Effectiveness and safety of eltrombopag in connective tissue disease patients with refractory immune thrombocytopenia: a retrospective study

Abstract

Objectives We aimed to investigate the safety and effectiveness of eltrombopag for adult patients with refractory immune thrombocytopenia (ITP) secondary to connective tissue disease (CTD). Methods This is a single center, retrospective cohort and propensity score-matched study. Data from CTD-ITP patients treated with eltrombopag between January 2019 and January 2023 were retrospectively analyzed. Baseline characteristics and follow-up information were recorded. CTD patients without ITP were matched to identify the risk factors associated with CTD-ITP performed by Logistic regression analysis. Results Twenty patients were enrolled, including 5 systemic lupus erythematosus (SLE), 9 Sjögren's syndrome (SS) and 6 undifferentiated connective tissue disease (UCTD). Nineteen (95%) patients were female and the median age was 59 years. Logistic regression analysis showed that anaemia (OR = 8.832, p = 0.007) was associated with increased risk of ITP, while non-erosive arthritis (OR = 0.045, p = 0.001) and interstitial lung disease (OR = 0.075, p = 0.031) were associated with reduced risk. Fourteen patients (70%) achieved a complete response (CR) and one (5%) achieved a partial response (PR). The median response time was 14 days. The median platelet count was 8.5 × 109/l at baseline of eltrombopag, and increased to 122 × 109/l after 4 weeks. No adverse events were observed. Conclusions Eltrombopag appears to be effective, safe, and well-tolerated in refractory ITP patients with CTD; larger studies are needed to confirm the generalizability of these findings.