IDH2 mutation

This case highlights the potential clinical relevance of IDH-directed therapy in IDH2-mutant cholangiocarcinoma and demonstrates feasibility in an immunosuppressed post-transplant setting. These findings are hypothesis-generating and support further evaluation of IDH inhibition in this population.

Compared to supratentorial tumors, non-R132H IDH1 mutations are significantly more frequent in infratentorial tumors. IDH2-mutant gliomas almost exclusively occur in adults and in supratentorial locations, with a significantly higher proportion in oligodendrogliomas than astrocytoma.

P1, N=24, Not yet recruiting, Megan Mantica

P3, N=57, Active, not recruiting, Servier | Trial primary completion date: Oct 2025 --> May 2026

This case establishes the shared clonal origin between AITL and BL arising in the setting of CH and provides additional biological insight into the development of B-cell lymphoma associated with AITL.

This review consolidates current evidence on candidate biomarkers and therapeutic targets for ONB, highlighting the vital role of omics-based approaches in elucidating its molecular mechanisms. To advance the field, future research should focus on standardizing methods, validating findings in larger, more diverse groups, and integrating multi-omics techniques with AI-driven analyses to enhance diagnostic precision and develop targeted treatments.

This included impaired expression of Cebpe, which encodes a key transcription factor regulating neutrophil differentiation. Reactivation of Cebpe expression, by overexpression of its upstream regulator Cebpa or following treatment with hypomethylating agents restored differentiation, indicating that the differentiation block is reversible.In summary, we found a reversible, pre-leukemic impairment of neutrophil differentiation in IDH1-mutant hematopoiesis that correlates with elevated IDH1 expression in myeloid progenitors and likely explains the strong association of IDH1 mutations with myeloid neoplasms.

Not available.

Given the role of IDH1 and IDH2 mutations in leukemic transformation, targeted therapies with IDH1 or IDH2 inhibitors are another option for selected patients. Current studies are investigating the role of next-generation Janus Kinase (JAK) inhibition, inhibition of epigenetic modulators, and blockade of anti-apoptotic pathways as paradigm shifts to make MPN-AP/BP a more manageable disease.

Standard of care (SOC) uses 5-day decitabine (Dec-5) or 7-day azacitidine (Aza-7), while extended HMA regimens (e.g., 10-day decitabine [Dec-10] during cycle 1) have also been explored, although direct comparative data between these approaches are limited. Our findings suggest that Dec-10/Ven may lead to improved transplant rates and survival, providing valuable real-world support for this intensified approach in appropriate patients. Prospective randomized evaluation is warranted.

IDH1 mutations were associated with global transcriptional repression and evidence of metabolic and epigenetic reprogramming. These results depict IDH-mutant prostate cancer as a subtype that can present with high tumor stage and grade, but is associated with favorable outcomes.

P1, N=40, Active, not recruiting, National Cancer Institute (NCI) | Trial completion date: May 2026 --> Sep 2026 | Trial primary completion date: May 2026 --> Sep 2026