Hydroxyurea treatment and neurocognitive functioning in sickle cell disease from school age to young adulthood

Andrew M. Heitzer, Jennifer Longoria, Victoria Okhomina, Winfred C. Wang, Darcy Raches, Brian Potter, Lisa M. Jacola, Jerlym Porter, Jane E. Schreiber, Allison A. King, Guolian Kang, Jane S. Hankins

Research output: Contribution to journalArticlepeer-review

18 Scopus citations


Neurocognitive impairment is common in sickle cell disease (SCD) and is associated with significant functional limitations. In a cross-sectional analysis, we examined the association between hydroxyurea (HU) treatment and neurocognitive functioning from school-age to young adulthood in individuals with SCD. A total of 215 patients with HbSS/HbSβ0-thalassaemia (71% HU treated) and 149 patients with HbSC/HbSβ+-thalassaemia (20% HU treated) completed neurocognitive measures at one of four developmental stages: school-age (age 8–9 years), early adolescence (age 12–13 years), late adolescence (age 16–17 years) and young adulthood (ages 19–24 years). For participants with multiple assessments, only the most recent evaluation was included. In multivariable analysis adjusted for social vulnerability, HU treatment and sex, older age was associated with a reduction in overall intelligence quotient (IQ) of 0·55 points per year of life [standard error (SE) = 0·18, false discovery rate adjusted P value (PFDR) = 0.01] for patients with HbSS/HbSβ0-thalassaemia. Earlier initiation of HU (n = 152) in HbSS/HbSβ0-thalassaemia was associated with higher scores on neurocognitive measures across most domains, including IQ [estimate (SE) 0·77 (0·25)/year, PFDR = 0·01], after adjusting for social vulnerability, sex and treatment duration. These results support the early use of HU to limit the detrimental neurocognitive effects of SCD, while highlighting the need for additional measures to further mitigate neurocognitive deterioration.

Original languageEnglish
Pages (from-to)256-266
Number of pages11
JournalBritish Journal of Haematology
Issue number2
StatePublished - Oct 2021


  • aging
  • fetal haemoglobin
  • hydroxyurea
  • neurocognition
  • sickle cell disease


Dive into the research topics of 'Hydroxyurea treatment and neurocognitive functioning in sickle cell disease from school age to young adulthood'. Together they form a unique fingerprint.

Cite this