1.1 Gemtuzumab ozogamicin, with daunorubicin and cytarabine, is recommended as an option for untreated de novo CD33-positive acute myeloid leukaemia (AML), except acute promyelocytic leukaemia, in people 15 years and over, only if:
they start induction therapy when either the cytogenetic test confirms that the disease has favourable, intermediate or unknown cytogenetics (that is, because the test was unsuccessful) or when their cytogenetic test results are not yet available and
they start consolidation therapy when their cytogenetic test confirms that the disease has favourable, intermediate or unknown cytogenetics (because the test was unsuccessful) and
the company provides gemtuzumab ozogamicin according to the commercial arrangement.
1.2 These recommendations are not intended to affect treatment with gemtuzumab ozogamicin that was started in the NHS before this guidance was published. People having treatment outside these recommendations may continue without change to the funding arrangements in place for them before this guidance was published, until they and their NHS clinician consider it appropriate to stop. For young people aged 15 to 17, this decision should be made jointly by the young person and their parents or carers and the clinician.
Why the committee made these recommendations
AML is currently treated with daunorubicin plus cytarabine. Cytogenetic testing is used to look for specific gene mutations in certain types of leukaemia, which might predict how a person's disease responds to treatment and affect treatment options. People whose disease has favourable or intermediate cytogenetics have a better prognosis than those whose disease has unfavourable cytogenetics in terms of treatment response, risk of relapse and survival. However, for some patients the cytogenetic test results are not available at the start of induction treatment.
Evidence from a randomised clinical trial shows that, for people whose disease has favourable or intermediate cytogenetics, gemtuzumab ozogamicin with daunorubicin and cytarabine is more clinically effective than daunorubicin and cytarabine. People are more likely to live longer without the disease relapsing or symptoms returning.
Because no clinical- or cost-effectiveness analysis is presented for people whose disease has unfavourable cytogenetics, gemtuzumab ozogamicin cannot be recommended for this group.
The most plausible cost-effectiveness estimates (including the stopping rule for consolidation therapy in people who have unfavourable cytogenetics) for gemtuzumab ozogamicin compared with daunorubicin and cytarabine in people whose disease has favourable, intermediate or unknown cytogenetics (because the cytogenetic test is unsuccessful) are within the range that NICE normally considers an acceptable use of NHS resources. Therefore gemtuzumab ozogamicin can be recommended for these groups of people.