ReviewExpert review of hematology2020
What is the future of patient-reported outcomes in sickle-cell disease?
Review in Expert review of hematology, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 13 papers, 1 of them a synthesis that pooled it.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
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
13 citing papers in PubMed, 1 synthesis or guideline pooled it, 20 citations in OpenAlex.
- Health-related quality of life in hemoglobinopathies: A systematic review from a global perspective.Frontiers in pediatrics · 2022Pooled it
- Arginine Therapy for Pain in Sickle Cell Disease: A Phase-2 Randomized, Placebo-Controlled Trial.American journal of hematology · 2025Trial
- Article
- A Scoping Review of Qualitative Studies on Inpatient Care for Individuals with Sickle Cell Disease.Journal of general internal medicine · 2026Article
- Development of a Novel Patient-Reported Outcome Diary to Capture and Monitor Home-Managed Pain Crises in Patients with Sickle Cell Disease.Advances in therapy · 2026Article
- Emergency care for children with sickle cell disease: a focus on pain and fever.Expert review of hematology · 2025Review
- Long-term outcomes of avascular necrosis in sickle cell disease using joint-specific patient-reported outcome measures: Results from a multicentre study.British journal of haematology · 2025Article
- Patient-reported outcomes in children with sickle cell disease at presentation for an acute pain episode.Blood advances · 2023Article
- Article
- Patient-Reported Outcome Measures in Patients with Thrombotic Thrombocytopenic Purpura: A Systematic Review of the Literature.Journal of clinical medicine · 2023Review
- Intraindividual pain variability metrics for youth with sickle cell disease: Relations to health outcomes.Pediatric blood & cancer · 2023Article
- Health-related quality of life and fatigue in children and adults with pyruvate kinase deficiency.Blood advances · 2022Article
- Patient-reported neurocognitive symptoms influence instrumental activities of daily living in sickle cell disease.American journal of hematology · 2021Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
4 authors at 2 institutions in 1 country.
Funding
Abstract
introductionSickle cell disease (SCD) is a complex, chronic disease caused by abnormal polymerization of hemoglobin, which leads to severe pain episodes, fatigue, and end-organ damage. Patient reported outcomes (PROs) have emerged as a critical tool for measuring SCD disease severity and response to treatment. AREAS COVERED: Authors review the key issues involved when deciding to use a PRO in a clinical trial. We describe the most highly recommended generic and disease-specific PRO tools in SCD and discuss the challenges of incorporating them in clinical practice. EXPERT OPINION: PRO measures are essential to incorporate into SCD clinical trials either as primary or secondary outcomes. The use of PRO measures in SCD facilitates a patient-centered approach, which is likely to lead to improved outcomes. Significant challenges remain in adapting PRO tools to routine clinical use and in developing countries.
Indexed as
Identifiers
What OpenQuestion holds
Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.