Clinical and immunological outcome after paediatric stem cell transplantation in inborn errors of immunity

Abstract

This thesis examines the clinical and immunological outcome after paediatric stem cell transplantation in inborn errors of immunity (IEI). First part reviews the general principles of haematopoietic cell transplantation (HCT) in IEI. The second part of this thesis focuses on practice pattern changes over the past three decades in the field of transplant for non-SCID IEI. The third part of this thesis analyses the outcome after HCT in IEI using ex-vivo T cell depleted mismatched grafts and compares the HCT outcomes between T deplete HLA-mismatched grafts and T-replete HLA-matched family/unrelated grafts. Part 4 focuses on late effects of HCT, particularly post-transplant autoimmunity and malignancy post-HCT in IEI. The final part explores the potential future directions of research into the diversity in the use of HCT in IEI.