MORTALITY IN SICKLE-CELL DISEASE - LIFE EXPECTANCY AND RISK-FACTORS FOR EARLY DEATH
MORTALITY IN SICKLE-CELL DISEASE - LIFE EXPECTANCY AND RISK-FACTORS FOR EARLY DEATH
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DOI：
10.1056/nejm199406093302303
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发表时间：
1994-06-09
影响因子：
158.5
通讯作者：
KLUG, PP
中科院分区：
文献类型：
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作者：
PLATT, OS;BRAMBILLA, DJ;KLUG, PP
背景镰状细胞病（镰状细胞贫血、镰状细胞血红蛋白C病和镰状细胞β地中海贫血）患者的预期寿命和早期死亡的危险因素的信息是为患者提供咨询、靶向治疗和设计临床试验所必需的。我们随访了3764例患者，年龄范围从出生到66岁，以确定预期寿命并计算死亡时的中位年龄。此外，我们调查了研究期间死亡的所有209例成人患者的死亡情况，并使用比例风险回归分析确定了964例随访至少两年的镰状细胞性贫血成人患者早期死亡的危险因素。在患有镰状细胞性贫血（镰状血红蛋白纯合子）的儿童和成人中，男性死亡的中位年龄为42岁，女性为48岁。在患有镰状细胞血红蛋白C疾病的患者中，男性死亡的中位年龄为60岁，女性为68岁。在患有镰状细胞病的成年人中，18%的死亡发生在明显的器官衰竭患者中，主要是肾衰竭。33%的人在临床上没有器官衰竭，但在急性镰状危象中死亡（78%的人有疼痛、胸部综合征或两者兼而有之; 22%的人有中风）。模型显示，镰状细胞性贫血患者的急性胸部综合征、肾功能衰竭、癫痫发作、基线白细胞计数高于15，000个细胞/立方毫米以及胎儿血红蛋白水平低与早期死亡风险增加相关。50%的镰状细胞性贫血患者存活超过50年。大部分死亡者没有明显的慢性器官衰竭，但死于疼痛、胸部综合征或中风的急性发作。早期死亡率在有症状的患者中最高。高水平的胎儿血红蛋白预示着存活率的提高，并且可能是一个可靠的儿童期成人预期寿命的预测者。
Background. Information on life expectancy and risk factors for early death among patients with sickle cell disease (sickle cell anemia, sickle cell-hemoglobin C disease, and the sickle cell-P-thalassemias) is needed to counsel patients, target therapy, and design clinical trials.Methods. We followed 3764 patients who ranged from birth to 66 years of age at enrollment to determine the life expectancy and calculate the median age at death. In addition, we investigated the circumstances of death for all 209 adult patients who died during the study, and used proportional-hazards regression analysis to identify risk factors for early death among 964 adults with sickle cell anemia who were followed for at least two years.Results. Among children and adults with sickle cell anemia (homozygous for sickle hemoglobin), the median age at death was 42 years for males and 48 years for females. Among those with sickle cell-hemoglobin C disease, the median age at death was 60 years for males and 68 years for females. Among adults with sickle cell disease, 18 percent of the deaths occurred in patients with overt organ failure, predominantly renal. Thirty-three percent were clinically free of organ failure but died during an acute sickle crisis (78 percent had pain, the chest syndrome, or both; 22 percent had stroke). Modeling revealed that in patients with sickle cell anemia, the acute chest syndrome, renal failure, seizures, a base-line white-cell count above 15,000 cells per cubic millimeter, and a low level of fetal hemoglobin were associated with an increased risk of early death.Conclusions. Fifty percent of patients with sickle cell anemia survived beyond the fifth decade. A large proportion of those who died had no overt chronic organ failure but died during an acute episode of pain, chest syndrome, or stroke. Early mortality was highest among patients whose disease was symptomatic. A high level of fetal hemoglobin predicted improved survival and is probably a reliable childhood forecaster of adult life expectancy.