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

I. Weitz, Gabrielle 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 journalArticle

    9 Citations (Scopus)

    Abstract

    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
    Volume43
    Issue number3
    DOIs
    StatePublished - Mar 2013

    Fingerprint

    Paroxysmal Hemoglobinuria
    Validation Studies
    Cross-Sectional Studies
    Fatigue
    Quality of Life
    Organizations
    Chronic Disease
    Research
    Therapeutics
    Neoplasms
    Patient Reported Outcome Measures
    Surveys and Questionnaires
    Hemolysis
    Thrombosis
    Stem Cells
    Morbidity

    Keywords

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

    ASJC Scopus subject areas

    • Internal Medicine

    Cite this

    Cross-sectional validation study of patient-reported outcomes in patients with paroxysmal nocturnal haemoglobinuria. / Weitz, I.; Meyers, Gabrielle; Lamy, T.; Cahn, J. Y.; Uranga, M. T.; García Vela, J. A.; Sanz, M. A.; Severino, B.; Kelly, R. J.; Hillmen, P.; Hill, A.

    In: Internal Medicine Journal, Vol. 43, No. 3, 03.2013, p. 298-307.

    Research output: Contribution to journalArticle

    Weitz, I, Meyers, G, Lamy, T, Cahn, JY, Uranga, MT, García Vela, JA, Sanz, MA, Severino, B, Kelly, RJ, Hillmen, P & Hill, A 2013, 'Cross-sectional validation study of patient-reported outcomes in patients with paroxysmal nocturnal haemoglobinuria', Internal Medicine Journal, vol. 43, no. 3, pp. 298-307. https://doi.org/10.1111/j.1445-5994.2012.02924.x
    Weitz, I. ; Meyers, Gabrielle ; Lamy, T. ; Cahn, J. Y. ; Uranga, M. T. ; García Vela, J. A. ; Sanz, M. A. ; Severino, B. ; Kelly, R. J. ; Hillmen, P. ; Hill, A. / Cross-sectional validation study of patient-reported outcomes in patients with paroxysmal nocturnal haemoglobinuria. In: Internal Medicine Journal. 2013 ; Vol. 43, No. 3. pp. 298-307.
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    abstract = "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.",
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    AU - Meyers, Gabrielle

    AU - Lamy, T.

    AU - Cahn, J. Y.

    AU - Uranga, M. T.

    AU - García Vela, J. A.

    AU - Sanz, M. A.

    AU - Severino, B.

    AU - Kelly, R. J.

    AU - Hillmen, P.

    AU - Hill, A.

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    KW - Haemolysis

    KW - Paroxysmal

    KW - Quality of life

    KW - Soliris

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