NICE approves arsenic trioxide for use in acute promyelocytic leukaemia (APL) treatment

NICE have today (Friday 4 May) recommended the use of arsenic trioxide in the treatment of acute promyelocytic leukaemia (APL).

NICE have today (Friday 4 May) recommended the use of arsenic trioxide in the treatment of acute promyelocytic leukaemia (APL).

Brand name Trisenox® (produced by Teva), Arsenic Tioxide is recommended for use in adult APL patients who have:

  • Untreated, low-to-intermediate risk disease in combination with ATRA (all-trans-retinoic acid)


  • Relapsed or refractory disease, after ATRA and chemotherapy

Current therapy for untreated, low-to-intermediate risk APL involves ATRA plus chemotherapy, known as AIDA. ATRA is laboratory-made like the natural product made in the body from vitamin A. It inhibits the activity of the PML-RARA gene that defines an APL cell.

When arsenic trioxide plus ATRA treatment was compared to AIDA treatment in clinical trials, there were significant improvements in long term outcomes for previously untreated patients. In one trial, patients were followed up for 50 months (5 years). After this time, 97.5% of patients had event-free survival after treatment with arsenic trioxide plus ATRA. This means patients achieved, and sustained, remission.

Arsenic trioxide has been used in the treatment of relapsed or refractory APL patients for over 10 years. It is expected that using it for untreated, low-to-intermediate risk patients there will be a significant reduction in the number of patients relapsing or having refractory disease (does not respond to treatment).

There are also benefits of removing chemotherapy from treatment, which has risks of secondary cancers and loss of fertility treatment in younger people. In trials, there were generally fewer adverse events using arsenic trioxide, however, the European Medicines Agency has previously recommended that long-term safety studies should be explored.

The recommendation from NICE approving the use of arsenic trioxide for untreated, low-to-intermediate risk patients is, however, a welcome alternative to chemotherapy that could ultimately improve quality of life by reducing the risk of relapse and side-effects.

To read the Final Appraisal Document from NICE visit: 

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