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http://dx.doi.org/10.1111/bjh.17199 | DOI Listing |
Background: Nucleophosmin 1 (NPM1) mutations represent one of the most frequent genetic alterations in acute myeloid leukemia (AML). However, the prognostic significance of concurrent molecular abnormalities and clinical features in NPM1-mutated AML remains to be fully elucidated.
Methods: We retrospectively analyzed 73 adult AML patients with NPM1 mutations.
Ther Adv Hematol
September 2025
Department of Hematology, The Affiliated Hospital of Qingdao University, 16 Jiangsu Road, Qingdao, Shandong 266000, China.
Myelodysplastic syndromes (MDS), particularly in older adults aged 60 years and above, present significant therapeutic challenges due to poor prognosis and limited treatment options. Higher-risk MDS (HR-MDS), defined by the Revised International Prognostic Scoring System score of ⩾3.5, is characterized by increased myeloblasts, severe cytopenia, and a median survival of <2 years.
View Article and Find Full Text PDFClin Cancer Res
September 2025
United States Food and Drug Administration, Silver Spring, Maryland, United States.
On August 6, 2024, the U.S. Food and Drug Administration (FDA) granted traditional approval to vorasidenib (VORANIGO, Servier Pharmaceuticals, LLC) for the treatment of adult and pediatric patients 12 years and older with Grade 2 astrocytoma or oligodendroglioma with a susceptible isocitrate dehydrogenase-1 or 2 (IDH1 or IDH2) mutation following surgery including biopsy, sub-total resection, or gross total resection.
View Article and Find Full Text PDFBr J Haematol
September 2025
Laboratory of Hematology, Biology and Pathology Center, CHU Lille, Lille, France.
Measurable residual disease (MRD) is a strong prognostic factor in acute myeloid leukaemia (AML). Next-generation sequencing (NGS) offers promise but must distinguish true signal from background. We assessed MRD in 98 adult AML patients in first complete remission after intensive chemotherapy using a duplex unique molecular identifier (UMI)-based NGS capture panel.
View Article and Find Full Text PDFmedRxiv
August 2025
Department of Neurosurgery, Massachusetts General Hospital, Harvard Medical School, Boston, MA, USA.
Gliomas are biologically heterogeneous brain tumors with marked differences in clinical behavior based on the IDH1 mutation status. While epigenetic dysregulation is well characterized, the contribution of RNA modifications, particularly N6-methyladenosine (m6A), remains underexplored. Using direct RNA nanopore sequencing of patient-derived gliomas, we generated the first isoform-resolved m6A maps across IDH1-mutant and wild-type tumors.
View Article and Find Full Text PDF