5610614 ASSESSING PHYSICAL, SOCIAL AND EMOTIONAL IMPACT OF SICKLE CELL DISEASE IN PORTUGUESE PATIENTS: AN OBSERVATIONAL, MULTICENTER STUDY (ASCEND)

Abstract

Background: Sickle cell disease (SCD) is a chronic, multisystem autosomal recessive blood disorder that is characterized by painful episodes caused by vaso-occlusion. SCD patients can face social, economic and academic challenges, as well as barriers to accessing quality heath care. Evidence about the impact of SCD and related treatments on the lives of patients is lacking, with a broad definition of health as a combination of physical, mental and social well-being. The purpose of this observational study is to characterize the physical, social and emotional impact of SCD on adult patients with confirmed diagnosis and to characterize patient pathway in the healthcare system. Objectives: The primary objective of ASCEND is to characterize the physical impact of SCD on patients, through collection of PROs that will assess the impact of pain and disease complications in patients’ QoL. As secondary objectives the study aims to characterize SCD patients’ demographics and clinical history, as well as to describe the social and emotional impact of SCD on patients; to describe SCD patient pathway in the healthcare system. In this abstract the authors intend to describe the results obtained for QoL assessment through PRO questionnaires Methods: ASCEND is non-interventional cross-sectional study of 2 cohorts of SCD adult patients with SCD. The study sample is 200 (recruited by hospital sites) + 50 patients (recruited by patient association). PRO questionnaires include both EQ-5D-5L and ASCQ-Me (Pain Episodes Frequency and Severity). Portuguese patients and Social Studies investigators participated in the validation process. Results: Study recruitment is terminating in the current week as 194 have been included already. We expect to have QoL results evaluated through EQ-5D-5L and ASQC-Me for 200 patients by mid September 2022, in due time to be presented in ASCAT. Conclusions: Conclusions will be elaborated upon data analyses