Quality of Life and Social and Psychological Outcomes in Adulthood Following Allogeneic HSCT in Childhood for Inborn Errors of Immunity

The aim of this study was to examine the social and psychological outcomes in adults following hematopoietic stem cell transplant (HSCT) for inborn errors of immunity (IEIs) in childhood.

This study included 83 patients, aged 16 and over, who underwent HSCT for IEI at 2 hospitals in the United Kingdom at least 5 years prior. The control group comprised 46 healthy individuals, matched for age and gender, nominated by the patients (friends or siblings).

This was a retrospective cross-sectional study that included a prospective cohort design stage to compare the long-term functioning of adult HSCT patients with healthy controls and population norms. Data collection comprised medical notes, questionnaires, telephone assessments, and psychological evaluations. Quality of life (QoL), anxiety, depression, psychological and cognitive functioning, and social circumstances were measured. Statistical tests were used to compare the patient group with population norms and to analyze differences between patients and controls.

The median age at transplant was 3 years and the median time since transplant was 17.1 years. Patients were transplanted for severe combined immunodeficiency, combined immunodeficiency, and phagocyte disorders with variable genetic causes. Regarding measurable outcomes, the patient and control groups had similar scores in QoL to the general population. They both had better physical health-related QoL than the general population, however, the patients had significantly worse physical health-related QoL compared with matched controls (P = .02). Patients had higher levels of anxiety and depressed mood than the general population, with 25% and 13% meeting clinical criteria for anxiety and depression, respectively. Patients had significantly worse self-reported health status (P = .01). When a learning disability was not expected with their condition, cognitive function was comparable between groups and within normal range.

Adult patients who underwent HSCT for IEI in childhood have variable social and psychological outcomes, including higher rates of anxiety and depression but generally good quality of life.

HSCT is the only curative therapy for many IEIs, and survival outcomes are better when HSCT is conducted earlier in life before patients have recurrent and/or severe infections. There has been little research into the long-term effects on mental health into adulthood for those who underwent HSCT in childhood for IEI. This study reveals mixed social and psychological outcomes in adulthood after transplantation. Although patients had higher levels of anxiety and depressed mood than the general population, QoL was comparable to population norms. Population bias could explain the comparable QoL, as expectations of health in patients with IEI may be lower than the general population. Although this study was limited by a small sample size and not all participants having a matched control, it emphasizes the need for further research into long-term outcomes, routine mental health screening post-transplant, and directed interventions to optimize outcomes in those at greatest risk of morbidity. The general pediatrician and subspecialists who care for transplant patients need to be cognizant of these potential outcomes, proactively discuss the possible negative outcomes early, and establish effective transitions of care into adult healthcare for this at-risk population.