{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,2]],"date-time":"2026-10-02T00:02:09Z","timestamp":1790899329405,"version":"4.1.0"},"reference-count":85,"publisher":"MDPI AG","issue":"4","license":[{"start":{"date-parts":[[2023,2,12]],"date-time":"2023-02-12T00:00:00Z","timestamp":1676160000000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["Cancers"],"abstract":"<jats:p>Acute myeloid leukemia (AML) represents 80% of acute leukemia in adults and is characterized by clonal expansion of hematopoietic stem cells secondary to genomic mutations, rendering a selective growth advantage to the mutant clones. NPM1mut is found in around 30% of AML and clinically presents with leukocytosis, high blast percentage and extramedullary involvement. Considered as a \u201cgate-keeper\u201d mutation, NPM1mut appears to be a \u201cfirst hit\u201d in the process of leukemogenesis and development of overt leukemia. Commonly associated with other mutations (e.g., FLT 3, DNMT3A, TET2, SF3B1), NPM1 mutation in AML has an important role in diagnosis, prognosis, treatment and post-treatment monitoring. Several novel therapies targeting NPM1 are being developed in various clinical phases with demonstration of efficacy. In this review, we summarize the pathophysiology of the NPM1 gene mutation in AML, clinical implications and the novel targeted therapies to date.<\/jats:p>","DOI":"10.3390\/cancers15041177","type":"journal-article","created":{"date-parts":[[2023,2,13]],"date-time":"2023-02-13T01:48:56Z","timestamp":1676252936000},"page":"1177","update-policy":"https:\/\/doi.org\/10.3390\/mdpi_crossmark_policy","source":"Crossref","is-referenced-by-count":52,"title":["NPM 1 Mutations in AML\u2014The Landscape in 2023"],"prefix":"10.3390","volume":"15","author":[{"given":"Naman","family":"Sharma","sequence":"first","affiliation":[{"name":"Department of Hematology and Oncology, University of Massachusetts-Baystate Medical Center, Springfield, MA 01199, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jane L.","family":"Liesveld","sequence":"additional","affiliation":[{"name":"Department of Medicine, Hematology\/Oncology, James P. Wilmot Cancer Institute, University of Rochester, Rochester, NY 14642, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"1968","published-online":{"date-parts":[[2023,2,12]]},"reference":[{"key":"ref_1","doi-asserted-by":"crossref","first-page":"e441","DOI":"10.1038\/bcj.2016.50","article-title":"Acute myeloid leukemia: A comprehensive review and 2016 update","volume":"6","year":"2016","journal-title":"Blood Cancer J."},{"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 ELN Recommendations from an International Expert Panel","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. Engl. J. 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