N-Acetylcistein for thrombotic thrombocytopenic purpura: an observational case series study
Abstract Acquired thrombotic thrombocytopenic purpura (TTP) is a life-threatening disorder. N-Acetylcysteine (NAC) rapidly degrades ultra-large von Willebrand factor multimers by disrupting the disulfide bonds. We report a series of twelve consecutive patients diagnosed with acquired TTP successfully treated with high-dose NAC (150 mg/kg/day) in combination with plasma exchange and steroids. Eight patients also received rituximab. Two patients presented refractory TTP. All patients achieved a quick clinical response in a median time of 5.5 days after starting NAC and are alive after a median follow-up of 29 months. The treatment was feasible and well tolerated. These data provide further evidence of the potential benefit and safety of adding NAC to the standard of care..
| Medienart: |
Artikel |
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| Erscheinungsjahr: |
2023 |
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| Erschienen: |
2023 |
| Enthalten in: |
Zur Gesamtaufnahme - volume:102 |
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| Enthalten in: |
Annals of hematology - 102(2023), 8 vom: 12. Mai, Seite 2069-2075 |
| Sprache: |
Englisch |
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| Beteiligte Personen: |
Español, Ignacio [VerfasserIn] |
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| Links: |
Volltext [lizenzpflichtig] |
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| Themen: |
Acquired thrombotic thrombocytopenic purpura |
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| Anmerkungen: |
© The Author(s), under exclusive licence to Springer-Verlag GmbH Germany, part of Springer Nature 2023. Springer Nature or its licensor (e.g. a society or other partner) holds exclusive rights to this article under a publishing agreement with the author(s) or other rightsholder(s); author self-archiving of the accepted manuscript version of this article is solely governed by the terms of such publishing agreement and applicable law. |
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| doi: |
10.1007/s00277-023-05248-9 |
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| funding: |
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| Förderinstitution / Projekttitel: |
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| 520 | |a Abstract Acquired thrombotic thrombocytopenic purpura (TTP) is a life-threatening disorder. N-Acetylcysteine (NAC) rapidly degrades ultra-large von Willebrand factor multimers by disrupting the disulfide bonds. We report a series of twelve consecutive patients diagnosed with acquired TTP successfully treated with high-dose NAC (150 mg/kg/day) in combination with plasma exchange and steroids. Eight patients also received rituximab. Two patients presented refractory TTP. All patients achieved a quick clinical response in a median time of 5.5 days after starting NAC and are alive after a median follow-up of 29 months. The treatment was feasible and well tolerated. These data provide further evidence of the potential benefit and safety of adding NAC to the standard of care. | ||
| 650 | 4 | |a N-Acetylcysteine | |
| 650 | 4 | |a Acquired thrombotic thrombocytopenic purpura | |
| 650 | 4 | |a Disease treatment | |
| 700 | 1 | |a Leal, Juan Diego |4 aut | |
| 700 | 1 | |a Blanquer, Miguel |4 aut | |
| 700 | 1 | |a García-Candel, Faustino |4 aut | |
| 700 | 1 | |a Heredia, Angela |4 aut | |
| 700 | 1 | |a Gómez-Espuch, Joaquín |4 aut | |
| 700 | 1 | |a González, Celia |4 aut | |
| 700 | 1 | |a Montserrat, Jorge |4 aut | |
| 700 | 1 | |a Díaz-Carrasco, María Sacramento |4 aut | |
| 700 | 1 | |a Martínez, Antonio |4 aut | |
| 700 | 1 | |a Moraleda, José M. |4 aut | |
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