{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,22]],"date-time":"2026-09-22T05:06:20Z","timestamp":1790053580750,"version":"4.0.1"},"reference-count":128,"publisher":"MDPI AG","issue":"18","license":[{"start":{"date-parts":[[2026,9,19]],"date-time":"2026-09-19T00:00:00Z","timestamp":1789776000000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"funder":[{"name":"AIRC","award":["IG-ID 30522"],"award-info":[{"award-number":["IG-ID 30522"]}]},{"award":["IG-ID 30522"],"award-info":[{"award-number":["IG-ID 30522"]}],"id":[{"id":"https:\/\/ror.org\/02g2x7380","id-type":"ROR","asserted-by":"publisher"}]}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["Cancers"],"abstract":"<jats:p>NPM1-mutated acute myeloid leukemia (AML) represents one of the most frequent and biologically distinct AML entities, characterized by the aberrant cytoplasmic localization of the NPM1 mutant protein. The recent advances in molecular biology and translational research have progressively redefined the clinical management of this disease. This narrative review summarizes the evolution of NPM1-mutated AML over the last two decades, focusing on the transition from immunohistochemical observations to modern genetics-based and measurable residual disease (MRD)-driven approaches. We discuss the biological role of mutant NPM1, the prognostic impact of co-occurring genetic alterations, and the integration of molecular monitoring into routine clinical practice. Particular attention is given to the prognostic and therapeutic implications of the co-mutational landscape. We also discuss emerging targeted therapies directed against key pathogenic pathways, including the menin\u2013KMT2A axis and nuclear export machinery. Despite significant advances in risk stratification and treatment, considerable heterogeneity in clinical outcomes persists among patients with NPM1-mutated AML, indicating that genomic information obtained at diagnosis alone is insufficient to fully capture prognostic differences. Future advances will likely depend on the integration of genetic, transcriptional, and broader cellular-state information to better define biologically relevant disease states and guide more personalized therapeutic strategies.<\/jats:p>","DOI":"10.3390\/cancers18183046","type":"journal-article","created":{"date-parts":[[2026,9,21]],"date-time":"2026-09-21T12:50:03Z","timestamp":1789995003000},"page":"3046","update-policy":"https:\/\/doi.org\/10.3390\/mdpi_crossmark_policy","source":"Crossref","is-referenced-by-count":0,"title":["Two Decades of NPM1-Mutated Acute Myeloid Leukemia: From Molecular Insights to Clinical Decision-Making"],"prefix":"10.3390","volume":"18","author":[{"ORCID":"https:\/\/orcid.org\/0000-0003-4879-7824","authenticated-orcid":false,"given":"Gaetano","family":"Cimino","sequence":"first","affiliation":[{"name":"Hematology and Clinical Immunology Section, Center for Hemato-Oncology Research, Department of Medicine and Surgery, University of Perugia, 06132 Perugia, Italy"},{"name":"Hematology Division, \u2018Santa Maria Della Misericordia\u2019 Hospital, Azienda Ospedaliera di Perugia, 06156 Perugia, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Matteo","family":"Caridi","sequence":"additional","affiliation":[{"name":"UOSD Mieloproliferative, Policlinico Tor Vergata, 00133 Rome, Italy"},{"name":"Department of Physiology and Pharmacology, Sapienza University, 00185 Rome, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Rosanna","family":"Celenza","sequence":"additional","affiliation":[{"name":"Hematology and Clinical Immunology Section, Center for Hemato-Oncology Research, Department of Medicine and Surgery, University of Perugia, 06132 Perugia, Italy"},{"name":"Department of Pharmaceutical Services and Clinical Pathology, USL Umbria 2, 05100 Terni, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Francesco","family":"Millucci","sequence":"additional","affiliation":[{"name":"Hematology and Clinical Immunology Section, Center for Hemato-Oncology Research, Department of Medicine and Surgery, University of Perugia, 06132 Perugia, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Martina","family":"Crocioni","sequence":"additional","affiliation":[{"name":"Hematology and Clinical Immunology Section, Center for Hemato-Oncology Research, Department of Medicine and Surgery, University of Perugia, 06132 Perugia, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sofia","family":"Sciabolacci","sequence":"additional","affiliation":[{"name":"Hematology Division, \u2018Santa Maria Della Misericordia\u2019 Hospital, Azienda Ospedaliera di Perugia, 06156 Perugia, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Valeria","family":"Cardinali","sequence":"additional","affiliation":[{"name":"Hematology Division, \u2018Santa Maria Della Misericordia\u2019 Hospital, Azienda Ospedaliera di Perugia, 06156 Perugia, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-9139-1729","authenticated-orcid":false,"given":"Maria Paola","family":"Martelli","sequence":"additional","affiliation":[{"name":"Hematology and Clinical Immunology Section, Center for Hemato-Oncology Research, Department of Medicine and Surgery, University of Perugia, 06132 Perugia, Italy"},{"name":"Hematology Division, \u2018Santa Maria Della Misericordia\u2019 Hospital, Azienda Ospedaliera di Perugia, 06156 Perugia, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"1968","published-online":{"date-parts":[[2026,9,19]]},"reference":[{"key":"ref_1","doi-asserted-by":"crossref","first-page":"1200","DOI":"10.1182\/blood.2022015850","article-title":"International Consensus Classification of Myeloid Neoplasms and Acute Leukemias: Integrating Morphologic, Clinical, and Genomic Data","volume":"140","author":"Arber","year":"2022","journal-title":"Blood"},{"key":"ref_2","doi-asserted-by":"crossref","first-page":"1345","DOI":"10.1182\/blood.2022016867","article-title":"Diagnosis and Management of AML in Adults: 2022 Recommendations from an International Expert Panel on Behalf of the ELN","volume":"140","author":"Wei","year":"2022","journal-title":"Blood"},{"key":"ref_3","doi-asserted-by":"crossref","first-page":"254","DOI":"10.1056\/NEJMoa041974","article-title":"Cytoplasmic Nucleophosmin in Acute Myelogenous Leukemia with a Normal Karyotype","volume":"352","author":"Falini","year":"2005","journal-title":"N. 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