Evidence map›Paper›PMID 39672600›Full record

Trial reportThe Journal of clinical endocrinology and metabolism2025

Adult Height Following Prepubertal Treatment With Antiandrogen, Aromatase Inhibitor, and Reduced Hydrocortisone in CAH.

Deborah P Merke, Ashwini Mallappa, Megan Parker, Charles Sukin, Sarah E Kulkarni, Margaret F Keil, Carol Van Ryzin, Suvimol Chirathivat Hill, James C Reynolds, Gordon B Cutler and 1 more

Erratum issued Registry-linked trialAbstract readRandomized Controlled Trial
PubMed Full text at PMC Publisher
In one paragraph

Trial report in The Journal of clinical endocrinology and metabolism, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. An erratum has been issued. It is linked to trial NCT00001521 (An Open, Randomized, Long-Term Clinical Trial of Flutamide, Testolactone, and Reduced Hydrocortisone Dose vs. Conventional Treatment of Children With Congenital Adrenal Hyperplasia), which is not on this map. Cited by 3 papers.

0numbers the graph read from it
0cells of the map it votes in
3citing 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.

NCT00001521 phase3completednot on this map

An Open, Randomized, Long-Term Clinical Trial of Flutamide, Testolactone, and Reduced Hydrocortisone Dose vs. Conventional Treatment of Children With Congenital Adrenal Hyperplasia

TypeinterventionalSponsorEunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)Ran1995 to 2024Enrolled66ConditionsCongenital Adrenal Hyperplasia (CAH)ArmsFludrocortisone, Hydrocortisone, Letrozole, Flutamide, Testolactone
3 · Its place in the literature

Who cites it

3 citing papers in PubMed.

  1. Review
  2. Article
  3. Article
4 · The record

Corrections and comments

5 · Who and what money

Authors and funding

11 authors.

Deborah P MerkeDepartment of Pediatrics, National Institutes of Health Clinical Center, Bethesda, MD 20892, USA.ORCID 0000-0002-3746-0460
Ashwini MallappaDepartment of Pediatrics, National Institutes of Health Clinical Center, Bethesda, MD 20892, USA.ORCID 0000-0003-0637-4559
Megan ParkerDepartment of Pediatrics, National Institutes of Health Clinical Center, Bethesda, MD 20892, USA.
Charles SukinDepartment of Pediatrics, National Institutes of Health Clinical Center, Bethesda, MD 20892, USA.
Sarah E KulkarniDepartment of Pediatrics, National Institutes of Health Clinical Center, Bethesda, MD 20892, USA.
Margaret F KeilEunice Kennedy Shriver National Institute of Child Health and Human Development, National Institutes of Health, Bethesda, MD 20892, USA.ORCID 0000-0001-5077-2218
Carol Van RyzinDepartment of Pediatrics, National Institutes of Health Clinical Center, Bethesda, MD 20892, USA.
Suvimol Chirathivat HillDepartment of Pediatrics, National Institutes of Health Clinical Center, Bethesda, MD 20892, USA.
James C ReynoldsDepartment of Pediatrics, National Institutes of Health Clinical Center, Bethesda, MD 20892, USA.
Gordon B CutlerEunice Kennedy Shriver National Institute of Child Health and Human Development, National Institutes of Health, Bethesda, MD 20892, USA.ORCID 0000-0001-8925-5728
Ninet SinaiiDepartment of Pediatrics, National Institutes of Health Clinical Center, Bethesda, MD 20892, USA.ORCID 0000-0003-4045-1386

Funding

National Institute of Child Health and Human Development
6 · The paper itself

Abstract

contextHeight outcome in patients with classic congenital adrenal hyperplasia (CAH) is suboptimal due to glucocorticoid and androgen excess.

methodsIn an open, randomized, controlled trial, children with classic CAH were randomized to receive a combination regimen of antiandrogen, aromatase inhibitor, reduced hydrocortisone, and fludrocortisone prior to puberty or standard therapy (hydrocortisone, fludrocortisone). Females continued on antiandrogen during puberty. The primary endpoint was adult height.

resultsOf 62 children randomized, 45 completed the study. Adult height SDS did not differ between the investigational and control groups (-0.34 [0.93] vs -0.60 [0.89], respectively), mean difference 0.26 [95% CI -0.29, 0.82], P = .35), irrespective of midparental height, but was greater than the predicted adult height pretreatment in both groups (P < .001). Growth rate and rate of bone maturation were reduced in the investigational group prior to puberty, despite lower hydrocortisone dose (7.6 [1.5] vs 15.0 [3.6] mg/m2/day, P < .001), and improvement in predicted adult height appeared greater at pubertal onset (P = .049) compared to standard therapy. Antiandrogen treatment during puberty in girls allowed for lower-dose glucocorticoid, and improved height outcome (adult minus midparental height: -0.7 [4.6] vs -5.6 [5.2] cm, mean difference 4.9 [95% CI 0.09, 9.7], P = .046). Those who received GnRHa had lower growth rate (P = .023) and longer years of unchanged bone age (P = .017), regardless of treatment.

conclusionPrepubertal antiandrogen, aromatase inhibitor combination with reduced hydrocortisone improves short-term predicted height for children with CAH but does not result in taller adult stature than those treated with standard therapy, and is not recommended. Females may benefit from antiandrogen treatment during puberty.

Indexed as

Adrenal Hyperplasia, CongenitalAndrogen AntagonistsAromatase InhibitorsBody HeightHydrocortisoneAdolescentAdultChildChild, PreschoolDrug Therapy, CombinationFemaleFludrocortisoneHumansMalePubertyTreatment OutcomeAndrogen AntagonistsAromatase InhibitorsFludrocortisoneHydrocortisoneadult heightantiandrogenaromatase inhibitorCAHcongenital adrenal hyperplasia

Identifiers

PMID39672600
PMCPMC12187330

What Socratic holds

Textmetadata
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 Socratic graph.