Upadacitinib in a Refractory Case of Alpha-1 Antitrypsin Deficiency-Related Panniculitis

乌帕替尼治疗难治性α1-抗胰蛋白酶缺乏相关性脂膜炎

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Abstract

Alpha-1 antitrypsin (AAT) deficiency is a rare inherited disorder that can lead to emphysema, liver cirrhosis, or, more rarely, panniculitis. Treatment for AAT deficiency-associated panniculitis is limited and often unsatisfactory. We report the case of a 62-year-old man with recurrent, painful panniculitis lesions on the thighs and hips, associated with fever and weight loss. Laboratory tests revealed low serum AAT levels (0.56 g/L) and elevated C-reactive protein (11.3 mg/L). PiSZ phenotyping confirmed the diagnosis of AAT deficiency. Standard treatments were ineffective or contraindicated. Upadacitinib, a JAK1 inhibitor, was initiated at 45 mg/day for three months, leading to a substantial reduction in flare frequency and pain, with lesions healing more rapidly, and a sustained clinical benefit persisting for three months after discontinuation. This is the first reported case of AAT deficiency-associated panniculitis treated with upadacitinib. Although full remission was not achieved, symptom improvement and lack of adverse events suggest a potential role for JAK inhibitors in such cases. Upadacitinib may represent a novel therapeutic approach for AAT deficiency-related panniculitis.

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