TABLE 4.
Working Group 4: design of pregnancy/birth longitudinal cohorts that leverage ‘omics’, existing phenotypic data, and in silico modelling to study FVIII immunogenicity, as well as inhibitor development and eradication
Co-chairs | Deborah Brown, MD and Jill Johnsen, MD |
Charge | The design of prospective pregnancy/birth longitudinal cohorts that leverage multi-“omics” science, existing phenotypic data and in silico protein modelling to study FVIII immunogenicity, inhibitor development and eradication |
Goals | Design data capture and mechanistic studies, based on translational scientific priorities required to build precision (personalized) medicine-based • Clinical decision-making algorithms that can be applied across the lifespan to either avoid or provoke clinical phenotype for the purpose of diagnosis and/or appropriate time-sensitive intervention, include the design of potential antenatal/neonatal interventions • Address challenges associated with, and successful models for building lifespan/intergenerational cohorts, as well as resources and partners required • For success, including unique challenges in sample procurement and banking • Optimization of private-public partnerships to fund longitudinal cohorts • Engage the patient community in longitudinal cohort participation • Training models and opportunities engendered by novel cross-disciplinary science |