University of Missouri-Kansas City School of Medicine, Children's Mercy Kansas City, Kansas City, MO, 64108, USA.
Department of Pediatrics, University of Virginia School of Medicine, Charlottesville, VA, USA.
Pediatr Nephrol. 2018 Jan;33(1):125-137. doi: 10.1007/s00467-017-3758-5. Epub 2017 Aug 17.
Darbepoetin alfa is a commonly prescribed erythropoiesis-stimulating agent (ESA) for correcting anemia in pediatric chronic kidney disease (CKD) patients. However, little information exists on its use in ESA-naïve patients. This study evaluated the efficacy and safety of darbepoetin alfa in pediatric patients initiating ESA therapy.
One-hundred sixteen pediatric ESA-naïve subjects (aged 1-18 years) with CKD stages 3-5D and hemoglobin (Hb) <10 g/dl from 43 centers in the US, Europe, and Mexico were randomized by age (three groups) and dialysis status (yes vs. no) to receive darbepoetin alfa once weekly (QW) or every 2 weeks (Q2W) subcutaneously (not on dialysis and peritoneal dialysis subjects) and intravenously (hemodialysis subjects). The drug was titrated to achieve Hb levels of 10.0-12.0 g/dl over 25 weeks. Patient- and parent-reported health-related outcomes were measured by the Pediatric Quality of Life Inventory (PedsQL™) in children ≥2 years.
In both groups, mean Hb concentrations increased to ≥11.0 g/dl over the first 3 months of treatment and remained stable within the 10.0-12.0 g/dl target range. The median time to achieve hemoglobin ≥10 g/dl was slightly longer for subjects <12 years (QW and Q2W, both 28 days) vs. those ≥12 years (23 and 22 days, respectively). Adverse event profiles were similar between groups, with QW, four (7%) and Q2W, five (9%). PedsQL™ scores showed modest increases.
Darbepoetin alfa can be safely administered either QW or Q2W to ESA-naïve pediatric patients with CKD-related anemia to achieve Hb targets of 10.0-12.0 g/dl.
达贝泊汀α是一种常用于治疗儿科慢性肾脏病（CKD）患者贫血的促红细胞生成素刺激剂（ESA）。然而，对于 ESA 初治患者，其使用信息较少。本研究评估了达贝泊汀α在开始 ESA 治疗的儿科患者中的疗效和安全性。
116 名来自美国、欧洲和墨西哥 43 个中心的 ESA 初治的 CKD 3-5D 期且血红蛋白（Hb）<10g/dl 的儿科患者（年龄 1-18 岁），按年龄（三组）和透析状态（是/否）随机分组，每周一次（QW）或每两周一次（Q2W）皮下（未透析和腹膜透析患者）或静脉内（血液透析患者）接受达贝泊汀α治疗。在 25 周内将药物滴定至 Hb 水平达到 10.0-12.0g/dl。≥2 岁的儿童通过儿科生活质量量表（PedsQL™）评估患者和家长报告的健康相关结局。
在两组中，治疗的前 3 个月内，平均 Hb 浓度均增加到≥11.0g/dl，并在 10.0-12.0g/dl 的目标范围内保持稳定。与≥12 岁的患者相比，<12 岁的患者达到血红蛋白≥10g/dl 的中位时间更长（QW 和 Q2W 分别为 28 天和 23 天）。两组的不良事件谱相似，QW 组有 4 例（7%），Q2W 组有 5 例（9%）。PedsQL™评分略有增加。
达贝泊汀α可安全地每周一次或每两周一次给药用于 ESA 初治的 CKD 相关贫血儿科患者，以实现 10.0-12.0g/dl 的 Hb 目标。
