Prevention and Management of Thromboembolism in Patients with Paroxysmal Nocturnal Hemoglobinuria in Asia: A Narrative Review

Abstract

Thromboembolism (TE) is a major cause of morbidity and mortality in patients with paroxysmal nocturnal hemoglobinuria (PNH). This narrative review summarizes available evidence on TE in Asian patients with PNH and discusses practical considerations and challenges for preventing and managing PNH-associated TE in Asian populations. Evidence suggests that, compared with non-Asians, fewer Asian patients have a history of TE (3.6% vs. 8.9%, p < 0.01), receive anticoagulants (8.5% vs. 16.2%, p = 0.002), or die from TE (6.9% vs. 43.7%, p = 0.000). Independent predictors of TE include lactate dehydrogenase ≥ 1.5 × upper limit of normal, pain, and male sex. Clone size alone does not appear to be a reliable estimate of TE risk. D-dimer levels are a useful marker of hemostatic activation, although they are not specific to PNH. Complement inhibition reduces the incidence of TE, although it does not wholly eliminate TE risk. Eligibility criteria and access to complement inhibitors vary across Asia, with limited availability in some countries. Anticoagulation is required to treat acute TE events and for primary or secondary prophylaxis in selected patients. Physicians and patients must stay alert to the signs and symptoms of TE to ensure prompt and appropriate treatment.