Definition and research guide
This page combines a MeSH definition, publication statistics and selected research abstracts. It is a starting point for reading the literature, not a medical textbook or a systematic evidence review.
Content available: 3 complete abstracts (576 words), 9 references. Full article text is not stored or reproduced here; follow the PMID, DOI or PMC links to read the original sources and their access terms.
Definition
A progressive, malignant disease of the blood-forming organs, characterized by distorted proliferation and development of leukocytes and their precursors in the blood and bone marrow. Leukemias were originally termed acute or chronic based on life expectancy but now are classified according to cellular maturity. Acute leukemias consist of predominately immature cells; chronic leukemias are composed of more mature cells. (From The Merck Manual, 2006)
Definition: NLM Medical Subject Headings (MeSH) scope note (Public domain (U.S. National Library of Medicine); attribution requested). View the MeSH record.
Also known as: Leukemias; Leucocythaemia; Leucocythaemias; Leucocythemia; Leucocythemias.
Where it fits in the MeSH classification
- Broader topics
- Neoplasms by Histologic Type; Hematologic Diseases
- Narrower topics
- Blastic Plasmacytoid Dendritic Cell Neoplasm; Enzootic Bovine Leukosis; Leukemia, Experimental; Leukemia, Feline; Leukemia, Hairy Cell; Leukemia, Lymphoid; Leukemia, Mast-Cell; Leukemia, Myeloid; Leukemia, Plasma Cell; Leukemia, Radiation-Induced
- See also
- Leukemic Infiltration
Research landscape
PubMed indexes 270,380 records tagged with the MeSH heading “Leukemia” (counts retrieved 2026-10-29). 38,381 of them (14.2%) were published from 2020 onward, and the busiest period so far is through 1999 with 128,661 records.
Reviews account for 30,385 records (11.2%) and meta-analyses for 1,052. Randomized controlled trials number 3,685 (1.4%) as a share of all records on the topic. Randomized trials make up only a small share of this literature, so much of what is known comes from laboratory, observational or descriptive work.
Show the data
| Period | Records |
|---|---|
| through 1999 | 128,661 |
| 2000-2009 | 45,740 |
| 2010-2019 | 57,598 |
| 2020 onward | 38,381 |
Publication-type chart data
| Type | Records | Share |
|---|---|---|
| Reviews | 30,385 | 11.2% |
| Meta-analyses | 1,052 | 0.4% |
| Randomized controlled trials | 3,685 | 1.4% |
Method: PubMed query "Leukemia"[MeSH Terms] with publication-type and date filters, retrieved 2026-10-29.
Reading the evidence in cancer and oncology
Cancer research spans very different kinds of evidence. Laboratory studies in cell lines and animal models reveal mechanisms but often do not translate to patients. Early-phase trials mainly test safety and dosing, while phase 3 randomized trials and their meta-analyses are the usual basis for treatment conclusions. Outcomes also differ in meaning: tumour shrinkage, progression-free survival and overall survival are not interchangeable, and screening studies can be distorted by lead-time and overdiagnosis. When reading a result, check the cancer type and stage studied, the comparison treatment, and which outcome was measured.
Evidence from open-access studies
These open-access studies come from PubMed’s best-match results for this heading (papers whose main topic it is), ranked here by citation count, with one recent addition. Each abstract is reproduced in full under its recorded open licence so that you can read the authors’ own words. They illustrate the literature; they are not a systematic summary of it. Citation counts measure attention, not reliability or clinical benefit.
| Study / authors | Year | Source label / design | Europe PMC citations | Licence / source |
|---|---|---|---|---|
| Optimizing venetoclax duration with hypomethylating agents for newly diagnosed acute myeloid leukemia: impact on response and survival | 2026 | Research article Design not recorded in structured metadata | 2 | cc by PMID 42734838 · DOI · PMC13574922 |
| Five-year follow-up of OPTIC: long-term efficacy, safety, and mutation analyses of ponatinib in chronic-phase chronic myeloid leukemia from a randomized phase 2 trial | 2026 | Randomized controlled trial Design not recorded in structured metadata | 2 | cc by PMID 42498834 · DOI · PMC13506327 |
| Targeting oncogenic FLT3 uncovers a ferroptosis vulnerability through selenocysteine recoding in acute myeloid leukaemia | 2026 | Research article Design not recorded in structured metadata | 1 | cc by PMID 42576015 · DOI · PMC13457094 |
Optimizing venetoclax duration with hypomethylating agents for newly diagnosed acute myeloid leukemia: impact on response and survival [1]
Abstract reading summary — source excerpts
- Opening excerpt
- The addition of venetoclax to hypomethylating agents (HMAs) is standard for older adults with newly diagnosed acute myeloid leukemia (AML).
- Closing excerpt
- These findings support further prospective evaluation of reduced VEN durations in this population.
- Interpretation limits
- Verbatim excerpts from the authors’ abstract; the closing excerpt may describe a finding, limitation or recommendation. Design, population, effect size and uncertainty have not been independently extracted or appraised. Read the complete abstract and linked paper before interpreting the findings.
Read complete abstract (229 words)
The addition of venetoclax to hypomethylating agents (HMAs) is standard for older adults with newly diagnosed acute myeloid leukemia (AML). However, prolonged venetoclax (VEN) exposure can cause cytopenias and infections, raising questions about optimal duration. We conducted a single‑center retrospective study of 102 patients treated between 2018 and 2025 with azacitidine or decitabine plus VEN administered for 14 (n = 22), 21 (n = 48), or 28 (n = 32) days per 28‑day cycle. Baseline cytogenetic and molecular profiling, responses by 2022/2024 ELN criteria, measurable residual disease (MRD), cumulative incidence of relapse (CIR), disease‑free survival (DFS), and overall survival (OS) were analyzed using competing‑risks regression and Cox models. Median age was 68 years and patients received a median of 4 cycles. Composite remission (CR/CRi/CRh/MLFS) was 66.7%, with MRD negativity in 75.0% of responders. Remission and MRD negativity rates did not differ significantly across VEN‑duration cohorts. Median OS and DFS were 17.3 and 12.3 months, respectively, with no significant differences in OS, DFS, and CIR between 14‑, 21‑, and 28‑day cohorts. Grade ≥ 3 cytopenias and transfusion independence rates also did not differ significantly. In this cohort, shortening VEN exposure to 14 or 21 days did not show a significant difference in response, survival, relapse risk, and toxicity profiles relative to a 28‑day schedule in our cohort. These findings support further prospective evaluation of reduced VEN durations in this population.
Abstract reproduced from the open-access article under the recorded cc by licence; © the authors. PMID 42734838 · DOI · PMC13574922
Five-year follow-up of OPTIC: long-term efficacy, safety, and mutation analyses of ponatinib in chronic-phase chronic myeloid leukemia from a randomized phase 2 trial [2]
Abstract reading summary — source excerpts
- Opening excerpt
- The primary analysis of the phase 2 OPTIC trial (NCT02467270) demonstrated optimal benefit:risk with response-based ponatinib dosing (45 mg once daily (QD) reduced to 15 mg QD) upon achieving ≤1% BCR::ABL1IS in patients with tyrosine kinase inhibitor-resistant or T315I-positive chronic-phase chronic myeloid leukemia (CP-CML).
- Closing excerpt
- These findings support long-term clinical benefit of response-based ponatinib dosing in third-line CP-CML, especially in patients with the T315I mutation.
- Interpretation limits
- Verbatim excerpts from the authors’ abstract; the closing excerpt may describe a finding, limitation or recommendation. Design, population, effect size and uncertainty have not been independently extracted or appraised. Read the complete abstract and linked paper before interpreting the findings.
Read complete abstract (201 words)
The primary analysis of the phase 2 OPTIC trial (NCT02467270) demonstrated optimal benefit:risk with response-based ponatinib dosing (45 mg once daily (QD) reduced to 15 mg QD) upon achieving ≤1% BCR::ABL1IS in patients with tyrosine kinase inhibitor-resistant or T315I-positive chronic-phase chronic myeloid leukemia (CP-CML). Here, we report 5-year long-term outcomes. Overall, 283 patients were randomized to 45-mg, 30-mg, or 15-mg QD starting doses (n = 94, 95, and 94, respectively), with dose reduction to 15 mg QD upon response in the 45-mg and 30-mg cohorts. At data cutoff, 61 patients remained on trial. Median follow-up time was 75-78 months. By 5 years, 60%, 41%, and 40% of patients in the 45-mg, 30-mg, and 15-mg cohorts, respectively, achieved ≤1% BCR::ABL1IS. Five-year progression-free survival rates were 63%, 57%, and 60%, respectively, by cohort; overall survival rates exceeded 80%. In patients with a T315I mutation, 5-year rates of ≤1% BCR::ABL1IS, PFS, and OS were highest in the 45-mg cohort. Exposure-adjusted rates of adjudicated arterial occlusive events were 4.1, 3.8, and 2.0 patients per 100 patient-years, respectively, by cohort; results were comparable in T315I-positive patients. These findings support long-term clinical benefit of response-based ponatinib dosing in third-line CP-CML, especially in patients with the T315I mutation.
Abstract reproduced from the open-access article under the recorded cc by licence; © the authors. PMID 42498834 · DOI · PMC13506327
Targeting oncogenic FLT3 uncovers a ferroptosis vulnerability through selenocysteine recoding in acute myeloid leukaemia [3]
Abstract reading summary — source excerpts
- Opening excerpt
- Ferroptosis, an iron-dependent form of cell death driven by lipid peroxidation, has emerged as a potential therapeutic strategy for therapy-resistant cancers.
- Closing excerpt
- This study highlights ferroptosis as a vulnerability in FLT3-mutant acute myeloid leukaemia and suggests that high vitamin E intake may compromise tyrosine kinase inhibitor efficacy partly by suppressing ferroptosis.
- Interpretation limits
- Verbatim excerpts from the authors’ abstract; the closing excerpt may describe a finding, limitation or recommendation. Design, population, effect size and uncertainty have not been independently extracted or appraised. Read the complete abstract and linked paper before interpreting the findings.
Read complete abstract (146 words)
Ferroptosis, an iron-dependent form of cell death driven by lipid peroxidation, has emerged as a potential therapeutic strategy for therapy-resistant cancers. Glutathione peroxidase 4 and the selenoprotein biosynthesis pathway essential for its translation are key regulators of ferroptosis but lack effective therapeutic targeting. In a drug screening using a selenoprotein translation reporter, here we identify FMS-like tyrosine kinase 3 (FLT3) inhibitors as suppressors of selenoprotein translation that induce ferroptosis in FLT3-mutant acute myeloid leukaemia. Mechanistically, FLT3 inhibition disrupts selenocysteine recoding, in which a UGA stop codon is recoded as selenocysteine via the SECIS element and associated binding proteins. Notably, the antileukemic efficacy of the FLT3 inhibitor gilteritinib was markedly reduced by dietary vitamin E, which attenuated ferroptosis. This study highlights ferroptosis as a vulnerability in FLT3-mutant acute myeloid leukaemia and suggests that high vitamin E intake may compromise tyrosine kinase inhibitor efficacy partly by suppressing ferroptosis.
Abstract reproduced from the open-access article under the recorded cc by licence; © the authors. PMID 42576015 · DOI · PMC13457094
Key reviews and meta-analyses
The most cited review-type papers among PubMed’s best-match results for this heading, listed with bibliographic details only. Follow the links for the full text and access terms.
- Research article Association of Measurable Residual Disease With Survival Outcomes in Patients With Acute Myeloid Leukemia: A Systematic Review and Meta-analysis [4]
- Research article CT Scans and Cancer Risks: A Systematic Review and Dose-response Meta-analysis [5]
- Research article Association between Outdoor Air Pollution and Childhood Leukemia: A Systematic Review and Dose-Response Meta-Analysis [6]
- Research article Ultra-processed food consumption and cancer risk: A systematic review and meta-analysis [7]
- Research article Therapy for children and adults with mixed phenotype acute leukemia: a systematic review and meta-analysis [8]
- Research article A systematic literature review and meta-analysis of minimal residual disease as a prognostic indicator in adult B-cell acute lymphoblastic leukemia [9]
References
- Ginosyan AA, Ashouri K, Cheng J, Ford L, Baya M, Humayun L, et al.. Optimizing venetoclax duration with hypomethylating agents for newly diagnosed acute myeloid leukemia: impact on response and survival. . 2026. PMID 42734838 · DOI · PMC13574922
- Kantarjian H, Deininger M, Apperley JF, Arthur CK, Chuah C, Hochhaus A, et al.. Five-year follow-up of OPTIC: long-term efficacy, safety, and mutation analyses of ponatinib in chronic-phase chronic myeloid leukemia from a randomized phase 2 trial. . 2026. PMID 42498834 · DOI · PMC13506327
- Li M, Zhu Y, Kageyama Y, Furudate K, Kitano A, Tan T, et al.. Targeting oncogenic FLT3 uncovers a ferroptosis vulnerability through selenocysteine recoding in acute myeloid leukaemia. . 2026. PMID 42576015 · DOI · PMC13457094
- Short NJ, Zhou S, Fu C, Berry DA, Walter RB, Freeman SD, et al.. Association of Measurable Residual Disease With Survival Outcomes in Patients With Acute Myeloid Leukemia: A Systematic Review and Meta-analysis. JAMA Oncol. 2020. PMID 33030517 · DOI · PMC7545346
- Cao CF, Ma KL, Shan H, Liu TF, Zhao SQ, Wan Y, et al.. CT Scans and Cancer Risks: A Systematic Review and Dose-response Meta-analysis. BMC Cancer. 2022. PMID 36451138 · DOI · PMC9710150
- Filippini T, Hatch EE, Rothman KJ, Heck JE, Park AS, Crippa A, et al.. Association between Outdoor Air Pollution and Childhood Leukemia: A Systematic Review and Dose-Response Meta-Analysis. Environ Health Perspect. 2019. PMID 31017485 · DOI · PMC6785230
- Isaksen IM, Dankel SN. Ultra-processed food consumption and cancer risk: A systematic review and meta-analysis. Clin Nutr. 2023. PMID 37087831 · DOI
- Maruffi M, Sposto R, Oberley MJ, Kysh L, Orgel E. Therapy for children and adults with mixed phenotype acute leukemia: a systematic review and meta-analysis. Leukemia. 2018. PMID 29550836 · DOI · PMC7508489
- Bassan R, Brüggemann M, Radcliffe HS, Hartfield E, Kreuzbauer G, Wetten S. A systematic literature review and meta-analysis of minimal residual disease as a prognostic indicator in adult B-cell acute lymphoblastic leukemia. Haematologica. 2019. PMID 30890593 · DOI · PMC6886415
About this article
- Sources
- NLM Medical Subject Headings (definition, synonyms); NCBI PubMed (counts, identifiers); Europe PMC (open-access abstracts and citation counts).
- Verification
- Every reference was resolved against PubMed on 2026-10-31; title, year and DOI matched.
- Review state
- automated source verified; no human expert review has been recorded yet.
- Version
- 1, last updated 2026-10-29.
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Evidence strength
EvidenceHelix's computed view of the study designs behind this topic: how common meta-analyses, randomized trials and reviews are, how recent the literature is, and a plain-language reading. It describes the design mix of the literature. It is not a quality appraisal of any study, and it is not medical advice.