Cognitive and behavioural abnormalities in children following haematopoietic stem cell transplantation for severe congenital immunodeficiencies

Cognitive and behavioural abnormalities in children following haematopoietic stem cell transplantation for severe congenital immunodeficiencies

Penny Titman*, Elizabeth Pink, Emily Skucek, Katherine O’Hanlon, Tim J Cole, Jane Gaspar, JinHua Xu-Bayford, Alison Jones, Adrian J. Thrasher, E Graham Davies, Paul A. Veys, and H. Bobby Gaspar

Psychosocial Services, Great Ormond Street Hospital, London, United Kingdom
Centre for Paediatric Epidemiology and Biostatistics, UCL Institute of Child Health, London, United Kingdom
Immunology Department, Great Ormond Street Hospital, London, United Kingdom
Molecular Immunology Unit, UCL Institute of Child Health, London, United Kingdom
Dept of Bone Marrow Transplant, GOSH, London, United Kingdom

* Corresponding author; email: titmap@gosh.nhs.uk

Haematopoietic stem cell transplantation (HSCT) is a highly successful treatment for severe congenital immunodeficiencies. However, some studies have suggested that children may experience cognitive difficulties following HSCT. This large scale study assessed cognitive and behavioural function for the cohort of children treated by HSCT at one centre between 1979 – 2003 to determine the frequency and severity of problems and to identify risk factors. 105 patients were assessed on standardized measures of cognitive and emotional and behavioural function together with a control group of unaffected siblings. The average IQ for the cohort was 85 (95 percent confidence interval 81 – 90), significantly lower than both the population average of 100 (p<0.001) and unaffected siblings. Multivariate analysis indicated that the underlying genetic defect, diagnosis of ADA deficient severe combined immunodeficiency and consanguinity were associated with worse outcome, but that age at transplant and chemotherapy conditioning were not. Children treated by HSCT for severe immunodeficiency have an increased risk of long term cognitive difficulties and associated emotional and behavioural difficulties. The specific genetic diagnosis, consanguinity and severe clinical course are associated with poor outcome. Long term follow up of these patients should include screening to identify and manage these problems more effectively.

linkback url: http://bloodjournal.hematologylibrary.org/cgi/content/short/blood-2008-04-151332v1?rss=1

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