Fetal hemoglobin levels in African American and Hispanic children with sickle cell disease at baseline and in response to hydroxyurea.
Fetal hemoglobin levels in African American and Hispanic children with sickle cell disease at baseline and in response to hydroxyurea.
复制标题
非裔美国人和西班牙裔镰状细胞病患儿的胎儿血红蛋白水平基线和对羟基脲的反应。
DOI：
10.1097/mph.0b013e31822dcc21
复制
发表时间：
2011-10
期刊：
影响因子：
--
通讯作者：
Green NS
中科院分区：
文献类型：
--
作者：
Ender KL;Lee MT;Sheth S;Licursi M;Crotty J;Barral S;Green NS
胎儿血红蛋白（HbF）表达的程度是镰状细胞病（SCD）表型严重程度的主要决定因素。HbF产生的遗传调节是复杂的，并且可以在种族群体之间变化。我们机构的儿科镰状细胞患者约有一半是西班牙裔，几乎全部来自多米尼加共和国。羟基脲（HU）是FDA批准的唯一一种改善SCD症状的药物。我们回顾性比较了4-21岁的HbSS或HbSβ 0地中海贫血非洲裔美国人（AA）和西班牙裔（H）患者的基线和HU诱导的%HbF。AA（N=48）和H（N=58）患者之间的平均基线%HbF无显著差异（p=0.63）。在服用达到最大耐受剂量（MTD）的羟基脲的儿童亚组中，在实验室药物应答方面未发现种族组间差异，通过MTD时的%HbF（p=0.28）、%HbF（p=0.31）或平均红细胞体积（MCV）（p=0.93）或HU的MTD（p=0.95）测量。在我们的中心，西班牙裔和非洲裔美国人之间基线时和对HU的反应中HbF的调节是相当的。如果可推广，我们的结果支持在未来的临床和翻译分析中将这两组合并，重点关注该种族混合患者人群中的HbF和对HU的反应。
The degree of fetal hemoglobin (HbF) expression is a major determinant of phenotypic severity of sickle cell disease (SCD). Genetic regulation of HbF production is complex and can vary among ethnic groups. The pediatric sickle cell population at our institution is approximately half Hispanic, nearly all from the Dominican Republic. Hydroxyurea (HU) is the only FDA-approved drug to ameliorate symptoms of SCD. We retrospectively compared baseline and HU-induced %HbF in African American (AA) and Hispanic (H) patients aged 4–21 years with HbSS or HbSβ0Thalassemia. No significant differences were detected in average baseline %HbF between AA (N=48) and H (N=58) patients (p=0.63). In the subset of children taking hydroxyurea who reached maximum tolerated dose (MTD), no differences were found between the ethnic groups in laboratory response to drug, measured by %HbF at MTD (p=0.28), the increase in %HbF (p=0.31) or mean red cell volume (MCV) (p=0.93), or the MTD of HU (p=0.95). Regulation of HbF at baseline and in response to HU are comparable between Hispanics and African Americans at our center. If generalizable, our results support combining these two groups in future clinical and translational analyses focused on HbF and response to HU in this ethnically-mixed patient population.