Evidence map›Paper›PMID 34735372›Full record

ReviewEuropean journal of endocrinology2021

Clinical advances in the pharmacotherapy of congenital adrenal hyperplasia.

Alessandro Prete, Richard J Auchus, Richard J Ross

Open access · hybridAbstract readReview
PubMed Full text at PMC Publisher
In one paragraph

Review in European journal of endocrinology, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 24 papers.

0numbers the graph read from it
0cells of the map it votes in
24citing papers in PubMed
2.4field-weighted citation impact, top 10% 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

24 citing papers in PubMed, 44 citations in OpenAlex.

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  10. Approach to the Child and Adolescent With Adrenal Insufficiency.The Journal of clinical endocrinology and metabolism · 2025
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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

3 authors at 3 institutions in 2 countries.

Alessandro PreteInstitute of Metabolism and Systems Research, University of Birmingham, Birmingham, UK.
Richard J AuchusDivision of Metabolism, Endocrinology and Diabetes, Departments of Pharmacology and Internal Medicine, University of Michigan, Ann Arbor, Michigan, USA.
Richard J RossDepartment of Oncology and Metabolism, University of Sheffield, Sheffield, UK.
Queen Elizabeth Hospital Birmingham · GBUniversity of Michigan–Ann Arbor · USUniversity of Sheffield · GB

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

backgroundPatients with 21-hydroxylase deficiency congenital adrenal hyperplasia (21OHD-CAH) have poor health outcomes with increased mortality, short stature, impaired fertility, and increased cardiovascular risk factors such as obesity. To address this, there are therapies in development that target the clinical goal of treatment, which is to control excess androgens with an adrenal replacement dose of glucocorticoid.

methodsNarrative review of publications on recent clinical developments in the pharmacotherapy of congenital adrenal hyperplasia. SUMMARY: Therapies in clinical development target different levels of the hypothalamo-pituitary-adrenal axis. Two corticotrophin-releasing factor type 1 (CRF1) receptor antagonists, Crinecerfont and Tildacerfont, have been trialled in poorly controlled 21OHD-CAH patients, and both reduced ACTH and androgen biomarkers while patients were on stable glucocorticoid replacement. Improvements in glucocorticoid replacement include replacing the circadian rhythm of cortisol that has been trialled with continuous s.c. infusion of hydrocortisone and Chronocort, a delayed-release hydrocortisone formulation. Chronocort optimally controlled 21OHD-CAH in 80% of patients on an adrenal replacement dose of hydrocortisone, which was associated with patient-reported benefits including restoration of menses and pregnancies. Adrenal-targeted therapies include the steroidogenesis-blocking drug Abiraterone acetate, which reduced adrenal androgen biomarkers in poorly controlled patients.

conclusionsCRF1 receptor antagonists hold promise to avoid excess glucocorticoid replacement in patients not controlled on standard or circadian glucocorticoid replacement such as Chronocort. Gene and cell therapies are the only therapeutic approaches that could potentially correct both cortisol deficiency and androgen excess.

Indexed as

Adrenal Hyperplasia, CongenitalAndrogensCircadian RhythmEndocrinologyGlucocorticoidsHormone Replacement TherapyHumansHydrocortisoneHypothalamo-Hypophyseal SystemAndrogensGlucocorticoidsHydrocortisone

Identifiers

PMID34735372
PMCPMC8679847
OpenAlexW3210321037

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.