Successful management of three patients with autoimmune thrombotic thrombocytopenic purpura with paradigm-changing therapy : Caplacizumab, steroids, plasma exchange, rituximab, and intravenous immunoglobulins (CASPERI)
Copyright © 2020 Elsevier Ltd. All rights reserved..
Autoimmune thrombotic thrombocytopenic purpura (aTTP) is a severe disease caused by the production of autoantibodies against von Willebrand factor (vWF)-cleaving ADAMTS13 (a disintegrin and metalloproteinase with thrombospondin-1 motifs; 13th member of the family). In 2018, caplacizumab was approved for the treatment of patients with acute aTTP in conjunction with plasma exchange (PE) and immunosuppressive therapy. Immunosuppressive standard of care includes mainly steroids whereas rituximab is usually reserved for refractory cases. We report three patients with a first acute episode of aTTP who were successfully treated with a paradigm-changing scheme including standard of care (caplacizumab, steroids and PE) plus upfront therapy with rituximab and intravenous immunoglobulins (CASPERI). Rituximab was added 1-4 days after diagnosis, when ADAMTS13 autoantibodies were detected and intravenous immunoglobulins were administered after performing PE using albumin as replacement solution. Successful outcome was observed in all three patients: platelet recovery (>150 × 109/L) was observed after 3, 4, and 5 days from diagnosis; ADAMTS13 activity >5% and ADAMTS13 autoantibodies were negative after 14, 15, and 21 days from diagnosis. In conclusion, caplacizumab, steroids, PE (using fresh frozen plasma or albumin as replacement solution and adding intravenous immunoglobulins) plus upfront rituximab therapy was a safe and efficient combination to induce remission in case of acute aTTP.
| Medienart: |
E-Artikel |
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| Erscheinungsjahr: |
2021 |
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| Erschienen: |
2021 |
| Enthalten in: |
Zur Gesamtaufnahme - volume:60 |
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| Enthalten in: |
Transfusion and apheresis science : official journal of the World Apheresis Association : official journal of the European Society for Haemapheresis - 60(2021), 1 vom: 15. Feb., Seite 103011 |
| Sprache: |
Englisch |
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| Beteiligte Personen: |
Cid, Joan [VerfasserIn] |
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| Links: |
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| Anmerkungen: |
Date Completed 14.10.2021 Date Revised 14.10.2021 published: Print-Electronic Citation Status MEDLINE |
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| doi: |
10.1016/j.transci.2020.103011 |
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| funding: |
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| Förderinstitution / Projekttitel: |
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| PPN (Katalog-ID): |
NLM317881442 |
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| 100 | 1 | |a Cid, Joan |e verfasserin |4 aut | |
| 245 | 1 | 0 | |a Successful management of three patients with autoimmune thrombotic thrombocytopenic purpura with paradigm-changing therapy |b Caplacizumab, steroids, plasma exchange, rituximab, and intravenous immunoglobulins (CASPERI) |
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| 500 | |a published: Print-Electronic | ||
| 500 | |a Citation Status MEDLINE | ||
| 520 | |a Copyright © 2020 Elsevier Ltd. All rights reserved. | ||
| 520 | |a Autoimmune thrombotic thrombocytopenic purpura (aTTP) is a severe disease caused by the production of autoantibodies against von Willebrand factor (vWF)-cleaving ADAMTS13 (a disintegrin and metalloproteinase with thrombospondin-1 motifs; 13th member of the family). In 2018, caplacizumab was approved for the treatment of patients with acute aTTP in conjunction with plasma exchange (PE) and immunosuppressive therapy. Immunosuppressive standard of care includes mainly steroids whereas rituximab is usually reserved for refractory cases. We report three patients with a first acute episode of aTTP who were successfully treated with a paradigm-changing scheme including standard of care (caplacizumab, steroids and PE) plus upfront therapy with rituximab and intravenous immunoglobulins (CASPERI). Rituximab was added 1-4 days after diagnosis, when ADAMTS13 autoantibodies were detected and intravenous immunoglobulins were administered after performing PE using albumin as replacement solution. Successful outcome was observed in all three patients: platelet recovery (>150 × 109/L) was observed after 3, 4, and 5 days from diagnosis; ADAMTS13 activity >5% and ADAMTS13 autoantibodies were negative after 14, 15, and 21 days from diagnosis. In conclusion, caplacizumab, steroids, PE (using fresh frozen plasma or albumin as replacement solution and adding intravenous immunoglobulins) plus upfront rituximab therapy was a safe and efficient combination to induce remission in case of acute aTTP | ||
| 650 | 4 | |a Case Reports | |
| 650 | 4 | |a Journal Article | |
| 650 | 4 | |a Caplacizumab | |
| 650 | 4 | |a Intravenous immunoglobulins | |
| 650 | 4 | |a Plasma exchange | |
| 650 | 4 | |a Rituximab | |
| 650 | 4 | |a Steroids | |
| 650 | 4 | |a Thrombotic thrombocytopenic purpura | |
| 650 | 7 | |a Immunoglobulins, Intravenous |2 NLM | |
| 650 | 7 | |a Immunosuppressive Agents |2 NLM | |
| 650 | 7 | |a Single-Domain Antibodies |2 NLM | |
| 650 | 7 | |a caplacizumab |2 NLM | |
| 650 | 7 | |a 2R27AB6766 |2 NLM | |
| 650 | 7 | |a Rituximab |2 NLM | |
| 650 | 7 | |a 4F4X42SYQ6 |2 NLM | |
| 700 | 1 | |a Pérez-Valencia, Amanda Isabel |e verfasserin |4 aut | |
| 700 | 1 | |a Torrente, Miguel Ángel |e verfasserin |4 aut | |
| 700 | 1 | |a Ávarez-Larrán, Alberto |e verfasserin |4 aut | |
| 700 | 1 | |a Díaz-Ricart, Maribel |e verfasserin |4 aut | |
| 700 | 1 | |a Esteve, Jordi |e verfasserin |4 aut | |
| 700 | 1 | |a Lozano, Miquel |e verfasserin |4 aut | |
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