Highlights from ASH 2016: Abstract 100 – A Phase II Study of Pracinostat and Azacitidine in Elderly Patients with AML Not Eligible for Induction Chemotherapy: Response and Long-Term Survival Benefit

On Saturday 3^rd December, at the 58th Annual Meeting & Exposition of the American Society of Hematology (ASH) in San Diego, CA, there was a gripping session focusing on “Acute Myeloid Leukemia: Novel Therapy, excluding Transplantation.”

Guillermo Garcia-Manero, MD, of the Department of Leukemia at The University of Texas M.D. Anderson Cancer Center, Houston, USA, presented the key findings from a phase II study of pracinostat and azacitidine. Pracinostat is a potent oral Class I, II, IV Histone Deacetylase (HDAC) inhibitor and azacitidine (AZA) is a novel hypomethylating agent. Garcia-Manero et al. investigated the combination of these agents in 50 older patients (median age 75 years) unable to undergo induction chemotherapy.

The findings were:

• Complete Response (CR) was achieved in 21 patients
• CR with incomplete blood count recovery (CRi) in 2 patients
• Morphologic Leukemia Free status (MLFs) in 3 patients
• cCR rate of 52%
• Median duration of cCR was 13.2 months

• Median Overall Survival (OS) was 19.1 months, 1-year and 2-year OS were 62% and 41%, respectively

•  OS by cytogenetic risk group, high vs intermediate; 13.5 months vs 24.1 months

• OS in de novo vs secondary AML, 13.0 months vs 19.6 months

•  OS in ≥ 75 years vs < 75 years; 13.5 months vs 22.8 months 

• OS by ECOG Performance Status (PS), PS 0–1 vs PS 2; 19.1 months vs 13.0 months

• Thrombocytopenia was the most common grade ≥ 3 hematologic adverse event (46%)

The authors concluded that pracinostat + AZA led to a high rate of response in elderly patients with AML. Furthermore, the responses were durable and observed irrespective of age, cytogenetic risk, ECOG performance status, and de novo or secondary AML.