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Record W4415452223 · doi:10.1210/jendso/bvaf149.278

OR18-06 A Global Approach to the Long-Term Follow-Up of 17 Families Affected by Bilateral Macronodular Adrenal Disease

2025· article· en· W4415452223 on OpenAlexaff
Helaine Laiz Silva Charchar, Guilherme Asmar Alencar, Beatriz Marinho de Paula Mariani, Aliny Weber Kuhn, Lucas Bouys, Patricia Vaduva, Mirian Yumie Nishi, Felipe L Ledesma, Madson Q. Almeida, Victor Srougi, Fábio Y Tanno, Jose L Chambô, Ana Cláudia Latronico, Maria Adelaide Albergaria Pereira, Jérôme Bertherat, Berenice Bilharinho Mendonca, Maria Candida Barisson Villares Fragoso

Bibliographic record

VenueJournal of the Endocrine Society · 2025
Typearticle
Languageen
FieldMedicine
TopicPituitary Gland Disorders and Treatments
Canadian institutionsWiLAN (Canada)
Fundersnot available
KeywordsDiseaseRare diseaseGermlineRetrospective cohort studyClinical diseaseGenetic predisposition

Abstract

fetched live from OpenAlex

Abstract Disclosure: H.L. Charchar: None. G.A. Alencar: None. B.M. Mariani: None. A.W. Kuhn: None. L. Bouys: None. P. Vaduva: None. M.Y. Nishi: None. F.L. Ledesma: None. M.Q. Almeida: None. V. Srougi: None. F.Y. Tanno: None. J.L. Chambô: None. A. Latronico: None. M.A. Pereira: None. J. Bertherat: None. B.B. Mendonca: None. M.C. Fragoso: None. Context: Bilateral macronodular adrenocortical disease (BMAD) is a rare and often underdiagnosed cause of adrenal Cushing’s syndrome (CS), presenting a spectrum of manifestations ranging from mild autonomous cortisol secretion (MACS) to overt CS. ARMC5 and KDM1A are the most important and frequent genes associated with BMAD-families, however,long-term follow-up of affected patients remain scarce. Objective: This retrospective study analyzed clinical-hormonal variability, genetic profiles, treatment modalities, and outcomes in 17 families and nine sporadic cases of BMAD over 8 to 410 months at a Brazilian tertiary center. Methods: A total of 250 individuals (50 index cases [IC] and 200 relatives [R]) were included. Clinical, hormonal, and imaging data, along with histological and genetic analyses of ARMC5 and KDM1A, were evaluated. Results: Among 250 individuals, 104 (26 IC and 78 R) carried germline pathogenic/likely pathogenic ARMC5 variants. No KDM1A (likely) pathogenic variants were identified in ARMC5-wild-type patients. ARMC5-positive index-cases exhibited severe clinical manifestations, evidenced by elevated cortisol levels (urinary, salivary, and post-dexamethasone suppression test) and reduced ACTH and DHEAS levels (p = 0.005, p = 0.042, p = 0.005, p = 0.041, and p = 0.007, respectively). ICs had larger adrenal nodules (p < 0.0001). Adrenal-sparing surgery achieved 100% remission, contrasting with a 40% remission rate for unilateral adrenalectomy. Central nervous system meningiomas were observed in BMAD-patients independently to ARMC5 status. Interestingly, malignant neoplasms were notably prevalent among ARMC5-altered individuals. Conclusion: A proposed management flowchart emphasizes genetic screening and the monitoring to mitigate the adrenal insufficiency period, MACS recurrence, and tumor risks development, underscoring the need for tailored therapeutic strategies in BMAD, depending on genetic alterations / ARMC5 status. Presentation: Sunday, July 13, 2025

Fetched live from OpenAlex and de-inverted. Abstracts are not stored in this database: the inverted indexes are 8.6 GB of the frame’s 9.3 GB of text, and the host has 13 GB free.

How this classification was reachedexpand

Full frame distilled prediction

Teacher imitation

Not calibrated prevalence, not ground truth. Human validation pending. Learned from the 10,348 direct Codex labels and 10,348 direct Gemma labels. Candidate is the union of thresholded teacher heads; consensus is their intersection. These outputs are machine_predicted_unvalidated and are not human labels or direct frontier model labels.

metaresearch head score (Codex)0.000
metaresearch head score (Gemma)0.000
Version: codex-gemma-dda1882f352aValidation status: machine_predicted_unvalidated
Candidate categoriesnone
Consensus categoriesnone
DomainCandidate signal: none · Consensus signal: none
Study designCandidate signal: Observational · Consensus signal: Observational
GenreCandidate signal: Empirical · Consensus signal: Empirical
Teacher disagreement score0.264
Threshold uncertainty score0.360

Codex and Gemma teacher scores by category

CategoryCodexGemma
Metaresearch0.0000.000
Meta-epidemiology (narrow)0.0000.000
Meta-epidemiology (broad)0.0000.001
Bibliometrics0.0000.000
Science and technology studies0.0000.000
Scholarly communication0.0000.000
Open science0.0000.000
Research integrity0.0000.000
Insufficient payload (model declined to judge)0.0000.000

Machine scores (provisional)

The two teacher heads of the student model, read on this work. A score orders the frame for review; it never asserts a category, and the validation status ships verbatim with every row.

Baseline scores from an immature model (maturity gate not passed, 7 training rounds). Scores rank; they never assert a category.

Opus teacher head0.007
GPT teacher head0.263
Teacher spread0.256 · how far apart the two teachers sit on this one work
Validation statusscore_only:v0-immature-baseline · verbatim from the scoring run: score_only means the number may rank works, and no category label ships from it

Classification

machine, unvalidated

Machine predicted; a candidate call from one teacher head, not a consensus.

The models applied no category: nothing in the taxonomy fit this work.
Study designObservational
Domainnot available
GenreEmpirical

How this classification was reached, model by model and score by score, is at the end of the page under "How this classification was reached".

Quick stats

Citations0
Published2025
Admission routes1
Has abstractyes

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