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Expert Point of View: Steven D. Gore, MD, and Rami Komrokji, MD



Survival in previously untreated patients seems to be inferior to what we achieved with azacitidine and decitabine in the registration trials. Is this drug [guadecitabine] any different from azacitidine?
— Steven D. Gore, MD

Steven D. Gore, MD, Director of Hematologic Malignancies at Yale School of Medicine, New Haven, Connecticut, applauded Dr. Stein for “accruing a terrible patient population, mutation-wise” and called the preliminary findings for the benefit of enasidenib in patients with ASXL1 mutations “exciting.” He also encouraged the wider research community to rethink the meaning of marrow complete remission, which both studies reported.

“In our multivariate analysis of the AZA-001 data, we showed that stable disease on azacitidine was not associated with improved overall survival,” revealed Dr. Gore. “Right now, we don’t have data that marrow complete remission is a meaningful outcome. It’s good only for activity-finding, in my opinion.”

Questions Remain

For the guadecitabine study, Dr. Gore questioned whether the outcomes were much better than those achieved with current hypomethylating agents. “I am concerned that survival in previously untreated patients seems to be inferior to what we achieved with azacitidine and decitabine in the registration trials. Is this drug any different from azacitidine?” he asked.

Dr. Montalban-Bravo pointed out that the median follow-up of only 6 months does not allow for a definitive overall survival assessment. “Also, based on the fact that these patients were all high risk, their expected overall survival may be shorter than we’ve seen with previous experience. We can’t draw definite conclusions yet,” he responded.

Additional Comments

Rami Komrokji, MD

Rami Komrokji, MD

Rami Komrokji, MD, Clinical Director of the Malignant Hematology Department at Moffitt Cancer Center, Tampa, added to the conversation by noting that in the AZA-001 study data, poor-prognosis patients had a median overall survival of approximately 15 to 17 months. “In our real-life experience, no one has been able to duplicate the 24-month overall survival with azacitidine,” he admitted. “This study population is very high risk, with P53 mutations and so forth, and so I would not be discouraged by these outcomes.” ■

Disclosure: Dr. Gore has received research funding from Celgene and Genentech/Roche and is a consultant for Celgene.


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