QUAZAR AML-001
QUAZAR AML-001 showed that a tablet form of azacitidine taken as maintenance after chemotherapy helped older people with acute myeloid leukaemia live about ten months longer than placebo, and it became the first approved maintenance treatment for the disease.
Overview
QUAZAR AML-001 was a double-blind, placebo-controlled phase 3 trial in 472 patients aged 55 and over with acute myeloid leukaemia in first complete remission (with or without count recovery) after induction with or without consolidation, who were not candidates for stem-cell transplant. Patients were randomised to oral azacitidine 300 mg or placebo once daily for 14 days per 28-day cycle. The primary endpoint was overall survival.
Median overall survival was 24.7 months with oral azacitidine against 14.8 months with placebo, and relapse-free survival was also longer. Side effects were mainly gastrointestinal symptoms and neutropenia, and quality-of-life scores were maintained. Oral azacitidine (Onureg) was approved in the United States in September 2020 for continued treatment of adults with acute myeloid leukaemia in first remission who cannot complete intensive curative therapy.
- Median 24.7 vs 14.8 months with Oral azacitidine + best supportive care compared with Placebo + best supportive care; about 9.9 months longer for half the group.
- A median is a midpoint: half the people did better than this and half did worse, so it is not a prediction for any one person.
- Put another way, the treated group had about 31 percent lower chance of the event at any given time (hazard ratio 0.69, likely range 0.55 to 0.86).
- Overall survival counts deaths from any cause, so it is the most direct measure of whether a treatment helps people live longer.
- Median 10.2 vs 4.8 months with Oral azacitidine + best supportive care compared with Placebo + best supportive care; about 5.4 months longer for half the group.
- A median is a midpoint: half the people did better than this and half did worse, so it is not a prediction for any one person.
- Put another way, the treated group had about 35 percent lower chance of the event at any given time (hazard ratio 0.65, likely range 0.52 to 0.8).
- This is a surrogate endpoint: it measures the cancer being controlled or absent on scans and tests, which often, but not always, translates into living longer.
- These results apply to the people the trial enrolled: Acute myeloid leukaemia in first remission after intensive chemotherapy in patients aged 55 or older who were not going to transplant: oral azacitidine (CC-486) or placebo for 14 days of each 28-day cycle as maintenance. People in a different situation may not see the same effect.
Numbers are from the trial as recorded here; see the source links in the table below. This is orientation, not medical advice: ask your team how closely the trial population matches you.
472 enrolled.
95% CI 18.7 to 30.5; median follow-up 41.2 months · 95% CI 11.7 to 17.6
Source| Endpoint | Arm | n | Value | HR (95% CI) | p | Source |
|---|---|---|---|---|---|---|
| Overall survivalprimary | Oral azacitidine + best supportive care | 238 | 24.7 months | 0.69 (0.55 to 0.86) | 0.0009 | link |
| Placebo + best supportive care | 234 | 14.8 months | ||||
| Relapse-free survival (registry, August 2024 cut-off) | Oral azacitidine + best supportive care | - | 10.2 months | 0.65 (0.52 to 0.8) | <0.0001 | link |
| Placebo + best supportive care | - | 4.8 months |
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