At the 65th American Society of Hematology (ASH) Annual Meeting in San Diego, California, Sylvain Garciaz, MD, Institut Paoli-Calmettes, Marseille, France, shared insights on the potential benefit of stopping venetoclax and/or azacitidine treatment for responding patients with acute myeloid leukemia, which was associated with sustained responses and long-term survival among patients with AML. 

Dr Garciaz noted that results from a FILO study indicated that resuming these treatments was associated with a second remission in some patients. 

Transcript:

Hello, I'm Sylvain Garciaz. I'm a hematologist in Marseille at Institut Paoli-Calmettes in France. I'm here at the ASH meeting because I presented the data from a retrospective study of patients who stopped [venetoclax-azacitidine] (ven-aza) treatment, not because of progression, but because of poor tolerance. This is [regarding] AML patients. 

The question is [whether] venetoclax-azacitidine is associated with a good response rate, sometimes associated with long-term survival. In approximately 25% of case[s] the patients survive more than 3 years with [minimal residual disease] (MRD) negative[ity]. As they are older, we can reasonably ask whether we can stop or not the treatment to improve quality of life in this population. So, as we don't have data in this setting, we decided to analyze the patients who had to stop treatment because of poor tolerance in order to see if we observe a rapid relapse or if they remain in remission for a long time. 

We included 62 patients with newly diagnosed AML and 22 patients with relapsed/refractory AML collected in 2 fellow centers in France and one center in the US Moffitt Center. In the first cohort of 62 patients with newly diagnosed AML, we observed that, as they were 75-year-old patients mainly with good cytogenetics profile and good molecular risk, including [isocitrate dehydrogenase] (IDH) mutation, they received 4 cycles of ven-aza [as the] median, and they had to stop because of hematological toxicity for most of them. 

This first group of patients received 4 cycles of ven-aza before stopping, in median. Interestingly, most of them remain in remission, and the time without treatment was longer than the time with treatment or with disease recurrence for 75% of them. The other point is approximately 25% of the deaths were, in fact, non-relapse mortalit[ies]. 

Overall, [the] median treatment-free remission for these patients [who] had to stop ven-aza was 16 months and overall survival was 44 months. That means that at some point in this population of patients with a good response---some of them a negative MRD--- you can stop [ven-aza] and these patients remain in remission for a long time. 

It was not exactly the same with [the] relapsed/refractory cohort of patients---they remain[ed] in remission for approximately 10 months. But, overall, we are planning now to do a prospective study in order to see whether the patient in remission can stop ven-aza treatment. This study will enroll patients who have received 1 year of ven-aza who are in remission with negative MRD, and we will stop ven[etoclax] and aza[citadine] treatment and see what's going on during follow-up.

Source:

Garciaz S, Bertoli S, Sallman DA, et al. Acute myeloid leukemia patients who stopped venetoclax or/and azacytidine for other reasons than progression have a prolonged treatment free remission and overall survival. a Filo study. Presented at the ASH 65th Annual Meeting & Exposition; December 9-12 2023; San Diego, California. Abstract 161