弹性假性黄瘤(PXE)的特征是低水平的无机焦磷酸盐(PPi)和高活性的组织非特异性碱性磷酸酶(TNAP)。兰索拉唑是TNAP的部分抑制剂。目的是研究兰索拉唑是否会增加PXE患者的血浆PPi水平。我们进行了2×2随机分组,双盲,PXE患者的安慰剂对照交叉试验。患者在8周的两个序列中被分配30毫克/天的兰索拉唑或安慰剂。主要结果是安慰剂和兰索拉唑阶段之间血浆PPi水平的差异。29名患者被纳入研究。第一次就诊后,有8人死于大流行封锁,还有1人死于胃不耐受,所以20名患者完成了试验。采用广义线性混合模型评价兰索拉唑的疗效。总的来说,兰索拉唑使血浆PPi水平从0.34±0.10µM升高至0.41±0.16µM(p=0.0302),TNAP活性无统计学显著变化。无重要不良事件发生。30mg/天的兰索拉唑能够显着增加PXE患者的血浆PPi;尽管如此,这项研究应该在多中心试验的大量参与者中重复进行,以临床终点为主要结局。
Pseudoxanthoma elasticum (PXE) is characterized by low levels of inorganic pyrophosphate (PPi) and a high activity of tissue-nonspecific alkaline phosphatase (TNAP). Lansoprazole is a partial inhibitor of TNAP. The aim was to investigate whether lansoprazole increases plasma PPi levels in subjects with PXE. We conducted a 2 × 2 randomized, double-blind, placebo-controlled crossover
trial in patients with PXE. Patients were allocated 30 mg/day of lansoprazole or a placebo in two sequences of 8 weeks. The primary outcome was the differences in plasma PPi levels between the placebo and lansoprazole phases. 29 patients were included in the
study. There were eight drop-outs due to the pandemic lockdown after the first visit and one due to gastric intolerance, so twenty patients completed the
trial. A generalized linear mixed model was used to evaluate the effect of lansoprazole. Overall, lansoprazole increased plasma PPi levels from 0.34 ± 0.10 µM to 0.41 ± 0.16 µM (p = 0.0302), with no statistically significant changes in TNAP activity. There were no important adverse events. 30 mg/day of lansoprazole was able to significantly increase plasma PPi in patients with PXE; despite this, the
study should be replicated with a large number of participants in a multicenter
trial, with a clinical end point as the primary outcome.