Central European Journal of Medicine Development and validation of QOL-E© instrument for the assessment of health-related quality of life in myelodysplastic syndromes * E-mail: estheroliva@hotmail.com Received 27 February 2013; Accepted 27 April 2013 Abstract: Objective. Supportive care and sustained health-related quality of life (HRQoL) are essential in the management of myelodysplastic syndromes (MDS), yet specifc instruments for the measurement of HRQoL in MDS are lacking. We report on the development and validation of a psychometric questionnaire assessing HRQoL in MDS patients (QOL-E©). Methods. The questionnaire was developed in three stages. First, a Medline search and interviews in focus groups generated a list of concepts important to MDS patients. Second, pilot (derivation) study was performed in a cross-sectional sample of 52 MDS patients. Third, feld (validation) testing in a clinical setting investigated psychometric properties in 147 MDS patients from six cohorts. Results. Forty-eight items were identifed, and fne-tuned to a 37-item list, then a fnal 29-item questionnaire containing a general well-being dimension, four general health dimensions (physical, functional, social, and sexual), and disease-related dimensions (fatigue and MDS-related disturbances). Conclusion. Cognitive debriefng and psychometric analyses demonstrated good internal validity and patient acceptability. The QOL-E© is the frst HRQoL instrument developed specifcally for MDS patients. © Versita Sp. z o.o Keywords: Myelodysplastic syndromes • Anaemia • QOL-E© questionnaire • Health-related quality of lif 1 Hematology Unit, Azienda Ospedaliera ‘Bianchi-Melacrino-Morelli’, Reggio Calabria, Italy 2 Division of Population Health Sciences, Royal College of Surgeons in Ireland, Dublin, Ireland 3 Academic Unit of Primary Care and Population Sciences, University of Southampton, Southampton, United Kingdom Esther N Oliva* 1 , Francesco Nobile 1 , Borislav D Dimitrov 2,3 Research Article 1. Introduction Myelodysplastic syndromes (MDS, ICD-10-CM D46) are a heterogeneous group of myeloid neoplasms characterized by ineffective haematopoiesis, peripheral blood cytopenias and abnormal cellular morphologies that typically occur in elderly individuals. According to the presence or absence of certain MDS features, which include severity of anaemia, transfusion-dependence, number of peripheral cytopenias, blast counts, cytoge- netics, bone marrow fbrosis, and molecular biomarkers, patients are at a variable risk of progression to acute my- eloid leukaemia [1,2]. Myelodysplastic syndromes may be cured with allogeneic hematopoietic stem cell trans- plantation, but advanced age and patient comorbidities at diagnosis often preclude this therapeutic option [3]. Although treatments, such as erythropoiesis-stimulating agents, lenalidomide, decitabine, and azacytidine, have improved myelodysplastic syndromes management, treatment ultimately fails in most patients [4]. Thus, supportive care and sustained health-related quality of life (HRQoL) are essential in MDS management. The main treatment for myelodysplastic syndromes is red blood cell transfusion to attenuate the most important clinical features of chronic anaemia, which manifest as fatigue and frequent infections [5-7]. Disease burden Cent. Eur. J. Med. • 8(6) • 2013 • 835-844 DOI: 10.2478/s11536-013-0196-z 835 Unauthenticated Download Date | 7/24/18 4:57 PM