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一名青少年患者的急性间歇性卟啉病:诊断与治疗挑战

Acute Intermittent Porphyria in an Adolescent Patient: Diagnostic and Treatment Challenges.

作者信息

Rudnick Samuel B, West Nicole E, Fong Kelly, Beaven Simon W, VanderVeen Nathan T

机构信息

Pediatrics, University of California Los Angeles, Los Angeles, USA.

Internal Medicine, University of California Los Angeles Vatche & Tamar Manoukian Division of Digestive Diseases, Los Angeles, USA.

出版信息

Cureus. 2024 Nov 29;16(11):e74784. doi: 10.7759/cureus.74784. eCollection 2024 Nov.

Abstract

Acute intermittent porphyria (AIP) is a rare inherited metabolic disorder caused by decreased activity of the enzyme porphobilinogen deaminase in the heme synthesis pathway. This leads to the accumulation of toxic porphyrin precursors, such as porphobilinogen and δ-aminolevulinic acid. Clinical manifestations typically include episodic bouts of severe neurovisceral pain and autonomic dysfunction. These events have various triggers, including fasting, dehydration, hormonal fluctuations, and certain medications, while treatment involves dextrose infusions for mild attacks and intravenous hemin for severe cases. This case highlights a 17-year-old female patient with numerous prior presentations to the healthcare system for intense bouts of abdominal pain, vomiting, and seizure-like activity who was ultimately diagnosed with AIP. Her course was complicated by diagnostic delays and challenges managing severe refractory pain requiring prolonged courses of hemin in addition to a multimodal pain plan. This case highlights the diagnostic complexities and treatment challenges that patients with AIP face when navigating this challenging clinical syndrome and identifies an opportunity for increased awareness to guide the testing and treatment for AIP.

摘要

急性间歇性卟啉病(AIP)是一种罕见的遗传性代谢紊乱疾病,由血红素合成途径中胆色素原脱氨酶活性降低引起。这导致有毒的卟啉前体物质积累,如胆色素原和δ-氨基乙酰丙酸。临床表现通常包括反复发作的严重神经内脏疼痛和自主神经功能障碍。这些发作有多种诱因,包括禁食、脱水、激素波动和某些药物,而治疗轻度发作时采用葡萄糖输注,严重病例则采用静脉注射血红素。本病例突出了一名17岁女性患者,她此前多次因剧烈腹痛、呕吐和癫痫样发作就诊于医疗系统,最终被诊断为AIP。她的病程因诊断延误以及处理严重难治性疼痛面临挑战而复杂化,除了多模式疼痛治疗方案外,还需要长期使用血红素。本病例突出了AIP患者在应对这一具有挑战性的临床综合征时所面临的诊断复杂性和治疗挑战,并确定了提高认识以指导AIP检测和治疗的机会。

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