Leukemia

P1/2, N=196, Recruiting, The First Affiliated Hospital of Soochow University | Active, not recruiting --> Recruiting | Trial completion date: Dec 2025 --> Dec 2027 | Trial primary completion date: Aug 2024 --> Aug 2027

P=N/A, N=650, Completed, Institut Paoli-Calmettes | Recruiting --> Completed

P3, N=40, Active, not recruiting, Fujian Medical University Union Hospital

In conclusion, our data support a role for VAP1 in driving fibroblast activation and cardiac fibrosis. Therefore, targeting VAP1 can be considered as a reasonable approach for the intervention of heart failure.

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.

In most dual-driver cases, one subtype gene expression signature predominated, consistent with oncogenic hierarchies, but also with the possibility of technical artifacts, which should prompt individual orthogonal validations. IntegrateALL provides an adaptable fully reproducible workflow for molecular B-ALL characterization by systematically integrating genomic drivers and downstream gene regulation.

Our study identified cellular and molecular biomarkers, notably CD56, that predict resistance to Rux + Ven, but further work is needed to understand and validate their effect in AML. This trial was registered at www.clinicaltrials.gov as #NCT03874052.

Azacitidine/venetoclax is the standard treatment for patients with acute myeloid leukemia (AML) unfit for intensive chemotherapy. The potentiated antileukemic activity positions cobicistat as a promising complementary agent in AML therapy. This trial was registered at www.clinicaltrials.gov as NCT06014489.

Aberrant expression of CD5 and CD23 is extremely rare. We present a case of classical HCL expressing both markers, along with a brief review of the literature emphasizing the diagnostic complexities associated with such aberrancy.

He was initially treated with azacitidine and venetoclax but demonstrated disease progression. In the setting of a KMT2A::ELL fusion, therapy was transitioned to the menin inhibitor revumenib, resulting in short-term clinical stability and tolerability under continued supportive care.

A 25-year-old woman with pre-existing Hashimoto's thyroiditis developed progressive bilateral nodular scleritis with anterior uveitis that proved completely refractory to high-dose corticosteroids, methotrexate, and conventional disease-modifying antirheumatic drugs, along with papilledema and granulomatous rosacea, suggesting systemic inflammation. Recognition of this molecular target enabled precision therapy with fostamatinib, a spleen tyrosine kinase (Syk) inhibitor, combined with adalimumab, a tumor necrosis factor-alpha (TNF-α) inhibitor, resulting in complete and sustained remission of all inflammatory manifestations over 18 months of follow-up. This case underscores the critical importance of maintaining high clinical suspicion for paraneoplastic autoimmune syndromes in patients with treatment-refractory inflammatory conditions, particularly when accompanied by atypical systemic or unexplained laboratory findings, and demonstrates that molecularly targeted precision medicine approaches can transform treatment outcomes in complex paraneoplastic rheumatic disorders.