azacitidine

P2, N=400, Not yet recruiting, Australasian Leukaemia and Lymphoma Group (ALLG) | Phase classification: P --> P2 | N=50 --> 400

This review compared the efficacy and safety of low-dose cytarabine (LDAC), azacitidine (AZA), 5- and 10-day decitabine (DEC), and gemtuzumab ozogamicin, alone or combined with drugs such as venetoclax (VEN), in older adults with AML ineligible for conventional chemotherapy. Treatment decisions should consider patient goals and functional status. These findings informed eight recommendations in updated ASH-AML guidelines.

Among the combination regimens tested, cytarabine or hypomethylating agents (HMA) drive strong blast reduction in patient samples previously exposed to venetoclax and yield improved survival in AML patient-derived xenograft models with prior venetoclax/5-azacytidine treatment. These preclinical findings support the clinical evaluation of dual BCL-2/BCL-XL inhibition in AML patients, particularly, those who do not respond to venetoclax.

Initial treatment began with azacitidine, a hypomethylating agent...However, there is no consensus on its management, and the role of systemic chemotherapy remains debated. Continuous monitoring for progression to acute myeloid leukemia (AML) is crucial, as early detection significantly impacts prognosis and informs treatment decisions.

P=N/A, N=20, Active, not recruiting, Hikma Pharmaceuticals LLC | Not yet recruiting --> Active, not recruiting

P2, N=147, Recruiting, National Cancer Institute (NCI) | Trial completion date: Oct 2025 --> May 2029 | Trial primary completion date: Oct 2025 --> May 2029

P1/2, N=34, Active, not recruiting, M.D. Anderson Cancer Center | Trial completion date: Dec 2025 --> Mar 2026 | Trial primary completion date: Dec 2025 --> Mar 2026

The 15-day venetoclax plus azacitidine regimen demonstrated high efficacy and manageable toxicity in treatment-naïve HR-MDS. It may be particularly beneficial for patients with high neutrophil counts, adverse cytogenetics, or those eligible for HSCT, supporting further investigation in larger trials.

However, the need for longer follow-up and expanded cohorts remains critical to validate these outcomes and optimize patient selection. These findings support the further exploration of venetoclax and azacitidine as a viable treatment option for patients with refractory or high-risk multiple myeloma, warranting additional prospective trials before consideration for standard-of-care implementation.

P2, N=28, Recruiting, University of California, Davis | Not yet recruiting --> Recruiting

These findings indicate that DNMT1 and DNMT3A drive hepatocellular carcinoma through epigenetic regulation and are viable prognostic biomarkers. Targeting these enzymes with 5-azacytidine offers a promising therapeutic strategy for hepatocellular carcinoma management.

P=N/A, N=200, Not yet recruiting, Peking University People's Hospital