A Retrospective Analysis of Characteristics and Outcomes of Paroxysmal Nocturnal Hemoglobinuria Patients, not Having Access to Standard of Care.

UNLABELLED: This study aims to investigate the clinical characteristics and outcomes of Paroxysmal Nocturnal Hemoglobinuria (PNH) patients in a tertiary care center in South India, where access to C5 inhibitors is limited. A retrospective observational study was conducted between January 2018 and July 2024. Data from 45 PNH patients, diagnosed with flow cytometry, were collected from electronic medical records. Clinical information, laboratory results, treatment details, and follow-up data were analyzed using appropriate statistical methods, including Kaplan-Meier survival analysis. The median age at symptom onset was 35 years. Common presentations included pancytopenia (59%), thrombosis (13.3%), and hemoglobinuria (11.1%). The mean time to diagnosis was 1.88 years. Most patients had hypocellular marrow (74%). The mean Hemoglobin was 7.3 ± 2.3gm/dL. Patients received supportive care, including transfusions (66.67%), steroids (28.8%), calcineurin inhibitors (42.2%), and danazol (64.4%). The 5-year overall survival was 57.6%. Infections and hemorrhage were the leading causes of death. PNH in this resource-limited setting is characterized by delayed diagnosis, significant morbidity, and high mortality. Pancytopenia and thrombosis were common. Supportive care, in the absence of C5 inhibitors, is associated with poor outcomes, highlighting the need for improved access to standard of care treatments. SUPPLEMENTARY INFORMATION: The online version contains supplementary material available at https://doi.org/10.1007/s12288-025-02263-w.