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2 weeks ago | 
nature.com | Jayastu Senapati |Guillermo Garcia-Manero |Courtney DiNardo |Gautam Borthakur |Tapan M Kadia |Elias Jabbour | +8 more
Despite the improvement in overall outcomes of patients with acute myeloid leukemia (AML), some high-risk disease subsets continue to fare dismally. AML with TP53 aberrations (mutations, deletions) is one such subset of high-risk AML with a median survival of about 6–9 months [1,2,3].
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3 weeks ago | 
nature.com | Mahesh Swaminathan |Courtney DiNardo |Naveen Pemmaraju |Guillermo Garcia-Manero |Ghayas C. Issa |Gautam Borthakur | +9 more
To the Editor:The advent of VEN (VEN) has changed the treatment paradigm of acute myeloid leukemia (AML). Currently, venetoclax (VEN), in combination with a hypomethylating agent (HMA), is approved for the treatment of adult patients with AML unsuitable for intensive chemotherapy or frontline treatment of patients ≥75 years [1].
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3 weeks ago | 
nature.com | Alex Bataller |Koji Sasaki |Danielle Hammond |Mahesh Swaminathan |Ghayas C. Issa |Nicholas Short | +6 more
AbstractHypomethylating agents (HMA) are indicated in the treatment of higher-risk myelodysplastic syndromes (MDS) and chronic myelomonocytic leukemia (CMML). The combination of hypomethylating agents with venetoclax (Ven) has demonstrated promising results in these diseases, although randomized clinical trials are needed for validation.
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1 month ago | 
nature.com | Courtney DiNardo |Wei-Ying Jen |Koichi Takahashi |Tapan M Kadia |Sanam Loghavi |Patrick K. Reville | +19 more
AbstractIntensive chemotherapy remains the standard for newly diagnosed (ND) acute myeloid leukemia (AML); however, relapse risk remains high. Additionally, most patients with relapsed/refractory (RR) AML have poor outcomes. We report the long-term experience of 138 patients, 77 ND and 61 RR, treated with FLAG-IDA in combination with venetoclax.
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2 months ago | 
onlinelibrary.wiley.com | Farhad Ravandi
Significant progress in the characterization of molecular pathogenic events in acute myeloid leukemia (AML) has led to better characterization of prognosis and identification of subsets that are more likely to benefit from currently available strategies. Furthermore, deciphering these molecular pathogenic events has led to the development of a number of effective molecularly targeted agents that have significantly improved our armamentarium in managing patients with AML.
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Jan 6, 2025 | 
onlinelibrary.wiley.com | Hagop Kantarjian |Nicholas Short |NITIN JAIN |Fadi Haddad |Tapan Mahendra Kadia |Musa Yilmaz | +8 more
Conflicts of Interest H.K. received research grants from AbbVie, Amgen, Ascentage, BMS, Daiichi-Sankyo, Immunogen, Jazz, Novartis, Pfizer; and honoraria from AbbVie, Amgen, Aptitude Health, Ascentage, Astellas Health, Astra Zeneca, Ipsen, Pharmaceuticals, KAHR Medical Ltd., NOVA Research, Novartis, Pfizer, Precision Biosciences, and Taiho Pharmaceutical Canada.
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Nov 12, 2024 | 
nature.com | Douglas Tremblay |Clifford M. Csizmar |Courtney DiNardo |Danielle Hammond |Tapan M Kadia |Farhad Ravandi | +10 more
Chronic myelomonocytic leukemia (CMML) is a rare hematologic malignancy with overlapping features of myelodysplastic neoplasm (MDS) and myeloproliferative neoplasms characterized by peripheral blood monocytosis [1]. There is a predisposition for transformation to acute myeloid leukemia (AML), termed CMML with blast transformation (CMML-BT) [2, 3].
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Sep 25, 2024 | 
nature.com | Farhad Ravandi |Jayastu Senapati |Nicholas Short |Tapan M Kadia |Gautam Borthakur |Marina Konopleva | +5 more
AbstractOptimal frontline use of active agents in T-cell acute lymphoblastic leukemia/lymphoma (T-ALL/LBL) is prudent to improve outcomes. We report the long-term follow-up of the phase 2 trial of HyperCVAD with nelarabine and pegylated asparaginase (Original cohort). In the latest protocol iteration venetoclax was added to the induction/consolidation regimen (Venetoclax cohort). Eligible patients were adults with untreated T-ALL/LBL or after minimal therapy and with adequate organ function.
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Aug 23, 2024 | 
nature.com | Branko Cuglievan |Hagop Kantarjian |Courtney DiNardo |Tapan M Kadia |Erin Guest |Nicholas Short | +18 more
AbstractAberrant expression of HOX and MEIS1 family genes, as seen in KMT2A-rearranged, NUP98-rearranged, or NPM1-mutated leukemias leads to arrested differentiation and leukemia development. HOX family genes are essential gatekeepers of physiologic hematopoiesis, and their expression is regulated by the interaction between KMT2A and menin.
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Sep 11, 2023 | 
nature.com | Nicholas Short |Farhad Ravandi
AbstractFLT3 is the most frequently mutated gene in acute myeloid leukemia (AML), with FLT3 internal tandem duplication (ITD) mutations being associated with a more aggressive clinical course. While two large, randomized clinical trials have shown a survival benefit with the frontline use of an oral FLT3 inhibitor (midostaurin or quizartinib) in patients with FLT3-mutated AML, the role of FLT3 inhibitors in older adults with newly diagnosed FLT3-mutated AML remains unclear.
