Division of Pediatric Nephrology, University of Miami Miller School of Medicine, P.O. Box 016960 (M714), Miami, FL, 33101, USA.
Pediatr Nephrol. 2017 Jul;32(7):1103-1108. doi: 10.1007/s00467-017-3675-7. Epub 2017 Apr 27.
The complex pathophysiology of progressive chronic kidney disease (CKD) and the development of mineral and bone disorder, abbreviated as CKD-MBD, is of vital importance to a pediatric patient. Paricalcitol, the 19 nor-1,25(OH)D analogue was shown to be effective and safe in the treatment of secondary hyperparathyroidism (SHPT) in adults almost two decades ago. It also significantly improved survival in dialysis patients compared to the standard calcitriol. The successful treatment of CKD-MBD in children is essential if they are to grow and survive into adulthood. It can be argued that it is more important for children with CKD than adults since they have early and prolonged disease risk exposure. In this issue of Pediatric Nephrology, Webb et.al. report a dual trial of the safety, efficacy, and pharmacokinetics of paricalcitol in children aged 10-16 years with moderate but significant efficacy in meeting the endpoint of >30% decrease in parathyroid hormone (PTH) levels from baseline with minimal adverse events. Much more research needs to be done to expand and develop clinical pharmaceutical trials in the use of paricalcitol in children, especially in the younger age categories. This current study has done much to open the doors for future studies, with the caveat that it has been long coming and much more needs to be done to compensate for this delay in the treatment of children with CKD-MBD and cardiovascular and renal disease progression.
进行性慢性肾脏病(CKD)的复杂病理生理学和矿物质及骨代谢紊乱(CKD-MBD)的发生,对儿科患者至关重要。近二十年前,19-去甲-1,25(OH)2D 类似物帕立骨化醇被证明可有效且安全地治疗成人继发性甲状旁腺功能亢进症(SHPT)。与标准的骨化三醇相比,它还显著改善了透析患者的生存率。如果要让儿童成长并存活到成年期,就必须成功治疗 CKD-MBD。可以说,对于患有 CKD 的儿童来说,这比成人更为重要,因为他们面临着更早和更长时间的疾病风险暴露。在本期《儿科肾脏病学》中,Webb 等人报告了一项帕立骨化醇在 10-16 岁中度但有显著疗效的儿童中的安全性、疗效和药代动力学的双试验,该试验达到了主要终点,即甲状旁腺激素(PTH)水平较基线降低>30%,且仅有轻微不良反应。需要开展更多的研究来扩大和开发帕立骨化醇在儿童中的临床药物试验,特别是在年龄较小的类别中。这项当前的研究为未来的研究打开了大门,但有一个警告,那就是它已经姗姗来迟,为了弥补治疗 CKD-MBD 儿童以及心血管和肾脏疾病进展方面的延误,还需要做更多的工作。