Personalised care for young people with dilated cardiomyopathy

Dilated cardiomyopathy (DCM) is the most common paediatric heart muscle disease and is associated with malignant arrhythmias, cardiac transplantation, and heart failure death. In adults, an understanding of the interplay between cause of disease, phenotype and outcomes has allowed patients to benefit from disease specific management and risk prediction tools leading to personalised care. In contrast, in children the underlying cause is often undetermined, the natural history is poorly understood, and we have no way of predicting response to therapies or outcomes. Together, these evidence gaps prevent personalised management and prognostication. During this fellowship, I will use international collaborations developed during my BHF clinical research training fellowship (CRTF), to develop the largest research cohort of children with DCM in the world and answer 3 questions; (1) Can we predict long-term outcomes and provide personalised risk estimates? (2) Can we predict disease progression and response to common therapies? (3) Can we use biomarkers to help diagnose and predict progression? Building on my substantial track record in risk modelling, I will take advantage of world leading expertise in data science and proteomic bioinformatics at UCL to develop the first paediatric tools for DCM allowing evidence-based personalisation of care for the first time.