Two-year Results of an Open Pilot Study of a 2-treatment Course with Rituximab in Patients with Early Systemic Sclerosis with Diffuse Skin Involvement

• Published in: Journal of rheumatology 2013, Vol.40 (1), p.52-57
• Main Authors: SMITH, Vanessa, PIETTE, Yves, VAN PRAET, Jens T, DECUMAN, Saskia, DESCHEPPER, Ellen, ELEWAUT, Dirk, DE KEYSER, Filip
• Format: Article
• Language: English
• Subjects: Aged; Antibodies, Monoclonal, Murine-Derived - adverse effects; Antibodies, Monoclonal, Murine-Derived - pharmacology; Antibodies, Monoclonal, Murine-Derived - therapeutic use; Antirheumatic Agents - adverse effects; Antirheumatic Agents - pharmacology; Antirheumatic Agents - therapeutic use; Biological and medical sciences; Diseases of the osteoarticular system; Drug Therapy, Combination; Female; Humans; Immunomodulators; Male; Medical sciences; Methylprednisolone - adverse effects; Methylprednisolone - pharmacology; Methylprednisolone - therapeutic use; Middle Aged; Pharmacology. Drug treatments; Pilot Projects; Rituximab; Sarcoidosis. Granulomatous diseases of unproved etiology. Connective tissue diseases. Elastic tissue diseases. Vasculitis; Scleroderma, Diffuse - drug therapy; Severity of Illness Index; Skin - drug effects; Treatment Outcome
• ISSN: 0315-162X; 1499-2752
• DOI: 10.3899/jrheum.120778

To study safety and potential efficacy of a 2-treatment course (month 0/6) with rituximab (RTX) in early diffuse systemic sclerosis (dcSSc). Two years' followup (open-label study) was done of 8 patients with early dcSSc. Patients received an infusion of 1000 mg RTX 2 times at months 0 and 6, with 100 mg methylprednisolone. Clinical measurements, Disease Activity Score, functional status, and CD19+ peripheral blood count were performed at months 0, 3, 6, 12, 15, 18, and 24 and histopathological evaluation of the skin at months 0, 3, 12, and 24. There was a clinically significant change in skin score, with a mean Modified Rodnan skin score of 24.8 at baseline (SD 3.4) and 13.6 at Month 24 [SD 5.6; mixed models analyses (MMA) p < 0.0001] and a significant decrease in Disease Activity Score (DAS), with a median of 4.5 at baseline (range 1.5-7.5) and 0.5 at Month 24 (range 0.0-5.5; MMA p < 0.0001). Indices of internal organ involvement remained stable throughout the study. RTX induced effective B cell depletion at baseline and Month 6 (< 5 CD19+ cells/μl blood). The blindly assessed hyalinized collagen score changed significantly over time (MMA p = 0.009), with a mean of 69.3 at baseline (SD 22.8) and 33.1 at 24 months (SD 27.0). Five serious adverse events were considered unrelated to the RTX treatment. A 2-treatment course (months 0/6) with RTX appears to be well tolerated and may have potential efficacy for skin disease and stabilization of internal organ status in early dcSSc. Clinical Trials Registration NCT00379431.