ArticleBlood advances2019
End points for sickle cell disease clinical trials: renal and cardiopulmonary, cure, and low-resource settings.
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 20 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
20 citing papers in PubMed, 1 synthesis or guideline pooled it.
- Systematic review of outcome reporting in studies of SCD and pregnancy: marked heterogeneity hinders meaningful data synthesis.Blood advances · 2023Pooled it
- Hydroxyurea for Children and Adults with Hemoglobin SC Disease.NEJM evidence · 2025Trial
- Baseline characteristics of Ghanaian children and adults enrolled in PIVOT, a randomised clinical trial of hydroxyurea in HbSC disease in sub-Saharan Africa.British journal of haematology · 2024Trial
- Trial
- Hydroxyurea in the management of sickle cell disease-associated priapism: a scoping review.International journal of impotence research · 2026Article
- Hydroxyurea pharmacokinetics in children with sickle cell anemia across different global populations.Blood advances · 2026Article
- Sickle Cell Diastolic Cardiomyopathy and Mortality Risk: A Novel Echocardiographic Framework for Prognostic Stratification.American journal of hematology · 2025Article
- A Patient Centric Model for Vaso-Occlusive Crises in Sickle Cell Disease-Outcomes of a Consensus Exercise Conducted Across Patients and Experts.Clinical and translational science · 2025Article
- Successes and pitfalls in orphan drug development for sickle cell disease.Blood advances · 2024Article
- Article
- Natural history and variability in albuminuria in pediatric and murine sickle cell anemia.Blood advances · 2023Article
- Multi-center retrospective study of children with sickle cell disease admitted to pediatric intensive care units in the United States.Scientific reports · 2023Article
- Organ function indications and potential improvements following curative therapy for sickle cell disease.Hematology. American Society of Hematology. Education Program · 2022Article
- Revisiting anemia in sickle cell disease and finding the balance with therapeutic approaches.Blood · 2022Article
- Evaluation of left ventricular systolic function in children with sickle cell anemia: contribution of 2D strain.F1000Research · 2022Article
- Article
- Clinical trial considerations in sickle cell disease: patient-reported outcomes, data elements, and the stakeholder engagement framework.Hematology. American Society of Hematology. Education Program · 2021Review
- coreSCD: multi-stakeholder consensus on core outcomes for sickle cell disease clinical trials.BMC medical research methodology · 2021Article
- What is the future of patient-reported outcomes in sickle-cell disease?Expert review of hematology · 2020Review
- End points for sickle cell disease clinical trials: patient-reported outcomes, pain, and the brain.Blood advances · 2019Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
27 authors.
Funding
Abstract
To address the global burden of sickle cell disease 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 outcome, pain (non-patient-reported outcomes), the brain, end-organ considerations, biomarkers, measurement of cure, and low-resource settings. This article presents the findings and recommendations of the end-organ considerations, measurement of cure, and low-resource settings panels as well as relevant findings and recommendations from the biomarkers panel.
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.