Infant acute lymphoblastic leukaemia—Progress from worldwide clinical efforts

Infant acute lymphoblastic leukaemia (ALL) is a rare and aggressive type of leukaemia, especially when a KMT2A rearrangement is involved. Historical treatment strategies have included intensification of conventional ALL chemotherapy in combination with acute myeloid leukaemia treatment elements. However, outcomes have remained consistently poor compared to the advances that have been seen in childhood ALL. The emergence of novel immunotherapies has transformed treatment strategies, with blinatumomab now incorporated into clinical trials for infants with KMT2A-rearranged ALL, promising significant advancements. Additionally, deeper insights into the unique biology of KMT2A-rearranged ALL have facilitated the development of targeted therapies, such as venetoclax and menin inhibitors, which are under clinical evaluation. These efforts offer renewed hope for better outcomes. This review highlights the substantial progress in understanding and treating KMT2A-rearranged ALL, fuelling optimism for future therapeutic success. In addition, we highlight the challenges that might be faced by using immunotherapy.