Sinnassamy P, Andre J L, Treize G, Leroy B
Service de Néphrologie Pédiatrique, Hôpital d'Enfants Armand-Trousseau, Paris.
Arch Fr Pediatr. 1993 Mar;50(3):201-8.
The production of recombinant erythropoietin, has made large quantities of pure protein available for clinical studies. Published reports have concentrated on patients with end-stage renal disease, who develop transfusion-dependent anemia.
A total of 58 children aged less than 18 years (mean age: 11.48 +/- 4.62 years) with end-stage renal disease (hereditary in 9, congenital in 21 and acquired in 28) treated between July 1987 and February 1990, were included in the study. The mean duration of dialysis (hemodialysis in 56, peritoneal dialysis in 2) at the onset of the study was 35 +/- 28 months. 3 children had undergone bilateral nephrectomy, and 2 were infected with HIV. 54 children were given a phosphate-binder, 10 were given iron and 20 were given folic acid supplements. During the 6 months preceding the study, 51 patients received at least one blood transfusion (40 received 1 to 4 and 11 received 5 to 9 transfusions of packed red blood cells). Recombinant human erythropoietin (rHu EPO) (40 units/kg) was given intravenously three times per week for at least 6 weeks, with the exception of the first 9 patients who were given 80-100 units/kg. When the hematocrit increased less than 0.5% per week (or 3% for 6 weeks), the dose of rHu EPO was increased in stages, without exceeding 200 units/kg/injection. When the hematocrit reached 30 to 35%, the dose of rHu EPO was decreased by half, then gradually adjusted to maintain the hematocrit within this range.
The mean value of reticulocytosis increased from 4.88 to 10.58% and the hematocrit increased from 19.34 to 29.95% during the study. The patients also reported that their appetites and general condition improved. The need for transfusion dramatically decreased after the first month of treatment. The main adverse effect of the rHu EPO administration was an increase in the number of patients with hypertension (31/45 versus 19/45), indicating the need for good control of blood pressure before treatment.
Erythropoietin treatment increases packed blood volume in a dose-dependent fashion. Most patients need no further transfusion and the quality of life clearly improves.
重组促红细胞生成素的生产使得大量纯蛋白可用于临床研究。已发表的报告主要集中在患有终末期肾病且发展为依赖输血性贫血的患者身上。
本研究纳入了1987年7月至1990年2月期间治疗的58名18岁以下儿童(平均年龄:11.48±4.62岁),他们患有终末期肾病(遗传性9例,先天性21例,后天性28例)。研究开始时透析的平均时长(56例为血液透析,2例为腹膜透析)为35±28个月。3名儿童接受了双侧肾切除术,2名感染了HIV。54名儿童服用了磷结合剂,10名服用了铁剂,20名服用了叶酸补充剂。在研究前的6个月里,51名患者至少接受了一次输血(40名接受了1至4次,11名接受了5至9次浓缩红细胞输血)。重组人促红细胞生成素(rHu EPO)(40单位/千克)每周静脉注射三次,至少持续6周,但最初的9名患者给予的剂量为80 - 100单位/千克。当血细胞比容每周增加少于0.5%(或6周内增加少于3%)时,rHu EPO的剂量逐步增加,但每次注射不超过200单位/千克。当血细胞比容达到30%至35%时,rHu EPO的剂量减半,然后逐渐调整以将血细胞比容维持在该范围内。
在研究期间,网织红细胞增多的平均值从4.88%增加到10.58%,血细胞比容从19.34%增加到29.95%。患者还报告说他们的食欲和总体状况有所改善。治疗第一个月后对输血的需求显著下降。rHu EPO给药的主要不良反应是高血压患者数量增加(31/45对19/45),这表明在治疗前需要良好地控制血压。
促红细胞生成素治疗以剂量依赖的方式增加红细胞压积。大多数患者无需进一步输血,生活质量明显改善。