Premium
Cross‐sectional validation study of patient‐reported outcomes in patients with paroxysmal nocturnal haemoglobinuria
Author(s) -
Weitz I.,
Meyers G.,
Lamy T.,
Cahn J.Y.,
Uranga M. T.,
García Vela J. A.,
Sanz M. A.,
Severino B.,
Kelly R. J.,
Hillmen P.,
Hill A.
Publication year - 2013
Publication title -
internal medicine journal
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 0.596
H-Index - 70
eISSN - 1445-5994
pISSN - 1444-0903
DOI - 10.1111/j.1445-5994.2012.02924.x
Subject(s) - medicine , quality of life (healthcare) , population , cross sectional study , physical therapy , pathology , environmental health , nursing
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 F unctional A ssessment of C hronic I llness T herapy F atigue subscale version 4 ( FACIT ‐Fatigue) and the E uropean O rganization for R esearch and T reatment of C ancer Q uality‐of‐ L ife Q uestionnaire‐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 ‐ C 30 instruments to be relevant and adequate in assessing the level of PNH ‐associated fatigue and other quality‐of‐life measures. The FACIT ‐ F atigue questionnaire was considered to be clear and to comprehensively cover PNH ‐related fatigue. The EORTC QLQ ‐ C 30 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 ‐ C 30 versions to make it more relevant to PNH . Conclusions This study confirms the validity of the FACIT ‐Fatigue and the EORTC QLQ ‐ C 30 questionnaires in this patient population and their routine use should be considered in the management of patients with PNH .