ArticleBlood advances2019
End points for sickle cell disease clinical trials: patient-reported outcomes, pain, and the brain.
Article in Blood advances, 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 45 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
45 citing papers in PubMed, 1 synthesis or guideline pooled it.
- Health State Utilities for Sickle Cell Disease: A Catalog Prepared From a Systematic Review.Value in health : the journal of the International Society for Pharmacoeconomics and Outcomes Research · 2022Pooled it
- Trial
- Introduction: A SEQUINS inductee recalls taking aim at inequities.Equity neuroscience · 2026Article
- Cerebral Blood Transit in Sickle Cell Anemia.Journal of magnetic resonance imaging : JMRI · 2026Article
- Cure Sickle Cell Initiative recommendations on common data elements for sickle cell disease gene therapy trials.Blood advances · 2026Article
- Gene expression profiling identifies potential biomarkers for vaso-occlusive episodes in sickle cell disease.JCI insight · 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
- The ASH Research Collaborative Sickle Cell Disease Research Network: past, present, and future.Blood advances · 2026Review
- Sickle cell disease and thalassemia go to the PROm.Blood advances · 2025Article
- Prospective cohort study of eDiary acute pain assessments in sickle cell disease during third trimester and postpartum.Blood advances · 2025Article
- A road map for uniform, comprehensive long-term follow-up after curative therapy for sickle cell disease.Blood advances · 2025Review
- Can we use biomarkers to identify those at risk of acute pain from sickle cell disease?Expert review of hematology · 2024Review
- Successes and pitfalls in orphan drug development for sickle cell disease.Blood advances · 2024Article
- Article
- Patient-reported outcomes in children with sickle cell disease at presentation for an acute pain episode.Blood advances · 2023Article
- Preliminary construct validity of patient-reported outcomes to assess chronic pain in adults with sickle cell disease.Blood advances · 2023Article
- Pharmacogenetics of pain management in Zimbabwean patients with sickle cell disease.Pharmacogenomics · 2023Article
- Intraindividual pain variability metrics for youth with sickle cell disease: Relations to health outcomes.Pediatric blood & cancer · 2023Article
- Comparison of CAT and short forms for PROMIS pain and physical health domains in children with sickle cell disease.Journal of patient-reported outcomes · 2023Article
- Association of Cerebral Hemodynamics and Anemia on Processing Speed in Adults with Sickle Cell Disease.Journal of neurology and experimental neural science · 2023Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
26 authors.
Funding
Abstract
To address the global burden of sickle cell disease (SCD) and the need for novel therapies, the American Society of Hematology partnered with the US Food and Drug Administration to engage the work of 7 panels of clinicians, investigators, and patients to develop consensus recommendations for clinical trial end points. The panels conducted their work through literature reviews, assessment of available evidence, and expert judgment focusing on end points related to: patient-reported outcomes (PROs), pain (non-PROs), the brain, end-organ considerations, biomarkers, measurement of cure, and low-resource settings. This article presents the findings and recommendations of the PROs, pain, and brain panels, as well as relevant findings and recommendations from the biomarkers panel. The panels identify end points, where there were supporting data, to use in clinical trials of SCD. In addition, the panels discuss where further research is needed to support the development and validation of additional clinical trial end points.
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.