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卟啉症研究进展。

Update on the Porphyrias.

机构信息

Division of Pulmonary and Critical Care Medicine, Department of Medicine, Massachusetts General Hospital, Boston, Massachusetts, USA; email:

Harvard Medical School, Boston, Massachusetts, USA.

出版信息

Annu Rev Med. 2024 Jan 29;75:321-335. doi: 10.1146/annurev-med-042921-123602. Epub 2023 Aug 4.

Abstract

The porphyrias are a group of rare diseases, each resulting from a defect in a different enzymatic step of the heme biosynthetic pathway. They can be broadly divided into two categories, hepatic and erythropoietic porphyrias, depending on the primary site of accumulation of heme intermediates. These disorders are multisystemic with variable symptoms that can be encountered by physicians in any specialty. Here, we review the porphyrias and describe their clinical presentation, diagnosis, and management. We discuss novel therapies that are approved or in development. Early diagnosis is key for the appropriate management and prevention of long-term complications in these rare disorders.

摘要

卟啉病是一组罕见疾病,每种疾病都是由于血红素生物合成途径中不同酶促步骤的缺陷引起的。它们可以根据血红素中间产物的主要积累部位广泛分为两类,肝性卟啉病和红细胞生成性卟啉病。这些疾病是多系统的,具有各种症状,任何科室的医生都可能遇到。在这里,我们综述了卟啉病,并描述了它们的临床表现、诊断和治疗。我们讨论了已批准或正在开发的新疗法。早期诊断对于这些罕见疾病的适当管理和预防长期并发症至关重要。

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