Open Access

Hans-Peter Tony, Gerd-Rüdiger Burmester, Hendrik Schulze-Koops, Mathias Grunke, Jörg Christoph Henes, Ina Kötter, Judith Haas, Leonore Unger, Svjetlana Lovric, Marion Haubitz, Rebecca Fischer-Betz, Gamal Chehab, Andrea Rubbert-Roth, Christof Specker, Jutta Weinerth, Julia Ulrike Holle, Ulf Müller-Ladner, Ramona König, Christoph Fiehn, Philip Burgwinkel, Klemens Budde, Helmut Sörensen, Michael Meurer, Martin Aringer, Bernd Kieseier, Cornelia Erfurt-Berge, Michael Sticherling, Roland Veelken, Ulf Ziemann, Frank Strutz, Praxis von Wussow, Florian M. P. Meier, Nico Hunzelmann, Nico Schmidt, Raoul Bergner, Andreas Schwarting, Rüdiger Eming, Michael Hertl, Rudolf Stadler, Michael Schwarz-Eywill, Siegfried Wassenberg, Martin Fleck, Claudia Metzler, Uwe Klaus Zettl, Jens Westphal, Stefan Heitmann, Anna Laura Herzog, Heinz Wiendl, Waltraud Jakob, Enno Schmidt, Klaus Freivogel, Thomas Dörner

• Introduction: Evidence from a number of open-label, uncontrolled studies has suggested that rituximab may benefit patients with autoimmune diseases who are refractory to standard-of-care. The objective of this study was to evaluate the safety and clinical outcomes of rituximab in several standard-of-care-refractory autoimmune diseases (within rheumatology, nephrology, dermatology and neurology) other than rheumatoid arthritis or non-Hodgkin's lymphoma in a real-life clinical setting. Methods: Patients who received rituximab having shown an inadequate response to standard-of-care had their safety and clinical outcomes data retrospectively analysed as part of the German Registry of Autoimmune Diseases. The main outcome measures were safety and clinical response, as judged at the discretion of the investigators. Results: A total of 370 patients (299 patient-years) with various autoimmune diseases (23.0% with systemic lupus erythematosus, 15.7% antineutrophil cytoplasmic antibody-associated granulomatous vasculitides, 15.1% multiple sclerosis and 10.0% pemphigus) from 42 centres received a mean dose of 2,440 mg of rituximab over a median (range) of 194 (180 to 1,407) days. The overall rate of serious infections was 5.3 per 100 patient-years during rituximab therapy. Opportunistic infections were infrequent across the whole study population, and mostly occurred in patients with systemic lupus erythematosus. There were 11 deaths (3.0% of patients) after rituximab treatment (mean 11.6 months after first infusion, range 0.8 to 31.3 months), with most of the deaths caused by infections. Overall (n = 293), 13.3% of patients showed no response, 45.1% showed a partial response and 41.6% showed a complete response. Responses were also reflected by reduced use of glucocorticoids and various immunosuppressives during rituximab therapy and follow-up compared with before rituximab. Rituximab generally had a positive effect on patient well-being (physician's visual analogue scale; mean improvement from baseline of 12.1 mm). Conclusions: Data from this registry indicate that rituximab is a commonly employed, well-tolerated therapy with potential beneficial effects in standard of care-refractory autoimmune diseases, and support the results from other open-label, uncontrolled studies.