A Pilot Open-Label Phase II Trial of Rituximab for Non-Criteria Manifestations of Antiphospholipid Syndrome

Associate Professor of Medicine, Weill Medical College of Cornell University, New York, NY. .
Arthritis & Rheumatology (Impact Factor: 7.87). 02/2013; 65(2). DOI: 10.1002/art.37759
Source: PubMed

ABSTRACT BACKGROUND: Few studies address the treatment of non-criteria manifestations of antiphospholipid syndrome (APS). METHODS: The primary objective of this 12-month pilot, Phase II study was to evaluate the safety of rituximab in antiphospholipid antibody (aPL)-positive patients with non-criteria APS manifestations. The secondary objectives were to evaluate the effect of rituximab on aPL profile and non-criteria APS manifestations. Adult aPL-positive patients with thrombocytopenia, cardiac valve disease (CVD), skin ulcer, aPL-nephropathy (aPLN), and/or cognitive dysfunction (CD) received two doses of rituximab (1000mg) on days one and 15. Antiphospholipid antibody profiles and clinical outcome measures (COMs) were analyzed at preset time points, which were categorized as: "complete response (CR)", "partial response (PR)", "no response (NR)", and "recurrence (RC)". RESULTS: Two of 19 patients developed infusion reactions resulting in early termination. There were 12 serious and 49 non-serious adverse events. All patients with baseline positive lupus anticoagulant as well as anticardiolipin and anti-β(2) -glycoprotein-I antibody IgG tests remained positive at 24 and 52 weeks. The number of patients with CR/PR/NR/RC for COMs at 24 weeks was: 1/1/2/0 for thrombocytopenia; 0/0/3/- for CVD; 3/1/0/1 for skin ulcer; 0/1/0/0 for aPLN; and 3/1/1/- for CD. CONCLUSION: Our uncontrolled and non-randomized pilot study suggests that safety is consistent with rituximab's safety profile in aPL-positive patients; and despite no substantial change in aPL profiles, rituximab may be effective in controlling some but not all non-criteria aPL manifestations. © 2012 American College of Rheumatology.