The effect of hydroxcarbamide therapy on survival of children with sickle cell disease
The effect of hydroxcarbamide therapy on survival of children with sickle cell disease
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DOI：
10.1111/bjh.12323
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发表时间：
2013-06-01
影响因子：
6.5
通讯作者：
Hankins, Jane S.
中科院分区：
文献类型：
--
作者：
de Castro Lobo, Clarisse Lopes;Pinto, Jorge F. C.;Hankins, Jane S.
尽管越来越多的证据表明羟基脲可降低镰状细胞病 (SCD) 成人患者的死亡率，但尚无关于羟基脲对儿童死亡率影响的公开数据。儿科羟基脲计划旨在治疗 3-18 岁患有 SCD 且符合疾病严重程度标准的儿童。回顾性收集了该计划前 9 年的死亡率数据和羟基脲的临床/实验室影响。将接受羟基脲的儿童的死亡率与未经治疗的儿童的死亡率进行比较。在 1760 名受试者中，267 名受试者接受羟基脲，中位剂量为 20 中心点 8mg/kg/d（范围 10-32），中位剂量为 2 年（范围 0 中心点 1-6 中心点 5）。接受羟基脲治疗的儿童的存活率显着高于未治疗的儿童（99 中心点 5% 与 94 中心点 5%，P=0 中心点 01），这主要是由于急性胸部综合征和感染导致的死亡较少。羟基脲治疗与血红蛋白浓度、胎儿血红蛋白、平均红细胞体积的增加以及血小板计数、网织红细胞和中性粒细胞的减少显着相关。毒性很小，主要是轻度可逆性中性粒细胞减少症。与治疗开始前 12 个月相比，接受羟基脲治疗的受试者住院和急诊室就诊次数显着减少，入院时间也缩短。羟基脲治疗可降低疾病严重程度，并可能与 SCD 儿童死亡率降低有关。
Although evidence is accumulating that hydroxycarbamide decreases mortality among adults with sickle cell disease (SCD), there are no published data regarding the effect of hydroxycarbamide on mortality among children. The Paediatric Hydroxycarbamide Program was established to treat children with SCD aged 3-18years if they met disease severity criteria. Mortality data and clinical/laboratorial effects of hydroxycarbamide were retrospectively collected for the first 9years of the Program. Mortality among those who received hydroxycarbamide was compared to that of untreated children. Among 1760 subjects, 267 received hydroxycarbamide at a median dose of 20 center dot 8mg/kg/d (range 10-32) for a median of 2years (range 0 center dot 1-6 center dot 5). Survival among hydroxycarbamide-treated children was significantly greater than that among untreated ones (99 center dot 5% vs. 94 center dot 5%, P=0 center dot 01), due primarily to fewer deaths from acute chest syndrome and infection. Hydroxycarbamide therapy was significantly associated with increases in haemoglobin concentration, fetal haemoglobin, mean corpuscular volume, and reduction in platelet counts, reticulocytes and neutrophils. Toxicity was minimal and predominantly mild reversible neutropenia. Significantly fewer hospitalizations and emergency room visits, and shorter admissions were observed among hydroxycarbamide-treated subjects, when compared to the 12-month period prior to treatment initiation. Hydroxycarbamide therapy reduces disease severity and is probably associated with decreased mortality among children with SCD.