Title: Asciminib for treating chronic myeloid leukaemia after 2 or more tyrosine kinase inhibitors [ID3813]
Funder: Department of Health and Social Care

Abstract: Chronic myeloid leukaemia (CML) is a type of cancer that affects the blood-forming cells, called myeloid cells, found in the bone marrow[1],[2] . People with CML have a genetic mutation in their bone marrow cells which causes an abnormal chromosome to form, known as the Philadelphia chromosome[1] . The Philadelphia chromosome is made up of 2 genes which join to make a single fusion gene called BCR-ABL[1] . The BCR-ABL gene causes the myeloid cells to produce a protein, called tyrosine kinase, that encourages white blood cells to grow and multiply[1] . CML usually develops and progresses slowly. It is diagnosed in one of three distinct phases; chronic, accelerated or blast phase [3] ._x000D_ _x000D_ The incidence of CML is approximately 1.3 per 100,000 in the UK [4] , it is a rare condition with around 760 people diagnosed in the UK each year [4] . The median age at diagnosis is between 60 and 65 years of age. Around 90% of CML cases are diagnosed at the chronic phase [2] ._x000D_ _x000D_ NICE technology appraisal 401 recommends bosutinib as an option, for chronic, accelerated and blast phase Philadelphia chromosome positive chronic myeloid leukaemia in adults, when they have previously had 1 or more tyrosine kinase inhibitor and imatinib, nilotinib and dasatinib are not appropriate. NICE technology appraisal guidance 425 recommends dasatinib and nilotinib as options for treating only chronic- or accelerated-phase Philadelphia-chromosome-positive chronic myeloid leukaemia in adults, if they cannot have imatinib, or their disease is imatinibresistant. NICE technology appraisal guidance 451 recommends ponatinib as an option for treating chronic , accelerated or blast phase chronic myeloid leukaemia in adults when the disease is resistant to dasatinib or nilotinib or they cannot tolerate dasatinib or nilotinib and for whom subsequent treatment with imatinib is not clinically appropriate or the T315I gene mutation is present._x000D_ _x000D_ References_x000D_ 1. Hochhaus, A., Saussele S., Rosti G., Mahon F. X., Janssen J. J. W. M., HjorthHansen H., et al. Chronic myeloid leukaemia: ESMO Clinical Practice Guidelines for diagnosis, treatment and follow-up. Annals of oncology : official journal of the European Society for Medical Oncology. 2017;28(suppl_4):iv41-iv51. Available from: https://10.1093/annonc/mdx219_x000D_ 2. Leukaemia Foundation. Chronic myeloid leukaemia (CML). 2021. Available from: https://www.leukaemia.org.au/disease-information/leukaemias/chronic-myeloidleukaemia/ [Accessed 16 April 2021]._x000D_ 3. Cancer Research UK. Chronic myeloid leukaemia (CML): Stages. 2020. Available from: https://www.cancerresearchuk.org/about-cancer/chronic-myeloid-leukaemiacml/stages [Accessed 27 April 2021]._x000D_ 4. Cancer Research UK. Chronic myeloid leukaemia (CML) incidence statistics. Available from: https://www.cancerresearchuk.org/health-professional/cancerstatistics/statistics-by-cancer-type/leukaemia-cml/incidence [Accessed 16 April 2021].

Lay abstract: To appraise the clinical and cost effectiveness of asciminib within its marketing authorisation for treating Philadelphia-chromosome-positive chronic myeloid leukaemia in the chronic phase after two or more tyrosine kinase inhibitors.

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