STAT3 mutation

Our findings support the role of immune dysregulation and genetic predisposition in L-HES and underscore the importance of broader genomic profiling in familial cases. Functional validation and long-term monitoring are essential for risk stratification and early detection of malignant transformation.

This study provides a comprehensive genomic profile of T-LGLL, identifying recurrent somatic mutations and commonly affected pathways. Notably, frequent alterations were observed in the FAS-FASL signaling pathway, underscoring its potential as a target for therapeutic development.

Selinexor and azacitidine offer a promising strategy to overcome therapeutic resistance and improve outcomes in TET2/RHOA-mutated PTCL, supporting further clinical evaluation.

This work provides the first genomic characterisation of a diagnosed MDS cohort in China and establishes the first risk prediction model for MDS-to-AML transformation, alongside novel prognostic models for OS and PFS. These tools offer improved prognostic prediction and potential guidance for therapeutic strategies in Chinese patients with MDS.

A significant correlation between microbiome abundance and VEGF and MMP9 was observed. This study illustrated that gut microbiomes contribute to HCC and HCV-related cirrhosis pathogenesis, opening approaches for cancer management and prevention.

This study enhances our understanding of how STAT3 mutations drive immunological dysregulation in HIES. The identified changes in immunological signature and transcriptional mechanisms offer new insights into therapeutic targets for HIES.

P1/2, N=11, Active, not recruiting, Jonathan Brammer | Recruiting --> Active, not recruiting | N=27 --> 11

Altogether, incorporation of CCL22 mutations reduced the fraction of unclassified patients, improved diagnostic sensitivity without compromising specificity, and may decrease reliance on invasive procedures. These revised international criteria represent a step toward standardized, molecularly guided NK-LGLL diagnosis.

Moreover, RA treatments such as methotrexate (MTX), Janus kinase (JAK) inhibitors, and anti-TNF therapies have complex implications, with some studies suggesting potential contributions to leukemia risk through immune suppression and hematopoietic alterations...Advances in multiomics and artificial intelligence-driven risk modeling may facilitate personalized treatment strategies, improving both RA management and leukemia prevention. Given the rising burden of RA and its associated complications, a comprehensive understanding of its link to leukemias is critical for enhancing patient outcomes and guiding clinical decision-making.

We evaluate clinical progression of breakthrough candidates such as ARV-110 for prostate cancer, ARV-471 for breast cancer, and BTK degraders, while discussing critical challenges including the "hook effect" and oral bioavailability limitations. This review provides essential foundations for rational target selection, molecular optimization, and clinical translation strategies. By integrating mechanistic insights with clinical realities, this analysis offers perspectives on PROTAC technology advancement and identifies opportunities for transforming treatment of complex diseases resistant to conventional therapies.

Underlying T-cell dysregulations, often associated with clonality and/or STAT3 gene mutation, have been a rationale for using cyclosporin and other directed immunosuppressive therapies in most of the disease subtypes, namely, thymoma-associated PRCA, large granular lymphocytic leukemia-associated PRCA, and idiopathic PRCA. Although the epidemiologic rarity of PRCA has precluded comparative clinical trials that could demonstrate the superiority of one immunosuppressive agent over another, some retrospective studies have demonstrated the significance of maintenance therapies to avoid blood transfusion dependency, a factor that may lead to poorer prognosis in patients with PRCA. This review primarily discusses clinical aspects of PRCA in line with recently updated clinical guidelines.

Despite receiving various treatments such as methotrexate, cyclosporin, cyclophosphamide, and thalidomide, the patient exhibited treatment refractoriness. This case highlights the clinical relevance of golidocitinib in developing novel therapeutic strategies for NK-LGLL. Moreover, this treatment option presents the potential as either a bridging therapy or alternative to allo-HSCT.