Raming Kristin, Meinke Jonathan, Ansari Georg, Cancian Giuseppe, Stürzebecher Katja, Staub Daniel, Feltgen Nicolas, Holz Frank G, Pfau Maximilian, Pfau Kristina
Universitäts-Augenklinik Bonn, Universität Bonn, Venusberg-Campus-1, 53127, Bonn, Deutschland.
Augenklinik, Universitätsspital Basel, Universität Basel, Basel, Schweiz.
Ophthalmologie. 2025 Jun;122(6):424-431. doi: 10.1007/s00347-025-02261-3. Epub 2025 Jun 2.
Pseudoxanthoma elasticum (PXE) is an autosomal-recessive multisystem disease characterized by elastic fiber calcification in the skin, the cardiovascular system, and eyes. PXE is caused by biallelic mutations in the ABCC6 gene on chromosome 16, resulting in low inorganic pyrophosphate plasma levels. Typical ocular manifestations result from calcification of Bruch's membrane and include peau d'orange, angioid streaks, and comet-tail lesions. Advances in multimodal imaging have significantly improved the detection of these features. Ocular complications in PXE include the development of secondary neovascularization, which can be treated with anti-vascular endothelial growth factor (VEGF) injections. In cases of suspected PXE, genetic testing and genetic counseling should be performed for diagnostic confirmation. Early referral to a specialized center is essential. Interdisciplinary care and management of complications, particularly in collaboration with angiologists, are crucial for treating this multisystem disorder. Although no approved causal therapy for PXE currently exists to date, accurate and early diagnosis using phenotypic characteristics is critical. Early identification of other affected family members and timely treatment of secondary complications allow for better disease management and offer patients the opportunity to participate in ongoing clinical trials. Promising therapeutic options are currently emerging and may significantly improve management and prognosis in the future.
弹性假黄瘤(PXE)是一种常染色体隐性多系统疾病,其特征为皮肤、心血管系统和眼睛中的弹性纤维钙化。PXE由16号染色体上ABCC6基因的双等位基因突变引起,导致血浆无机焦磷酸水平降低。典型的眼部表现源于布鲁赫膜钙化,包括橘皮样改变、血管样条纹和彗星尾病变。多模态成像技术的进步显著提高了对这些特征的检测能力。PXE的眼部并发症包括继发性新生血管形成,可通过抗血管内皮生长因子(VEGF)注射进行治疗。对于疑似PXE的病例,应进行基因检测和遗传咨询以确诊。尽早转诊至专业中心至关重要。跨学科护理和并发症管理,特别是与血管病专家合作,对于治疗这种多系统疾病至关重要。尽管目前尚无批准用于PXE的病因治疗方法,但利用表型特征进行准确早期诊断至关重要。尽早识别其他受影响的家庭成员并及时治疗继发性并发症,有助于更好地管理疾病,并为患者提供参与正在进行的临床试验的机会。目前正在出现有前景的治疗选择,未来可能会显著改善管理和预后。