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. 2025 Aug 23.
doi: 10.1007/s40801-025-00512-y. Online ahead of print.

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

Rong Fu  1 , Hui Liu  2 , Pin-Tzu Su  3 , Xuefei Liu  3 , Karl-Johan Myrén  4 , Francesca Gatta  5 , Lucy Earl  6 , Yasmin Taylor  6 , Alice Simons  6 , Elmira Lechat  5
Affiliations

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

Rong Fu et al. Drugs Real World Outcomes. .

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.

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Conflict of interest statement

Declarations. Funding: Data collection was undertaken by Adelphi Real World as part of an independent survey, entitled the Adelphi Real World PNH DSP. Alexion did not influence the original survey through either contribution to the design of questionnaires or data collection. The DSP is a wholly owned Adelphi Real World product. Alexion is one of multiple subscribers to the DSP. Publication of survey results was not contingent on the subscriber’s approval or censorship of the manuscript. Conflicts of Interest: Xuefei Liu is a previous employee of AstraZeneca China. All authors declare no competing financial interests related to this work. Availability of Data and Material: All data, i.e. methodology, materials, data and data analysis, that support the findings of this survey are the intellectual property of Adelphi Real World. All requests for access should be addressed directly to Yasmin Taylor at yasmin.taylor@omc.com. Yasmin Taylor is an employee of Adelphi Real World. Ethics Approval and Consent to Participate: Patients provided informed consent to take part in the survey using a checkbox. The PNH DSP was submitted to the Pearl Institutional Review Board (study protocol number no. 21-ADRW-127/AG9119), and exemption from ethical approval was granted. Consent for Publication: Not applicable. Code Availability: Not applicable. Authors’ Contributions: All authors were contributors in writing the manuscript. Rong Fu, Hui Liu, Pin-Tzu Su, Xuefei Liu, Karl-Johan Myrén, Francesca Gatta and Elmira Lechat contributed to the study conceptualization and design. Lucy Earl, Yasmin Taylor and Alice Simons contributed to data analysis and interpretation. All authors read and approved the final manuscript.

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