Evidence map›Paper›PMID 35781728›Full record

ArticleClinical endocrinology2022

Analysis of therapy monitoring in the International Congenital Adrenal Hyperplasia Registry.

Neil Lawrence, Irina Bacila, Jeremy Dawson, Jillian Bryce, Salma R Ali, Erica L T van den Akker, Tânia A S S Bachega, Federico Baronio, Niels H Birkebaek, Walter Bonfig and 22 more

Open access · hybridAbstract read
In one paragraph

Article in Clinical endocrinology, 2022. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 3 papers.

0numbers the graph read from it
0cells of the map it votes in
3citing papers in PubMed
0.5field-weighted citation impact, top 39% 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

3 citing papers in PubMed, 8 citations in OpenAlex.

  1. Article
  2. Review
  3. 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

32 authors at 20 institutions in 18 countries.

Neil LawrenceDepartment of Oncology and Metabolism, University of Sheffield, Sheffield, UK.ORCID 0000-0002-7686-6172
Irina BacilaDepartment of Oncology and Metabolism, University of Sheffield, Sheffield, UK.
Jeremy DawsonInstitute of Work Psychology, Management School, University of Sheffield, Sheffield, UK.
Jillian BryceOffice for Rare Conditions, Royal Hospital for Children & Queen Elizabeth University Hospital, Glasgow, UK.
Salma R AliOffice for Rare Conditions, Royal Hospital for Children & Queen Elizabeth University Hospital, Glasgow, UK.
Erica L T van den AkkerDepartment of Pediatric Endocrinology, Sophia Children's Hospital, Erasmus Medical Centre, Rotterdam, the Netherlands.
Tânia A S S BachegaHormones and Molecular Genetics Laboratory LIM 42, Department of Internal Medicine, University of Sao Paulo, Sao Paulo, Brazil.
Federico BaronioDepartment of Medical and Surgical Sciences, Pediatric Unit, Endo-ERN Center for Rare Endocrine Diseases, S. Orsola-Malpighi University Hospital, Bologna, Italy.
Niels H BirkebaekDepartment of Pediatrics, Aarhus University Hospital, Aarhus, Denmark.
Walter BonfigDepartment of Pediatrics, Technical University Munich, Munich, Germany.ORCID 0000-0002-6797-7604
Hedi C van der GrintenDepartment of Pediatric Endocrinology, Radboud University Medical Centre, Nijmegen, the Netherlands.
Eduardo C CostaPediatric Surgery Service, Hospital de Clínicas de Porto Alegre, Porto Alegre, Brazil.
Liat de VriesInstitute for Diabetes and Endocrinology, Schneider's Children Medical Center of Israel, Petah-Tikvah, Israel.
Heba ElsedfyPediatrics Department, Ain Shams University, Cairo, Egypt.
Ayla GüvenBaskent University Istanbul Hospital, Pediatric Endocrinology, Istanbul, Turkey.
Sabine HannemaDepartment of Paediatric Endocrinology, Erasmus MC, Sophia Children's Hospital, University Medical Center Rotterdam, Rotterdam, the Netherlands.ORCID 0000-0002-8996-0993
Violeta IotovaDepartment of Paediatrics, Medical University of Varna, Varna, Bulgaria.
Hetty J van der KampPediatric Endocrinology Wilhelmina Children's Hospital, University Medical Centre Utrecht, Utrecht, the Netherlands.
María ClementePaediatric Endocrinology, Hospital Universitario Vall d'Hebron, CIBER de Enfermedades Raras (CIBERER) ISCIII, Barcelona, Spain.
Corina R LichiardopolDepartment of Endocrinology, University of Medicine and Pharmacy Craiova, Craiova, Romania.
Tatjana MilenkovicDepartment of Endocrinology, Institute for Mother and Child Healthcare of Serbia "Dr Vukan Čupić", Belgrade, Serbia.
Uta NeumannInstitute for Experimental Pediatric Endocrinology and Center for Chronically Sick Children, Charite-Universitätsmedizin, Berlin, Germany.ORCID 0000-0003-3892-0577
Ana NordenströmDepartment of Women's and Children's Health, Karolinska Institutet, Stockholm, Sweden.ORCID 0000-0003-0405-3401
Şukran PoyrazoğluIstanbul Faculty of Medicine, Paediatric Endocrinology Unit, Istanbul University, Istanbul, Turkey.
Ursina Probst-ScheideggerPediatric Department, Kantonsspital Winterthur, Winterthur, Switzerland.
Luisa De SanctisPaediatric Endocrinology, Regina Margherita Children's Hospital, Torino, Italy.
Rieko Tadokoro-CuccaroDepartment of Pediatrics, University of Cambridge, Cambridge, United Kingdom Biomedical Campus, Cambridge, UK.
Ajay ThankamonyDepartment of Pediatrics, University of Cambridge, Cambridge, United Kingdom Biomedical Campus, Cambridge, UK.
Ana VieitesCentro de Investigaciones Endocrinológicas (CEDIE-CONICET), Hospital de Niños Ricardo Gutiérrez, Buenos Aires, Argentina.
Zehra YavaşPediatric Endocrinology and Diabetes, Marmara University, Istanbul, Turkey.
Syed Faisal AhmedOffice for Rare Conditions, Royal Hospital for Children & Queen Elizabeth University Hospital, Glasgow, UK.ORCID 0000-0003-0689-5549
Nils KroneDepartment of Oncology and Metabolism, University of Sheffield, Sheffield, UK.ORCID 0000-0002-3402-4727
Royal Hospital for Children · GBSheffield Children's NHS Foundation Trust · GBUniversity of Cambridge · GBUniversity of Sheffield · GB5T Torino (Italy) · ITAarhus University Hospital · DKAin Shams University · EGBaşkent University · TRCharité - Universitätsmedizin Berlin · DEConsejo Nacional de Investigaciones Científicas y Técnicas · ARErasmus MC - Sophia Children’s Hospital · NLHospital de Clínicas de Porto Alegre · BRInstitute of Public Health of Serbia · RSInstituto de Salud Carlos III · ESIRCCS Azienda Ospedliero-Universitaria di Bologna Policlinico di Sant'Orsola · ITIstanbul University · TRKantonsspital Winterthur · CHKarolinska University Hospital · SEKlinikum Wels-Grieskirchen · ATLeiden University · NL

Funding

Department of HealthMedical Research Council G1100236
6 · The paper itself

Abstract

objectiveCongenital adrenal hyperplasia (CAH) requires exogenous steroid replacement. Treatment is commonly monitored by measuring 17-OH progesterone (17OHP) and androstenedione (D4).

designRetrospective cohort study using real-world data to evaluate 17OHP and D4 in relation to hydrocortisone (HC) dose in CAH patients treated in 14 countries. PATIENTS: Pseudonymized data from children with 21-hydroxylase deficiency (21OHD) recorded in the International CAH Registry. MEASUREMENTS: Assessments between January 2000 and October 2020 in patients prescribed HC were reviewed to summarise biomarkers 17OHP and D4 and HC dose. Longitudinal assessment of measures was carried out using linear mixed-effects models (LMEM).

resultsCohort of 345 patients, 52.2% female, median age 4.3 years (interquartile range: 3.1-9.2) were taking a median 11.3 mg/m DISCUSSION: Registry data show large variability in 17OHP and D4 between centres. 17OHP correlates with D4 well when accounting for age. Prescribed HC dose per body surface area decreased with weight gain.

Indexed as

Adrenal Hyperplasia, Congenital17-alpha-HydroxyprogesteroneAndrostenedioneChildChild, PreschoolFemaleHumansHydrocortisoneMaleProgesteroneRegistriesRetrospective Studies17-alpha-HydroxyprogesteroneAndrostenedioneHydrocortisoneProgesteronebiomarkerscongenital adrenal hyperplasiahydrocortisonelinear mixed-effects models

Identifiers

PMID35781728
PMCPMC9796837
OpenAlexW4283790260

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.