Clinical Outcomes and Disease Management for Patients with Paroxysmal Nocturnal Haemoglobinuria in China: Results from a Real-World Study.

Abstract

Background: Paroxysmal nocturnal haemoglobinuria (PNH) is a chronic haematological disorder caused by uncontrolled complement activation, leading to intravascular haemolysis, indicated by increased lactate dehydrogenase levels (LDH). Standard-of-care complement factor 5 inhibitors have only recently been approved in China. Using real-world data, this study aimed to understand treatment pattern and burden of disease, including thrombosis risk, for Chinese patients with PNH.

Methods: Data were from the Adelphi Real World PNH Disease Specific Programme (DSP)™, a cross-sectional retrospective survey of haematologists/haematologist-oncologists and patients with PNH, conducted July-November 2022. Physicians reported demographic and clinical data on one to ten consecutively consulted patients with PNH aged ≥ 16 years. Patients were stratified by LDH level [cutoff of 1.5 times upper level of normal (ULN, 250 U/L)] and compared using bivariate analysis.

Results: Out of 113 patients, 49.5% had LDH levels ≥1.5 ULN (high-LDH), these had greater physician-perceived disease severity (p<0.001). Anticoagulation treatments (39.8%), prednisone (31.0%), and cyclosporine (27.4%) were most prescribed. More high-LDH patients received rivaroxaban (p=0.002) or vitamin B12 supplements (p=0.008). Overall, the most common symptoms were fatigue and anaemia (92.9% and 74.3%, respectively). Haemolytic crises were reported for 39.3% of all patients, thrombotic events were more common in high-LDH patients (50.0%, compared with 23.2%, p=0.006). Patients reported moderate fatigue levels in the FACIT-Fatigue scale.

Conclusions: Patients were mainly treated with anticoagulants, prednisone or cyclosporine, and had frequent thrombotic events and haemolytic crises. This demonstrates the current management of PNH in China, focusing on mitigating anaemia and managing haemolytic crises rather than addressing the underlying chronic complement-mediated haemolysis and associated morbidities.