Nissenson A R
Department of Medicine, UCLA School of Medicine 90024.
Am J Kidney Dis. 1991 Oct;18(4 Suppl 1):24-33.
Initial experience with recombinant human erythropoietin (rHuEPO, epoetin alfa) was gained through research protocols in which patient selection and management were tightly controlled. When epoetin alfa was approved for use by the Food and Drug Administration (FDA) these constraints were removed. The present study was designed to examine the medical and social impact of epoetin alfa therapy, as well as to document its safety and efficacy as it is used in clinical practice. From 1,000 to 2,000 patients at 100 to 200 centers will be enrolled and monitored for 1 year. Two groups of patients are being studied--those already receiving epoetin alfa at study entry and those new to epoetin alfa therapy. Demographic data are collected initially and detailed outcome data are collected monthly. This interim report presents data from 68 participating dialysis centers on 447 enrolled patients. To date, 89% of the patients are on in-center hemodialysis, while 9% are on home dialysis (79% continuous ambulatory peritoneal dialysis [CAPD], 14% continuous cyclic peritoneal dialysis [CCPD]). Preliminary analyses suggest this population is similar in age and cause of chronic renal failure to the overall US dialysis population, although whites and males are somewhat underrepresented. Of those already on epoetin alfa at study entry, 62% were receiving 3,000 to 9,000 U/wk. In addition, those already on epoetin alfa had better self-rated general health than those new to therapy. Outcomes and epoetin alfa usage patterns are currently being fully analyzed. Three-month follow-up data on a cohort of patients new to epoetin alfa indicate that the average dose in these patients is less than 100 U/kg/wk; two thirds of the doses were administered intravenously. Hematocrit levels increased over the 3 months from a level of 25.1% at entry to 30.6% by month 3. The percentage of cohort patients who were transfusion-independent increased from 85.1% at study entry to 95.7% by month 3. No increases in the incidence of adverse events occurred in the cohort group and no changes in the safety profile were indicated. These preliminary findings suggest that low doses of rHuEPO are being used in general, with a resultant low mean hematocrit. Thus, patients do not seem to be realizing the full potential beneficial effects of this remarkable agent.
对重组人促红细胞生成素（rHuEPO，阿法依泊汀）的初步经验是通过研究方案获得的，在这些方案中，患者的选择和管理受到严格控制。当阿法依泊汀被美国食品药品监督管理局（FDA）批准使用时，这些限制被取消。本研究旨在考察阿法依泊汀治疗的医学和社会影响，并记录其在临床实践中的安全性和疗效。将在100至200个中心招募1000至2000名患者并进行为期1年的监测。正在研究两组患者——研究开始时已接受阿法依泊汀治疗的患者和首次接受阿法依泊汀治疗的患者。最初收集人口统计学数据，每月收集详细的结果数据。这份中期报告展示了来自68个参与研究的透析中心的447名已登记患者的数据。迄今为止，89%的患者接受中心血液透析，而9%的患者接受家庭透析（79%为持续性非卧床腹膜透析[CAPD]，14%为持续性循环腹膜透析[CCPD]）。初步分析表明，尽管白人和男性的比例略低，但该人群在年龄和慢性肾衰竭病因方面与美国总体透析人群相似。在研究开始时已接受阿法依泊汀治疗的患者中，62%的患者每周接受3000至9000单位的治疗。此外，已接受阿法依泊汀治疗的患者自我评估的总体健康状况优于首次接受治疗的患者。目前正在对结果和阿法依泊汀的使用模式进行全面分析。对一组首次接受阿法依泊汀治疗的患者进行的3个月随访数据表明，这些患者的平均剂量低于100单位/千克/周；三分之二的剂量通过静脉给药。血细胞比容水平在3个月内从开始时的25.1%升至第3个月时的30.6%。该队列中无需输血的患者比例从研究开始时的85.1%升至第3个月时的95.7%。该队列组中不良事件的发生率没有增加，安全性方面也没有变化。这些初步研究结果表明，一般使用的是低剂量的rHuEPO，导致平均血细胞比容较低。因此，患者似乎没有充分发挥这种显著药物的潜在有益作用。
