Anemia and Patient‐Reported Outcomes in Patients With Paroxysmal Nocturnal Hemoglobinuria: A Real‐World Observational Study

Résumé fourni par la source

Introduction: Paroxysmal nocturnal hemoglobinuria (PNH) is a rare hematologic disorder characterized by complement-mediated hemolysis, thrombosis, bone marrow failure, and anemia. Although current treatments improve outcomes, patients still experience hemolysis and could remain anemic with a negative impact on quality of life (QoL). The aim of this study was to assess whether patient-reported outcomes (PROs) distinguish PNH patients with/without hemolysis and evaluate factors associated with PROs. Methods: In this international, multicenter, observational study, 97 PNH patients witfrom Italy and the UK completed the EORTC QLQ-C30 and QLQ-AA/PNH measures at baseline and after 2 weeks. Clinical, biochemical, and treatment data were collected. Hemolysis was defined as LDH ≥ 1.5 × upper normal limit. PRO scores were compared between patients above and below this threshold. Associations of PROs with patient and disease factors were analyzed. Results: > 0.7) and test-retest reliability (ICC: 0.72-0.95). Conclusions: Anemia, but not residual hemolysis, was associated with impaired QoL across multiple domains. Addressing anemia alongside hemolysis may improve PROs in the management of PNH. Trial Registration: The authors have confirmed clinical trial registration is not needed for this submission.

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