1 Recommendations

1.1 Gilteritinib monotherapy is recommended as an option for treating relapsed or refractory FLT3‑mutation-positive acute myeloid leukaemia (AML) in adults only if the company provides gilteritinib according to the commercial arrangement.

1.2 Gilteritinib should not be given as maintenance therapy after a haematopoietic stem cell transplant.

1.3 These recommendations are not intended to affect treatment with gilteritinib that was started in the NHS before this guidance was published. People having treatment outside this recommendation 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.

Why the committee made these recommendations

Relapsed or refractory FLT3‑mutation-positive AML is usually treated with salvage chemotherapy (a type of chemotherapy offered when a first course of chemotherapy has not worked, or the disease has come back after treatment). Gilteritinib is an alternative treatment taken as an oral tablet at home, which is an important quality-of-life benefit for patients.

Clinical evidence shows that people having gilteritinib live longer compared with people having salvage chemotherapy. However, there is considerable uncertainty about long-term survival, particularly after stem cell transplant. There is no robust evidence of further benefit if someone restarts gilteritinib after stem cell transplant when they have had it before the transplant.

Gilteritinib meets NICE's criteria for a life-extending treatment at the end of life. The most likely cost-effectiveness estimates are within the range that NICE normally considers an acceptable use of NHS resources for end-of-life treatments. Therefore, gilteritinib is recommended as an option for people with relapsed or refractory FLT3‑mutation-positive AML. However, if people then have a stem cell transplant, gilteritinib should not be restarted afterwards.