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The Lancet:羟基脲可缓解婴幼儿贫血并发症

来源:新华网 2011-05-17 18:30

正常血细胞与镰刀形贫血血细胞

英国医学期刊《柳叶刀》刊登的一项新研究成果显示,对于那些患镰刀型细胞贫血症的婴幼儿来说,可使用一种名为羟基脲的药物来减少疼痛等并发症。这是医学界首次试验该药对婴幼儿的疗效,结果显示副作用不大,可安全使用。

美国圣祖德儿童研究医院等机构的研究人员报告说,参加试验者为大约200名年龄在9个月到18个月的婴幼儿患者,其中一部分婴幼儿服用羟基脲,另一部分婴幼儿作为对照组。随后的跟踪调查发现,那些服用羟基脲的婴幼儿会显著减少因镰刀型细胞贫血症而出现疼痛、指趾炎等并发症的频率。

镰刀型细胞贫血症是一种遗传疾病,患者血液中的红细胞会呈镰刀型,而不是像正常人的红细胞那样呈圆饼型。镰刀型细胞也不像正常细胞那样有弹性,因此不能通过某些较窄的血管,会引起贫血及一系列并发症,如疼痛、脾和肾等器官受损,在婴幼儿中还会引起指趾炎。这种疾病目前不能治愈,只能使用药物来缓解症状。

此前羟基脲已被用于成人治疗镰刀型细胞贫血症,它可以帮助缓解一些并发症。但年龄很小的婴幼儿是否也能用这种药,此前缺少相关证据。本次研究显示,该药在婴幼儿身上引起的副作用主要是造成一种白细胞减少,影响不是很大,因此可以安全使用。(生物谷Bioon.com)

生物谷推荐原文:

The Lancet    DOI:10.1016/S0140-6736(11)60355-3

Hydroxycarbamide in very young children with sickle-cell anaemia: a multicentre, randomised, controlled trial (BABY HUG)

Prof Winfred C Wang MD , Prof Russell E Ware MD , Prof Scott T Miller MD , Prof Rathi V Iyer MD, Prof James F Casella MD , Caterina P Minniti MD, Prof Sohail Rana MD , Courtney D Thornburg MD , Prof Zora R Rogers MD , Ram V Kalpatthi MD, Prof Julio C Barredo MD , R Clark Brown MD , Prof Sharada A Sarnaik MD l, Prof Thomas H Howard MD , Lynn W Wynn MSN , Prof Abdullah Kutlar MD , Prof F Daniel Armstrong PhD , Beatrice A Files MD , Jonathan C Goldsmith MD, Myron A Waclawiw PhD, Xiangke Huang MD ,

Background Sickle-cell anaemia is associated with substantial morbidity from acute complications and organ dysfunction beginning in the first year of life. Hydroxycarbamide substantially reduces episodes of pain and acute chest syndrome, admissions to hospital, and transfusions in adults with sickle-cell anaemia. We assessed the effect of hydroxycarbamide therapy on organ dysfunction and clinical complications, and examined laboratory findings and toxic effects. Methods This randomised trial was undertaken in 13 centres in the USA between October, 2003, and September, 2009. Eligible participants had haemoglobin SS (HbSS) or haemoglobin Sβ0thalassaemia, were aged 9—18 months at randomisation, and were not selected for clinical severity. Participants received liquid hydroxycarbamide, 20 mg/kg per day, or placebo for 2 years. Randomisation assignments were generated by the medical coordinating centre by a pre-decided schedule. Identical appearing and tasting formulations were used for hydroxycarbamide and placebo. Patients, caregivers, and coordinating centre staff were masked to treatment allocation. Primary study endpoints were splenic function (qualitative uptake on 99Tc spleen scan) and renal function (glomerular filtration rate by 99mTc-DTPA clearance). Additional assessments included blood counts, fetal haemoglobin concentration, chemistry profiles, spleen function biomarkers, urine osmolality, neurodevelopment, transcranial Doppler ultrasonography, growth, and mutagenicity. Study visits occurred every 2—4 weeks. Analysis was by intention to treat. The trial is registered with ClinicalTrials.gov, number NCT00006400. Findings 96 patients received hydroxycarbamide and 97 placebo, of whom 83 patients in the hydroxycarbamide group and 84 in the placebo group completed the study. Significant differences were not seen between groups for the primary endpoints (19 of 70 patients with decreased spleen function at exit in the hydroxycarbamide group vs 28 of 74 patients in the placebo group, p=0·21; and a difference in the mean increase in DTPA glomerular filtration rate in the hydroxycarbamide group versus the placebo group of 2 mL/min per 1·73 m2, p=0·84). Hydroxycarbamide significantly decreased pain (177 events in 62 patients vs 375 events in 75 patients in the placebo group, p=0·002) and dactylitis (24 events in 14 patients vs 123 events in 42 patients in the placebo group, p<0·0001), with some evidence for decreased acute chest syndrome, hospitalisation rates, and transfusion. Hydroxyurea increased haemoglobin and fetal haemoglobin, and decreased white blood-cell count. Toxicity was limited to mild-to-moderate neutropenia. Interpretation On the basis of the safety and efficacy data from this trial, hydroxycarbamide can now be considered for all very young children with sickle-cell anaemia. Funding The US National Heart, Lung, and Blood Institute; and the National Institute of Child Health and Human Development.

 

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