azacitidine

P=N/A, N=30, Not yet recruiting, Sun Yat-sen University

We also demonstrated that, compared with that with each drug used as single agents, the combined 5-aza and HDAC3i treatment induced the epigenetic remodeling of DLBCL cells, which resulted in a more potent reexpression of differentiation genes, including XBP1 and ATF4. Our results highlight the importance of specifically targeting multiple layers of the epigenome to maximize the efficacy of epigenetic-based therapies.

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.

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.

P3, N=788, Not yet recruiting, Institute of Hematology & Blood Diseases Hospital, China

Given the patient's advanced age and underlying MF, two cycles of a low-intensity chemotherapy regimen primarily based on the "VP regimen (Vincristine + Prednisone) combined with Azacitidine" were administered...Patient tolerability to intensive chemotherapy and novel targeted agents (e.g., Venetoclax) is poor, leading to a dismal prognosis...This case not only serves as a unique model illustrating the complex evolution of a malignant clone but also profoundly reveals the unique therapeutic challenges and extremely poor survival outcome resulting from the convergence of advanced age, MF background, and MPAL transformation. It offers pivotal real-world evidence for the clinical management of this specific patient population and highlights the need to explore novel therapeutic strategies.

This case may preliminarily suggest ATRA's potential in AML1-MTG16⁺ AML, offering translational clues for ultra-rare leukemia subtypes, pending further validation.

After eight years and treatment with lenalidomide with excellent clinical response, she developed progressive cytopenias and transformation to acute myeloid leukemia, myelodysplasia-related (AML-MR)...The patient was treated with combination azacitidine and venetoclax and an investigational immunotherapy within a clinical trial...Our findings expand the spectrum of dmin-associated oncogenic amplifications in myeloid neoplasms and highlight FLI1 and ETS1 as recurrent targets of 11q24-derived ecDNA amplification. Recognition of such rare events underscores the importance of integrative cytogenomic profiling for uncovering novel mechanisms of leukemic transformation and potential therapeutic targets.

P2, N=52, Not yet recruiting, BioLite, Inc. | Trial completion date: Jun 2028 --> Dec 2028 | Trial primary completion date: Dec 2027 --> Dec 2028

The CLL-1 targeted NPs loaded with MDP5 and AZA demonstrated superior AML control and targeting of LSCs in TP53-mutant mice models, while sparing normal hematopoiesis in healthy NSG mice. These promising results highlight a potential efficacy of our novel CLL-1 targeted NP combination approach to treat AML, particularly those harboring TP53 mutation.

P1, N=12, Not yet recruiting, Centre Hospitalier Universitaire De Nantes

The patient underwent induction chemotherapy with cytarabine and mitoxantrone, followed by salvage therapy with venetoclax and azacitidine, resulting in morphologic remission. Subsequent haploidentical hematopoietic stem cell transplantation achieved remission, with ongoing hematologic recovery. This case underscores the limitations of conventional molecular assays in detecting cryptic fusions and highlights the critical role of comprehensive genomic profiling in refining subclassification and optimizing therapeutic strategies in pediatric AML.