Title: Oral azacitidine for maintenance treatment of acute myeloid leukaemia after induction therapy [ID3892]
Funder: Department of Health and Social Care

Abstract: Acute myeloid leukaemia is a cancer of the blood and bone marrow. It is characterised by the overproduction of early immature myeloid cells (blasts). Acute myeloid leukaemia is classified into different types. In most types of acute myeloid leukaemia, the leukaemia cells are immature white blood cells. In other less common types, too many immature platelets or immature red blood cells form leukaemia cells. Anaemia, bleeding problems and serious infections are common symptoms of acute myeloid leukaemia._x000D_ _x000D_ The incidence of acute myeloid leukaemia has increased by 7% in the UK over the last decade.[1] There are around 3,200 new diagnoses of acute myeloid leukaemia in the UK per year. [1] The incidence rate increases with age with the highest rates being in the 85 to 89 age group.[1] There are around 2,600 acute myeloid leukaemia deaths registered in the UK per year. [1]_x000D_ _x000D_ The aim of treatment for acute myeloid leukaemia is to cure it. For people who are fit enough to have intensive treatment, induction chemotherapy is initially given to achieve a remission. Up to 80% of younger adults and up to 60% of adults aged 60 years or older have complete remission with a standard induction therapy regimen of anthracycline and cytarabine. [2] For people with adverse risk factors, relapse rates are high and 5-year overall survival rates are low even after people have remission with chemotherapy. [3] After remission, further cycles of chemotherapy are sometimes given to reduce the risk of the leukaemia recurring (consolidation therapy). Maintenance therapy is long term treatment which may be given to prevent the cancer returning when it is in remission or to prolong remission. Although, maintenance therapy is not always given to people with AML. People with good general health may have a stem cell transplantation to replace damaged blood cells with healthy ones. However, the decision for transplantation includes response to chemotherapy, the individual’s fitness, donor source, and the individual’s choice. Therefore, some people may not be eligible or may choose not to have stem cell transplantation._x000D_ _x000D_ For people with untreated disease:_x000D_ _x000D_ • NICE technology appraisal 552 recommends liposomal cytarabinedaunorubicin as a treatment option._x000D_ _x000D_ • NICE technology appraisal 545 recommends gemtuzumab ozogamicin with daunorubicin and cytarabine as a treatment option for people with de novo CD33-positive acute myeloid leukaemia._x000D_ _x000D_ • NICE technology appraisal 523 recommends midostaurin as a treatment option for newly diagnosed acute FLT3-mutation positive myeloid leukaemia with standard daunorubicin and cytarabine as induction therapy, with highdose cytarabine as consolidation therapy, and alone after complete response as maintenance therapy._x000D_ _x000D_ • NICE technology appraisal 399 does not recommend subcutaneous azacitidine for treating acute myeloid leukaemia with more than 30% bone marrow blasts in people 65 years or older who are not eligible for haematopoietic stem cell transplant._x000D_ _x000D_ • NICE technology appraisal 218 recommends subcutaneous azacitidine as a treatment option for adults who are not eligible for haematopoietic stem cell transplant and have acute myeloid leukaemia with 20-30% blasts and multilineage dysplasia according to the World Health Organization classification. The only treatment option currently recommended by NICE as maintenance therapy for acute myeloid leukaemia is midostaurin, and it is only recommended for people who had complete response to midostaurin as induction and consolidation treatment in people with FLT-3 mutations._x000D_ _x000D_ References_x000D_ 1 Cancer Research UK (2016) Acute myeloid leukaemia (AML) statistics. Accessed June 2021._x000D_ 2 Dohner et al. (2017) Diagnosis and management of AML in adults: 2017 ELN recommendations from an international expert panel. Blood 129 (4): 424-447._x000D_ 3 Medeiros, BC et al. (2019) Optimizing survival outcomes with post-remission therapy in acute myeloid leukemia. American Journal of Hematology 94: 803-811.

Lay abstract: To appraise the clinical and cost effectiveness of oral azacitidine within its marketing authorisation as maintenance treatment for adults with acute myeloid leukaemia after induction therapy.

Research activity codes: 6.1 PHARMACEUTICALS
Health categories: CANCER AND NEOPLASMS