Department of Dermatology, Mount Sinai School of Medicine, New York, New York.
Department of Dermatopathology, Mount Sinai School of Medicine, New York, New York.
J Am Acad Dermatol. 2011 Aug;65(2):341-348. doi: 10.1016/j.jaad.2010.05.023. Epub 2011 Apr 15.
Pseudoxanthoma elasticum (PXE) is a rare connective tissue disorder involving fragmentation and mineralization of elastic fibers predominantly in the skin, eyes, and cardiovascular system.
The objective of this study was to assess the efficacy of sevelamer hydrochloride on the reversal of elastic fiber calcification and clinical lesions of PXE.
This was a randomized, double-blind, placebo-controlled, two-part prospective study. In the first year, 40 patients with PXE were randomized to receive either sevelamer hydrochloride (800 mg by mouth three times daily) or placebo in a 1:1 ratio. In the second year, all patients received sevelamer hydrochloride (800 mg by mouth three times daily).
In the first year, the placebo and treatment groups' mean calcium scores decreased from 29.52 to 15.97 (41.93% mean improvement) and 27.48 to 16.75 (38.37% mean improvement), respectively. In the second year, the mean calcium scores decreased to 13.36 (53.94%) and 14.03 (51.35%) in these groups. The mean clinical score in the placebo group decreased from 6.25 to 6.05 at year 1 (2% improvement) whereas the mean clinical score in the sevelamer hydrochloride group decreased from 7.10 to 6.55 (7% improvement). In year 2, the scores in the original placebo and sevelamer hydrochloride groups decreased to 5.33 (14% improvement) and 5.72 (19% improvement), respectively.
Magnesium stearate in our placebo and active drugs may have played a confounding role in this study, contributing to the small differences observed in these two groups.
Sevelamer hydrochloride produced a reduction in both calcification levels and clinical scores; however, this difference was not statistically significant compared with placebo. Future clinical studies should examine the inhibitory role and potential therapeutic effect of magnesium in PXE.
弹性假黄瘤（PXE）是一种罕见的结缔组织疾病，主要涉及弹性纤维的碎裂和矿化，发生于皮肤、眼睛和心血管系统。
本研究旨在评估盐酸司维拉姆对逆转 PXE 弹性纤维钙化和临床病变的疗效。
这是一项随机、双盲、安慰剂对照、两部分前瞻性研究。在第 1 年，40 名 PXE 患者被随机分为接受盐酸司维拉姆（800mg，口服，每日 3 次）或安慰剂，比例为 1:1。在第 2 年，所有患者均接受盐酸司维拉姆（800mg，口服，每日 3 次）治疗。
第 1 年，安慰剂组和治疗组的平均钙评分分别从 29.52 降至 15.97（平均改善 41.93%）和 27.48 降至 16.75（平均改善 38.37%）。第 2 年，这两组的平均钙评分分别降至 13.36（53.94%）和 14.03（51.35%）。安慰剂组的平均临床评分从第 1 年的 6.25 降至 6.05（改善 2%），而盐酸司维拉姆组从 7.10 降至 6.55（改善 7%）。第 2 年，原安慰剂组和盐酸司维拉姆组的评分分别降至 5.33（改善 14%）和 5.72（改善 19%）。
我们的安慰剂和活性药物中的硬脂酸镁可能在这项研究中起了混杂作用，导致这两组之间观察到的差异较小。
盐酸司维拉姆可降低钙水平和临床评分；然而，与安慰剂相比，这一差异无统计学意义。未来的临床研究应检查镁在 PXE 中的抑制作用和潜在治疗效果。
