Summary
Health-related quality-of-life (HRQoL) scores in patients treated with CC-486 were about the same as those given a placebo in the phase III QUAZAR AML-001 clinical trial, findings of which were reported at the 2020 ASCO Annual Meeting (Abstract 7533).
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Original Article
Positive Quality-of-Life Data Reported With CC-486 in AML Patients
Oncology Times
By Kurt Samson
Maintenance therapy with the experimental oral hypomethylating agent CC-486 resulted in very little change in health-related quality-of-life scores in older patients successfully treated for acute myeloid leukemia.
Health-related quality-of-life (HRQoL) scores in patients treated with CC-486 were about the same as those given a placebo in the phase III QUAZAR AML-001 clinical trial, findings of which were reported at the 2020 ASCO Annual Meeting (Abstract 7533).
A number of positive analyses from the trial were presented at the conference, including data showing better overall and relapse-free survival with treatment, as well as relatively low occurrences of adverse events. CC-486 appears to be both safe and well-tolerated as a maintenance therapy for older patients with acute myeloid leukemia (AML) in remission, as it is in younger persons, trial investigators reported.
On May 1, the FDA granted priority review for CC-486 as a maintenance treatment for adult AML patients in complete remission but incomplete blood count recovery (CRi) following induction therapy, with or without consolidation treatment, and who are not candidates for, or who choose not to proceed to, hematopoietic stem cell transplantation.
“I’ve been working on QUAZAR for over a decade and am thrilled to finally see all the work of this huge endeavor come to fruition,” said investigator Gail J. Roboz, MD, Professor and Director of Clinical and Translational Leukemia Programs at Weill Cornell Medicine and NewYork Presbyterian Hospital.
“The data show a clear prolongation of both [overall survival (OS) and relapse-free survival (RFS)] for AML patients who received CC-486 and this is the first time that a benefit in OS has been shown for AML maintenance. We anticipate that treatment with CC-486 will become part of the standard of care for selected AML patients,” she told Oncology Times.
The study included patients 55 years of age or older with intermediate- or poor-risk cytogenetics, ECOG scores of 3 or lower, and in CR/CRi after induction chemotherapy consolidation. Subjects were randomized to receive either 300 mg CC-486 or placebo, and health-related quality of life (HRQoL) was assessed during the first 2 weeks of each 28-day treatment cycle.
The researchers evaluated patients using the FACIT-Fatigue scale and the EQ-5D-3L health utility index on the first day of each cycle, then again at the end of treatment. Differences in mean changes and/proportions of patients with clinically meaningful changes from baseline were assessed, including improvement, no change, or deterioration.
All of the patients evaluated by the team had an HRQoL assessment at baseline and again once afterwards.
Out of 238 patients, 225 in the CC-486 arm and 219 out of 234 patients given placebo were evaluated, and patient characteristics were comparable between both groups. Most patients were between the ages of 65 and 74 years, and the median number of CC-486 cycles was 12, compared to 7 in the placebo arm.
At baseline, patients on both groups reported comparable low levels of fatigue and generally good HRQoL when compared to the general population. There were no meaningful differences between the CC-486 and placebo arms in mean changes from baseline in fatigue or EQ-5D-3L scores at any post-baseline visit.
“We found HRQoL and low levels of fatigue were preserved with CC-486 maintenance therapy, while CC-486 significantly improved overall and relapse-free survival,” Roboz noted.
The researchers also found no statistically significant difference between the treatment arms in proportion of patients with a clinically meaningful deterioration in FACIT-Fatigue scores at any post-baseline visit except at cycle 29. However, this was likely due to chance, said Roboz, who noted that no adjustment was made for multiple testing at any visit.
Median time to deterioration was not significantly different between CC-486 and placebo on the FACIT-Fatigue scale at 41 weeks and 44 weeks, respectively.
The data show that this therapy can be safely given in an ongoing manner, including much older patients, without compromising quality of life, Roboz said.
“This is extremely important—after fighting hard to get into remission, we of course want the longest possible survival for our AML patients, but we also want them to be able to enjoy their time in remission and not have to endure difficult side effects from post-remission treatment.”
Kurt Samson is a contributing writer.