Evidence map›Paper›PMID 40441700›Full record

ArticleBritish journal of clinical pharmacology2025

The feasibility of pharmacokinetic-based dosing of hydroxyurea for children with sickle cell anaemia in Uganda: Baseline results of the alternative dosing and prevention of transfusions trial.

Alexandra Power-Hays, Ruth Namazzi, Min Dong, Caroline Kazinga, Charles Kato, Sadat Aliwuya, Kathryn McElhinney, Andrea L Conroy, Adam Lane, Chandy John and 4 more

Registry-linked trialAbstract readClinical Trial, Phase I
In one paragraph

Article in British journal of clinical pharmacology, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT05662098 (Alternative Dosing And Prevention of Transfusions), which is not on this map. Cited by 2 papers.

0numbers the graph read from it
0cells of the map it votes in
2citing papers in PubMed
–field-weighted citation impact
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.

NCT05662098 early_phase1active not recruitingnot on this map

Alternative Dosing And Prevention of Transfusions (ADAPT): A Prospective Study to Reduce Transfusion Requirements for Children With Sickle Cell Anemia Using Pharmacokinetics-based Hydroxyurea Dosing

TypeinterventionalSponsorChildren's Hospital Medical Center, CincinnatiRan2022 to 2027Enrolled100ConditionsSickle Cell DiseaseArmsHydroxyurea
3 · Its place in the literature

Who cites it

2 citing papers in PubMed.

  1. Article
  2. Article
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

14 authors.

Alexandra Power-HaysDivision of Hematology, Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio, USA.ORCID https://orcid.org/0000-0002-5823-0218
Ruth NamazziDepartment of Paediatrics and Child Health, Makerere School of Medicine, Kampala, Uganda.
Min DongDivision of Hematology, Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio, USA.ORCID https://orcid.org/0000-0002-8552-8298
Caroline KazingaGlobal Health Uganda, Kampala, Uganda.
Charles KatoGlobal Health Uganda, Kampala, Uganda.
Sadat AliwuyaGlobal Health Uganda, Kampala, Uganda.ORCID https://orcid.org/0009-0007-0334-4449
Kathryn McElhinneyDivision of Hematology, Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio, USA.
Andrea L ConroyGlobal Health Uganda, Kampala, Uganda.
Adam LaneDivision of Hematology, Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio, USA.
Chandy JohnDivision of Pediatric Infectious Disease, Indiana University School of Medicine, Indianapolis, Indiana, USA.
Alexander A VinksDepartment of Pediatrics, University of Cincinnati, Cincinnati, Ohio, USA.ORCID https://orcid.org/0000-0003-4224-2967
Teresa LathamDivision of Hematology, Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio, USA.
Robert O OpokaDepartment of Paediatrics, Aga Khan University, Nairobi, Kenya.
Russell E WareDivision of Hematology, Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio, USA.

Funding

Realizing Effectiveness Across Continents with Hydroxyurea(REACH): A Phase I/II Pilot Study of Hyroxyurea for Children with Sickle Cell AnemiaU01HL133883 · NHLBI · CINCINNATI CHILDRENS HOSP MED CTR · PI Russell E Ware · 2017 to 2026
$10.6M
T32 Cincinnati Pediatric Clinical Pharmacology Training ProgramT32HD069054 · NICHD · CINCINNATI CHILDRENS HOSP MED CTR · PI TRACY A GLAUSER, Sonya C Tang Girdwood · 2011 to 2026
$3.0M
American Society of Haematology Minority Haematology Fellow AwardCincinnati Children's Research FoundationDoris Duke Charitable Foundation Physician-Scientist Award 2021097National Institute of Child Health and Human Development 3T32HD069054-12S1NHLBI NIH HHS U01 HL133883NICHD NIH HHS T32 HD069054
6 · The paper itself

Abstract

Pharmacokinetic (PK)-guided dosing of hydroxyurea for children with sickle cell anaemia (SCA) could optimize dosing and improve outcomes, but its feasibility has not been demonstrated in low-resource settings where the majority of affected children live. Alternative Dosing And Prevention of Transfusions (ADAPT) is a prospective trial evaluating blood transfusions and the feasibility of determining PK-guided, hydroxyurea maximum tolerated doses (MTD) for children with SCA in Uganda, using portable high-performance liquid chromatography (HPLC) and a novel PK software programme (HdxSim). ADAPT enrolled 106 participants, and 100% completed PK testing. PK-guided doses were generated for 78%, of which 38% were within the protocol-defined range. Accurately, measuring serum hydroxyurea concentrations via HPLC and the potential for hydroxyurea degradation impacted the feasibility. Ensuring that people with SCA globally have access to hydroxyurea is imperative, and improving treatment strategies requires ongoing innovation including PK-guided dosing. ADAPT is registered at ClinicalTrials.gov (NCT05662098).

Indexed as

Anemia, Sickle CellAntisickling AgentsHydroxyureaAdolescentBlood TransfusionChildChild, PreschoolChromatography, High Pressure LiquidDose-Response Relationship, DrugFeasibility StudiesFemaleHumansMaleMaximum Tolerated DoseProspective StudiesUgandaAntisickling AgentsHydroxyureaclinical pharmacologyhaematologyHPLC < drug analysispaediatricspharmacokinetics

Identifiers

PMID40441700
PMCPMC12122129

What OpenQuestion holds

Textmetadata
LicenceCC BY-NC-ND
Read underepoch 390

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.