1 Recommendations

1.1 Arsenic trioxide is recommended, within its marketing authorisation, as an option for inducing remission and consolidation in acute promyelocytic leukaemia (characterised by the presence of the t[15;17] translocation or the PML/RAR-alpha gene) in adults with:

untreated, low-to-intermediate risk disease (defined as a white blood cell count of 10x10³ per microlitre or less), when given with all-trans-retinoic acid (ATRA)
relapsed or refractory disease, after a retinoid and chemotherapy.

Why the committee made these recommendations

People with untreated, low-to-intermediate risk acute promyelocytic leukaemia are given ATRA plus chemotherapy (together called AIDA). Clinical trial evidence shows that arsenic trioxide plus ATRA is effective for untreated disease compared with AIDA. Some assumptions in the model, such as the costs of stem cell transplant and the long-term effect of treatment, lead to the cost-effectiveness analyses being uncertain. However, the most plausible cost-effectiveness estimate is likely to be less than £20,000 per quality-adjusted life year gained, so arsenic trioxide plus ATRA is cost effective compared with AIDA in untreated disease.

Arsenic trioxide is already used to treat relapsed or refractory acute promyelocytic leukaemia. The clinical- and cost-effectiveness evidence for arsenic trioxide in relapsed or refractory disease is uncertain, because the clinical trial was small and did not compare arsenic trioxide with AIDA. However, it is likely that arsenic trioxide is clinically effective and represents a cost-effective use of NHS resources in relapsed or refractory disease. Therefore, arsenic trioxide is recommended for both untreated and relapsed or refractory disease.