Background/Purpose: There is increasing evidence of Tocilizumab (TCZ) efficacy in refractory auto-immune diseases. The present study aimed at evaluating the real-world experience of using TCZ in patients with refractory autoimmune diseases.

Methods: This is a retrospective, descriptive and multicenter study from 9 departments of Internal Medicine. Demographic, clinical and therapeutic data were collected. Glucocorticoid sparing effect was evaluated with Fisher’s exact test, with statistical significance set at 0.05.

Results: Fifty five patients were included (20 men, 35 women). Mean age was 56 ± 16.7 years (range 23 – 80).
TCZ was used in:
– 15 connective tissue diseases (27%): relapsing polychondritis (n=6), systemic sclerosis (n=5), anti-synthetase syndrome (n=1), and unclassified connective tissue disease (n=3).
– 10 vasculitis (18%): Takayasu arteritis (n=7), Cogan disease (n=1), panarteritis nodosa (n=1), unclassified vasculitis (n=1).
– 12 ophthalmologic conditions (21.8%): non infectious posterior uveitis (n=10), sympathetic ophtalmia (n=1), Basedow orbitopathy (n=1).
– 8 adult-onset Still’s diseases (AOSD) (14.5%).
– 7 cases of polymyalgia rheumatica (12.7%)
– 3 miscellaneous diseases (5.5%): idiopathic AA amyloidosis, multicentric non HHV8 Castelmann disease, Erdheim Chester disease (1 case each).
Mean disease duration of the disease at TCZ initiation was 7 ± 6.3 years. In 48 cases (87%) TCZ was administered for refractory diseases to corticosteroids and/or immunosuppressive drugs. Previous therapies included corticosteroids (83%) , methotrexate (66%), TNF inhibitor drug (44%), azathioprine (20.8%), mycophenolate (12%), cyclophosphamide (8%), rituximab (10%), hydroxychloroquine (6%), anakinra in 2 patients and interferon, dapsone, etoposide, leflunomide, abatacept, salazopyrin or intra-venous immunoglobulin in 1 patient each.
TCZ was associated with methotrexate in 3 cases (5.4%) and route of TCZ administration was mainly intravenous (96%).
At the end of the follow up, 43 patients (78%) were still using TCZ, with a mean follow up period of 21.7 ± 22.6 months (range 1-90). Daily corticosteroid use significantly decrease from 15.3 ± 15.5 mg to 4.47 ± 4.9 mg (p< 0.005).
A complete response was observed in 5 patients (71%) with polymyalgia rheumatica, in 6 patients (75%) with AOSD, in 4 patients (66%) with relapsing polychondritis, in 7 patients (70%) with uveitis, in six patients (85%) with Takayasu arteritis, in 3 patients (75%) with unclassified connective tissue disease and in 3 patients (60%) with systemic sclerosis.
TCZ was withdrawn in 12 patients (2%), because of treatment failure in 3 patients, loss of efficacy in 2 patients or side effect in 7 patients. Side effects occured in 15 patients. They were infection (2 pneumonias, zona, sinus infection), pruritus (n=2), urticaria (n=1), dyslipidemia (n=1), high blood pressure (n=2), infusion-related reaction (n=1), bullous dermatitis (n=1), acute renal failure (n=1), angioedema (n=1), mouth ulcers (n=1).

Conclusion: In conclusion, TCZ is used off-label in daily practice to treat various refractory autoimmune diseases with a good short-term efficacy and tolerance.

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ACR Meeting Abstracts - https://acrabstracts.org/abstract/effectiveness-and-safety-of-off-label-use-of-tocilizumab-in-refractory-autoimmune-diseases-a-multicenter-study/