Chanyuan Wu1, Qian Wang1, Dong Xu1, Mengtao Li1, Xiaofeng Zeng1. 1. Department of Rheumatology, Peking Union Medical College Hospital, Key Laboratory of Rheumatology and Clinical Immunology, Ministry of Education, Peking Union Medical College and Chinese Academy of Medical Sciences, National Clinical Research Center for Dermatologic and Immunologic Disease (NCRC-DID), Beijing, China.
Abstract
OBJECTIVES: Connective tissue disease-related thrombocytopenia (CTD-TP) is a problematic disorder in clinical practice. Because the first-line therapy of glucocorticoid and/or immunosuppressants is not effective for refractory cases, alternative treatment approaches are urgently needed. The present study investigated the efficacy and safety of sirolimus in refractory CTD-TP patients. METHODS: This single-centre, single-arm, phase II study enrolled 20 refractory CTD-TP patients between September 2017 and September 2018 (registered on ClinicalTrials.gov: NCT03688191). Oral sirolimus administration was dose-adjusted to maintain a therapeutic range of 6-15 ng/ml for 6 months. The primary endpoints were partial and complete remission rates at 6 months. RESULTS: Twelve (60%) patients achieved the primary end point with a 50% complete remission rate after 6 months. Among the 14 SLE patients, the overall response rate was 71.4%, with a complete remission rate of 64.3%. None of the primary Sjögren's syndrome cases responded to sirolimus. There was no significant difference in baseline clinical characteristics or lymphocyte subpopulations between responders and non-responders. No severe side effect was detected during the study. CONCLUSION: Sirolimus is an effective and safe treatment option for refractory CTD-TP patients. TRIAL REGISTRATION: https://clinicaltrials.gov, NCT03688191.
OBJECTIVES: Connective tissue disease-related thrombocytopenia (CTD-TP) is a problematic disorder in clinical practice. Because the first-line therapy of glucocorticoid and/or immunosuppressants is not effective for refractory cases, alternative treatment approaches are urgently needed. The present study investigated the efficacy and safety of sirolimus in refractory CTD-TP patients. METHODS: This single-centre, single-arm, phase II study enrolled 20 refractory CTD-TP patients between September 2017 and September 2018 (registered on ClinicalTrials.gov: NCT03688191). Oral sirolimus administration was dose-adjusted to maintain a therapeutic range of 6-15 ng/ml for 6 months. The primary endpoints were partial and complete remission rates at 6 months. RESULTS: Twelve (60%) patients achieved the primary end point with a 50% complete remission rate after 6 months. Among the 14 SLEpatients, the overall response rate was 71.4%, with a complete remission rate of 64.3%. None of the primary Sjögren's syndrome cases responded to sirolimus. There was no significant difference in baseline clinical characteristics or lymphocyte subpopulations between responders and non-responders. No severe side effect was detected during the study. CONCLUSION:Sirolimus is an effective and safe treatment option for refractory CTD-TP patients. TRIAL REGISTRATION: https://clinicaltrials.gov, NCT03688191.