Myelodysplastic Syndrome

In conclusion, the addition of Quizartinib to intensive chemotherapy, delayed until chemotherapy course 2, prolonged OS in older patients with FLT3-mutated AML but did not improve OS in non-FLT3 selected patients. ISRCTN-31682779, EudraCR-2013-002730-21.

The risk of late-onset MDS/AML was low. These results can guide counseling on the duration of maintenance PARP inhibitors in patients with exceptional response and suggest that functional cure may be possible in patients with PS-ROC and exceptional response to PARP inhibitors.

Thematic analysis identified research hotspots across multiple dimensions, including 5q deletion, trisomy 8, germline predisposition, TP53 mutation, clonal hematopoiesis, inflammation, venetoclax, luspatercept, targeted therapy, immunotherapy, pediatric population, VEXAS syndrome, IPSS-R, relapse, prognosis, overall survival, and artificial intelligence. Future advances are expected to rely on multi-ethnic cohort building, mechanism exploration, combined treatment regimen optimization and novel drug development. Strengthening multidisciplinary collaboration is anticipated to facilitate clinical translation and foster more precise, equitable and clinically practical management of MDS globally.

P2, N=300, Recruiting, Masonic Cancer Center, University of Minnesota | Trial completion date: Jun 2027 --> Jun 2028 | Trial primary completion date: Jun 2026 --> Jun 2027

Transcriptomic profiling of CD34+ bone marrow cells revealed unique, homogeneous gene expression patterns, particularly within the AML-like, biTET2, SF3B1, and TP53-complex subgroups. Further integration of multi-omics data may refine MDS classification, improving clinical decision-making and guiding the development of targeted therapies.

P1, N=18, Suspended, National Cancer Institute (NCI) | N=30 --> 18

P1, N=15, Not yet recruiting, Regeneron Pharmaceuticals Inc.

P=N/A, N=1000, Not yet recruiting, The First Affiliated Hospital of Soochow University

P2, N=216, Recruiting, The First Affiliated Hospital of Soochow University | Not yet recruiting --> Recruiting

Although these findings required an urgent repeat marrow evaluation, cytogenetic/molecular testing, and hematology clearance due to the high risk of an indeterminate or pre-leukemic state, the presence of active, refractory gastrointestinal bleeding prompted the immediate initiation of infliximab induction...Severe systemic inflammation can create a reactive bone marrow phenotype indistinguishable from early myeloid neoplasia or myelodysplastic syndrome, posing a life-threatening diagnostic trap. Comprehensive hematologic evaluation-including repeat bone marrow assessment under controlled inflammatory conditions-should be considered mandatory before initiating biologic therapy in patients with CD and unexplained cytopenias or borderline blast counts.

Cases harboring biallelic TET2 inactivation or TET2+SRSF2 co-mutation show the highest bone marrow monocyte burden, frequent classical monocyte (MO1; CD14+/CD16-) elevation, and highest progression risk representing biologically true OM-CMML, whereas SF3B1-mutated and biallelic TP53 mutated cases show MDS-directed biology and warrant reclassification. This review synthesizes current diagnostic frameworks, molecular heterogeneity, risk stratification approaches, and evolving classification proposals, thereby providing a practical guide for pathologists navigating OM-CMML in the modern genomic era.

Overexpression of CD123 and CD86 in CBFB::MYH11 may have diagnostic and therapeutic relevance. The online version contains supplementary material available at 10.1007/s12288-025-02150-4.