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Expert Point of View: Jacqueline S. Garcia, MD


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Jacqueline S. Garcia, MD

Jacqueline S. Garcia, MD

Jacqueline S. Garcia, MD, of Dana-Farber Cancer Institute, who was not involved in this study, agreed that “novel therapies for relapsed/refractory AML that are biomarker-directed are a dire unmet need and may help patients avoid ineffective therapies and unnecessary toxicity.”

She noted that 36% of patients treated in the study had treatment-related grade 3 neutropenic fever, “comparable to historical [hypomethylating agent] monotherapy. A concerning expected nonhematologic toxicity was grade 3 hypertriglyceridemia, which was observed in 21% of patients and needs to be monitored. Of the 21 patients, 4 (19%) achieved an objective response, with a swift response in 1.4 months (range = 1–5.6 months),” she commented.

“A major highlight of this abstract is the availability of a treatment-directed biomarker with a rapid 2- to 3-day turnaround time. It is encouraging to see early activity with this novel agent, although a few questions remain on feasibility and response, some of which are challenging to answer given the small population assessed,” Dr. Garcia continued.

“The screen failure rate of patients who consented to the trial whose bone marrow did not demonstrate RARA overexpression was not reported. Was it more than expected? Do certain mutational subsets have more frequent RARA overexpression? Further delineation of the subset may help to enrich responses,” she said.

“Although the study included patients who were heavily pretreated [median of two prior therapies (range = 1–9)] including some with a transplantation history [21%], the rate of response among those with prior [hypomethylating agent] therapy was not robust [although one of the four responders had received [hypomethylating agent] /venetoclax prior to study entry]. Early discontinuation among the few responders should also be noted, although this is limited by the sample size,” Dr. Garcia noted. 

DISCLOSURE: Dr. Garcia has received institutional trial support from Pfizer, Genentech, AbbVie, Lilly, and SAB Biotherapeutics.


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