N-acetylcysteine in Thrombotic Thrombocytopenic Purpura: A Systematic Review.
Level 4 - case-series / case-control
Systematic review limited entirely to uncontrolled case reports and case series
PubMed 42338865 · doi:10.7759/cureus.109497
What was done
A systematic review following PRISMA 2020 guidelines searched PubMed from database inception to December 31, 2025, for case reports and case series evaluating therapeutic N-acetylcysteine (NAC) use in patients with thrombotic thrombocytopenic purpura (TTP). Two reviewers independently screened records and extracted study-level data on treatment regimens, clinical outcomes, and safety. The review protocol was not prospectively registered.
What was found
Six publications comprising 22 adult patients were included. NAC was used as an adjunctive therapy, primarily for refractory or relapsing TTP, alongside therapeutic plasma exchange, corticosteroids, rituximab, and other immunosuppressive agents. Platelet recovery occurred after NAC initiation across the included cases, accompanied by improvements in hemolysis markers and neurological symptoms in some reports. No serious NAC-related adverse events were reported. Specific numerical rates and effect sizes were not provided in the abstract.
Why it matters
NAC represents a biologically plausible adjunctive candidate for targeting ultralarge von Willebrand factor multimers in refractory TTP. However, current clinical evidence remains purely observational and hypothesis-generating.
Limits
The total sample is limited to 22 patients across 6 publications, carrying substantial risk of publication bias. All included data are from uncontrolled reports where patients received multiple concurrent standard therapies, making it impossible to isolate the specific therapeutic contribution of NAC. The search was restricted to a single database (PubMed).
Cited by
- supports N-acetylcysteine (NAC) inhibits von Willebrand factor to prevent microclots.