Article
Safety and efficacy of biological therapies in large vessel vasculitis and polymyalgia rheumatica – results from a nationwide german registry (GRAID2)
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Published: | September 1, 2015 |
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Introduction: To evaluate the safety and clinical outcome of biological therapies in patients with large-vessel vasculitides (LVV) or polymyalgia rheumatica (PMR) refractory to standard of care therapy in a real life setting in Germany.
Methods: GRAID 2 (German Register in Autoimmune Diseases 2) is a retrospective, non-interventional multicentre online registry collecting data from all patients with inflammatory rheumatic diseases treated with an initial biological off-label therapy between August 2006 and December 2013. The retrospective documentation comprised case history, diagnosis, course of disease including safety and global efficacy. In LVV and PMR patients, clinical parameters included bilateral proximal myalgia, headache, jaw-claudicatio, scalp-tenderness, visual-deterioration, tender and swollen joint counts, morning stiffness, fatigue, depression, B symptoms and general wellbeing. The laboratory parameters included ESR, CRP, leukocytes and thrombocytes. Additionally, the investigators rated their patients as non- (NR), partial (PR) or complete responders (CR) based on clinical judgements.
Results: Data from 14 patients were collected, 11 (78.6%) with LVV and 3 (21.4%) with isolated PMR, with 10 (71.4%) female and 4 (28.6%) male patients and a mean age of 55.54 +/- 18.40 years. The mean follow-up period was 10.8 +/- 8.52 months resulting in 12.7 patient years of follow-up. 10 patients (71.4%) were treated with tocilizumab (TOC), while 3 patients (21.4%) had infliximab(IFX)-infusions and 1 patient (7%) was treated with rituximab (RTX). On average, all clinical as well as laboratory efficacy parameters improved substantially. After the first application, tolerability of biologicals was assessed as "very good"/"good" by physicians in 92.3% of 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 SAE, requiring ICU-treatment representing a rate of SAEs of 7.9 per 100 patient years.
Conclusion: With known limitations of a retrospective data base, the results of this survey confirm data of smaller case series, suggest a substantial response to biological therapies in patients with treatment refractory LVV or PMR with an overall favorable safety profile.