SynthesisHealth and quality of life outcomes2018
Systematic literature review and assessment of patient-reported outcome instruments in sickle cell disease.
Synthesis in Health and quality of life outcomes, 2018. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 12 papers.
What it found
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
12 citing papers in PubMed.
- Patient-reported outcomes in sickle cell disease and association with clinical and psychosocial factors: Report from the sickle cell disease implementation consortium.American journal of hematology · 2020Trial
- Measuring and valuing spillover effects in caregivers and families: A scoping review.PloS one · 2026Article
- Understanding the role of asthma in acute chest syndrome: a comparative analysis of patients with and without asthma.Annals of hematology · 2025Article
- An evaluation of patient-reported outcomes in sickle cell disease within a conceptual model.Quality of life research : an international journal of quality of life aspects of treatment, care and rehabilitation · 2022Article
- Treatment Options That Reduce the Duration of Sickle Cell Vaso-Occlusive Crises: A Systematic Review.Cureus · 2022Review
- Health-Related Quality of Life Assessments by Children and Adolescents with Sickle Cell Disease and Their Parents in Portugal.Children (Basel, Switzerland) · 2022Article
- External validation of the priapism impact profile in a Jamaican cohort of patients with sickle cell disease.PloS one · 2021Article
- What is the future of patient-reported outcomes in sickle-cell disease?Expert review of hematology · 2020Review
- Quality of life assessments in a cohort of Mozambican children with sickle cell disease.The Pan African medical journal · 2020Article
- Patient-Reported Outcomes and Economic Burden of Adults with Sickle Cell Disease in the United States: A Systematic Review.International journal of general medicine · 2020Article
- A preliminary investigation of the psychometric properties of PROMIS® scales in emerging adults with sickle cell disease.Health psychology : official journal of the Division of Health Psychology, American Psychological Association · 2019Article
- The New Paradigms in Clinical Research: From Early Access Programs to the Novel Therapeutic Approaches for Unmet Medical Needs.Frontiers in pharmacology · 2019Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
7 authors.
Funding
No grant is acknowledged in the PubMed record.
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.
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