PapersFlow Research Brief
Acute Myeloid Leukemia Research
Research Guide
What is Acute Myeloid Leukemia Research?
Acute Myeloid Leukemia Research is the scientific investigation into the classification, genetic basis, cellular hierarchy, diagnosis, and management of acute myeloid leukemia, a hematologic malignancy characterized by uncontrolled proliferation of myeloid precursor cells.
Acute Myeloid Leukemia Research encompasses 112,772 published works focused on myeloid neoplasms and acute leukemias. Key contributions include WHO classifications revised in 2008, 2009, and 2016 that incorporate genetic biomarkers for precise categorization. Studies have established AML as originating from a hierarchy of primitive hematopoietic cells, with at least one driver mutation identified in nearly all cases.
Research Sub-Topics
WHO Classification of Acute Myeloid Leukemia
This sub-topic updates integrated genomic, immunophenotypic, and morphologic criteria for AML subtypes. Researchers incorporate revisions from 2008, 2016 for prognostication.
Leukemia Stem Cell Hierarchy in AML
Studies identify primitive CD34+CD38- cells initiating AML in xenografts and hierarchies. Focuses on self-renewal, quiescence, and therapeutic targeting.
Genomic and Epigenomic Landscapes of De Novo AML
Comprehensive sequencing reveals mutations (DNMT3A, FLT3, NPM1) and epigenetic alterations. Researchers profile subtypes and clonal evolution.
ELN Recommendations for AML Diagnosis and Management
Harmonizes European LeukemiaNet guidelines on diagnostics, induction, consolidation, and MRD. Includes updates for older adults and novel agents.
Clonal Hematopoiesis in AML Precursor States
Examines age-related CH with DNMT3A/TET2/ASXL1 mutations progressing to myeloid neoplasms. Links to therapy-related AML and prognostic scores.
Why It Matters
Acute Myeloid Leukemia Research directly informs clinical guidelines that improve patient outcomes through standardized diagnosis and risk stratification. The 2017 ELN recommendations by Döhner et al. (2016) integrate genetic discoveries to guide therapy, while genomic analyses by Ley (2013) reveal driver mutations enabling targeted interventions. Recent developments include FDA breakthrough status for ICT01 plus venetoclax in AML and a $13 million National Cancer Institute grant to UVA for treatment-resistant cases, alongside $1 million from Blood Cancer United for escalated approaches.
Reading Guide
Where to Start
"The 2016 revision to the World Health Organization classification of myeloid neoplasms and acute leukemia" by Arber et al. (2016), as it provides the foundational updated classification incorporating biomarkers essential for understanding modern AML categorization.
Key Papers Explained
Arber et al. (2016) builds on Vardiman et al. (2009) and the 2008 WHO framework by integrating post-2008 biomarker advances for myeloid neoplasms. Döhner et al. (2016) applies these classifications in ELN recommendations for AML management. Bonnet and Dick (1997) and Lapidot et al. (1994) establish the primitive cell hierarchy underpinning genomic findings in Ley (2013). Jaiswal et al. (2014) links clonal hematopoiesis to AML risk.
Paper Timeline
Most-cited paper highlighted in red. Papers ordered chronologically.
Advanced Directions
Preprints profile NPM1 subtypes via single-cell RNA-seq on 120 AMLs and venetoclax combinations for chemotherapy-free management. FDA granted breakthrough status to ICT01 plus venetoclax; $13M NCI grant to UVA targets resistant AML. ASH 2025 features ECOG-ACRIN data; tools like Tazi et al.'s aml-risk-model.com and SCEMILA enable subtype classification.
Papers at a Glance
| # | Paper | Year | Venue | Citations | Open Access |
|---|---|---|---|---|---|
| 1 | The 2016 revision to the World Health Organization classificat... | 2016 | Blood | 10.0K | ✓ |
| 2 | Human acute myeloid leukemia is organized as a hierarchy that ... | 1997 | Nature Medicine | 6.9K | ✕ |
| 3 | Diagnosis and management of AML in adults: 2017 ELN recommenda... | 2016 | Blood | 5.7K | ✓ |
| 4 | Proposals for the Classification of the Acute Leukaemias F<scp... | 1976 | British Journal of Hae... | 5.6K | ✕ |
| 5 | Genomic and Epigenomic Landscapes of Adult De Novo Acute Myelo... | 2013 | New England Journal of... | 5.0K | ✓ |
| 6 | A cell initiating human acute myeloid leukaemia after transpla... | 1994 | Nature | 4.7K | ✕ |
| 7 | World Health Organization Classification of Tumours: Pathology... | 2002 | Annals of Oncology | 4.6K | ✓ |
| 8 | Age-Related Clonal Hematopoiesis Associated with Adverse Outcomes | 2014 | New England Journal of... | 4.5K | ✓ |
| 9 | International Scoring System for Evaluating Prognosis in Myelo... | 1997 | Blood | 4.4K | ✓ |
| 10 | The 2008 revision of the World Health Organization (WHO) class... | 2009 | Blood | 4.4K | ✓ |
In the News
$1 Million Grant from Blood Cancer United to Escalate ...
approach for patients with treatment-resistant acute myeloid leukemia (AML).
FDA Grants Breakthrough Status to ICT01 Plus Venetoclax ...
(AML).1
Guide to ASH 2025: ECOG-ACRIN and PrECOG are set to ...
--------------------- The PrE0405 study was sponsored by PrECOG, LLC. All of the other research listed above was supported by the National Cancer Institute, part of the National Institutes of Health.
Breakthrough blood cancer research from UChicago ...
myeloid leukemia (AML).
$13 Million Grant Funds Battle Against Acute Myeloid ...
UVA Comprehensive Cancer Center researchers have received $13 million from the National Cancer Institute to advance innovative new approaches to treating acute myeloid leukemia (AML), the deadliest...
Code & Tools
Github Repository for Tazi et al. : Unified classification and risk-stratification in Acute Myeloid Leukemia. Please check our online tool : https:...
# Introduction The Peter Moss Acute Lymphoblastic Leukemia classifiers are a collection of projects that use computer vision to classify Acute Lym...
Single cell based explainable multiple instance learning algorithm (SCEMILA) for genetic acute myeloid leukemia subtype classification. ### Licens...
Artificial Intelligence-based Prediction of Acute Leukemia: a free and open-source software package built in R, with a user-friendly interface prov...
## Repository files navigation ## A cellular hierarchy framework for understanding heterogeneity and predicting drug response in AML Publication:...
Recent Preprints
The AML cellular state space unveils NPM1 immune evasion subtypes with distinct clinical outcomes
Acute myeloid leukemia is a genetically and cellularly heterogeneous disease. We characterize 120 AMLs using genomic and transcriptomic analyses, including single-cell RNA sequencing. Our results r...
Comprehensive view on chemotherapy-free management of acute myeloid leukemia by using venetoclax in combination with targeted and/or immune therapies
A hallmark of cancer biology is resistance to apoptosis. BCL-2 is an anti-apoptotic molecule that is being overexpressed in several myeloid diseases, such as acute myeloid leukemia and myelodysplas...
Emerging strategies and novel therapeutic targets in acute myeloid leukemia: current advances and future directions
Acute myeloid leukemia (AML) is an aggressive hematologic malignancy characterized by dysregulated differentiation and uncontrolled proliferation of myeloid precursor cells. AML is the second most ...
Emerging strategies and novel therapeutic targets in acute ...
Acute myeloid leukemia (AML) is an aggressive hematologic malignancy characterized by dysregulated differentiation and uncontrolled proliferation of myeloid precursor cells. AML is the second most ...
Leukemia
Classification and nomenclature of hematologic diseases
Latest Developments
Recent developments in AML research include the FDA approval of ziftomenib (Komzifti™) in November 2025 for adults with advanced AML and NPM1 mutations, and ongoing clinical trials exploring investigational drugs like ziftomenib in combination with standard therapies, as well as new targeted treatments such as quizartinib for FLT3-ITD mutations (bloodcancerunited.org, clinicaltrials.ucsd.edu, uvahealth.com).
Sources
Frequently Asked Questions
What is the WHO classification system for acute myeloid leukemia?
The World Health Organization classification of myeloid neoplasms and acute leukemia was revised in 2016 by Arber et al. to include unique biomarkers identified since 2008. The 2009 revision by Vardiman et al. updated the 4th edition with rationale for changes in categorization. These systems provide standards for recording cases in clinical trials.
How is acute myeloid leukemia organized at the cellular level?
Bonnet and Dick (1997) demonstrated that human AML is organized as a hierarchy originating from a primitive hematopoietic cell. Lapidot et al. (1994) identified a cell initiating human AML after transplantation into SCID mice. These findings establish the leukemia stem cell model.
What are the ELN recommendations for AML management?
Döhner et al. (2016) published 2017 ELN recommendations for diagnosis and management of AML in adults, building on the 2010 edition with advances in genetic discoveries. These guidelines have broad acceptance among physicians treating AML patients. They emphasize risk-adapted therapy.
What genetic features define de novo AML?
Ley (2013) mapped genomic and epigenomic landscapes of adult de novo AML, identifying at least one potential driver mutation in nearly all samples. A complex interplay of genetic events contributes to pathogenesis in individual patients. The study's databases support further AML investigations.
How has FAB classification influenced AML research?
Bennett et al. (1976) proposed the French-American-British (FAB) classification for acute leukemias to standardize nomenclature and distribution in clinical trials. It serves as a reference for newly developed cell-surface markers. This system facilitated early comparative studies.
What is the current state of AML classification?
Recent preprints like 'The AML cellular state space unveils NPM1 immune evasion subtypes with distinct clinical outcomes' (2025) use single-cell RNA sequencing on 120 AMLs to reveal cellular heterogeneity. Tools such as Tazi et al.'s unified classification model at aml-risk-model.com integrate risk-stratification. News highlights ASH 2025 presentations on ECOG-ACRIN studies.
Open Research Questions
- ? How do NPM1 immune evasion subtypes identified in single-cell analyses of 120 AMLs influence distinct clinical outcomes?
- ? What cellular hierarchies predict drug response in AML patient samples?
- ? Can chemotherapy-free regimens combining venetoclax with targeted therapies overcome BCL-2 overexpression in AML?
- ? Which genetic events interplay to drive pathogenesis in therapy-resistant AML cases?
- ? How do age-related clonal hematopoiesis mutations progress to full AML?
Recent Trends
Preprints from late 2025 emphasize single-cell analyses revealing NPM1 immune evasion subtypes in 120 AMLs and venetoclax-based chemotherapy-free strategies targeting BCL-2 overexpression.
News reports $13M NCI funding to UVA and $1M Blood Cancer United grant (2026) for resistant AML, plus FDA breakthrough for ICT01-venetoclax.
2025ASH 2025 previews PrECOG studies; GitHub tools like Tazi et al.'s unified risk model reflect classification advances.
Research Acute Myeloid Leukemia Research with AI
PapersFlow provides specialized AI tools for your field researchers. Here are the most relevant for this topic:
AI Literature Review
Automate paper discovery and synthesis across 474M+ papers
Deep Research Reports
Multi-source evidence synthesis with counter-evidence
Paper Summarizer
Get structured summaries of any paper in seconds
AI Academic Writing
Write research papers with AI assistance and LaTeX support
Start Researching Acute Myeloid Leukemia Research with AI
Search 474M+ papers, run AI-powered literature reviews, and write with integrated citations — all in one workspace.