A phase 2 randomised trial published by the New England Journal of Medicine found that azacitidine combined with venetoclax produced longer event-free survival than standard induction chemotherapy in patients with acute myeloid leukaemia who were eligible for intensive treatment.
The New England Journal of Medicine has posted findings from a trial known as the PARADIGM trial, which tested whether a two-drug combination — azacitidine (a drug that affects how genes are switched on in cancer cells) and venetoclax (a drug that triggers cancer cell death) — could outperform the intensive chemotherapy that has been the standard starting treatment for acute myeloid leukaemia (AML), a fast-moving cancer of the blood and bone marrow, for decades.
The trial was randomised and conducted at phase 2 level, meaning it was designed to test whether the treatment works well enough to justify larger studies, rather than to confirm final approval for widespread use. Patients enrolled were classed as induction-eligible — that is, fit enough to receive intensive chemotherapy — making the comparison directly relevant to a group who would ordinarily receive the standard treatment.
Event-free survival — the length of time a patient lives without their disease worsening, returning, or requiring a change in treatment — was much longer in the azacitidine–venetoclax group, according to the journal’s summary of the findings.
The NHS does not currently list azacitidine–venetoclax as a standard first-line treatment for induction-eligible AML patients in England. Decisions on NHS availability are made by the National Institute for Health and Care Excellence (NICE) after reviewing trial data, cost-effectiveness, and patient outcomes. Phase 2 results typically feed into that process but do not automatically change prescribing practice.
Patients with AML or their families who have questions about current treatment options should speak to their haematology team or contact NHS 111 for guidance on accessing specialist advice.
Key information
- AML is a cancer of the blood and bone marrow that progresses quickly and requires prompt treatment
- This trial was phase 2 — an earlier stage of testing; it does not mean the combination is yet available on the NHS as a standard first-line option
- Patients with questions about their own treatment should contact their specialist team or call NHS 111
Source: @NEJM