Evidence map›Paper›PMID 41782560›Full record

ArticleEndocrine connections2026

Glucocorticoid-prescribing trends in congenital adrenal hyperplasia, 2017 to 2023.

A Roxas, A Gihawi, M Makarchuk, J Bryce, M Chen, S F Ahmed, S R Ali, A Drake, M Casipe, L Groves and 61 more

Abstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Endocrine connections, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 1 paper.

0numbers the graph read from it
0cells of the map it votes in
1citing 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.

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

1 citing paper in PubMed.

  1. 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

71 authors.

A RoxasNorwich Medical School, University of East Anglia, Norwich, UK.ORCID https://orcid.org/0009-0007-2326-5131
A GihawiNorwich Medical School, University of East Anglia, Norwich, UK.
M MakarchukNorwich Medical School, University of East Anglia, Norwich, UK.
J BryceOffice for Rare Conditions, Royal Hospital for Children & Queen Elizabeth University Hospital, Glasgow, United Kingdom.
M ChenOffice for Rare Conditions, Royal Hospital for Children & Queen Elizabeth University Hospital, Glasgow, United Kingdom.
S F AhmedOffice for Rare Conditions, Royal Hospital for Children & Queen Elizabeth University Hospital, Glasgow, United Kingdom.ORCID https://orcid.org/0000-0003-0689-5549
S R AliOffice for Rare Conditions, Royal Hospital for Children & Queen Elizabeth University Hospital, Glasgow, United Kingdom.ORCID https://orcid.org/0000-0003-2178-269X
A DrakeNorwich Medical School, University of East Anglia, Norwich, UK.
M CasipeRoyal Devon University Healthcare NHS Foundation Trust, Exeter, UK.
L GrovesNorwich Medical School, University of East Anglia, Norwich, UK.
J IdkowiakDepartment of Paediatric Endocrinology, Birmingham Women's & Children's Hospital, Birmingham, UK.
R KroneDepartment of Paediatric Endocrinology, Birmingham Women's & Children's Hospital, Birmingham, UK.
C FlueckPediatric Endocrinology, Diabetology and Metabolism, Department of Pediatrics and Department of Biomedical Research, Bern University Hospital Inselspital, University of Bern, Bern, Switzerland.
A NordenströmDepartment of Women's and Children's Health, Karolinska Institute, Stockholm, Sweden.
N ReischMedizinische Klinik IV, Ludwig-Maximillian University Hospital Munich, Munich, Germany.ORCID https://orcid.org/0000-0002-7469-6069
H L Claahsen-van der GrintenDepartment of Paediatrics, Division of Paediatric Endocrinology, Radboud University Medical Centre, Amalia Children's Hospital, Nijmegen, The Netherlands.
B P H AdriaansenDepartment of Paediatrics, Division of Paediatric Endocrinology, Radboud University Medical Centre, Amalia Children's Hospital, Nijmegen, The Netherlands.
N H BirkebaekDepartment of Paediatrics, Aarhus University Hospital, Aarhus, Denmark.
S HannemaDepartment of Pediatric Endocrinology, Amsterdam UMC location Vrije Universiteit Amsterdam, Amsterdam, The Netherlands.
M W O ReillyBeaumont Hospital and RCSI University of Medicine and Health Sciences, Dublin, Ireland.
L CussenBeaumont Hospital and RCSI University of Medicine and Health Sciences, Dublin, Ireland.
S P ZaricDepartment of Endocrinology, Institute for Mother and Child Healthcare of Serbia, Belgrade, Serbia.
U NeumannInstitute for Experimental Paediatric Endocrinology, Charité Universitätsmedizin Berlin, Berlin, Germany.ORCID https://orcid.org/0000-0003-3892-0577
F BaronioDepartment Hospital of Woman and Child, Pediatric Unit, Center for Rare Endocrine Conditions (Endo-ERN), IRCCS - S.Orsola-Malpighi University Hospital, Bologna, Italy.
A VieitesCentro de Investigaciones Endocrinológicas, División de Endocrinología, Hospital de Niños Ricardo Gutiérrez, Buenos Aires, Argentina.
G F AlonsoSección Endocrinología Pediátrica, Hospital Italiano de Buenos Aires, Buenos Aires, Argentina.
H ElsedfyDepartment of Pediatrics, Ain Shams University, Cairo, Egypt.
I MazenClinical Genetics Department, Human Genetics and Genome Research Institute, National Research Centre, Cairo, Egypt.
A ThankamonyDepartment of Paediatrics, University of Cambridge, Cambridge, UK.
J WitczakNeuroscience and Mental Health Innovation Institute, School of Medicine, Cardiff University, Cardiff, UK.
D A ReesNeuroscience and Mental Health Innovation Institute, School of Medicine, Cardiff University, Cardiff, UK.
N AtapattuEndocrinology, Lady Ridgeway Hospital for Children, Colombo, Sri Lanka.
S N SeneviratneDepartment of Paediatrics, Faculty of Medicine, University of Colombo, Colombo, Sri Lanka.
M CoolsDepartment of Internal Medicine and Paediatrics, Ghent University, and Department of Paediatric Endocrinology, Ghent University Hospital, Ghent, Belgium.
Hayat El KaddouriDepartment of Internal Medicine and Paediatrics, Ghent University, and Department of Paediatric Endocrinology, Ghent University Hospital, Ghent, Belgium.
Alegria Ferri PerezDepartment of Internal Medicine and Paediatrics, Ghent University, and Department of Paediatric Endocrinology, Ghent University Hospital, Ghent, Belgium.
A GuvenEmeritus Professor of Health Science University, İstanbul, Turkey.
S PoyrazogluIstanbul Faculty of Medicine, Pediatric Endocrinology Unit, Istanbul University, Istanbul, Turkey.
A FuDepartment of Paediatrics, Princess Margaret Hospital, Kowloon, Hong Kong.
D JanusDepartment of Paediatric and Adolescent Endocrinology, University Children's Hospital, Jagiellonian University, Krakow, Poland.
E GlobaUkrainian Research Centre of Endocrine Surgery, Endocrine Organs and Tissue Transplantation, MoH of Ukraine, Kyiv, Ukraine.
S ShenoyDepartment of General Paediatrics and Endocrinology, Leicester Children's Hospital, University Hospitals of Leicester NHS Trust, Leicester, UK.
C de BruinDivision of Endocrinology, Department of Pediatrics, Willem-Alexander Children's Hospital, Leiden University Medical Center, Leiden, The Netherlands.
M KorbonitsCentre for Endocrinology, William Harvey Research Institute, Barts and The London School of Medicine and Dentistry, Queen Mary University of London, London, UK.
S AdamDepartment of Endocrinology, The Christie Hospital NHS Foundation Trust, Manchester, UK.
M WasniewskaUnit of Pediatrics, Department of Human Pathology of Adulthood and Childhood, University of Messina, Messina, Italy.ORCID https://orcid.org/0000-0002-8299-2677
G RussoIRCCS Ospedale San Raffaele, Milan, Italy.
F Phan-HugPediatric Endocrinologist, Hôpital de Morges, Morges, Switzerland.
W BonfigDepartment of Pediatrics, Technical University of Munich, TUM School of Medicine, Munich, Germany.
M SalernoDepartment of Translational Medical Sciences, Paediatric Endocrinology Unit, University of Naples 'Federico II', Naples, Italy.
J W TomlinsonOxford Center for Diabetes, Endocrinology and Metabolism, University of Oxford, Oxford, UK.ORCID https://orcid.org/0000-0002-3170-8533
S Leka-EmiriDepartment of Endocrinology-Growth and Development, 'P & A Kyriakou' Children's Hospital, Athens, Greece.
L de VriesThe Jesse Z and Sara Lea Shafer Institute for Endocrinology and Diabetes, National Center for Childhood Diabetes, Schneider Children's Medical Center of Israel, Petah Tikva, Israel.
I YarhereEndocrinology Unit, Paediatrics Department, University of Port Harcourt Teaching Hospital, Port Harcourt, Nigeria.
G Guaranga-FilhoUniversidade Feberal do Rio Grande dol Sul (UFRDS), Porto Alegre, Brazil.
J van EckDepartment of Pediatrics, Div Pediatric Endocrinology, Erasmus MC Center of Expertise for DSD, Erasmus Medical Center-Sophia Children's Hospital, Rotterdam, Netherlands.
T A S S BachegaUnidade de Endocrinologia do Desenvolvimento, Laboratório de Hormônios e Genética Molecular/LIM42, Disciplina de Endocrinologia, Hospital Das Clinicas, Faculdade De Medicina, Universidade de Sao Paulo, São Paulo, Brazil.
N KroneDepartment of Oncology and Metabolism, University of Sheffield, Sheffield, UK.
M De BonoDepartment of Endocrinology, Sheffield Teaching Hospitals NHS Foundation Trust, Sheffield, UK.
J H DaviesFaculty of Medicine, University of Southampton, Southampton, UK.
A Segev-BeckerSackler Faculty of Medicine, Tel Aviv University, Tel Aviv, Israel.
V IotovaMedical University Varna, Varna, Bulgaria.ORCID https://orcid.org/0000-0003-4160-1200
N Lenherr-TaubeDivision of Pediatric Endocrinology and Diabetology, University Children's Hospital, University of Zurich, Zurich, Switzerland.
A GermanThe Ruth and Bruce Rappaport Faculty of Medicine, Technion-Israel Institute of Technology, Haifa, Israel.
R GiordanoDepartment of Biological and Clinical Sciences, University of Turin, Turin, Italy.
L De SanctisPediatric Endocrinology Unit, Regina Margherita Children's Hospital, University of Turin, Turin, Italy.
U ProbstPediatric Department, Kantonsspital Winterthur, Winterthur, Switzerland.
R MarkosyanEndocrinology, Yerevan State Medical University Endocrinology Clinic, Yerevan, Armenia.
D S BrewerMetabolic Health Research Centre, Norwich Medical School, University of East Anglia, Norwich, UK.
E C CostaHospital de Clinical de Porto Alegre, Porto Alegre, Brazil.
E A WebbNorwich Medical School, University of East Anglia, Norwich, UK.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Objective: This study investigates the utilisation of modern glucocorticoid medications (Acecort®, Alkindi®, Efmody®, and Plenadren®) for congenital adrenal hyperplasia due to 21-hydroxylase deficiency, examining prescribing patterns, barriers to adoption, and geographical and temporal trends. Methods: A two-part study was conducted: a retrospective analysis of treatment regimens from the International Congenital Adrenal Hyperplasia Registry across 46 centres in 20 countries (2017-2023) and a qualitative survey of 39 centres regarding barriers to prescribing modern medications. Patients included both paediatric and adult populations. Data analysed included regional prescription trends, timing of modern glucocorticoid adoption, and identified barriers. Results: From 2017 to 2023, 44 of 790 (5%) patients transitioned from traditional to modern glucocorticoid therapy, with the highest adoption in high-income Western European countries. Alkindi® was exclusively prescribed to patients under 8 years, while 97% of Efmody® users were 7 years or older. By 2023, modern glucocorticoid availability varied among centres: Alkindi® (54%), Efmody® (46%), Plenadren® (33%), and Acecort® (15%). Conclusion: Adoption of modern glucocorticoid medications for congenital adrenal hyperplasia remains limited, with only approximately 5% of patients transitioning from traditional therapies. Significant barriers include legislative approval, supply chain challenges, and elevated costs. Plain language summary: This international study looked at how new medications for congenital adrenal hyperplasia are used globally. We found that despite increasing availability of new medications during the study time period, only a small number of patients (5%) switched to these newer treatments. This limited use is mainly due to high costs, problems with getting legal approval, and supply issues, highlighting unequal access to care worldwide.

Indexed as

congenital adrenal hyperplasiaglucocorticoidhydrocortisoneprescribing

Identifiers

PMID41782560
PMCPMC13034519

What OpenQuestion holds

Textmetadata
LicenceCC BY-NC-ND
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.