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Updated: Aug 13, 2026
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A Point-of-Care Method with Integrated Decision Support Tool to Estimate Anemia at Population Level
Published on: January 19, 2024
Anemia and Patient-Reported Outcomes in Patients With Paroxysmal Nocturnal Hemoglobinuria: A Real-World Observational
Esther Natalie Oliva1, Antonio M Risitano2, Eros Di Bona3
1Hematology Department London North West University Healthcare NHS Trust London UK.
Ejhaem
|August 12, 2026
Summary
Anemia significantly impacts quality of life in paroxysmal nocturnal hemoglobinuria (PNH) patients, more so than residual hemolysis. Addressing anemia is key to improving patient outcomes and overall well-being.
Area of Science:
- Hematology
- Patient-Reported Outcomes
- Quality of Life Research
Background:
- Paroxysmal nocturnal hemoglobinuria (PNH) is a rare disorder with hemolysis, thrombosis, and bone marrow failure.
- Current PNH treatments improve outcomes but residual hemolysis and anemia can persist, impacting quality of life (QoL).
Purpose of the Study:
- To assess if patient-reported outcomes (PROs) differentiate PNH patients with or without hemolysis.
- To evaluate factors associated with PROs in PNH patients.
Main Methods:
- International, multicenter, observational study of 97 PNH patients.
- Collected clinical, biochemical, and treatment data.
- Used EORTC QLQ-C30 and QLQ-AA/PNH measures; hemolysis defined as LDH ≥ 1.5 × upper normal limit.
Main Results:
- Anemia (Hb < 10 g/dL) was present in 24.7% of patients; 16% had hemolysis.
- Anemia, not hemolysis, significantly correlated with lower QoL scores across multiple domains (global health, physical, role, social, emotional, cognitive).
- Anemic patients reported more fatigue, dyspnea, pain, illness intrusiveness, and stigmatization.
Conclusions:
- Anemia, rather than residual hemolysis, is strongly associated with impaired quality of life in PNH patients.
- Managing anemia alongside hemolysis may enhance PROs and improve PNH patient care.
