Hydroxycarbamide therapy may confer neurocognitive benefits to adolescents with sickle cell disease (SCD), according to results from a study published in the British Journal of Haematology.

Researchers screened neurocognitive functioning in 103 adolescents with SCD who were grouped by sickle genotype severity and compared outcomes between those treated with hydroxycarbamide (12 patients with HbSC/HbSβ+ thalassemia; 52 patients with HbSS/HbSβ0 thalassemia) and those not treated with hydroxycarbamide (31 patients with HbSC/HbSβ+ thalassemia; 8 patients with HbSS/HbSβ0 thalassemia). Demographic distributions were similar between the groups. Patients with different sickle genotype severity were analyzed separately.

Patients treated with hydroxycarbamide exhibited higher scores on measures of nonverbal IQ (HbSC/HbSβ+, P =.036), working memory (HbSC/HbSβ+, P =.034), verbal memory (HbSC/HbSβ+, P =.038), reaction speed (HbSS/HbSβ0, P =.002), and sustained attention (HbSS/HbSβ0, P =.014) compared with patients not receiving hydroxycarbamide.

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Longer treatment duration with hydroxycarbamide was associated with better scores on measures of verbal memory (P =.009) and reading (P =.002) in HbSS/HbSβ0 adolescents. Patients receiving hydroxycarbamide tended to have higher fetal hemoglobin (HbF), higher mean corpuscular volume (MCV), and lower white blood cell count (WBC), which were associated with better verbal fluency (HbF, P =.014;  MCV, P =.006; WBC, P =.047) and reading (MCV, P =.021; WBC, P =.037) scores.

These results suggest hydroxycarbamide therapy has disease-modifying effects that may affect cognitive function. “Additionally, we showed that impairment exist across neurocognitive domains, and screening for deficits is thus necessary to identify patients who need further evaluation or intervention,” the researchers noted.


Partanen M, Kang G, Wang WC, et al. Association between hydroxycarbamide exposure and neurocognitive function in adolescents with sickle cell disease [published online February 26, 2020]. Br J Haematol. doi: 10.1111/bjh.16519