目的：初步探索羧基麦芽糖铁（FCM）治疗非透析慢性肾脏病（ND-CKD）贫血患者的有效性与安全性，为FCM在我国ND-CKD贫血患者中的临床应用提供参考。方法：本研究为单中心、前瞻性、单臂研究，纳入25例ND-CKD贫血患者，给予FCM 500 mg或1000 mg单次静脉输注，分别于基线和 FCM治疗的1周后、1月后采集患者外周血，检测血红蛋白、血清铁蛋白、转铁蛋白饱和度，同时观察、记录不良事件发生情况。结果：（1）患者经FCM单次输注后，1周后及1月后的血红蛋白、血清铁蛋白、转铁蛋白饱和度均显著升高（P<0.05）。与FCM治疗1周后相比，1月后的血红蛋白显著升高（P<0.05），血清铁蛋白、转铁蛋白饱和度均显著降低（P<0.05）。（2）2例患者发生低磷血症，1例患者出现过敏性皮疹，1例患者出现输注侧上肢酸胀不适。结论：FCM作为新型快速补铁制剂，可有效改善ND-CKD患者的贫血及铁代谢，短期安全性整体可控。

Objective: To preliminarily explore the efficacy and safety of ferric carboxymaltose (FCM) in the treatment of anemia in patients with non-dialysis chronic kidney disease (ND-CKD), and to provide a reference for the clinical application of FCM in Chinese ND-CKD patients with anemia. Methods: This was a single-center, prospective, single-arm study. A total of 25 ND-CKD patients with anemia were enrolled and received a single intravenous infusion of FCM at a dose of 500 mg or 1000 mg. Peripheral blood samples were collected from the patients at baseline, 1 week, and 1 month after FCM treatment to measure hemoglobin, serum ferritin, and transferrin saturation. Meanwhile, adverse events were observed and recorded. Results: (1) After a single infusion of FCM, the levels of hemoglobin, serum ferritin, and transferrin saturation were significantly increased at one week and one month post-treatment (P<0.05). Compared with the values at one week after FCM treatment, hemoglobin levels at one month were significantly higher (P<0.05), while serum ferritin and transferrin saturation levels were significantly lower (P<0.05). (2) Two patients developed hypophosphatemia, one patient experienced an allergic rash, and one patient reported soreness and discomfort in the upper limb on the infusion side. Conclusion: As a novel and rapid iron supplement preparation, FCM can effectively improve anemia and iron metabolism in patients with ND-CKD, with overall manageable short-term safety.

慢性肾脏病是一类具有高发病率、高死亡率的慢性疾病群。临床上一般采用血液透析和肾脏移植治疗终末期的慢性肾脏病。研究表明,小分子药物或核酸类药物在慢性肾脏病治疗中极具潜力,但是缺乏特异性导致肾脏纤维化治疗效果有限,亟需开发新的治疗策略。纳米载体因具有良好的理化性质,被广泛应用于生物医学领域。本文综述了近年来纳米载体递送小分子或核酸类药物在慢性肾脏病中的研究进展。

Chronic kidney disease (CKD) is a series of chronic disease groups associated with high morbidity and mortality. Hemodialysis and kidney transplantation are the only choices for end-stage renal disease. According to the literature report, it is shown that small molecule and nucleic acid drugs have great potential in CKD treatments with an unsatisfied therapeutic efficacy because of the lack of specific targeting. Thus, it is necessary to develop a new strategy. Nanocarriers have been widely used in biomedical fields due to their excellent physical and chemical properties. In this review, we have summarized the recent advances in applying functional nanocarriers to deliver the small molecules and nucleic acid drugs in the treatment of CKD.