Neonatal-onset multisystem inflammatory disease responsive to interleukin-1beta inhibition.
Обработка статьи · этапы, ошибки и управление
Ошибки и ограничения
История · было → стало
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Goldbach-Mansky Raphaela, Dailey Natalie J, Canna Scott W, Gelabert Ana, Jones Janet, Rubin Benjamin I, Kim H Jeffrey, Brewer Carmen, Zalewski Christopher, Wiggs Edythe, Hill Suvimol, Turner Maria L, Karp Barbara I, Aksentijevich Ivona, Pucino Frank, Penzak Scott R, Haverkamp Margje H, Stein Leonard, Adams Barbara S, Moore Terry L, Fuhlbrigge Robert C, Shaham Bracha, Jarvis James N, O'Neil Kathleen, Vehe Richard K, Beitz Laurie O, Gardner Gregory, Hannan William P, Warren Robert W, Horn William, Cole Joe L, Paul Scott M, Hawkins Philip N, Pham Tuyet Hang, Snyder Christopher, Wesley Robert A, Hoffmann Steven C, Holland Steven M, Butman John A, Kastner Daniel L
Аннотация
METHODS: We selected 18 patients with neonatal-onset multisystem inflammatory disease (12 with identifiable CIAS1 mutations) to receive anakinra, an interleukin-1-receptor antagonist (1 to 2 mg per kilogram of body weight per day subcutaneously). In 11 patients, anakinra was withdrawn at three months until a flare occurred. The primary end points included changes in scores in a daily diary of symptoms, serum levels of amyloid A and C-reactive protein, and the erythrocyte sedimentation rate from baseline to month 3 and from month 3 until a disease flare.
RESULTS: All 18 patients had a rapid response to anakinra, with disappearance of rash. Diary scores improved (P<0.001) and serum amyloid A (from a median of 174 mg to 8 mg per liter), C-reactive protein (from a median of 5.29 mg to 0.34 mg per deciliter), and the erythrocyte sedimentation rate decreased at month 3 (all P<0.001), and remained low at month 6. Magnetic resonance imaging showed improvement in cochlear and leptomeningeal lesions as compared with baseline. Withdrawal of anakinra uniformly resulted in relapse within days; retreatment led to rapid improvement. There were no drug-related serious adverse events.
CONCLUSIONS: Daily injections of anakinra markedly improved clinical and laboratory manifestations in patients with neonatal-onset multisystem inflammatory disease, with or without CIAS1 mutations. (ClinicalTrials.gov number, NCT00069329 [ClinicalTrials.gov].).