Efficacy of eltrombopag on recurrent connective tissue disease related thrombocytopenia patients who failed to respond to rhTPO or recombinant human thrombopoietin: a report of 4 cases and literature review / 中华风湿病学杂志

ABSTRACT

Objective:To evaluate the efficacy and safety of eltrombopag standard therapy in the treatment of patients with connective tissue disease (CTD) associated with thrombocytopenia who were non-responder to recombinant human thrombopoietin (rhTPO) or relapsed after discontinuation of rhTPO.Methods:Retrospectively analysis of clinical data of 4 cases presented with CTD associated with thrombocytopenia who were non-responder to rhTPO or relapsed after discontinuation of rhTPO, and then treated with eltrombopag from November 2017 to June 2022. The literature were reviewed on eltrombopag in the treatment of CTD associated with thrombocytopenia.Results:The platelet count of 4 patients increased to more than 30×10 9/L after 2 weeks administration of eltrombopag. The median time was 32 (14, 41) days for platelet counts returning to normal level. The dosage of eltrombopag was gradually reduced to maintain normal platelet count, which was helpful for tapering of glucocorticoid and other immunosuppressant. No adverse event was observed during the treatment. Conclusion:Eltrombopag is safe and effective for CTD associated with thrombocytopenia, for patients who are non-responders to rhTPO or relapsed after discontinuation of rhTPO treatment in particular.