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Caplacizumab for immune thrombotic thrombocytopenic purpura: real-world multicenter data
- Source :
- Frontiers in Medicine, Vol 10 (2023)
- Publication Year :
- 2023
- Publisher :
- Frontiers Media S.A., 2023.
-
Abstract
- Given the limited real-world data of caplacizumab, our multicenter real-world study was designed to assess the safety and efficacy of caplacizumab in immune thrombotic thrombocytopenic pupura (iTTP), compared to historic controls. We have studied 70 patients: 23 in the caplacizumab and 47 in the historic control group. Plasma exchange was applied in all episodes except for two patients that denied plasma exchange. Rituximab as first-line treatment was more common in the caplacizumab group compared to historic control. Caplacizumab (10 mg daily) was given at a median on day 7 (1–43) from initial diagnosis for 32 (6–47) dosages. In the caplacizumab group, a median of 12 (8–23) patients required plasma exchange sessions versus 14 (6–32) in the control group. Caplacizumab administration did not produce any grade 3 complications or major hemorrhagic events. After a median of 19.0 (2.6–320) months since the iTTP diagnosis, 5 deaths occurred (4 in the control group and 1 in the caplacizumab group, p = 0.310). Caplacizumab patients achieved early platelet normalization and ADAMTS13 activity normalization at the end of treatment. Relapse was observed only in 2/23 (9%) caplacizumab patients, compared to 29/47 (62%) historic controls (p
Details
- Language :
- English
- ISSN :
- 2296858X
- Volume :
- 10
- Database :
- Directory of Open Access Journals
- Journal :
- Frontiers in Medicine
- Publication Type :
- Academic Journal
- Accession number :
- edsdoj.0e47eee9cc9b45b8b1af651681d65d33
- Document Type :
- article
- Full Text :
- https://doi.org/10.3389/fmed.2023.1226114