Sustained response and prevention of damage progression in patients with neonatal-onset multisystem inflammatory disease treated with anakinra: a cohort study to determine three- and five-year outcomes.
Sustained response and prevention of damage progression in patients with neonatal-onset multisystem inflammatory disease treated with anakinra: a cohort study to determine three- and five-year outcomes.
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通过Anakinra治疗的新生儿发作多系统炎症性疾病患者的持续反应和预防损伤进展：一项同类研究，以确定三年和五年结局。
DOI：
10.1002/art.34409
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发表时间：
2012-07
影响因子：
--
通讯作者：
Goldbach-Mansky, Raphaela
中科院分区：
文献类型：
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作者：
Sibley, Cailin H.;Plass, Nikki;Snow, Joseph;Wiggs, Edythe A.;Brewer, Carmen C.;King, Kelly A.;Zalewski, Christopher;Kim, H. Jeffrey;Bishop, Rachel;Hill, Suvimol;Paul, Scott M.;Kicker, Patrick;Phillips, Zachary;Dolan, Joseph G.;Widemann, Brigitte;Jayaprakash, Nalini;Pucino, Frank;Stone, Deborah L.;Chapelle, Dawn;Snyder, Christopher;Butman, John A.;Wesley, Robert;Goldbach-Mansky, Raphaela
阿那白滞素阻断自身炎性综合征患者的白细胞介素-1可减少全身性和器官特异性炎症。然而，长期治疗的影响尚未确定。本研究旨在评估阿那白滞素对NOMID患者临床和实验室结局的长期影响以及安全性。我们对26名年龄在0.80-42.17岁之间的NOMID患者进行了一项队列研究，这些患者在NIH接受了随访，并接受了阿那白滞素1-5 mg/kg/天的治疗，持续至少36个月。通过每日日记、问卷调查和C反应蛋白水平评估疾病活动性。评价了中枢神经系统（CNS）炎症、听力、视力和安全性。观察到日记评分、父母/患者和医生的疾病活动总体评分、父母/患者的疼痛评分和炎症标志物的持续改善（36个月和60个月时均P < 0.001）。在36个月和60个月时，CNS炎症受到抑制，脑脊液白色血细胞计数（分别为P = 0.0026和P = 0.0076）、白蛋白水平和开放压（分别为P = 0.0012和P < 0.001）降低。大多数患者听力稳定或改善。磁共振成像上的耳蜗强化与持续性听力损失相关。视力和周边视力稳定。视神经小与视野差相关。骨病变进展。除病毒感染外的不良事件很少，所有患者都继续接受药物治疗。这些发现表明，阿那白滞素在治疗NOMID中提供了长达5年的持续疗效，需要剂量递增。中枢神经系统、耳和眼的损害进展是可以预防的，但骨的损害进展不是可以预防的。阿那白滞素总体耐受性良好。
Blocking interleukin-1 with anakinra in patients with the autoinflammatory syndrome neonatal-onset multisystem inflammatory disease (NOMID) reduces systemic and organ-specific inflammation. However, the impact of long-term treatment has not been established. This study was undertaken to evaluate the long-term effect of anakinra on clinical and laboratory outcomes and safety in patients with NOMID. We conducted a cohort study of 26 NOMID patients ages 0.80–42.17 years who were followed up at the NIH and treated with anakinra 1–5 mg/kg/day for at least 36 months. Disease activity was assessed using daily diaries, questionnaires, and C-reactive protein level. Central nervous system (CNS) inflammation, hearing, vision, and safety were evaluated. Sustained improvements in diary scores, parent’s/patient’s and physician’s global scores of disease activity, parent’s/patient’s pain scores, and inflammatory markers were observed (all P < 0.001 at 36 and 60 months). At 36 and 60 months, CNS inflammation was suppressed, with decreased cerebrospinal fluid white blood cell counts (P = 0.0026 and P = 0.0076, respectively), albumin levels, and opening pressures (P = 0.0012 and P < 0.001, respectively). Most patients showed stable or improved hearing. Cochlear enhancement on magnetic resonance imaging correlated with continued hearing loss. Visual acuity and peripheral vision were stable. Low optic nerve size correlated with poor visual field. Bony lesions progressed. Adverse events other than viral infections were rare, and all patients continued to receive the medication. These findings indicate that anakinra provides sustained efficacy in the treatment of NOMID for up to 5 years, with the requirement of dose escalation. Damage progression in the CNS, ear, and eye, but not bone, is preventable. Anakinra is well tolerated overall.
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慢性婴儿神经系统，皮肤，关节综合征的患者NLRP3体细胞镶嵌的发病率很高：国际多中心协作研究的结果。
影响因子：
--
作者：
Tanaka, Naoko;Izawa, Kazushi;Saito, Megumu K.;Sakuma, Mio;Oshima, Koichi;Ohara, Osamu;Nishikomori, Ryuta;Morimoto, Takeshi;Kambe, Naotomo;Goldbach-Mansky, Raphaela;Aksentijevich, Ivona;de Saint Basile, Genevieve;Neven, Benedicte;van Gijn, Marielle;Frenkel, Joost;Arostegui, Juan I.;Yaguee, Jordi;Merino, Rosa;Ibanez, Mercedes;Pontillo, Alessandra;Takada, Hidetoshi;Imagawa, Tomoyuki;Kawai, Tomoki;Yasumi, Takahiro;Nakahata, Tatsutoshi;Heike, Toshio
通讯作者：
Heike, Toshio
影响因子：
2.5
作者：
Crossman, David C.;Morton, Allison C.;Gunn, Julian P.;Greenwood, John P.;Hall, Alistair S.;Fox, Keith Aa;Lucking, Andrew J.;Flather, Marcus D.;Lees, Belinda;Foley, Claire E.
通讯作者：
Foley, Claire E.
影响因子：
158.5
作者：
Lachmann, Helen J.;Kone-Paut, Isabelle;Hawkins, Philip N.
通讯作者：
Hawkins, Philip N.
影响因子：
3.3
作者：
Fox, Elizabeth;Jayaprakash, Nalini;Goldbach-Mansky, Raphaela
通讯作者：
Goldbach-Mansky, Raphaela
DOI：
10.1084/jem.20050473
发表时间：
2005-05-02
期刊：
The Journal of experimental medicine
影响因子：
--
作者：
Pascual V;Allantaz F;Arce E;Punaro M;Banchereau J
通讯作者：
Banchereau J