Many primary immunodeficiencies (PIDs) start in childhood and are life-threatening. Treatment includes haematopoietic stem cell transplant (HSCT). Due to improved techniques, more transplants are undertaken and patients are living longer. However, long-term complications can significantly affect future health and quality of life.
Previous research has focused on short-term medical outcomes and little is known about health or psychosocial outcomes in adulthood. This project is the largest and most comprehensive evaluation of long-term physical, social and psychological outcomes for adults who underwent HSCT for PID during childhood.
83 adult patients, who underwent HSCT for PID at Great Ormond Street Hospital during childhood, and at least five years previously, were recruited. Questionnaires and practical tasks assessed their current functioning and circumstances. Information was also gathered from medical notes. Data was compared with population norms and a control group of participant-nominated siblings or friends.
The majority of participants reported satisfaction with their overall wellbeing. However, patients reported significantly poorer physical health-related quality of life than peers, and decreased cognitive and social functioning in some domains. These and other psychosocial, physical and cost-effectiveness outcomes will be presented.
Implications and suggestions for future research and service development will be discussed.
This abstract presents independent research funded by the NIHR under its Research for Patient Benefit Programme (Grant Reference Number PB-PG-0215-36145). The views expressed are those of the authors and not necessarily those of the NIHR or the Department of Health and Social Care.