, Koji, Bueso-Ramos, Carlos E., Patel, Keyur P., Routbort, Mark J., Loghavi, Sanam, Ok, Chi Young, Quesada, Andres, Khoury, Joseph D., Konoplev, Sergej N., Kantarjian, Hagop P., Garcia-Manero, Guillermo, Medeiros, L. Jeffrey, and Kanagal-Shamanna, Rashmi

Published
2022
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28. Azacitidine plus venetoclax in patients with high-risk myelodysplastic syndromes or chronic myelomonocytic leukaemia: phase 1 results of a single-centre, dose-escalation, dose-expansion, phase 1–2 study

Author

Bazinet, Alexandre, Darbaniyan, Faezeh, Jabbour, Elias, Montalban-Bravo, Guillermo, Ohanian, Maro, Chien, Kelly, Kadia, Tapan, Takahashi, Koichi, Masarova, Lucia, Short, Nicholas, Alvarado, Yesid, Yilmaz, Musa, Ravandi, Farhad, Andreeff, Michael, Kanagal-Shamanna, Rashmi, Ganan-Gomez, Irene, Colla, Simona, Qiao, Wei, Huang, Xuelin, McCue, Deborah, Mirabella, Bailey, Kantarjian, Hagop, and Garcia-Manero, Guillermo

Published
2022
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30. Marrow ring sideroblasts are highly predictive for TP53 mutation in MDS with excess blasts

Author

Swoboda, David M., Kanagal-Shamanna, Rashmi, Brunner, Andrew M., Cluzeau, Thomas, Chan, Onyee, Al Ali, Najla, Montalban-Bravo, Guillermo, Gesiotto, Quinto J., Gavralidis, Alexander, Hunter, Anthony M., Lee, Jung-Hoon, Kuykendall, Andrew T., Talati, Chetasi, Sweet, Kendra L., Lancet, Jeffrey E., Padron, Eric, Hussaini, Mohammad, Song, Jinming, Garcia-Manero, Guillermo, Komrokji, Rami S., and Sallman, David A.

Published
2022
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31. Cladribine-Based Therapy for Acute Myeloid Leukemia in Child, Adolescent, and Early Young Adult Patients: The MD Anderson Cancer Center Experience.

Author

McCall, David, Alqahtani, Shaikha, Budak, Moriah, Sheikh, Irtiza, Fan, Aaron E., Ramakrishnan, Ramya, Nunez, Cesar, Roth, Michael, Garcia, Miriam B., Gibson, Amber, Daver, Naval, Garces, Sofia, Short, Nicholas J., Issa, Ghayas C., Ravandi, Farhad, DiNardo, Courtney D., Montalban Bravo, Guillermo, Garcia-Manero, Guillermo, Cuglievan, Branko, and Kadia, Tapan

Subjects
CANCER treatment, FEBRILE neutropenia, RESEARCH funding, ADENOSINES, SALVAGE therapy, TREATMENT effectiveness, RETROSPECTIVE studies, DESCRIPTIVE statistics, CANCER chemotherapy, MEDICAL records, ACQUISITION of data, SEPSIS, CONFIDENCE intervals, SPECIALTY hospitals, DRUG tolerance, OVERALL survival, DISEASE progression, DISEASE risk factors
Abstract

Simple Summary: Cladribine is a chemotherapy used in combination with other chemotherapy drugs in adult patients with acute myeloid leukemia (AML) who relapse or are nonresponsive to treatment. It appeared to work well and is now used for newly diagnosed adult patients. While it is commonly used in adults, it has not been used routinely in children. We looked at medical records of pediatric and adolescent patients treated for AML at MD Anderson to analyze its safety and how well it works against AML. It was found to be safe and well tolerated in these patients and also showed improved survival outcomes. About half of the patients responded to cladribine either by itself or with other chemotherapy agents. This review also highlighted its safety in combination with other agents. We found these combinations to be well tolerated and could provide additional options for pediatric providers who are treating relapsed AML. Background: Cladribine-based combination chemotherapy has demonstrated promising efficacy in patients with relapsed/refractory adult acute myeloid leukemia (AML), prompting its increased utilization in the frontline; in pediatrics, it has been typically reserved for relapsed or refractory cases. While fludarabine has been used more commonly as a purine analog in intensive regimens, cladribine may be an important alternative. Methods: We performed a retrospective study at MD Anderson Cancer Center from January 2015 to July 2023, which included patients aged 1–21 years with refractory or relapsed AML who received cladribine outside of a transplant conditioning. Results: A total of 30 patients were included, with a median age of 20 years (range, 2–21), and 55% being male. Similar to adults, cladribine exhibited good tolerability in pediatric and adolescent patients, with the most common adverse events being febrile neutropenia and myelosuppression. The most common grade 3 or 4 adverse events included febrile neutropenia (55%) and sepsis (26%), and there were no treatment discontinuations due to adverse events. Among patients with a median number of 2 (0–7) prior treatments, the overall response rate (CR/CRi) was 45%, and median event-free and overall survival were 6 and 12 months, respectively. Disease progression resulted in 4 deaths within 30 days of treatment. Conclusions: Cladribine was tolerated in pediatrics. No new safety signals were seen with cladribine regimens in this cohort. Response assessment is limited due to the heavily pretreated cohort. Further prospective studies are warranted on the safety and efficacy of cladribine and establish its role in pediatric, adolescent, and early young adult patients with AML. [ABSTRACT FROM AUTHOR]

Published
2024
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32. NPM1‐mutated myeloid neoplasms are a unique entity not defined by bone marrow blast percentage.

Author

Gener‐Ricos, Georgina, Bataller, Alex, Rodriguez‐Sevilla, Juan Jose, Chien, Kelly S., Quesada, Andres E., Almanza‐Huante, Emmanuel, Hammond, Danielle, Sasaki, Koji, DiNardo, Courtney, Kadia, Tapan, Daver, Naval, Borthakur, Gautam, Issa, Ghayas C., Short, Nicholas J., Kanagal‐Shamanna, Rashmi, Kantarjian, Hagop M., Garcia‐Manero, Guillermo, and Montalban‐Bravo, Guillermo

Subjects
STEM cell transplantation, BONE marrow, MYELODYSPLASTIC syndromes, OVERALL survival, CLINICAL pathology
Abstract

Introduction: NPM1‐mutated (NPM1mut) myeloid neoplasms (MNs) with <20% bone marrow (BM) blasts (NPM1mut MNs<20) are uncommon, and their classification remains inconsistent. Methods: The clinicopathologic features of 54 patients with NPM1mut MNs <20 were evaluated and compared with wild‐type NPM1 MNs <20 and NPM1mut MNs≥20, respectively. Results: NPM1mut MNs had similar features regardless of blast percentage, except for higher IDH2 (29% vs 7%, p =.023) and FLT3 (70% vs 11%, p <.001) frequency in patients with ≥20% BM blasts. Thirty‐three (61%) patients with NPM1mut MNs <20 received low‐intensity chemotherapy (LIC) and 12 (22%) received intensive chemotherapy (IC). Higher complete remission rates (75% vs 27%, p =.006) and median overall survival (mOS) (not reached vs 30.4 months, p =.06) were observed with IC compared to LIC. Young patients (age <60 years) did not reach mOS either when treated with LIC or IC. Stem cell transplant was associated with increased survival only in patients treated with LIC (HR, 0.24; p =.025). No differences in mOS were observed by BM blast strata (32.2 months, not reached and 46.9 months for <10%, 10%–19%, and ≥20% blasts, p =.700) regardless of treatment modality (LIC: p =.900; IC: p =.360). Twenty‐three patients (43%) with NPM1mut MNs <20 had marrow blast progression to ≥20%. Conclusions: Overall, NPM1mut MNs define a unique entity independent of BM blast percentage. NPM1 mutations define a unique leukemia entity regardless of bone marrow blast percentage. Patients with NPM1‐mutated myeloid neoplasms benefit from intensive chemotherapy approaches even in the setting of low bone marrow blast percentage. [ABSTRACT FROM AUTHOR]

Published
2024
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33. Detection of PNH Clones can Aid in the Distinction of Aplastic Anemia vs Inherited BM Failure Syndromes: A Single Center Experience and Review of the Literature.

Author

Nasnas, Patrice, Ling, Jianhua, Gerstein, Yoheved, Wang, Sa A., Loghavi, Sanam, Hammond, Danielle, Montalban-Bravo, Guillermo, Senapati, Jayastu, Pemmaraju, Naveen, Corredor, Jessie, Pierce, Sherry, Roth, Michael, Ravandi, Farhad, Cuglievan, Branko, Kadia, Tapan, and DiNardo, Courtney D.

Published
2024
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34. Impact of frontline treatment approach on outcomes in patients with secondary AML with prior hypomethylating agent exposure

Author

Short, Nicholas J., Venugopal, Sangeetha, Qiao, Wei, Kadia, Tapan M., Ravandi, Farhad, Macaron, Walid, Dinardo, Courtney D., Daver, Naval, Konopleva, Marina, Borthakur, Gautam, Shpall, Elizabeth J., Popat, Uday, Champlin, Richard E., Mehta, Rohtesh, Al-Atrash, Gheath, Oran, Betul, Jabbour, Elias, Garcia-Manero, Guillermo, Issa, Ghayas C., Montalban-Bravo, Guillermo, Yilmaz, Musa, Maiti, Abhishek, and Kantarjian, Hagop

Published
2022
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35. The ABNL-MARRO 001 study: a phase 1–2 study of randomly allocated active myeloid target compound combinations in MDS/MPN overlap syndromes

Author

Moyo, Tamara K., Mendler, Jason H., Itzykson, Raphael, Kishtagari, Ashwin, Solary, Eric, Seegmiller, Adam C., Gerds, Aaron T., Ayers, Gregory D., Dezern, Amy E., Nazha, Aziz, Valent, Peter, van de Loosdrecht, Arjan A., Onida, Francesco, Pleyer, Lisa, Cirici, Blanca Xicoy, Tibes, Raoul, Geissler, Klaus, Komrokji, Rami S., Zhang, Jing, Germing, Ulrich, Steensma, David P., Wiseman, Daniel H., Pfeilstöecker, Michael, Elena, Chiara, Cross, Nicholas C. P., Kiladjian, Jean-Jacques, Luebbert, Michael, Mesa, Ruben A., Montalban-Bravo, Guillermo, Sanz, Guillermo F., Platzbecker, Uwe, Patnaik, Mrinal M., Padron, Eric, Santini, Valeria, Fenaux, Pierre, and Savona, Michael R.

Published
2022
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36. Outcomes of patients with bone marrow fibrosis in de novo and secondary acute myeloid leukemia.

Author

Urrutia, Samuel, primary, Kantarjian, Hagop M., additional, Ravandi-Kashani, Farhad, additional, Jabbour, Elias, additional, Kanagal-Shamanna, Rashmi, additional, Loghavi, Sanam, additional, Patel, Keyur P., additional, Montalban-Bravo, Guillermo, additional, Short, Nicholas James, additional, Daver, Naval Guastad, additional, Borthakur, Gautam, additional, Dinardo, Courtney Denton, additional, Kadia, Tapan M., additional, Masarova, Lucia, additional, Bose, Prithviraj, additional, Pemmaraju, Naveen, additional, Garcia-Manero, Guillermo, additional, and Sasaki, Koji, additional

Published
2024
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37. Supplemental Table 1 from Cytogenetic and Molecular Associations with Outcomes in Higher-Risk Myelodysplastic Syndromes Treated with Hypomethylating Agents plus Venetoclax

Author

Bazinet, Alexandre, primary, Desikan, Sai Prasad, primary, Li, Ziyi, primary, Rodriguez-Sevilla, Juan Jose, primary, Venugopal, Sangeetha, primary, Urrutia, Samuel, primary, Montalban-Bravo, Guillermo, primary, Sasaki, Koji, primary, Chien, Kelly S., primary, Hammond, Danielle, primary, Kanagal-Shamanna, Rashmi, primary, Ganan-Gomez, Irene, primary, Kadia, Tapan M., primary, Borthakur, Gautam, primary, DiNardo, Courtney D., primary, Daver, Naval G., primary, Jabbour, Elias J., primary, Ravandi, Farhad, primary, Kantarjian, Hagop, primary, and Garcia-Manero, Guillermo, primary

Published
2024
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38. Supplemental Table 4 from Cytogenetic and Molecular Associations with Outcomes in Higher-Risk Myelodysplastic Syndromes Treated with Hypomethylating Agents plus Venetoclax

Author

Bazinet, Alexandre, primary, Desikan, Sai Prasad, primary, Li, Ziyi, primary, Rodriguez-Sevilla, Juan Jose, primary, Venugopal, Sangeetha, primary, Urrutia, Samuel, primary, Montalban-Bravo, Guillermo, primary, Sasaki, Koji, primary, Chien, Kelly S., primary, Hammond, Danielle, primary, Kanagal-Shamanna, Rashmi, primary, Ganan-Gomez, Irene, primary, Kadia, Tapan M., primary, Borthakur, Gautam, primary, DiNardo, Courtney D., primary, Daver, Naval G., primary, Jabbour, Elias J., primary, Ravandi, Farhad, primary, Kantarjian, Hagop, primary, and Garcia-Manero, Guillermo, primary

Published
2024
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39. Supplemental Table 2 from Cytogenetic and Molecular Associations with Outcomes in Higher-Risk Myelodysplastic Syndromes Treated with Hypomethylating Agents plus Venetoclax

Author

Bazinet, Alexandre, primary, Desikan, Sai Prasad, primary, Li, Ziyi, primary, Rodriguez-Sevilla, Juan Jose, primary, Venugopal, Sangeetha, primary, Urrutia, Samuel, primary, Montalban-Bravo, Guillermo, primary, Sasaki, Koji, primary, Chien, Kelly S., primary, Hammond, Danielle, primary, Kanagal-Shamanna, Rashmi, primary, Ganan-Gomez, Irene, primary, Kadia, Tapan M., primary, Borthakur, Gautam, primary, DiNardo, Courtney D., primary, Daver, Naval G., primary, Jabbour, Elias J., primary, Ravandi, Farhad, primary, Kantarjian, Hagop, primary, and Garcia-Manero, Guillermo, primary

Published
2024
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40. Supplemental Figure 3 from Cytogenetic and Molecular Associations with Outcomes in Higher-Risk Myelodysplastic Syndromes Treated with Hypomethylating Agents plus Venetoclax

Author

Bazinet, Alexandre, primary, Desikan, Sai Prasad, primary, Li, Ziyi, primary, Rodriguez-Sevilla, Juan Jose, primary, Venugopal, Sangeetha, primary, Urrutia, Samuel, primary, Montalban-Bravo, Guillermo, primary, Sasaki, Koji, primary, Chien, Kelly S., primary, Hammond, Danielle, primary, Kanagal-Shamanna, Rashmi, primary, Ganan-Gomez, Irene, primary, Kadia, Tapan M., primary, Borthakur, Gautam, primary, DiNardo, Courtney D., primary, Daver, Naval G., primary, Jabbour, Elias J., primary, Ravandi, Farhad, primary, Kantarjian, Hagop, primary, and Garcia-Manero, Guillermo, primary

Published
2024
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41. Supplemental Figure 1 from Cytogenetic and Molecular Associations with Outcomes in Higher-Risk Myelodysplastic Syndromes Treated with Hypomethylating Agents plus Venetoclax

Author

Bazinet, Alexandre, primary, Desikan, Sai Prasad, primary, Li, Ziyi, primary, Rodriguez-Sevilla, Juan Jose, primary, Venugopal, Sangeetha, primary, Urrutia, Samuel, primary, Montalban-Bravo, Guillermo, primary, Sasaki, Koji, primary, Chien, Kelly S., primary, Hammond, Danielle, primary, Kanagal-Shamanna, Rashmi, primary, Ganan-Gomez, Irene, primary, Kadia, Tapan M., primary, Borthakur, Gautam, primary, DiNardo, Courtney D., primary, Daver, Naval G., primary, Jabbour, Elias J., primary, Ravandi, Farhad, primary, Kantarjian, Hagop, primary, and Garcia-Manero, Guillermo, primary

Published
2024
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42. Supplemental Figure 2 from Cytogenetic and Molecular Associations with Outcomes in Higher-Risk Myelodysplastic Syndromes Treated with Hypomethylating Agents plus Venetoclax

Author

Bazinet, Alexandre, primary, Desikan, Sai Prasad, primary, Li, Ziyi, primary, Rodriguez-Sevilla, Juan Jose, primary, Venugopal, Sangeetha, primary, Urrutia, Samuel, primary, Montalban-Bravo, Guillermo, primary, Sasaki, Koji, primary, Chien, Kelly S., primary, Hammond, Danielle, primary, Kanagal-Shamanna, Rashmi, primary, Ganan-Gomez, Irene, primary, Kadia, Tapan M., primary, Borthakur, Gautam, primary, DiNardo, Courtney D., primary, Daver, Naval G., primary, Jabbour, Elias J., primary, Ravandi, Farhad, primary, Kantarjian, Hagop, primary, and Garcia-Manero, Guillermo, primary

Published
2024
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43. Data from Cytogenetic and Molecular Associations with Outcomes in Higher-Risk Myelodysplastic Syndromes Treated with Hypomethylating Agents plus Venetoclax

Author

Bazinet, Alexandre, primary, Desikan, Sai Prasad, primary, Li, Ziyi, primary, Rodriguez-Sevilla, Juan Jose, primary, Venugopal, Sangeetha, primary, Urrutia, Samuel, primary, Montalban-Bravo, Guillermo, primary, Sasaki, Koji, primary, Chien, Kelly S., primary, Hammond, Danielle, primary, Kanagal-Shamanna, Rashmi, primary, Ganan-Gomez, Irene, primary, Kadia, Tapan M., primary, Borthakur, Gautam, primary, DiNardo, Courtney D., primary, Daver, Naval G., primary, Jabbour, Elias J., primary, Ravandi, Farhad, primary, Kantarjian, Hagop, primary, and Garcia-Manero, Guillermo, primary

Published
2024
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44. Supplemental Figure 4 from Cytogenetic and Molecular Associations with Outcomes in Higher-Risk Myelodysplastic Syndromes Treated with Hypomethylating Agents plus Venetoclax

Author

Bazinet, Alexandre, primary, Desikan, Sai Prasad, primary, Li, Ziyi, primary, Rodriguez-Sevilla, Juan Jose, primary, Venugopal, Sangeetha, primary, Urrutia, Samuel, primary, Montalban-Bravo, Guillermo, primary, Sasaki, Koji, primary, Chien, Kelly S., primary, Hammond, Danielle, primary, Kanagal-Shamanna, Rashmi, primary, Ganan-Gomez, Irene, primary, Kadia, Tapan M., primary, Borthakur, Gautam, primary, DiNardo, Courtney D., primary, Daver, Naval G., primary, Jabbour, Elias J., primary, Ravandi, Farhad, primary, Kantarjian, Hagop, primary, and Garcia-Manero, Guillermo, primary

Published
2024
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45. Supplemental Table 3 from Cytogenetic and Molecular Associations with Outcomes in Higher-Risk Myelodysplastic Syndromes Treated with Hypomethylating Agents plus Venetoclax

Author

Bazinet, Alexandre, primary, Desikan, Sai Prasad, primary, Li, Ziyi, primary, Rodriguez-Sevilla, Juan Jose, primary, Venugopal, Sangeetha, primary, Urrutia, Samuel, primary, Montalban-Bravo, Guillermo, primary, Sasaki, Koji, primary, Chien, Kelly S., primary, Hammond, Danielle, primary, Kanagal-Shamanna, Rashmi, primary, Ganan-Gomez, Irene, primary, Kadia, Tapan M., primary, Borthakur, Gautam, primary, DiNardo, Courtney D., primary, Daver, Naval G., primary, Jabbour, Elias J., primary, Ravandi, Farhad, primary, Kantarjian, Hagop, primary, and Garcia-Manero, Guillermo, primary

Published
2024
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47. Impact of splicing mutations in acute myeloid leukemia treated with hypomethylating agents combined with venetoclax

Author

Lachowiez, Curtis A., Loghavi, Sanam, Furudate, Ken, Montalban-Bravo, Guillermo, Maiti, Abhishek, Kadia, Tapan, Daver, Naval, Borthakur, Gautam, Pemmaraju, Naveen, Sasaki, Koji, Alvarado, Yesid, Yilmaz, Musa, Short, Nicholas J., Chien, Kelly, Ohanian, Maro, Pierce, Sherry, Patel, Keyur P., Jabbour, Elias, Ravandi, Farhad, Kantarjian, Hagop M., Garcia-Manero, Guillermo, Takahashi, Koichi, Konopleva, Marina Y., and DiNardo, Courtney D.

Published
2021
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48. Prognostic value of measurable residual disease after venetoclax and decitabine in acute myeloid leukemia

Author

Maiti, Abhishek, DiNardo, Courtney D., Wang, Sa A., Jorgensen, Jeffrey, Kadia, Tapan M., Daver, Naval G., Short, Nicholas J., Yilmaz, Musa, Pemmaraju, Naveen, Borthakur, Gautam, Bose, Prithviraj, Issa, Ghayas C., Ferrajoli, Alessandra, Jabbour, Elias J., Jain, Nitin, Garcia-Manero, Guillermo, Ohanian, Maro, Takahashi, Koichi, Montalban-Bravo, Guillermo, Masarova, Lucia, Burger, Jan A., Thompson, Philip A., Verstovsek, Srdan, Sasaki, Koji, Andreeff, Michael, Rausch, Caitlin R., Montalbano, Kathryn S., Pierce, Sherry, Qiao, Wei, Ning, Jing, Kantarjian, Hagop M., Konopleva, Marina Y., and Ravandi, Farhad

Published
2021
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49. TP53 mutation does not confer a poor outcome in adult patients with acute lymphoblastic leukemia who are treated with frontline hyper-CVAD-based regimens.

Author

Kanagal-Shamanna, Rashmi, Jain, Preetesh, Takahashi, Koichi, Short, Nicholas, Tang, Guilin, Issa, Ghayas, Ravandi, Farhad, Garcia-Manero, Guillermo, Yin, Cameron, Luthra, Rajyalakshmi, Patel, Keyur, Khoury, Joseph, Montalban-Bravo, Guillermo, Sasaki, Koji, Kadia, Tapan, Borthakur, Gautam, Konopleva, Marina, Jain, Nitin, Garris, Rebecca, Pierce, Sherry, Wierda, William, Estrov, Zeev, Cortes, Jorge, Kantarjian, Hagop, Jabbour, Elias, and OBrien, Susan

Subjects
acute lymphoblastic leukemia (ALL), hyperfractionated cyclophosphamide, vincristine, doxorubicin, and dexamethasone (hyper-CVAD), next-generation sequencing, tumor protein 53 (TP53), Adult, Age Factors, Aged, Aged, 80 and over, Alleles, Antineoplastic Combined Chemotherapy Protocols, Cyclophosphamide, Dexamethasone, Doxorubicin, Female, Genes, p53, Humans, Leukemia, Myeloid, Chronic, Atypical, BCR-ABL Negative, Male, Middle Aged, Mutation, Mutation, Missense, Precursor B-Cell Lymphoblastic Leukemia-Lymphoma, Precursor Cell Lymphoblastic Leukemia-Lymphoma, Sequence Analysis, DNA, Treatment Outcome, Tumor Suppressor Protein p53, Vincristine
Abstract

BACKGROUND: Tumor protein 53 (TP53) mutations are uncommon in adult patients with acute lymphoblastic leukemia (ALL) and predict a poor outcome. METHODS: TP53 mutation analysis was performed in 164 newly diagnosed adult patients with ALL using a combination of targeted amplicon-based next-generation sequencing and Sanger sequencing. RESULTS: TP53 mutations were detected in 25 patients (15%), with a median allelic frequency of 42.2% (range, 5.6%-93.8%). The majority of mutations were single-nucleotide variants of missense type and involved the DNA-binding domain. TP53-mutated (TP53mut ) ALL was found to be significantly associated with older age, lower median white blood cell and platelet counts, lower frequency of Philadelphia chromosome and a higher frequency of low hypodiploid karyotype compared with ALL with wild-type TP53 (TP53wt ). To evaluate the prognostic effect of TP53 mutations, the authors selected 146 patients with B-cell immunophenotype ALL (24 with TP53mut and 122 with TP53wt ) who were uniformly treated with frontline hyperfractionated cyclophosphamide, vincristine, doxorubicin, and dexamethasone (hyper-CVAD)-based regimens; >90% of these individuals also received a monoclonal antibody. Over a median follow-up duration of 15 months, there was no significant difference in the median overall survival, event-free survival, and duration of complete remission noted between patients with TP53mut ALL and those with TP53wt ALL. CONCLUSIONS: Hyper-CVAD-based regimens appear to negate the poor prognostic impact of TP53 mutations in patients with adult B-cell immunophenotype ALL. Cancer 2017;123:3717-24. © 2017 American Cancer Society.

Published
2017

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