{
  "abstract": "Core binding factor acute myeloid leukemia (CBF-AML)-defined by t(8;21) or inv(16) chromosomal rearrangements-is classified as favorable-risk disease per the European LeukemiaNet 2022 risk model and is typically treated with intensive chemotherapy. Patients unsuitable for intensive therapy increasingly receive hypomethylating agents plus venetoclax (HMA/ven), though CBF-AML was excluded from pivotal trials of this regimen. Furthermore, HMA/ven is typically administered as indefinite therapy in AML; however, there is increasing interest in exploring the feasibility of therapy cessation given persistent treatment-free remissions with this regimen. Here, we report the first case of sustained treatment-free remission following HMA/ven in CBF-AML. Features in this case that suggest a higher likelihood of achieving a durable treatment-free remission include inv(16) disease and rapid measurable residual disease (MRD) clearance. We summarize the current retrospective data comparing response rates of inv(16) vs. t(8;21) AML to HMA/ven: inv(16) AML consistently exhibits higher response rates compared with t(8;21) disease, although an overall survival benefit is not well established. Emerging data also suggest achieving MRD-negative complete remission after induction may predict more durable outcomes. This case provides proof-of-concept that carefully selected CBF-AML patients achieving sustained MRD-negative remission with HMA/ven can successfully discontinue therapy. Prospective evaluation of HMA/ven in unfit CBF-AML patients, weighed against dose-attenuated intensive chemotherapy, is needed to define its role and optimal duration.",
  "abstract_shown": true,
  "affiliations": [
    "Department of Medicine, Stanford Health Care, Stanford, CA, USA.",
    "Division of Hematology, Department of Medicine, Stanford Health Care, Stanford, CA, USA.",
    "Division of Hematology, Department of Medicine, Stanford Health Care, Stanford, CA, USA. gmannis@stanford.edu."
  ],
  "article_type": "case report",
  "arxiv": null,
  "author_total": 4,
  "authors": [
    {
      "affs": [
        0
      ],
      "name": "Morgan L Shannon",
      "orcid": "0000-0003-4941-7643"
    },
    {
      "affs": [
        1
      ],
      "name": "Jared Wallace",
      "orcid": "0009-0002-9427-221X"
    },
    {
      "affs": [
        1
      ],
      "name": "Amy Kuo"
    },
    {
      "affs": [
        2
      ],
      "name": "Gabriel N Mannis",
      "orcid": "0009-0000-8202-0870"
    }
  ],
  "bibtex": "https://research.draconitic.com/paper/entnh401f6br1.bib",
  "cited_by": 0,
  "doi": "10.1007/s00277-026-07237-0",
  "fulltext_url": "https://europepmc.org/articles/PMC13722771",
  "id": "entnh401f6br1",
  "issns": [
    "0939-5555",
    "1432-0584"
  ],
  "landing_url": "https://europepmc.org/article/MED/42963459",
  "language": "eng",
  "licence": "CC-BY-NC-ND",
  "licence_label": "CC BY-NC-ND",
  "licence_source": "article",
  "licence_url": "https://creativecommons.org/licenses/by-nc-nd/4.0/",
  "metadata_licence": null,
  "native_subjects": [
    [
      "heading",
      "Case Report"
    ],
    [
      "openalex",
      "2720|Acute Myeloid Leukemia Research"
    ]
  ],
  "openalex": "W7202070109",
  "pdf_url": "https://europepmc.org/articles/PMC13722771?pdf=render",
  "pmcid": "PMC13722771",
  "pmid": "42963459",
  "published": "2026-08-10",
  "publisher": null,
  "ris": "https://research.draconitic.com/paper/entnh401f6br1.ris",
  "sources": [
    {
      "first_seen": "2026-11-02T03:21:43Z",
      "native_id": "MED:42963459",
      "source": "europepmc",
      "url": "https://europepmc.org/article/MED/42963459"
    },
    {
      "first_seen": "2026-11-04T12:21:32Z",
      "native_id": "PMC13722771",
      "source": "pmc",
      "url": "https://doi.org/10.1007/s00277-026-07237-0"
    }
  ],
  "subjects": [
    {
      "field": "medicine",
      "origin": "openalex:2720|Acute Myeloid Leukemia Research",
      "subfield": "general"
    }
  ],
  "title": "Prolonged treatment-free remission after venetoclax in combination with a hypomethylating agent in acute myeloid leukemia with inv(16).",
  "title_text": "Prolonged treatment-free remission after venetoclax in combination with a hypomethylating agent in acute myeloid leukemia with inv(16).",
  "type": "peer-reviewed",
  "updated_at": "2026-11-04T12:21:32Z",
  "url": "https://research.draconitic.com/paper/entnh401f6br1",
  "venue": "Annals of hematology",
  "versions": []
}