Direct Oral Anticoagulants and Paroxysmal Nocturnal Hemoglobinuria: A Systematic Review and Update on Evidence.

Paroxysmal nocturnal hemoglobinuria (PNH) is a hematological disorder with an elevated risk of thrombosis. The primary cause of death in PNH patients is thrombosis. Thrombosis in patients with PNH typically occurs in unusual locations such as mesenteric, cerebral, or cutaneous veins; arterial occurrences are less frequent. There is no significant data regarding treating thrombotic events in patients with PNH with direct oral anticoagulants (DOACs). A systematic review following PRISMA guidelines was conducted, with searches in PubMed, Scopus, and Google Scholar for articles published up to December 10, 2024. The review included PNH patients over 18 who developed thrombotic events and were treated using DOACs. Non-English articles lacking sufficient detail and studies involving pregnant patients were excluded from the analysis. After applying inclusion and exclusion criteria, only seven articles were included. Most of the excluded articles involved patients who received standard therapy with heparin, low molecular weight heparin, and warfarin, but few patients were treated with DOACs. There is scarce data about the use of DOAC in patients with PNH. Our review showed that most patients were not treated with DOAC. Moreover, the absence of a well-defined duration for anticoagulation therapy in patients with PNH raises concerns about using vitamin K antagonists (VKA), particularly if treatment is lifelong. In such cases, DOACs offer a more convenient option for patients. Therefore, DOAC use needs to be investigated for treating patients with PNH. Large-scale studies are required to gather data on patients with PNH.