Sprague Stuart M, Crawford Paul W, Melnick Joel Z, Strugnell Stephen A, Ali Shaukat, Mangoo-Karim Roberto, Lee Sungchun, Petkovich P Martin, Bishop Charles W
NorthShore University Health System-University of Chicago, Pritzker School of Medicine, Evanston, Ill., USA.
Am J Nephrol. 2016;44(4):316-325. doi: 10.1159/000450766. Epub 2016 Sep 28.
BACKGROUND/AIMS: Vitamin D insufficiency and secondary hyperparathyroidism (SHPT) are associated with increased morbidity and mortality in chronic kidney disease (CKD) and are poorly addressed by current treatments. The present clinical studies evaluated extended-release (ER) calcifediol, a novel vitamin D prohormone repletion therapy designed to gradually correct low serum total 25-hydroxyvitamin D, improve SHPT control and minimize the induction of CYP24A1 and FGF23.
Two identical multicenter, randomized, double-blind, placebo-controlled studies enrolled subjects from 89 US sites. A total of 429 subjects, balanced between studies, with stage 3 or 4 CKD, SHPT and vitamin D insufficiency were randomized 2:1 to receive oral ER calcifediol (30 or 60 µg) or placebo once daily at bedtime for 26 weeks. Most subjects (354 or 83%) completed dosing, and 298 (69%) entered a subsequent open-label extension study wherein ER calcifediol was administered without interruption for another 26 weeks.
ER calcifediol normalized serum total 25-hydroxyvitamin D concentrations (>30 ng/ml) in >95% of per-protocol subjects and reduced plasma intact parathyroid hormone (iPTH) by at least 10% in 72%. The proportion of subjects receiving ER calcifediol who achieved iPTH reductions of ≥30% increased progressively with treatment duration, reaching 22, 40 and 50% at 12, 26 and 52 weeks, respectively. iPTH lowering with ER calcifediol was independent of CKD stage and significantly greater than with placebo. ER calcifediol had inconsequential impact on serum calcium, phosphorus, FGF23 and adverse events.
Oral ER calcifediol is safe and effective in treating SHPT and vitamin D insufficiency in CKD.
背景/目的:维生素D缺乏和继发性甲状旁腺功能亢进(SHPT)与慢性肾脏病(CKD)患者发病率和死亡率增加相关,且当前治疗对此处理不佳。本临床研究评估了缓释(ER)骨化二醇,这是一种新型维生素D原补充疗法,旨在逐步纠正血清总25-羟维生素D水平低下、改善SHPT控制并尽量减少CYP24A1和FGF23的诱导。
两项相同的多中心、随机、双盲、安慰剂对照研究在美国89个地点招募受试者。共有429名受试者在两项研究间均衡分配,患有3期或4期CKD、SHPT和维生素D缺乏,按2:1随机分组,于睡前每日口服一次ER骨化二醇(30或60μg)或安慰剂,共26周。大多数受试者(354名或83%)完成给药,298名(69%)进入后续开放标签延长研究,其中继续不间断服用ER骨化二醇26周。
在超过95%的符合方案受试者中,ER骨化二醇使血清总25-羟维生素D浓度恢复正常(>30 ng/ml),72%的受试者血浆完整甲状旁腺激素(iPTH)降低至少10%。接受ER骨化二醇治疗且iPTH降低≥30%的受试者比例随治疗时间逐渐增加,在12周、26周和52周时分别达到22%、40%和50%。ER骨化二醇降低iPTH与CKD分期无关,且显著优于安慰剂。ER骨化二醇对血清钙、磷、FGF23和不良事件影响不大。
口服ER骨化二醇治疗CKD患者的SHPT和维生素D缺乏安全有效。