Prediction of adverse outcomes in children with sickle cell disease
Prediction of adverse outcomes in children with sickle cell disease
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DOI：
10.1056/nejm200001133420203
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发表时间：
2000-01-13
影响因子：
158.5
通讯作者：
Kinney, TR
中科院分区：
文献类型：
--
作者：
Miller, ST;Sleeper, LA;Kinney, TR
背景：如果能够识别患有镰状细胞性贫血的婴儿，这些婴儿在以后的生活中可能会出现严重的并发症，这将有助于准确的预测和调整治疗，以匹配疾病相关的风险，并促进临床试验的规划。我们试图通过跟踪 392 名从婴儿期到 10 岁左右患有镰状细胞病的儿童的临床病程来定义此类婴儿的特征。 方法：我们分析了 392 名在 6 个月前被诊断为纯合镰状细胞贫血或镰状细胞-β(0)-地中海贫血的婴儿的记录，并前瞻性地记录了他们的全面临床和实验室数据；可用数据的平均值 (+/-SD) 为 10.0+/-4.8 年。对两岁前获得的结果进行评估，以确定它们是否可以预测以后的生活结果。结果：在队列中的 392 名婴儿中，70 名（18%）随后出现不良结果，定义为死亡（18 名患者[26%]）、中风（25 名患者[36%]）、频繁疼痛（17 名患者[24%]）或复发性急性胸部综合征（10 名患者[14%]）。通过多变量分析，我们发现了不良结果的三个具有统计学意义的预测因素：一岁之前发生指炎（定义为手或脚的疼痛和压痛）（不良结果的相对风险，2.55；95%置信区间，1.39至4.67），血红蛋白水平低于7克/分升（相对风险，2.47；95%置信区间， 1.14 至 5.33），以及在没有感染的情况下白细胞增多（相对风险，1.80；95% 置信区间，1.05 至 3.09）。结论：生命头两年可能出现的镰状细胞病的三种易于识别的表现（指炎、严重贫血和白细胞增多）有助于预测生命后期患严重镰状细胞病的可能性。 （N Engl J Med 2000；342：83-9。）(C) 2000，马萨诸塞州医学会。
Background: The ability to identify infants with sickle cell anemia who are likely to have severe complications later in life would permit accurate prognostication and tailoring of therapy to match disease-related risks and facilitate planning of clinical trials. We attempted to define the features of such babies by following the clinical course of 392 children with sickle cell disease from infancy to about the age of 10 years.Methods: We analyzed the records of 392 infants who received the diagnosis of homozygous sickle cell anemia or sickle cell-beta(0)-thalassemia before the age of six months and for whom comprehensive clinical and laboratory data were recorded prospectively; data were available for a mean (+/-SD) of 10.0+/-4.8 years. Results obtained before the age of two years were evaluated to determine whether they predicted the outcome later in life.Results: Of the 392 infants in the cohort, 70 (18 percent) subsequently had an adverse outcome, defined as death (18 patients [26 percent]), stroke (25 [36 percent]), frequent pain (17 [24 percent]), or recurrent acute chest syndrome (10 [14 percent]). Using multivariate analysis, we found three statistically significant predictors of an adverse outcome: an episode of dactylitis (defined as pain and tenderness in the hands or feet) before the age of one year (relative risk of an adverse outcome, 2.55; 95 percent confidence interval, 1.39 to 4.67), a hemoglobin level of less than 7 g per deciliter (relative risk, 2.47; 95 percent confidence interval, 1.14 to 5.33), and leukocytosis in the absence of infection (relative risk, 1.80; 95 percent confidence interval, 1.05 to 3.09).Conclusions: Three easily identifiable manifestations of sickle cell disease that may appear in the first two years of life (dactylitis, severe anemia, and leukocytosis) can help to predict the possibility of severe sickle cell disease later in life. (N Engl J Med 2000;342:83-9.) (C) 2000, Massachusetts Medical Society.