阿尔茨海默病和帕金森病杂志

抽象的

Understanding the Role of Chromosome 21 for Precision Treatment in Down Syndrome Acute Lymphoblastic Leukaemia

Elyse Page, Deborah White

Children with Down Syndrome (DS) are predisposed to developing Acute Lymphoblastic Leukaemia (ALL) and experience lower overall survival (75%) compared to children without Down syndrome (85-90%). The mortality rate for paediatric DS-ALL patients is four times higher than non-DS-ALL patients in the first two years after their diagnosis. Increased chemotherapy-related toxicity is experienced by DS-ALL patients, however, new immunotherapies including bi-specific T-cell engagers and chimeric antigen receptor T-cell therapies are being pursued in clinical trials. Fundamental research has identified 31 genes in the Down syndrome critical region of chromosome 21 which play a role in leukaemogenesis. Understanding these genes will be critical to identify the predisposition DS patients have for developing ALL, as well as discovering new targeted therapeutic approaches. The aim is to identify the role(s) of chromosome 21 genes to establish less toxic treatment options for DS-ALL patients.