Association between hydroxycarbamide exposure and neurocognitive function in adolescents with sickle cell disease
Association between hydroxycarbamide exposure and neurocognitive function in adolescents with sickle cell disease
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DOI：
10.1111/bjh.16519
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发表时间：
2020-02-26
影响因子：
6.5
通讯作者：
Jacola, Lisa M.
中科院分区：
文献类型：
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作者：
Partanen, Marita;Kang, Guolian;Jacola, Lisa M.
镰状细胞病（SCD）患者出现神经认知障碍的风险增加。虽然羟基脲（羟基脲）等疾病缓解治疗可能会降低这种风险，但尚未在患有 SCD 的儿童中进行系统研究。我们筛查了 103 名 SCD 青少年（16-17 岁，50% 女性）的神经认知功能，并比较了有羟基脲暴露史的患者（n = 12 例 HbSC/HbS β(+) 地中海贫血；n = 52 例 HbSS/HbS β(0) 地中海贫血）和从未接受过羟基脲治疗的患者（n = 31 例 HbSC/HbS）之间的结果β(+) 地中海贫血；n = 8 HbSS/HbS β(0) 地中海贫血)。各组之间的人口统计分布相似。调整社会经济状况后，羟基脲组在非语言智商（HbSC/HbS β 地中海贫血：P = 0 中心点 036，效应大小 [d] = 0 中心点 65）、反应速度（HbSS/HbS β(0) 地中海贫血：P = 0 中心点 002，d = 1 中心点 70）、持续注意力方面得分显着更高（HbSS/HbS beta(0) 地中海贫血：P = 0 中心点 014，d = 1 中心点 30）、工作记忆（HbSC/HbS beta(+) 地中海贫血：P = 0 中心点 034，d = 0 中心点 71）和言语记忆（HbSC/HbS beta(+) 地中海贫血：P = 0 中心点038，d = 0 中心点 84) 与未接受羟基脲的患者相比。在 HbSS/HbS beta(0) 地中海贫血患者中，较长的羟基脲治疗时间与更好的言语记忆（P = 0 中心点 009）和阅读（P = 0 中心点 002）相关。羟基脲效应的标志物，包括较高的胎儿血红蛋白 (HbF)、较高的平均红细胞体积 (MCV) 和较低的白细胞计数 (WBC)，与更好的言语流畅性（HbF：P = 0 中心点 014，MCV：P = 0 中心点 006，WBC：P = 0 中心点 047）和阅读（MCV：P = 0 中心点 021， WBC：P = 0 中心点 037)。患有 SCD 的青少年接触羟基脲可能会减轻认知障碍。
Patients with sickle cell disease (SCD) are at increased risk for neurocognitive impairments. While disease-modifying treatment, such as hydroxycarbamide (hydroxyurea), may decrease this risk, it has not been systematically investigated in children with SCD. We screened neurocognitive functioning in 103 adolescents with SCD (16-17 years, 50% female) and compared outcomes between patients with a history of exposure to hydroxycarbamide (n = 12 HbSC/HbS beta(+) thalassaemia; n = 52 HbSS/HbS beta(0) thalassaemia) and those never treated with hydroxycarbamide (n = 31 HbSC/HbS beta(+) thalassaemia; n = 8 HbSS/HbS beta(0) thalassaemia). Demographic distributions were similar between the groups. After adjusting for socioeconomic status, the hydroxycarbamide group had significantly higher scores on nonverbal IQ (HbSC/HbS beta thalassaemia: P = 0 center dot 036, effect size [d] = 0 center dot 65), reaction speed (HbSS/HbS beta(0) thalassaemia: P = 0 center dot 002, d = 1 center dot 70), sustained attention (HbSS/HbS beta(0) thalassaemia: P = 0 center dot 014, d = 1 center dot 30), working memory (HbSC/HbS beta(+) thalassaemia: P = 0 center dot 034, d = 0 center dot 71) and verbal memory (HbSC/HbS beta(+) thalassaemia: P = 0 center dot 038, d = 0 center dot 84) when compared to those who did not receive hydroxycarbamide. In patients with HbSS/HbS beta(0) thalassaemia, longer treatment duration with hydroxycarbamide was associated with better verbal memory (P = 0 center dot 009) and reading (P = 0 center dot 002). Markers of hydroxycarbamide effect, including higher fetal haemoglobin (HbF), higher mean corpuscular volume (MCV) and lower white blood cell count (WBC), were associated with better verbal fluency (HbF: P = 0 center dot 014, MCV: P = 0 center dot 006, WBC: P = 0 center dot 047) and reading (MCV: P = 0 center dot 021, WBC: P = 0 center dot 037). Cognitive impairment may be mitigated by exposure to hydroxycarbamide in adolescents with SCD.