PAIN IN SICKLE-CELL DISEASE - RATES AND RISK-FACTORS
PAIN IN SICKLE-CELL DISEASE - RATES AND RISK-FACTORS
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DOI：
10.1056/nejm199107043250103
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发表时间：
1991-07-04
影响因子：
158.5
通讯作者：
KINNEY, TR
中科院分区：
文献类型：
--
作者：
PLATT, OS;THORINGTON, BD;KINNEY, TR
研究背景和方法。急性发作的疼痛是镰状细胞病的主要症状，但人们对这些发作的流行病学特征或其危险因素知之甚少，也不知道此类发作发生率高的患者是否过早死亡。我们前瞻性地研究了3578名患者的镰状细胞疾病的自然病史，这些患者从新生儿到66岁以下的人，他们在美国各地的临床中心接受了跟踪调查。在18356个病人年中，有12290次疼痛发作。镰状细胞性贫血为0.8例/病人年，镰状β-0地中海贫血为1.0例/病人年，血红蛋白SC病和镰状β+地中海贫血为0.4例/病人年。在这四组中，这一比例差异很大--例如，39%的镰状细胞性贫血患者没有疼痛发作，1%的患者每年有六次以上的疼痛发作。每年发作3到10次的患者中有5.2%的人有32.9%的发作。在20岁以上的镰状细胞性贫血患者中，那些疼痛发作频率高的患者往往比那些疼痛发作频率低的患者更早死亡。高发病率与高红细胞压积和低胎儿血红蛋白水平有关。甲型地中海贫血除了与血细胞比容增加有关外，对疼痛没有影响。“疼痛率”(每年发作次数)是临床严重程度的衡量标准，与20岁以上镰状细胞性贫血患者的早期死亡相关。即使胎儿的血红蛋白水平较低，也可以预测，该水平的小幅增加可能会对疼痛发生率产生缓解作用，并最终可能提高存活率。这一结果对于研究羟基脲和其他旨在提高胎儿血红蛋白水平的治疗方法的研究人员来说尤其令人鼓舞。
Background and Methods. Acute episodes of pain are the principal symptom of sickle cell disease, but little is known about the epidemiologic features of these episodes or risk factors for them, nor is it known whether patients with high rates of such episodes die prematurely. We prospectively studied the natural history of sickle cell disease in 3578 patients ranging from newborns to persons up to 66 years old who were followed at clinical centers across the United States.Results. There were 12,290 episodes of pain in 18,356 patient-years. The average rate was 0.8 episode per patient-year in sickle cell anemia, 1.0 episode per patient-year in sickle beta-0-thalassemia, and 0.4 episode per patient-year in hemoglobin SC disease and sickle beta+-thalassemia. The rate varied widely within each of thesefour groups - e.g., 39 percent of patients with sickle cell anemia had no episodes of pain, and 1 percent had more than six episodes per year. The 5.2 percent of patients with 3 to 10 episodes per year had 32.9 percent of all episodes. Among patients with sickle cell anemia who were more than 20 years old, those with high rates of pain episodes tended to die earlier than those with low rates. High rates were associated with a high hematocrit and low fetal hemoglobin levels. Alpha-Thalassemia had no effect on pain apart from its association with an increased hematocrit.Conclusions. The "pain rate" (episodes per year) is a measure of clinical severity and correlates with early death in patients with sickle cell anemia over the age of 20. Even when the fetal hemoglobin level is low, one can predict that small increments in the level may have an ameliorating effect on the pain rate and may ultimately improve survival. This outcome is particularly encouraging to investigators studying hydroxyurea and other treatments designed to increase the fetal hemoglobin level.