Cross-sectional validation study of patient-reported outcomes in patients with paroxysmal nocturnal haemoglobinuria

I. Weitz, G. Meyers, T. Lamy, J. Y. Cahn, M. T. Uranga, J. A. García Vela, M. A. Sanz, B. Severino, R. J. Kelly, P. Hillmen, A. Hill

    Research output: Contribution to journalArticlepeer-review

    18 Scopus citations


    Background: Paroxysmal nocturnal haemoglobinuria (PNH) is a rare, acquired, clonal haemopoietic stem cell disorder that causes chronic intravascular haemolysis, increases the risk of thrombosis and results in significant patient morbidity and mortality. The symptoms of PNH may have a major impact on patient quality of life. Aims: To assess patient fatigue and health-related quality of life in 29 patients with PNH using the Functional Assessment of Chronic Illness Therapy Fatigue subscale version 4 (FACIT-Fatigue) and the European Organization for Research and Treatment of Cancer Quality-of-Life Questionnaire-C30, version 3 (EORTC QLQ-C30). Methods: Following completion of the questionnaires, patients were interviewed to assess the validity, clarity, relevance and comprehensiveness of the assessments. Results: Overall, patients considered both the FACIT-Fatigue and EORTC QLQ-C30 instruments to be relevant and adequate in assessing the level of PNH-associated fatigue and other quality-of-life measures. The FACIT-Fatigue questionnaire was considered to be clear and to comprehensively cover PNH-related fatigue. The EORTC QLQ-C30 instrument was considered to be easy to understand, but of an overall lower relevance, although some differences between countries were observed. Patients suggested additional questions that could be incorporated into future EORTC QLQ-C30 versions to make it more relevant to PNH. Conclusions: This study confirms the validity of the FACIT-Fatigue and the EORTC QLQ-C30 questionnaires in this patient population and their routine use should be considered in the management of patients with PNH.

    Original languageEnglish (US)
    Pages (from-to)298-307
    Number of pages10
    JournalInternal Medicine Journal
    Issue number3
    StatePublished - Mar 2013


    • Haemoglobinuria
    • Haemolysis
    • Paroxysmal
    • Quality of life
    • Soliris

    ASJC Scopus subject areas

    • Internal Medicine


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