Session Type: Poster Session (Monday)
Session Time: 9:00AM-11:00AM
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.
To cite this abstract in AMA style:Michaud M, Sene T, Chazerain P, Urbanski G, Retornaz F, Bienvenu B, Chiche L, Catros F, Sailler L, Alric L, Giraud J, Caudrelier L, Lassoued S, Ancellin S, Lidove O, Gaches F. Effectiveness and Safety of Off-label Use of Tocilizumab in Refractory Autoimmune Diseases: A Multicenter Study [abstract]. Arthritis Rheumatol. 2019; 71 (suppl 10). https://acrabstracts.org/abstract/effectiveness-and-safety-of-off-label-use-of-tocilizumab-in-refractory-autoimmune-diseases-a-multicenter-study/. Accessed May 8, 2021.
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