Abstract
BackgroundSickle cell disease (SCD) is a chronic condition associated with high mortality and morbidity. It is characterized by acute clinical symptoms such as painful vaso-occlusive crises, which can impair health-related quality of life (HRQL). This study was conducted to identify validated patient-reported outcome (PRO) instruments for use in future trials of potential treatments for SCD.MethodsA systematic literature review (SLR) was performed using MEDLINE and EMBASE to identify United States (US)-based studies published in English between 1997 and 2017 that reported on validated PRO instruments used in randomized controlled trials and real-world settings. The COnsensus-based Standards for the selection of health Measurement INstruments (COSMIN) checklist was used to assess the quality of PRO instruments.ResultsThe SLR included 21 studies assessing the psychometric properties of 24 PRO instruments. Fifteen of those instruments were developed and validated for adults and 10 for children (one instrument was used in both children and young adults aged up to 21 years). Only five of the 15 adult instruments and three of the 10 pediatric instruments were developed specifically for SCD. For most instruments, there were few or no data on validation conducted in SCD development cohorts. Of the 24 PRO instruments identified, 16 had strong internal reliability (Cronbach’s α ≥0.80). There was often insufficient information to assess the content validity, construct validity, responsiveness, or test-retest reliability of the instruments identified for both child and adult populations. No validated PRO instruments measuring caregiver burden in SCD were identified.ConclusionsThe evidence on the psychometric properties of PRO instruments was limited. However, the results of this SLR provide key information on such tools to help inform the design of future clinical trials for patients with SCD in the US.
Highlights
Sickle cell disease (SCD) is a chronic condition associated with high mortality and morbidity
The evidence on the psychometric properties of patient-reported outcome (PRO) instruments was limited. The results of this systematic literature review (SLR) provide key information on such tools to help inform the design of future clinical trials for patients with SCD in the United States (US)
Following full-text assessment, 19 studies reporting on the psychometric properties of PRO instruments were identified, and two more articles were added from manual searches of bibliographies of published SLRs
Summary
Sickle cell disease (SCD) is a chronic condition associated with high mortality and morbidity. It is characterized by acute clinical symptoms such as painful vaso-occlusive crises, which can impair health-related quality of life (HRQL). Sickle cell disease (SCD) is a lifelong, multisystem condition characterized by hemoglobin polymerization that leads to erythrocyte rigidity, hemolysis, and vasoocclusion. Vaso-occlusive crises (VOC) and pain associated with such crises are hallmark symptoms in SCD, and typically first manifest in infants around the age of 5 months. These painful episodes can occur without warning and have been described as sharp, intense stabbing or throbbing. Complications of SCD, such as anemia, infection, Sarri et al Health and Quality of Life Outcomes (2018) 16:99 stroke can have major physiological, cognitive, and emotional effects on patients [2, 3]
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