Observational Study Journal Article Ppublish 2 citations
PubMed: 37171598 DOI: 10.1007/s00277-023-05248-9
N-Acetylcistein for thrombotic thrombocytopenic purpura: an observational case series study.
Ann Hematol 102(8):2069-2075
Authors: Ignacio Español, Juan Diego Leal, Miguel Blanquer, Faustino García-Candel, Angela Heredia, Joaquín Gómez-Espuch, Celia González, Jorge Montserrat, María Sacramento Díaz-Carrasco, Antonio Martínez, José M Moraleda
Affiliations
- Hematology Department, University Hospital Virgen de la Arrixaca, IMIB-Pascual Parilla, Carretera Madrid-Cartagena s/n, 30120 El Palmar, Murcia, Spain.
- Hematology Department, University Hospital Virgen de la Arrixaca, IMIB-Pascual Parilla, Carretera Madrid-Cartagena s/n, 30120 El Palmar, Murcia, Spain.
- Pharmacy Department, IMIB-Pascual Parrilla, University of Murcia, Murcia, Spain.
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.
MeSH
Purpura, Thrombotic Thrombocytopenic, Hematology, Acetylcysteine, Purpura, Thrombocytopenic, Purpura, Hematologic Diseases