Evidence map›Paper›PMID 37737189›Full record

ArticleTrials2023

Prospective identification of variables as outcomes for treatment (PIVOT): study protocol for a randomised, placebo-controlled trial of hydroxyurea for Ghanaian children and adults with haemoglobin SC disease.

Luke R Smart, Catherine I Segbefia, Teresa S Latham, Susan E Stuber, Kwesi N Amissah-Arthur, Klenam Dzefi-Tettey, Adam C Lane, Yvonne A Dei-Adomakoh, Russell E Ware

Open access · goldAbstract readClinical Trial Protocol
PubMed Full text at PMC Publisher
In one paragraph

Article in Trials, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 4 papers.

0numbers the graph read from it
0cells of the map it votes in
4citing papers in PubMed
1.4field-weighted citation impact, top 16% of its field
1 · What the graph read from 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.

2 · The registry

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.

3 · Its place in the literature

Who cites it

4 citing papers in PubMed, 5 citations in OpenAlex.

  1. Trial
  2. Trial
  3. Article
  4. The right patient, the right treatment: tailoring sickle cell disease care.Hematology. American Society of Hematology. Education Program · 2025
    Review
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

9 authors at 3 institutions in 2 countries.

Luke R SmartDivision of Hematology, Cincinnati Children's Hospital Medical Center, Cincinnati, USA. luke.smart@cchmc.org.ORCID http://orcid.org/0000-0003-4083-0060
Catherine I SegbefiaDepartment of Child Health, University of Ghana Medical School, Accra, Ghana.
Teresa S LathamDivision of Hematology, Cincinnati Children's Hospital Medical Center, Cincinnati, USA.
Susan E StuberDivision of Hematology, Cincinnati Children's Hospital Medical Center, Cincinnati, USA.
Kwesi N Amissah-ArthurOphthalmology Unit, Department of Surgery, University of Ghana Medical School, Accra, Ghana.
Klenam Dzefi-TetteyDepartment of Radiology, Korle Bu Teaching Hospital, Accra, Ghana.
Adam C LaneDivision of Hematology, Cincinnati Children's Hospital Medical Center, Cincinnati, USA.
Yvonne A Dei-AdomakohDepartment of Haematology, University of Ghana Medical School, Accra, Ghana.
Russell E WareDivision of Hematology, Cincinnati Children's Hospital Medical Center, Cincinnati, USA.
Cincinnati Children's Hospital Medical Center · USUniversity of Ghana · GHKorle Bu Teaching Hospital · GH

Funding

Sickle Cell Anemia, Splenic Pathology, and Hydroxyurea in Sub-Saharan AfricaK23HL153763 · NHLBI · CINCINNATI CHILDRENS HOSP MED CTR · PI SMART, LUKE · 2020 to 2024
$848k
NHLBI NIH HHS K23 HL153763
6 · The paper itself

Abstract

backgroundHaemoglobin SC (HbSC) is a common form of sickle cell disease (SCD), especially among individuals of West African ancestry. Persons with HbSC disease suffer from the same clinical complications and reduced quality of life that affect those with sickle cell anaemia (HbSS/Sβ

methodsPIVOT is a randomised, placebo-controlled, double blind clinical trial of hydroxyurea. Approximately 120 children and 120 adults ages 5-50 years with HbSC disease will be enrolled, screened for 2 months, and then randomised 1:1 to once-daily oral hydroxyurea or placebo. Study treatment will be prescribed initially at 20 ± 5 mg/kg/day with an opportunity to escalate the dose twice over the first 6 months. After 12 months of blinded study treatment, all participants will be offered open-label hydroxyurea for up to 4 years. Safety outcomes include treatment-related cytopenias, whole blood viscosity, and adverse events. Efficacy outcomes include a variety of laboratory and clinical parameters over the first 12 months of randomised treatment, including changes in haemoglobin and fetal haemoglobin, intracranial arterial velocities measured by transcranial Doppler ultrasound, cerebral oxygenation using near infrared spectrometry, spleen volume and kidney size by ultrasound, proteinuria, and retinal imaging. Exploratory outcomes include functional erythrocyte analyses with ektacytometry for red blood cell deformability and point-of-sickling, patient-reported outcomes using the PROMIS questionnaire, and 6-min walk test. DISCUSSION: For children and adults with HbSC disease, PIVOT will determine the safety of hydroxyurea and identify measurable changes in laboratory and clinical parameters, suitable for future prospective testing in a definitive multi-centre phase III clinical trial.

trial registrationPACTR, PACTR202108893981080. Registered 24 August 2021, https://pactr.samrc.ac.za.

Indexed as

Anemia, Sickle CellHemoglobin SC DiseaseAdultChildGhanaHumansHydroxyureaQuality of LifeRandomized Controlled Trials as TopicRetrospective StudiesHydroxyureaAdultsChildrenHaemoglobin SC diseaseHydroxyureaSickle cell disease

Identifiers

PMID37737189
PMCPMC10515018
OpenAlexW4386945393

What OpenQuestion holds

Textmetadata
LicenceCC BY
Read underepoch 390

Registered trials

None linked

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.