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

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

Of 97 patients (mean age 49 ± 15), 24.7% were anemic (Hb < 10 g/dL) and 16% had hemolysis. QoL did not differ significantly by hemolysis status. However, anemia was associated with significantly lower EORTC QLQ-C30 global health and functional scores (p < 0.05), particularly in physical, role, and social domains, and with worse QLQ-AA/PNH emotional, cognitive, and physical functioning, as well as greater illness intrusiveness and stigmatization. Fatigue, dyspnea, and pain were common symptoms in anemic patients. QLQ-AA/PNH scores showed high internal consistency (Cronbach's α > 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.