Assessment of measurable residual disease (MRD) by quantitative reverse transcription polymerase chain reaction is strongly prognostic in patients with NPM1-mutated acute myeloid leukemia (AML) treated with intensive chemotherapy; however, there are no data regarding its utility in venetoclax-based nonintensive therapy, despite high efficacy in this genotype. We analyzed the prognostic impact of NPM1 MRD in an international real-world cohort of 76 previously untreated patients with NPM1-mutated AML who achieved complete remission (CR)/CR with incomplete hematological recovery following treatment with venetoclax and hypomethylating agents (HMAs) or low-dose cytarabine (LDAC). A total of 44 patients (58%) achieved bone marrow (BM) MRD negativity, and a further 14 (18%) achieved a reduction of ≥4 log10 from baseline as their best response, with no difference between HMAs and LDAC. The cumulative rates of BM MRD negativity by the end of cycles 2, 4, and 6 were 25%, 47%, and 50%, respectively. Patients achieving BM MRD negativity by the end of cycle 4 had 2-year overall of 84% compared with 46% if MRD was positive. On multivariable analyses, MRD negativity was the strongest prognostic factor. A total of 22 patients electively stopped therapy in BM MRD-negative remission after a median of 8 cycles, with 2-year treatment-free remission of 88%. In patients with NPM1-mutated AML attaining remission with venetoclax combination therapies, NPM1 MRD provides valuable prognostic information.
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http://dx.doi.org/10.1182/blood.2023021579 | DOI Listing |
Sultan Qaboos Univ Med J
November 2024
College of Medicine and Health Sciences, Sultan Qaboos University, Muscat, Oman.
Objectives: This study aimed to identify the impact of the COVID-19 pandemic on the frequency, clinical characteristics and outcomes of patients with acute myeloid leukaemia (AML) and acute lymphoblastic leukaemia (ALL).
Methods: This retrospective cohort study included all patients treated at Sultan Qaboos University Hospital in Muscat, Oman with AML or ALL from January 2017 to December 2021. Data were obtained from the electronic medical record, and patients diagnosed before the start of the COVID-19 pandemic were compared with those diagnosed during the pandemic using appropriate statistical tests.
Ann Hematol
December 2024
Division of Hematology, Department of Medicine, Faculty of Medicine, Chulalongkorn University and King Chulalongkorn Memorial Hospital, Rama IV Road, Patumwan, Bangkok, 10330, Thailand.
Hum Cell
November 2024
Department of Medical Oncology, Dr. B.R.A. IRCH, All India Institute of Medical Sciences, Room No. 401, 4th Floor, New Delhi, India.
Acute myeloid leukemia (AML) is characterized by impaired differentiation of myeloid cells leading to hematopoietic failure. Despite advances, the molecular mechanisms driving AML remain incompletely understood, limiting the identification and targeting of critical vulnerabilities in leukemic cells. Homeobox (HOX) genes, encoding transcription factors essential for myeloid and lymphoid differentiation, are distributed across four clusters: HOXA (chromosome 7), HOXB (chromosome 17), HOXC (chromosome 12), and HOXD (chromosome 2).
View Article and Find Full Text PDFBone Marrow Transplant
November 2024
Division of Hematology and Oncology, Rhode Island Hospital, The Warren Alpert Medical School of Brown University, Providence, RI, USA.
Leukemia
November 2024
Section of Hematology, Department of Internal Medicine, Yale School of Medicine and Yale Comprehensive Cancer Center, Yale University, New Haven, CT, USA.
The use of measurable residual disease (MRD) as a biomarker for prognostication, risk stratification, and therapeutic decision-making in acute myeloid leukemia (AML) is gaining prominence. MRD monitoring for NPM1-mutated and core-binding factor AML using PCR techniques is well-established for assessing disease after intensive chemotherapy. AML with persistent FLT3-ITD MRD post-intensive chemotherapy and pre-allogeneic hematopoietic cell transplantation (pre-allo-HCT) is associated with an increased risk of relapse and lower survival.
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