Haematopoietic stem cell transplantation for severe combined immunodeficiency: Long-term health outcomes and patient perspectives

Aim: To examine the long-term follow-up and health outcomes of patients who have undergone haematopoietic stem cell transplant (HSCT) forsevere combined immunodeficiency (SCID).

Methods: Through a structured questionnaire, we examined follow-up arrangements and long-term health outcomes in 22 children who havehad a successful HSCT for SCID during the period of 1984–2012 at the Sydney Children’s Hospital, Sydney, Australia.

Results: Most children considered themselves healthy and ‘cured’ from SCID. Whilst many children enjoy relatively good bio-social health out-comes, specific negative health outcomes and absenteeism from school were perceived negatively. Two-thirds of children see their general practi-tioner or specialist regularly; however, there did not appear to be consistency with the nature of this follow-up.

Conclusion: The findings from our study highlight the comple x bio-psychosocial health needs of post-HSC T SCID childr en and encour age SCID centr es toconsider a multidisciplinary approach to their follow -up . Further studies into the determinants of patients’ perceptions of their health are needed.