Arthritis & Rheumatism, Volume 63,
November 2011 Abstract Supplement

Abstracts of the American College of
Rheumatology/Association of Rheumatology Health Professionals
Annual Scientific Meeting
Chicago, Illinois November 4-9, 2011.

Off-Label Use of Biological Therapies in Systemic Autoimmune Diseases.

Araujo,  Ana Carolina, Noronha,  Carla, Grilo,  Ana, Moraes-Fontes,  Maria Francisca, Riso,  Nuno, Riscado,  Manuel Vaz


Patients with systemic autoimmune diseases (AID) may not respond to first line immunosupressants or relapse after initial clinical remission. The emergence of biological therapies has increased therapeutic options despite the lack of controlled trials.


Retrospective study of data from our Autoimmune Disease Clinic's database on the use of biological agents in patients with systemic autoimmune diseases (AID). The analysis included a total of 14 AID and by June 2011, the Database included 133 patients who had been treated with biological agents (16 received infliximab, 29 rituximab, 73 etanercept, 1 golimumab, 6 tocilizumab and 37 adalimumab).


Off-label use of biological agents in 21 patients with AID included systemic lupus erythematosus (eight cases with nephritis and neuropsychiatric manifestations), autoimmune thrombocytopenia (five cases), Behçet disease (two cases) and one patient in each of the following diseases (overlap syndrome consisting of Sjögren's Syndrome with rheumatoid arthritis, Wegener granulomatosis, adult-onset Still's disease, Churg Strauss vasculitis, ulcerative colitis and SAPHO syndrome). Patients calling for off-label use of biological drugs were also refractory to conventional therapies obtaining partial or no response to high dose conventional immunosupressants (namely steroids, full-dose cyclophosphamide and highest tolerated methotrexate dose). Regarding SLE, most patients had already been on cyclophosphamide for at least six months without adequate disease control. Both ANCA-vasculitis patients experienced only a partial response to the traditional treatment with cyclophosphamide and steroids.

The favorable response to Rituximab is highlighted. Five patients required re-treatment once and of these, one patient required a further re-treatment. Three were SLE patients and two had immune thrombocytopenic purpura. Of note, the Behçet's disease patients showed none or very small improvement with anti-TNF blockade.


Current evidence on the use of biological therapies in patients with refractory AID is mainly based on uncontrolled, observational data. In our experience, the best results have been observed in the use of rituximab for refractory SLE nephritis and ITP. Lack of efficacy was demonstrated for infliximab in Behçet's disease. Until future controlled trials confirm the potential use of biological therapies in patients with refractory AID, it is hoped that we can gather national data in a registry for refractory AID, from which further evidence can be drawn to support their use.

To cite this abstract, please use the following information:
Araujo, Ana Carolina, Noronha, Carla, Grilo, Ana, Moraes-Fontes, Maria Francisca, Riso, Nuno, Riscado, Manuel Vaz; Off-Label Use of Biological Therapies in Systemic Autoimmune Diseases. [abstract]. Arthritis Rheum 2011;63 Suppl 10 :1971

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