title: Cognitive and behavioral abnormalities in children after hematopoietic stem cell transplantation for severe congenital immunodeficiencies.
abstract: Hematopoietic stem cell transplantation (HSCT) is a highly successful treatment for severe congenital immunodeficiencies. However, some studies have suggested that children may experience cognitive difficulties after HSCT. This large-scale study assessed cognitive and behavioral function for the cohort of children treated by HSCT at one center between 1979 and 2003 to determine the frequency and severity of problems and to identify risk factors. A total of 105 patients were assessed on standardized measures of cognitive and emotional and behavioral function together with a control group of unaffected siblings. The average IQ for the cohort was 85 (95% confidence interval, 81-90), significantly lower than both the population average of 100 (P < .001) and unaffected siblings. Multivariate analysis indicated that the underlying genetic defect, diagnosis of adenosine deaminase-deficient severe combined immunodeficiency, and consanguinity were associated with worse outcome but that age at transplantation 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 behavioral 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.
authors.affiliation: Department of Psychosocial Services, Great Ormond Street Hospital National Health Service (NHS) Trust, London, UK. titmap@gosh.nhs.uk
authors.forename: Penny
authors.initials: P
authors.lastname: Titman
authors.forename: Elizabeth
authors.initials: E
authors.lastname: Pink
authors.forename: Emily
authors.initials: E
authors.lastname: Skucek
authors.forename: Katherine
authors.initials: K
authors.lastname: O'Hanlon
authors.forename: Tim J
authors.initials: TJ
authors.lastname: Cole
authors.forename: Jane
authors.initials: J
authors.lastname: Gaspar
authors.forename: Jinhua
authors.initials: J
authors.lastname: Xu-Bayford
authors.forename: Alison
authors.initials: A
authors.lastname: Jones
authors.forename: Adrian J
authors.initials: AJ
authors.lastname: Thrasher
authors.forename: E Graham
authors.initials: EG
authors.lastname: Davies
authors.forename: Paul A
authors.initials: PA
authors.lastname: Veys
authors.forename: H Bobby
authors.initials: HB
authors.lastname: Gaspar
mesh_terms: D000243:Adenosine Deaminase / Q000172:deficiency; D000293:Adolescent; D000328:Adult; D000342:Affective Symptoms / Q000209:etiology; D000367:Age Factors; D016022:Case-Control Studies; D002648:Child; D002653:Child Behavior Disorders / Q000209:etiology*; D002675:Child, Preschool; D003072:Cognition Disorders / Q000209:etiology*; D015331:Cohort Studies; D003241:Consanguinity; D005260:Female; D005500:Follow-Up Studies; D018380:Hematopoietic Stem Cell Transplantation / Q000009:adverse effects* / Q000523:psychology; D006801:Humans; D007153:Immunologic Deficiency Syndromes / Q000151:congenital / Q000235:genetics / Q000523:psychology* / Q000628:therapy*; D007360:Intelligence; D008297:Male; D016511:Severe Combined Immunodeficiency / Q000151:congenital / Q000235:genetics / Q000523:psychology / Q000628:therapy; D013997:Time Factors
chemical_list: D000243:Adenosine Deaminase