A flowchart illustrating the different disease journeys experienced by children with severe LAD-I. The process begins with LAD-I related infections, inflammatory complications, and hospitalization. All children initially receive anti-microbials. The paths then diverge: some children undergo gene therapy, leading to no new LAD-related infections. Others receive allogenic hematopoietic stem cell transplant, facing graft versus host challenges, and either stop experiencing infections or need a second transplant. One child remains in a loop of infection and anti-microbial treatment.
Participants’ children’s severe LAD-I disease journeys by treatment type. A graphical representation of the different disease journeys experienced by each of the children with severe LAD-I whose parents participated in the study. Each child started with the shared experience of severe, recurrent infections, inflammatory complications, and hospitalizations and was initially treated with antimicrobials. The journeys then diverge. Children who had gene therapy no longer experienced LAD-related infections, etc. Those who had allo-HSCT experienced graft-versus-host challenges and either stopped experiences infections or needed a second stem cell transfer. The child who was only treated using antimicrobials continued to be in a loop of infection/inflammation and antimicrobial treatment.