IDH1 (Isocitrate dehydrogenase (NADP(+)) 1)

P1, N=94, Recruiting, Mayo Clinic | Trial completion date: Feb 2029 --> May 2029 | Trial primary completion date: Feb 2028 --> May 2028

Real-world data from our cohort and the TriNetX database support the oncological outcomes reported in the ClarIDHy trial and other real-world cohorts.

We validated these clinical and molecular findings in an independent validation cohort of 302 NPM1 mut patients enrolled in the acute myeloid leukemia study group (AMLSG) 09-09 clinical trial, which included the administration of all-trans retinoic acid (ATRA) to all patients and a randomization for gemtuzumab ozogamicin. In this cohort, the APL-like immunophenotype was associated with events occurring within the first 15 days but did not influence mortality, likely due to protocol-driven patient selection. Our findings have important clinical implications that warrant the development of studies exploring disease-tailored clinical measures to mitigate the risk of early vascular events, as in current APL management.

Unlike HCC, roughly 45% of iCCA harbor alterations amenable to precision oncology approaches, including fibroblast growth factor receptor 2 (FGFR2) fusions, isocitrate dehydrogenase 1 (IDH1) mutations, ERBB2 alterations, and BRAF mutations. In this review, we explore how this framework has reshaped liver cancer care and discuss the resulting breakthroughs in management and emerging directions that may further improve therapeutic strategies.

Multivariable and LASSO analyses identified independent risk factors: male gender (OR=3.28, p = 0.025), higher blast percentage (OR=1.03, p = 0.006), elevated baseline uric acid (OR=1.01, p = 0.011), IDH1/2 mutation (OR=4.86, p = 0.005), and a trend with venetoclax-based therapy (OR=7.52, p = 0.072)...Adequate urine output reduced TLS risk, while excessive positive fluid balance correlated with severe TLS (p = 0.046). Individualized fluid management and 72-hour intensive monitoring are critical for high-risk groups.

The striking reductions in tumor 2HG and tCho levels early following initiation of targeted therapy using an IDH inhibitor suggest that MRS may provide an important tool for monitoring treatment response of lower-grade gliomas.

Key advancements include the integration of FLT3 inhibitors into intensive chemotherapy and the emergence of venetoclax...Innovations such as the liposomal formulation CPX-351 and oral formulations of CC-486 and oral decitabine/cedazuridine have further optimized treatment delivery and improved patient quality of life...Current research is actively exploring next-generation inhibitors, antibody-drug conjugates, and cellular immunotherapies such as BiTEs and CAR-T cells. This review summarizes the recent pharmacological evolution in AML and discusses how these emerging therapies bring us closer to the ultimate goal of achieving a definitive cure.

In this hospital-based cross-sectional study, IDH1 (R132H) IHC-negative status was more frequently observed in higher-grade tumors. These findings support the use of immunohistochemistry as an accessible adjunct in glioma diagnosis and clinicopathological stratification; outcome-based prognostic significance requires longitudinal follow-up.

P1, N=46, Active, not recruiting, Yale University | Trial primary completion date: Jul 2026 --> Mar 2026

Accurate distinction between distal CCA and pancreatic head cancer has become increasingly important, particularly in unresectable or borderline resectable cases, as systemic treatment strategies differ between these entities. Although these two neoplasms share many morphological and immunohistochemical features, the presence of clear or foamy cancer cells in biopsy specimens is uncommon in dCCA and may favour pancreatic ductal carcinoma.

Notably, the oncogenic idh1*R132H variant also rescued the pronephric defects induced by idh1 knockdown, indicating that its neomorphic activity is absent under embryonic metabolic conditions. These findings identify Idh1 and Idh2 as key regulators of pronephric morphogenesis and reveal a developmental function of Idh1 that is distinct from its canonical catalytic role.

KRAS G12C-mutated NSCLC is clinically aggressive and frequently shows solid growth with rhabdoid, plasmacytoid, or SCC-like morphology, which may lead to misclassification and missed genetic testing. Immunohistochemistry and molecular profiling are essential for accurate classification and enabling targeted therapy.