Proft F, Fleck M, Fiehn C, Schulze-Koops H, Witt M, Dörner T, Henes J C
Sektion Rheumatologie und Klinische Immunologie, Medizinische Klinik IV, Klinikum der Universität München, München, Deutschland.
Abteilung für Rheumatologie, Medizinische Klinik für Gastroenterologie, Infektiologie und Rheumatologie, Campus Benjamin Franklin, Charité Universitätsmedizin Berlin, Berlin, Deutschland.
Z Rheumatol. 2018 Feb;77(1):46-54. doi: 10.1007/s00393-017-0329-x.
To evaluate the safety and efficacy of therapy with biologics in patients with autoinflammatory diseases (AIF) or macrophage activating syndrome (MAS) in a real-life setting in Germany.
The German Register of Autoimmune Diseases 2 (GRAID2) is a retrospective, non-interventional, multicenter registry collecting data from all patients with inflammatory rheumatic diseases refractory to conventional therapy and treated with initial off-label biologics between August 2006 and December 2013. Patients with MAS could be included without prior treatment with a biologic agent.
Data from 26 patients with AIF and 5 with MAS were collected. Of the AIF patients 13 (50%) were diagnosed with adult onset Still's disease (AOSD), 6 (23%) with familial Mediterranean fever (FMF), 4 (15.4%) with tumor necrosis factor-associated periodic syndrome (TRAPS), 1 (3.8%) patient with cryopyrin-associated periodic syndrome (CAPS) and 2 (8%) with undifferentiated fever syndromes. The 5 MAS patients suffered from rheumatoid arthritis (RA) with chronic myeloid leukemia, systemic lupus erythematosus and in 2 cases AOSD. In 1 patient a chronic neurological disease was documented without further differentiaton. All patients with TRAPS were primarily treated with etanercept and all CAPS patients with canakinumab. The AOSD and FMF patients were treated with anakinra as the first line off-label biologic in 6 out of 13 and 5 out of 6 cases, respectively. The MAS patients responded very well or well to therapy in 40% and 60% had a moderate response. There were no non-responders. Within the group of AIF patients the physicians documented a very effective or effective treatment in 38.5%, a moderate response in 30.8% and no response in 30.7%. The tolerance was very good in 5 out of 5 of the MAS and in 92% of the AIF patients.
The data of this retrospective register provide indications for an effective and safe treatment with off-label biologic medication in patients with AIF and MAS in daily practice.
在德国的实际临床环境中评估生物制剂治疗自身炎症性疾病（AIF）或巨噬细胞活化综合征（MAS）患者的安全性和有效性。
德国自身免疫性疾病登记册2（GRAID2）是一项回顾性、非干预性、多中心登记研究，收集了2006年8月至2013年12月期间所有对传统治疗无效且初始接受非标签生物制剂治疗的炎性风湿性疾病患者的数据。MAS患者无需事先接受生物制剂治疗即可纳入。
收集了26例AIF患者和5例MAS患者的数据。在AIF患者中，13例（50%）被诊断为成人斯蒂尔病（AOSD），6例（23%）为家族性地中海热（FMF），4例（15.4%）为肿瘤坏死因子相关周期性综合征（TRAPS），1例（3.8%）为冷吡啉相关周期性综合征（CAPS），2例（8%）为未分化发热综合征。5例MAS患者分别患有类风湿关节炎（RA）合并慢性髓系白血病、系统性红斑狼疮，2例患有AOSD。1例患者记录有慢性神经系统疾病，但未进一步明确诊断。所有TRAPS患者主要接受依那西普治疗，所有CAPS患者接受卡那单抗治疗。AOSD和FMF患者分别有6/13例和5/6例将阿那白滞素作为一线非标签生物制剂治疗。MAS患者40%对治疗反应非常好或良好，60%有中度反应。无无反应者。在AIF患者组中，医生记录38.5%治疗非常有效或有效，30.8%有中度反应，30.7%无反应。5例MAS患者中有5例耐受性非常好，92%的AIF患者耐受性良好。
该回顾性登记研究的数据为AIF和MAS患者在日常临床实践中使用非标签生物制剂进行有效且安全的治疗提供了依据。
