Geary Denis F, Keating Laura E, Vigneux Annette, Stephens Derek, Hébert Diane, Harvey Elizabeth A
Division of Nephrology, and Department of Population Health Sciences, Hospital for Sick Children, Toronto, Ontario, Canada.
Kidney Int. 2005 Oct;68(4):1759-65. doi: 10.1111/j.1523-1755.2005.00592.x.
Darbepoetin alfa use has been reported in 7 children with chronic renal failure (CRF). Our objective was to evaluate the efficacy and safety of darbepoetin and determine a therapeutic dose in a larger sample of children with CRF.
Twenty-six children with chronic renal insufficiency (CRI) GFR <30 mL/min/1.73 m(2), on peritoneal dialysis (PD) or hemodialysis (HD) entered a prospective, open-label study of darbepoetin. Seven ineligible children who underwent the same evaluation were analyzed retrospectively. The starting dose was 0.45 microg/kg/week. IRB/REB approval and informed consent were obtained. The primary outcome measure was hemoglobin (Hb) response within a target range of 10.0 to 12.5 g/dL between 8 and 12 and 20 and 28 weeks.
Thirty-three children (15 CRI, 9 HD, 9 PD; aged 1-17 years) were enrolled in the study. Ten patients dropped out (3 before 12 weeks and 7 before 28 weeks), none due to darbepoetin. Mean Hbs were 11.8 and 11.4 between weeks 8 and 12 and 20 and 28, respectively; the proportion of patients with Hb values >10.0 g/dL was 97% and 91% in the same intervals. No effect of grouping patients into CRI, HD, or PD or prospective versus retrospective was observed. One of 13 serious adverse events (hypertension) was possibly related to darbepoetin; 8/14 children reported injection-site pain. At 12 and 28 weeks, respectively, 73% and 87% were receiving darbepoetin less than once weekly.
A dose approximating 0.5 microg/kg/week of darbepoetin effectively treats anemia in children with chronic renal failure; for many, this may be proportionately increased and injected less than once weekly.
据报道，7例慢性肾衰竭（CRF）儿童使用了阿法达贝泊汀。我们的目的是在更大样本的CRF儿童中评估阿法达贝泊汀的疗效和安全性，并确定治疗剂量。
26例慢性肾功能不全（CRI）、肾小球滤过率（GFR）<30 mL/min/1.73 m²、接受腹膜透析（PD）或血液透析（HD）的儿童进入一项关于阿法达贝泊汀的前瞻性、开放标签研究。对7例接受相同评估但不符合条件的儿童进行回顾性分析。起始剂量为0.45μg/kg/周。获得了机构审查委员会/研究伦理委员会的批准并取得了知情同意。主要结局指标是在第8至12周以及第20至28周期间血红蛋白（Hb）在10.0至12.5 g/dL目标范围内的反应。
33例儿童（15例CRI、9例HD、9例PD；年龄1至17岁）纳入研究。10例患者退出（3例在12周前，7例在28周前），均非因阿法达贝泊汀退出。在第8至12周以及第20至28周期间，平均Hb分别为11.8和11.4；在相同时间段内，Hb值>10.0 g/dL的患者比例分别为97%和91%。未观察到将患者分为CRI、HD或PD组以及前瞻性与回顾性分组的影响。13例严重不良事件中有1例（高血压）可能与阿法达贝泊汀有关；14例儿童中有8例报告有注射部位疼痛。在第12周和第28周时，分别有73%和87%的患者接受阿法达贝泊汀的频率低于每周一次。
阿法达贝泊汀剂量约为0.5μg/kg/周可有效治疗慢性肾衰竭儿童的贫血；对许多儿童而言，该剂量可按比例增加且注射频率低于每周一次。
