Efficacy of Anakinra in Refractory Adult-Onset Still's Disease: Multicenter Study of 41 Patients and Literature Review.
Efficacy of Anakinra in Refractory Adult-Onset Still's Disease: Multicenter Study of 41 Patients and Literature Review.
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DOI：
10.1097/md.0000000000001554
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发表时间：
2015-09
期刊：
影响因子：
1.6
通讯作者：
González-Gay MA
中科院分区：
文献类型：
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作者：
Ortiz-Sanjuán F;Blanco R;Riancho-Zarrabeitia L;Castañeda S;Olivé A;Riveros A;Velloso-Feijoo ML;Narváez J;Jiménez-Moleón I;Maiz-Alonso O;Ordóñez C;Bernal JA;Hernández MV;Sifuentes-Giraldo WA;Gómez-Arango C;Galíndez-Agirregoikoa E;Blanco-Madrigal J;Ortiz-Santamaria V;Del Blanco-Barnusell J;De Dios JR;Moreno M;Fiter J;Riscos ML;Carreira P;Rodriguez-Valls MJ;González-Vela MC;Calvo-Río V;Loricera J;Palmou-Fontana N;Pina T;Llorca J;González-Gay MA
成人发病的斯蒂尔氏病（AOSD）通常难以标准治疗。Anakinra （ANK）是一种白细胞介素-1受体拮抗剂，已证明对单个病例和小系列AOSD有效。我们评估了ANK在一系列AOSD患者中的疗效。多中心回顾性开放标签研究。ANK的使用是由于对标准合成免疫抑制药物缺乏疗效，在某些情况下也对至少一种生物制剂缺乏疗效。招募了41名患者（26名女性/15名男性）。他们的平均年龄为34.4±14岁，在ANK发作前，AOSD的中位[四分位间距（IQR）]持续时间为3.5[2-6]年。最常见的临床表现为关节表现（87.8%）、发热（78%）和皮疹（58.5%）。ANK取得了快速和持续的临床和实验室改善。治疗1年后，关节和皮肤表现的频率分别降至41.5%和7.3%，发热从78%降至14.6%，贫血从56.1%降至9.8%，淋巴结病从26.8%降至4.9%。实验室参数也有了显著的改善。强的松的中位[IQR]剂量也从ANK发病时的20 [11.3-47.5]mg/天减少到12个月时的5 [0-10]mg/天。中位[IQR]随访16[5 - 50]个月后，最重要的副作用是皮肤表现（n = 8），轻度白细胞减少（n = 3），肌病（n = 1）和感染（n = 5）。ANK与快速和持续的临床和实验室改善有关，即使在对其他生物制剂无反应的情况下也是如此。然而，关节表现比全身表现更难治性。
Adult-onset Still's disease (AOSD) is often refractory to standard therapy. Anakinra (ANK), an interleukin-1 receptor antagonist, has demonstrated efficacy in single cases and small series of AOSD. We assessed the efficacy of ANK in a series of AOSD patients. Multicenter retrospective open-label study. ANK was used due to lack of efficacy to standard synthetic immunosuppressive drugs and in some cases also to at least 1 biologic agent. Forty-one patients (26 women/15 men) were recruited. They had a mean age of 34.4 ± 14 years and a median [interquartile range (IQR)] AOSD duration of 3.5 [2–6] years before ANK onset. At that time the most common clinical features were joint manifestations 87.8%, fever 78%, and cutaneous rash 58.5%. ANK yielded rapid and maintained clinical and laboratory improvement. After 1 year of therapy, the frequency of joint and cutaneous manifestations had decreased to 41.5% and to 7.3% respectively, fever from 78% to 14.6%, anemia from 56.1% to 9.8%, and lymphadenopathy from 26.8% to 4.9%. A dramatic improvement of laboratory parameters was also achieved. The median [IQR] prednisone dose was also reduced from 20 [11.3–47.5] mg/day at ANK onset to 5 [0–10] at 12 months. After a median [IQR] follow-up of 16 [5–50] months, the most important side effects were cutaneous manifestations (n = 8), mild leukopenia (n = 3), myopathy (n = 1), and infections (n = 5). ANK is associated with rapid and maintained clinical and laboratory improvement, even in nonresponders to other biologic agents. However, joint manifestations are more refractory than the systemic manifestations.