Work

Expert guidance on when to screen, monitor, and initiate complement inhibition therapy in patients without classical PNH

Year: 2026

Type: article

Abstract: Paroxysmal nocturnal hemoglobinuria (PNH) is a rare, acquired hematological disease characterized by complement-mediated hemolysis, with or without overt hemoglobinuria, and is associated with bone marrow failure. PNH can be classified into three subcategories: classical (hemolytic) PNH, PNH with underlying bone marrow failure (BMF), and subclinical PNH. The optimal approach for patients with underlying BMF or subclinical PNH is not clearly defined. Additionally, recommendations for screening and monitoring non-classical patients are dated. A modified Delphi panel consisting of 10 hematologists and hematologist-oncologists evaluated 414 patient scenarios on two separate occasions and participated in a moderated in-person meeting. Consensus statements were developed based on the second round of ratings. Following the meeting, the panel agreed on 90% of the ratings and made recommendations on when to screen patients for a PNH clone, when to initiate complement inhibitor treatment, and when to reevaluate patients with a PNH clone who are not yet on treatment. For example, it is appropriate to initiate complement inhibition in patients with a PNH clone size ≥10% who had a recent thrombotic event (TE) with laboratory evidence of hemolysis. Further evidence is required to improve recommendations for patients with small (<10%) PNH clone sizes.

Source: Blood Red Cells & Iron

DOI: 10.1016/j.brci.2026.100103