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[在布拉柴维尔(刚果)接受羟基脲治疗的纯合子镰状细胞病患儿]

[Homozygous Sickle Cell Children Treated with Hydroxyurea in Brazzaville (Congo)].

作者信息

Mabiala-Babela J R, Nika E R, Ikobo L C Ollandzobo, Gnakingue A N Ofamalekou, Ngoulou B P S, Mandilou S V Missambou

机构信息

Faculté des sciences de la santé, université Marien-Ngouabi, Brazzaville, Congo.

Service de pédiatrie nourrisson, CHU de Brazzaville, Brazzaville, Congo.

出版信息

Bull Soc Pathol Exot. 2019;112(4):206-212. doi: 10.3166/bspe-2019-0096.

Abstract

The objective of this study is to reduce the morbidity of children with homozygous sickle cell disease presenting severe forms. We have conducted a longitudinal study between November 2015 and September 2017 at the Brazzaville University Hospital. Children with sickle cell disease requiring treatment with hydroxyurea were included. The variables studied were therapeutic compliance, evolutive profile of nutritional status, indications of hydroxyurea, electrophoresis of hemoglobin, blood count, and toxicity. The statistical test used was Student test with a significance threshold of less than 0.05. One thousand twenty-four children were monitored for sickle cell disease, 107 of which had received hydroxyurea (10.4%). The indications for hydroxyurea were recurrent anemic attacks (≥ 4) in 62 cases (57.9%), neurological crises 19 cases (17.8%), recurrent hyperalgesic crises in 17 cases (15.9%), priapism in 4 cases (3.7), and acute thoracic syndrome in 2 cases (1.9%). Therapeutic compliance was good in 89.5% of them. A rapid and lasting clinical improvement was noted in the majority of patients with hyperalgesic attacks (88.2%) and anemic attacks (88.7%), two recurrences for the cerebrovascular accidents, and an absence of recurrence of priapism and of the acute thoracic syndrome. From the biological point of view, there was a significant increase in fetal hemoglobin (1.2 to 16.2%;  < 0.05), hemoglobin (7 to 8.3 g/dl;  < 0.05), mean cell volume (80.8 to 96 fl;  < 0.05) and a significant decrease in mean white blood cell count (15,633 to 9,872/mm;  < 0.05) and platelets (387,002 to 324,400/mm;  < 0.05). The signs of toxicity observed were mainly vomiting and thrombocytopenia in two cases each, one case with headache and the other with neutropenia. Indications for use of hydroxyurea therapy in children with sickle cell disease in Brazzaville are common. These are dominated by recurrent anemic seizures, strokes, and hyperalgesic seizures. The excellent evolution of these complications under hydroxyurea represents an interesting alternative in our countries with limited resources.

摘要

本研究的目的是降低患有严重形式纯合子镰状细胞病儿童的发病率。我们于2015年11月至2017年9月在布拉柴维尔大学医院进行了一项纵向研究。纳入了需要使用羟基脲进行治疗的镰状细胞病儿童。所研究的变量包括治疗依从性、营养状况的演变情况、羟基脲的适应证、血红蛋白电泳、血细胞计数及毒性。所使用的统计检验为学生检验,显著性阈值小于0.05。对1024名镰状细胞病儿童进行了监测,其中107名接受了羟基脲治疗(10.4%)。羟基脲的适应证为:62例(57.9%)反复贫血发作(≥4次)、19例(17.8%)神经危象、17例(15.9%)反复痛性危象、4例(3.7%)阴茎异常勃起、2例(1.9%)急性胸综合征。其中89.5%的患儿治疗依从性良好。大多数痛性发作(88.2%)和贫血发作(88.7%)的患者出现了快速且持久的临床改善,脑血管意外复发2次,阴茎异常勃起和急性胸综合征未复发。从生物学角度来看,胎儿血红蛋白显著增加(从1.2%至16.2%;<0.05)、血红蛋白(从7至8.3g/dl;<0.05)、平均红细胞体积(从80.8至96fl;<0.05),平均白细胞计数(从15,633至9,872/mm;<0.05)和血小板显著减少(从387,002至324,400/mm;<0.05)。观察到的毒性体征主要为呕吐和血小板减少,各2例,1例头痛,另1例中性粒细胞减少。在布拉柴维尔,羟基脲疗法在镰状细胞病儿童中的使用适应证较为常见。主要为反复贫血发作、中风和痛性发作。在资源有限的我国,这些并发症在羟基脲治疗下的良好转归是一种有趣的选择。

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