Henes J C, Schulze-Koops H, Witt M, Tony H P, Mueller F, Grunke M, Czihal M, Dörner T, Proft F
Abteilung für Onkologie, Hämatologie, klinische Immunologie, Rheumatologie, Pulmologie, Medizinische Klinik II, Universität Tübingen, Tübingen, Deutschland.
Sektion Rheumatologie und Klinische Immunologie, Medizinische Klinik und Poliklinik IV, Klinikum, Universität München, München, Deutschland.
Z Rheumatol. 2018 Feb;77(1):12-20. doi: 10.1007/s00393-017-0325-1.
To evaluate the safety and clinical outcome of biological therapies in patients with large vessel vasculitis (LVV) or polymyalgia rheumatica (PMR) refractory to standard of care therapy in a real-life setting in Germany.
GRAID 2 (German Registry in Autoimmune Diseases 2) is a retrospective, noninterventional, multicenter registry collecting data from all patients with inflammatory rheumatic diseases refractory to conventional therapy treated with an initial off-label biological between August 2006 and December 2013. The retrospective documentation comprised case history, diagnosis, course of disease including safety and overall efficacy.
Data from 14 patients were collected, 11 with LVV (78.6%) and 3 with isolated PMR (21.4%). Ten patients were treated with tocilizumab (71.4%), while 3 patients received infliximab infusions (21.4%) and 1 patient was treated with rituximab (7.1%). All clinical as well as laboratory efficacy parameters improved substantially. After the first application, tolerability of biologicals was assessed as "very good"/"good" by the physicians in 92.3% of the patients. Altogether, 8 adverse events (AEs) occurred in 4 patients including 3 infections (1 urogenital infection, 2 diverticulitis) representing a rate of 23.6 infections per 100 patient-years. One of these infections (diverticulitis under infliximab treatment) was rated as serious AE, requiring ICU treatment representing a rate of serious AEs of 7.9 per 100 patient-years. No deaths occurred during the observation period.
With known limitations of a retrospective database, the results of this survey confirm data of smaller case series and proof-of-concept studies and suggest a substantial response to biological therapies in patients with otherwise refractory LVV or PMR with no new safety signals.
在德国的实际临床环境中，评估生物疗法对标准治疗无效的大血管血管炎（LVV）或风湿性多肌痛（PMR）患者的安全性和临床疗效。
GRAID 2（德国自身免疫性疾病登记2）是一项回顾性、非干预性、多中心登记研究，收集2006年8月至2013年12月期间所有对传统治疗无效的炎性风湿性疾病患者的数据，这些患者最初接受了未按药品说明书使用的生物制剂治疗。回顾性记录包括病史、诊断、疾病病程，包括安全性和总体疗效。
收集了14例患者的数据，其中11例为大血管血管炎（78.6%），3例为孤立性风湿性多肌痛（21.4%）。10例患者接受了托珠单抗治疗（71.4%），3例患者接受了英夫利昔单抗输注（21.4%），1例患者接受了利妥昔单抗治疗（7.1%）。所有临床和实验室疗效参数均有显著改善。首次用药后，92.3%的患者的医生将生物制剂的耐受性评估为“非常好”/“好”。共有4例患者发生了8起不良事件（AE），包括3起感染（1起泌尿生殖系统感染，2起憩室炎），每100患者年的感染率为23.6起。其中1例感染（英夫利昔单抗治疗下的憩室炎）被评为严重不良事件，需要重症监护治疗，严重不良事件发生率为每100患者年7.9起。观察期内无死亡病例。
尽管本回顾性数据库存在已知局限性，但本调查结果证实了较小病例系列和概念验证研究的数据，并表明生物疗法对其他治疗无效的大血管血管炎或风湿性多肌痛患者有显著疗效，且无新的安全信号。
