Efficacy and safety of belimumab versus rituximab for refractory immune thrombocytopenia in patients with connective tissue disease

Objectives Immune thrombocytopenia (ITP) is a haematological manifestation secondary to connective tissue disease (CTD). Many patients with CTD–ITP are refractory to glucocorticoids (GCs) plus immunosuppressant agents (ISAs); rituximab (RTX) is the recommended second-line therapy. Belimumab (BLM) sh...

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Main Authors: Min Wang, Yudong Liu, Tong Li, Xuan Zhang, Ying Jiang, Xiaoxia Zuo, Xiangning Yuan
Format: Article
Language:English
Published: BMJ Publishing Group 2025-05-01
Series:Lupus Science and Medicine
Online Access:https://lupus.bmj.com/content/12/1/e001501.full
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author Min Wang
Yudong Liu
Tong Li
Xuan Zhang
Ying Jiang
Xiaoxia Zuo
Xiangning Yuan
author_facet Min Wang
Yudong Liu
Tong Li
Xuan Zhang
Ying Jiang
Xiaoxia Zuo
Xiangning Yuan
author_sort Min Wang
collection DOAJ
description Objectives Immune thrombocytopenia (ITP) is a haematological manifestation secondary to connective tissue disease (CTD). Many patients with CTD–ITP are refractory to glucocorticoids (GCs) plus immunosuppressant agents (ISAs); rituximab (RTX) is the recommended second-line therapy. Belimumab (BLM) shows efficacy against CTD. We compared the efficacy and safety of RTX and BLM.Methods Data of patients with CTD–ITP refractory to GCs plus ISAs administered were collected. The data of 11 patients with refractory CTD–ITP who received BLM were compared with those of 15 patients treated with RTX.Results At week 2, BLM resulted in a better overall response (OR) than RTX (72.7% vs 26.7%, p=0.045). The OR rate was 60.0% (9/15), 66.7% (10/15) and 73.3% (11/15) at week 4, 8 and 12, respectively, in the RTX group. It remained at 72.7% (8/11) during week 4–12 in the BLM group. Excluding the data of three deceased patients, the OR rate dropped at week 24 in both groups (RTX vs BLM, 61.5% (8/13) vs 70.0% (7/10), p=1.000). At week 24, four patients with OR in both groups successfully withdrew GCs to <15 mg prednisone (RTX vs BLM, 40% (4/10) vs 66.7% (4/6), p=0.608). The serum C3 level did not significantly change, whereas the serum immunoglobulin G level significantly decreased at week 4, 8 and 12 in both groups. There were three patients with serious adverse effects who died of severe pneumonia during weeks 12–24.Conclusions BLM may be a safe and effective alternative to RTX for CTD–ITP refractory to GCs plus ISAs.
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spelling doaj-art-c97048e7afb74eb297b19c49e75e616e2025-08-20T01:50:23ZengBMJ Publishing GroupLupus Science and Medicine2053-87902025-05-0112110.1136/lupus-2025-001501Efficacy and safety of belimumab versus rituximab for refractory immune thrombocytopenia in patients with connective tissue diseaseMin Wang0Yudong Liu1Tong Li2Xuan Zhang3Ying Jiang4Xiaoxia Zuo5Xiangning Yuan6Department of Rheumatology, Beijing Hospital, National Center of Gerontology, Institute of Geriatric Medicine, Chinese Academy of Medical Sciences and Peking Union Medical College, Beijing, ChinaThe Key Laboratory of Geriatrics, Beijing Institute of Geriatrics, Beijing Hospital, Beijing, ChinaDepartment of Rheumatology, Xiangya Hospital Central South University, Changsha, Hunan, ChinaDepartment of Rheumatology, Beijing Hospital, National Center of Gerontology, Institute of Geriatric Medicine, Chinese Academy of Medical Sciences and Peking Union Medical College, Beijing, ChinaDepartment of Rheumatology, Xiangya Hospital Central South University, Changsha, Hunan, ChinaDepartment of Rheumatology, Xiangya Hospital Central South University, Changsha, Hunan, ChinaDepartment of Nephrology, Xiangya Hospital Central South University, Changsha, Hunan, ChinaObjectives Immune thrombocytopenia (ITP) is a haematological manifestation secondary to connective tissue disease (CTD). Many patients with CTD–ITP are refractory to glucocorticoids (GCs) plus immunosuppressant agents (ISAs); rituximab (RTX) is the recommended second-line therapy. Belimumab (BLM) shows efficacy against CTD. We compared the efficacy and safety of RTX and BLM.Methods Data of patients with CTD–ITP refractory to GCs plus ISAs administered were collected. The data of 11 patients with refractory CTD–ITP who received BLM were compared with those of 15 patients treated with RTX.Results At week 2, BLM resulted in a better overall response (OR) than RTX (72.7% vs 26.7%, p=0.045). The OR rate was 60.0% (9/15), 66.7% (10/15) and 73.3% (11/15) at week 4, 8 and 12, respectively, in the RTX group. It remained at 72.7% (8/11) during week 4–12 in the BLM group. Excluding the data of three deceased patients, the OR rate dropped at week 24 in both groups (RTX vs BLM, 61.5% (8/13) vs 70.0% (7/10), p=1.000). At week 24, four patients with OR in both groups successfully withdrew GCs to <15 mg prednisone (RTX vs BLM, 40% (4/10) vs 66.7% (4/6), p=0.608). The serum C3 level did not significantly change, whereas the serum immunoglobulin G level significantly decreased at week 4, 8 and 12 in both groups. There were three patients with serious adverse effects who died of severe pneumonia during weeks 12–24.Conclusions BLM may be a safe and effective alternative to RTX for CTD–ITP refractory to GCs plus ISAs.https://lupus.bmj.com/content/12/1/e001501.full
spellingShingle Min Wang
Yudong Liu
Tong Li
Xuan Zhang
Ying Jiang
Xiaoxia Zuo
Xiangning Yuan
Efficacy and safety of belimumab versus rituximab for refractory immune thrombocytopenia in patients with connective tissue disease
Lupus Science and Medicine
title Efficacy and safety of belimumab versus rituximab for refractory immune thrombocytopenia in patients with connective tissue disease
title_full Efficacy and safety of belimumab versus rituximab for refractory immune thrombocytopenia in patients with connective tissue disease
title_fullStr Efficacy and safety of belimumab versus rituximab for refractory immune thrombocytopenia in patients with connective tissue disease
title_full_unstemmed Efficacy and safety of belimumab versus rituximab for refractory immune thrombocytopenia in patients with connective tissue disease
title_short Efficacy and safety of belimumab versus rituximab for refractory immune thrombocytopenia in patients with connective tissue disease
title_sort efficacy and safety of belimumab versus rituximab for refractory immune thrombocytopenia in patients with connective tissue disease
url https://lupus.bmj.com/content/12/1/e001501.full
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