Characterization of Breakthrough Hemolysis in Patients With Paroxysmal Nocturnal Hemoglobinuria: An International Multicenter Experience.

ABSTRACT: Breakthrough hemolysis (BTH) is defined as a hemolytic exacerbation in a patient with paroxysmal nocturnal hemoglobinuria (PNH) treated with complement inhibitors (CIs). In the current era of several terminal and proximal inhibitors, there are no guidelines for defining BTH and its severity, and clinical management is not standardized. This retrospective, observational, and multicentric study evaluated BTH frequency and severity in PNH patients treated with complement inhibitors from 2007 until February 2025 at 10 centers across Italy (9 centers) and the United Kingdom (1 center). A total of 271 BTH events occurred in 102 out of 198 patients (51%), with 36/198 (18%) patients experiencing 3 or more BTH episodes over the 18-year follow-up. Most events (55%) were prompted by infections, and 2

SUBMITTER: Fattizzo B 

PROVIDER: S-EPMC12516651 | biostudies-literature | 2025 Nov

REPOSITORIES: biostudies-literature