Abstract
Only few data are available regarding the efficacy and safety of thrombopoietin receptor agonists (TPO-RAs) for treating systemic lupus erythematosus (SLE) and/or antiphospholipid syndrome (APS)-associated immune thrombocytopenia (ITP). We retrospectively assessed the efficacy and safety of TPO-RAs in 80 adults with ITP in three subgroups: (1) 37 patients with definite or incomplete SLE and no antiphospholipid antibodies (APAs), (2) 27 patients with definite or incomplete SLE associated with APAs and no history of arterial or venous thrombotic events (TEs) and (3) 16 patients with APS. In total, 39 (48.8%) patients received eltrombopag, 14 (17.5%) romiplostim and 27 (33.8%) both TPO-RAs sequentially. The overall response of ITP was 78.8%. In total, 17 (21.3%) patients had 21 TEs (7 venous and 14 arterials, including 3 catastrophic APS); 3 were fatal. The rate of TEs was 8.1% in the SLE or lupus without APAs group, 22.2% in the SLE or lupus with APAs group and 50% in the APS group. In 12 patients, TPO-RAs were continued after TE onset, combined with an anticoagulant (n = 12) and/or an antiplatelet agent (n = 3). Only one TE relapse occurred in the nine patients with TPO-RAs maintained and combined with an anticoagulant after TE. TPO-RAs may be effective safe in ITP associated with SLE and no APAs. Their use should be carefully considered in the presence of thrombotic risk factors and/or APA positivity and should be avoided in patients with definite APS. When a TE occurs, risk of relapse is low with maintenance of the TPO-RA combined with anticoagulation.