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Hiperplasia suprarrenal congênita
ORPHA:418CID-10 · E25.0CID-11 · 5A71.01PCDT · SUSDOENÇA RARA

A Hiperplasia Adrenal Congênita (HAC) é uma doença hormonal hereditária (passada de pais para filhos). Ela é causada pela falta de uma enzima importante para a produção de alguns hormônios no corpo. Essa condição faz com que as glândulas suprarrenais (que ficam acima dos rins) não funcionem corretamente e pode levar a sinais de excesso ou falta de hormônios masculinos (andrógenos), variando conforme o tipo e a gravidade da doença.

Mantido por Agente Raras·Colaborar como especialista →

Introdução

O que você precisa saber de cara

📋

A Hiperplasia Adrenal Congênita (HAC) é uma doença hormonal hereditária (passada de pais para filhos). Ela é causada pela falta de uma enzima importante para a produção de alguns hormônios no corpo. Essa condição faz com que as glândulas suprarrenais (que ficam acima dos rins) não funcionem corretamente e pode levar a sinais de excesso ou falta de hormônios masculinos (andrógenos), variando conforme o tipo e a gravidade da doença.

Pesquisas ativas
12 ensaios
185 total registrados no ClinicalTrials.gov
Publicações científicas
6.265 artigos
Último publicado: 2026
Medicamentos
12 registrados
PREDNISOLONE, CORTISONE ACETATE, DEXAMETHASONE SODIUM PHOSPHATE

Tem tratamento?

12 medicamentos registrados
Ver detalhes, fases e interações →
PREDNISOLONECORTISONE ACETATEDEXAMETHASONE SODIUM PHOSPHATEDEXAMETHASONEHYDROCORTISONEPREDNISONECRINECERFONTNEVANIMIBEFLUDROCORTISONE ACETATEABIRATERONE ACETATE

Escala de raridade

CLASSIFICAÇÃO ORPHANET · BRASIL 2024
1-9 / 100 000
Ultra-rara
<1/50k
Muito rara
1/20k
Rara
1/10k
Pouco freq.
1/5k
Incomum
1/2k
Prevalência
10.0
Europe
Herança
Autosomal recessive
🏥
SUS: Cobertura completaScore: 80%
PCDT disponívelTriagem neonatal (Fase 1)Centros em: PA, PR, SC, RS, ES +10CID-10: E25.0
🇧🇷Dados SUS / DATASUS2024
890
internações/ano
R$ 3.210
custo médio/internação
ESTADOS COM MAIS INTERNAÇÕES
SPMGRJBARS
PROCEDIMENTOS SIGTAP (1)
0202080013
Teste do pezinho (triagem neonatal)newborn_screening
Você se identifica com essa condição?
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Entender a doença

Do básico ao detalhe, leia no seu ritmo

Preparando trilha educativa...

Sinais e sintomas

O que aparece no corpo e com que frequência cada sintoma acontece

Partes do corpo afetadas

🦴
Ossos e articulações
26 sintomas
📏
Crescimento
18 sintomas
🫘
Rins
12 sintomas
😀
Face
8 sintomas
🫃
Digestivo
6 sintomas
🧬
Pele e cabelo
5 sintomas

+ 130 sintomas em outras categorias

Características mais comuns

Genitália externa anormal
Anormalidade da anti-hélice
Anormalidade da maturação esquelética
Hemangioma capilar frontomediano
Constipação
Morfologia vertebral anormal
220sintomas
Sem dados (220)

Os sintomas variam de pessoa para pessoa. Abaixo estão as 220 características clínicas mais associadas, ordenadas por frequência.

Genitália externa anormalAbnormal external genitalia
Anormalidade da anti-héliceAbnormality of the antihelix
Anormalidade da maturação esqueléticaAbnormality of skeletal maturation
Hemangioma capilar frontomedianoMidfrontal capillary hemangioma
ConstipaçãoConstipation

Linha do tempo da pesquisa

Publicações por ano — veja quando o interesse científico cresceu
Anos de pesquisa1desde 2026
Total histórico6.265PubMed
Últimos 10 anos200publicações
Pico2025119 papers
Linha do tempo
2026Hoje · 2026🧪 1993Primeiro ensaio clínico📈 2025Ano de pico
Publicações por ano (últimos 10 anos)

Triagem neonatal (Teste do Pezinho)

👶
Teste: 17-OH-Progesterona em sangue seco
Fase 1 do PNTNTriagem nacionalimplemented_nationally
Incidência no Brasil: 1:15.000

A triagem neonatal permite diagnóstico precoce e início imediato do tratamento.

Encontrou um erro ou informação desatualizada? Sugira uma correção →

Genética e causas

O que está alterado no DNA e como passa nas famílias

Genes associados

6 genes identificados com associação a esta condição. Padrão de herança: Autosomal recessive.

CYP21A2Steroid 21-hydroxylaseDisease-causing germline mutation(s) inTolerante
FUNÇÃO

A cytochrome P450 monooxygenase that plays a major role in adrenal steroidogenesis. Catalyzes the hydroxylation at C-21 of progesterone and 17alpha-hydroxyprogesterone to respectively form 11-deoxycorticosterone and 11-deoxycortisol, intermediate metabolites in the biosynthetic pathway of mineralocorticoids and glucocorticoids (PubMed:10602386, PubMed:16984992, PubMed:22014889, PubMed:25855791, PubMed:27721825). Mechanistically, uses molecular oxygen inserting one oxygen atom into a substrate, a

LOCALIZAÇÃO

Endoplasmic reticulum membraneMicrosome membrane

VIAS BIOLÓGICAS (3)
Glucocorticoid biosynthesisEndogenous sterolsMineralocorticoid biosynthesis
MECANISMO DE DOENÇA

Adrenal hyperplasia 3

A form of congenital adrenal hyperplasia, a common recessive disease due to defective synthesis of cortisol. Congenital adrenal hyperplasia is characterized by androgen excess leading to ambiguous genitalia in affected females, rapid somatic growth during childhood in both sexes with premature closure of the epiphyses and short adult stature. Four clinical types: 'salt wasting' (SW, the most severe type), 'simple virilizing' (SV, less severely affected patients), with normal aldosterone biosynthesis, 'non-classic form' or late-onset (NC or LOAH) and 'cryptic' (asymptomatic).

EXPRESSÃO TECIDUAL(Tecido-específico)
Glândula adrenal
1673.2 TPM
Fígado
17.5 TPM
Ovário
4.6 TPM
Baço
3.3 TPM
Fallopian Tube
3.2 TPM
OUTRAS DOENÇAS (3)
classic congenital adrenal hyperplasia due to 21-hydroxylase deficiencyclassic congenital adrenal hyperplasia due to 21-hydroxylase deficiency, simple virilizing formclassic congenital adrenal hyperplasia due to 21-hydroxylase deficiency, salt wasting form
HGNC:2600UniProt:P08686
STARGuanylyl cyclase CDisease-causing germline mutation(s) inTolerante
FUNÇÃO

Guanylyl cyclase that catalyzes synthesis of cyclic GMP (cGMP) from GTP (PubMed:11950846, PubMed:1718270, PubMed:22436048, PubMed:22521417, PubMed:23269669). Receptor for the E.coli heat-stable enterotoxin; E.coli enterotoxin markedly stimulates the accumulation of cGMP in mammalian cells expressing GUCY2C (PubMed:1680854, PubMed:1718270). Also activated by the endogenous peptides guanylin and uroguanylin (PubMed:8381596)

LOCALIZAÇÃO

Cell membraneEndoplasmic reticulum membrane

VIAS BIOLÓGICAS (1)
Pregnenolone biosynthesis
MECANISMO DE DOENÇA

Diarrhea 6

A relatively mild, early-onset chronic diarrhea that may be associated with increased susceptibility to inflammatory bowel disease, small bowel obstruction, and esophagitis.

EXPRESSÃO TECIDUAL(Tecido-específico)
Glândula adrenal
4912.2 TPM
Ovário
925.7 TPM
Testículo
73.5 TPM
Brain Frontal Cortex BA9
6.5 TPM
Hipotálamo
5.9 TPM
OUTRAS DOENÇAS (4)
congenital lipoid adrenal hyperplasia due to STAR deficencyfamilial glucocorticoid deficiencynon-classic congenital lipoid adrenal hyperplasia due to STAR deficencyclassic congenital lipoid adrenal hyperplasia due to STAR deficency
HGNC:11359UniProt:P25092
CYP11B1Cytochrome P450 11B1, mitochondrialDisease-causing germline mutation(s) inTolerante
FUNÇÃO

A cytochrome P450 monooxygenase involved in the biosynthesis of adrenal corticoids (PubMed:12530636, PubMed:1518866, PubMed:1775135, PubMed:18215163, PubMed:23322723). Catalyzes a variety of reactions that are essential for many species, including detoxification, defense, and the formation of endogenous chemicals like steroid hormones. Steroid 11beta, 18- and 19-hydroxylase with preferred regioselectivity at 11beta, then 18, and lastly 19 (By similarity). Catalyzes the hydroxylation of 11-deoxyc

LOCALIZAÇÃO

Mitochondrion inner membrane

VIAS BIOLÓGICAS (2)
Endogenous sterolsGlucocorticoid biosynthesis
MECANISMO DE DOENÇA

Adrenal hyperplasia 4

A form of congenital adrenal hyperplasia, a common recessive disease due to defective synthesis of cortisol. Congenital adrenal hyperplasia is characterized by androgen excess leading to ambiguous genitalia in affected females, rapid somatic growth during childhood in both sexes with premature closure of the epiphyses and short adult stature. Four clinical types: 'salt wasting' (SW, the most severe type), 'simple virilizing' (SV, less severely affected patients), with normal aldosterone biosynthesis, 'non-classic form' or late-onset (NC or LOAH) and 'cryptic' (asymptomatic).

EXPRESSÃO TECIDUAL(Tecido-específico)
Glândula adrenal
4787.3 TPM
Testículo
6.4 TPM
Cervix Ectocervix
0.9 TPM
Fallopian Tube
0.5 TPM
Bladder
0.5 TPM
OUTRAS DOENÇAS (2)
glucocorticoid-remediable aldosteronismcongenital adrenal hyperplasia due to 11-beta-hydroxylase deficiency
HGNC:2591UniProt:P15538
HSD3B23 beta-hydroxysteroid dehydrogenase/Delta 5-->4-isomerase type 2Disease-causing germline mutation(s) inTolerante
FUNÇÃO

3-beta-HSD is a bifunctional enzyme, that catalyzes the oxidative conversion of Delta(5)-ene-3-beta-hydroxy steroid, and the oxidative conversion of ketosteroids. The 3-beta-HSD enzymatic system plays a crucial role in the biosynthesis of all classes of hormonal steroids

LOCALIZAÇÃO

Endoplasmic reticulum membraneMitochondrion membrane

VIAS BIOLÓGICAS (3)
Androgen biosynthesisMineralocorticoid biosynthesisGlucocorticoid biosynthesis
MECANISMO DE DOENÇA

Adrenal hyperplasia 2

A form of congenital adrenal hyperplasia, a common recessive disease due to defective synthesis of cortisol. Congenital adrenal hyperplasia is characterized by androgen excess leading to ambiguous genitalia in affected females, rapid somatic growth during childhood in both sexes with premature closure of the epiphyses and short adult stature. Four clinical types: 'salt wasting' (SW, the most severe type), 'simple virilizing' (SV, less severely affected patients), with normal aldosterone biosynthesis, 'non-classic form' or late-onset (NC or LOAH) and 'cryptic' (asymptomatic). In AH2, virilization is much less marked or does not occur. AH2 is frequently lethal in early life.

EXPRESSÃO TECIDUAL(Tecido-específico)
Glândula adrenal
1702.3 TPM
Intestino delgado
7.9 TPM
Testículo
1.0 TPM
Cólon transverso
0.9 TPM
Rim - Medula
0.6 TPM
OUTRAS DOENÇAS (1)
congenital adrenal hyperplasia due to 3-beta-hydroxysteroid dehydrogenase deficiency
HGNC:5218UniProt:P26439
CYP17A1Steroid 17-alpha-hydroxylase/17,20 lyaseDisease-causing germline mutation(s) inTolerante
FUNÇÃO

A cytochrome P450 monooxygenase involved in corticoid and androgen biosynthesis (PubMed:22266943, PubMed:25301938, PubMed:27339894, PubMed:9452426). Catalyzes 17-alpha hydroxylation of C21 steroids, which is common for both pathways. A second oxidative step, required only for androgen synthesis, involves an acyl-carbon cleavage. The 17-alpha hydroxy intermediates, as part of adrenal glucocorticoids biosynthesis pathway, are precursors of cortisol (Probable) (PubMed:25301938, PubMed:9452426). Hyd

LOCALIZAÇÃO

Endoplasmic reticulum membraneMicrosome membrane

VIAS BIOLÓGICAS (2)
Androgen biosynthesisGlucocorticoid biosynthesis
MECANISMO DE DOENÇA

Adrenal hyperplasia 5

A form of congenital adrenal hyperplasia, a common recessive disease due to defective synthesis of cortisol. Congenital adrenal hyperplasia is characterized by androgen excess leading to ambiguous genitalia in affected females, rapid somatic growth during childhood in both sexes with premature closure of the epiphyses and short adult stature. Four clinical types: 'salt wasting' (SW, the most severe type), 'simple virilizing' (SV, less severely affected patients), with normal aldosterone biosynthesis, 'non-classic form' or late-onset (NC or LOAH) and 'cryptic' (asymptomatic).

EXPRESSÃO TECIDUAL(Tecido-específico)
Glândula adrenal
6921.5 TPM
Testículo
14.6 TPM
Tireoide
6.3 TPM
Rim - Medula
4.7 TPM
Rim - Córtex
4.7 TPM
OUTRAS DOENÇAS (2)
congenital adrenal hyperplasia due to 17-alpha-hydroxylase deficiency17,20-lyase deficiency, isolated
HGNC:2593UniProt:P05093
PORNADPH--cytochrome P450 reductaseDisease-causing germline mutation(s) inTolerante
FUNÇÃO

This enzyme is required for electron transfer from NADP to cytochrome P450 in microsomes. It can also provide electron transfer to heme oxygenase and cytochrome B5

LOCALIZAÇÃO

Endoplasmic reticulum membrane

VIAS BIOLÓGICAS (1)
Cytochrome P450 - arranged by substrate type
MECANISMO DE DOENÇA

Antley-Bixler syndrome, with genital anomalies and disordered steroidogenesis

A disease characterized by the association of Antley-Bixler syndrome with steroidogenesis defects and abnormal genitalia. Antley-Bixler syndrome is characterized by craniosynostosis, radiohumeral synostosis present from the perinatal period, midface hypoplasia, choanal stenosis or atresia, femoral bowing and multiple joint contractures.

EXPRESSÃO TECIDUAL(Ubíquo)
Glândula adrenal
164.2 TPM
Fígado
149.3 TPM
Tireoide
116.3 TPM
Pituitária
84.0 TPM
Pulmão
67.2 TPM
OUTRAS DOENÇAS (2)
congenital adrenal hyperplasia due to cytochrome P450 oxidoreductase deficiencyAntley-Bixler syndrome with genital anomalies and disordered steroidogenesis
HGNC:9208UniProt:P16435

Medicamentos e terapias

PREDNISOLONEPhase 4

Mecanismo: Glucocorticoid receptor agonist

CORTISONE ACETATEPhase 4

Mecanismo: Glucocorticoid receptor agonist

DEXAMETHASONE SODIUM PHOSPHATEPhase 4

Mecanismo: Glucocorticoid receptor agonist

DEXAMETHASONEPhase 4

Mecanismo: Glucocorticoid receptor agonist

HYDROCORTISONEPhase 4

Mecanismo: Glucocorticoid receptor agonist

PREDNISONEPhase 4

Mecanismo: Glucocorticoid receptor agonist

CRINECERFONTPhase 3

Mecanismo: Corticotropin releasing factor receptor 1 antagonist

NEVANIMIBEPhase 2

Mecanismo: Acyl coenzyme A:cholesterol acyltransferase 1 inhibitor

FLUDROCORTISONE ACETATEPhase 2

Mecanismo: Mineralocorticoid receptor agonist

ABIRATERONE ACETATEPhase 2

Mecanismo: Cytochrome P450 17A1 inhibitor

Ver mais no OpenTargets

Variantes genéticas (ClinVar)

686 variantes patogênicas registradas no ClinVar.

🧬 CYP21A2: NM_000500.9(CYP21A2):c.1381_1398del (p.Ser461_Pro466del) ()
🧬 CYP21A2: NM_000500.9(CYP21A2):c.710_719delinsAGGAGGAGAA (p.Ile237_Met240delinsLysGluGluLys) ()
🧬 CYP21A2: NM_000500.9(CYP21A2):c.1088C>T (p.Ala363Val) ()
🧬 CYP21A2: NM_000500.9(CYP21A2):c.738+2T>C ()
🧬 CYP21A2: NM_000500.9(CYP21A2):c.1301T>C (p.Leu434Pro) ()
Ver todas no ClinVar

Classificação de variantes (ClinVar)

Distribuição de 926 variantes classificadas pelo ClinVar.

139
93
694
Patogênica (15.0%)
VUS (10.0%)
Benigna (74.9%)
VARIANTES MAIS SIGNIFICATIVAS
POR: NM_001395413.1(POR):c.1841_1842del (p.Glu614fs) [Pathogenic]
POR: NM_001395413.1(POR):c.469C>T (p.Gln157Ter) [Pathogenic]
POR: NM_001395413.1(POR):c.1158del (p.Tyr387fs) [Pathogenic]
LOC126860075: NM_001395413.1(POR):c.854C>T (p.Ala285Val) [Uncertain significance]
LOC126860075: NM_001395413.1(POR):c.826T>G (p.Phe276Val) [Uncertain significance]

Diagnóstico

Os sinais que médicos procuram e os exames que confirmam

Carregando...

Tratamento e manejo

Remédios, cuidados de apoio e o que precisa acompanhar

Pipeline de tratamentos
Pipeline regulatório — de medicamentos já aprovados a drogas em pesquisa exploratória.
Aprovado7
3Fase 34
2Fase 211
1Fase 11
·Pré-clínico7
Medicamentos catalogadosEnsaios clínicos· 10 medicamentos · 20 ensaios
✓ Aprovados — podem ser usados hoje
PREDNISOLONECORTISONE ACETATEDEXAMETHASONE SODIUM PHOSPHATEDEXAMETHASONEHYDROCORTISONEPREDNISONE
Carregando informações de tratamento...

Onde tratar no SUS

Hospitais de referência no Brasil e o protocolo oficial do SUS (PCDT)

🇧🇷 Atendimento SUS — Hiperplasia suprarrenal congênita

Centros de Referência SUS

24 centros habilitados pelo SUS para Hiperplasia suprarrenal congênita

Centros para Hiperplasia suprarrenal congênita

Detalhes dos centros

Hospital Universitário Prof. Edgard Santos (HUPES)

R. Dr. Augusto Viana, s/n - Canela, Salvador - BA, 40110-060 · CNES 0003808

Serviço de Referência

Rota
Anomalias CongênitasErros Inatos do Metabolismo

Hospital Infantil Albert Sabin

R. Tertuliano Sales, 544 - Vila União, Fortaleza - CE, 60410-794 · CNES 2407876

Serviço de Referência

Rota
Anomalias CongênitasDeficiência Intelectual

Hospital de Apoio de Brasília (HAB)

AENW 3 Lote A Setor Noroeste - Plano Piloto, Brasília - DF, 70684-831 · CNES 0010456

Serviço de Referência

Rota
Anomalias CongênitasErros Inatos do MetabolismoDeficiência Intelectual

Hospital Estadual Infantil e Maternidade Alzir Bernardino Alves (HIABA)

Av. Min. Salgado Filho, 918 - Soteco, Vila Velha - ES, 29106-010 · CNES 6631207

Serviço de Referência

Rota
Anomalias CongênitasErros Inatos do MetabolismoDeficiência Intelectual

Hospital das Clínicas da UFG

Rua 235 QD. 68 Lote Área, Nº 285, s/nº - Setor Leste Universitário, Goiânia - GO, 74605-050 · CNES 2338424

Serviço de Referência

Rota
Anomalias CongênitasErros Inatos do Metabolismo

Hospital Universitário da UFJF

R. Catulo Breviglieri, Bairro - s/n - Santa Catarina, Juiz de Fora - MG, 36036-110 · CNES 2297442

Atenção Especializada

Rota
Anomalias Congênitas

Hospital das Clínicas da UFMG

Av. Prof. Alfredo Balena, 110 - Santa Efigênia, Belo Horizonte - MG, 30130-100 · CNES 2280167

Serviço de Referência

Rota
Anomalias CongênitasErros Inatos do MetabolismoDeficiência Intelectual

Hospital Universitário Julio Müller (HUJM)

R. Luis Philippe Pereira Leite, s/n - Alvorada, Cuiabá - MT, 78048-902 · CNES 2726092

Atenção Especializada

Rota
Anomalias Congênitas

Hospital Universitário João de Barros Barreto

R. dos Mundurucus, 4487 - Guamá, Belém - PA, 66073-000 · CNES 2337878

Serviço de Referência

Rota
Anomalias CongênitasErros Inatos do MetabolismoDeficiência Intelectual

Hospital Universitário Lauro Wanderley (HULW)

R. Tabeliao Estanislau Eloy, 585 - Castelo Branco, João Pessoa - PB, 58050-585 · CNES 0002470

Atenção Especializada

Rota
Anomalias Congênitas

Instituto de Medicina Integral Prof. Fernando Figueira (IMIP)

R. dos Coelhos, 300 - Boa Vista, Recife - PE, 50070-902 · CNES 0000647

Serviço de Referência

Rota
Anomalias CongênitasErros Inatos do MetabolismoDeficiência Intelectual

Hospital Pequeno Príncipe

R. Des. Motta, 1070 - Água Verde, Curitiba - PR, 80250-060 · CNES 3143805

Serviço de Referência

Rota
Anomalias CongênitasDeficiência Intelectual

Hospital Universitário Regional de Maringá (HUM)

Av. Mandacaru, 1590 - Parque das Laranjeiras, Maringá - PR, 87083-240 · CNES 2216108

Atenção Especializada

Rota
Anomalias Congênitas

Hospital de Clínicas da UFPR

R. Gen. Carneiro, 181 - Alto da Glória, Curitiba - PR, 80060-900 · CNES 2364980

Serviço de Referência

Rota
Anomalias CongênitasErros Inatos do MetabolismoDeficiência Intelectual

Hospital Universitário Pedro Ernesto (HUPE-UERJ)

Blvd. 28 de Setembro, 77 - Vila Isabel, Rio de Janeiro - RJ, 20551-030 · CNES 2280221

Serviço de Referência

Rota
Anomalias CongênitasErros Inatos do Metabolismo

Instituto Nacional de Saúde da Mulher, da Criança e do Adolescente Fernandes Figueira (IFF/Fiocruz)

Av. Rui Barbosa, 716 - Flamengo, Rio de Janeiro - RJ, 22250-020 · CNES 2269988

Serviço de Referência

Rota
Anomalias CongênitasErros Inatos do MetabolismoDeficiência Intelectual

Hospital São Lucas da PUCRS

Av. Ipiranga, 6690 - Jardim Botânico, Porto Alegre - RS, 90610-000 · CNES 2232928

Serviço de Referência

Rota
Anomalias CongênitasErros Inatos do Metabolismo

Hospital de Clínicas de Porto Alegre (HCPA)

Rua Ramiro Barcelos, 2350 Bloco A - Av. Protásio Alves, 211 - Bloco B e C - Santa Cecília, Porto Alegre - RS, 90035-903 · CNES 2237601

Serviço de Referência

Rota
Anomalias CongênitasErros Inatos do MetabolismoDeficiência Intelectual

Hospital Universitário da UFSC (HU-UFSC)

R. Profa. Maria Flora Pausewang - Trindade, Florianópolis - SC, 88036-800 · CNES 2560356

Serviço de Referência

Rota
Anomalias CongênitasErros Inatos do Metabolismo

Hospital das Clínicas da FMUSP

R. Dr. Ovídio Pires de Campos, 225 - Cerqueira César, São Paulo - SP, 05403-010 · CNES 2077485

Serviço de Referência

Rota
Anomalias CongênitasErros Inatos do MetabolismoDeficiência Intelectual

Hospital de Base de São José do Rio Preto

Av. Brg. Faria Lima, 5544 - Vila Sao Jose, São José do Rio Preto - SP, 15090-000 · CNES 2079798

Atenção Especializada

Rota
Anomalias Congênitas

Hospital de Clínicas da UNICAMP

R. Vital Brasil, 251 - Cidade Universitária, Campinas - SP, 13083-888 · CNES 2748223

Serviço de Referência

Rota
Anomalias CongênitasErros Inatos do MetabolismoDeficiência Intelectual

Hospital de Clínicas de Ribeirão Preto (HCRP-USP)

R. Ten. Catão Roxo, 3900 - Vila Monte Alegre, Ribeirão Preto - SP, 14015-010 · CNES 2082187

Serviço de Referência

Rota
Anomalias CongênitasErros Inatos do MetabolismoDeficiência Intelectual

UNIFESP / Hospital São Paulo

R. Napoleão de Barros, 715 - Vila Clementino, São Paulo - SP, 04024-002 · CNES 2688689

Serviço de Referência

Rota
Anomalias CongênitasErros Inatos do Metabolismo
Sobre os centros SUS: Estes centros são habilitados pelo Ministério da Saúde como Serviços de Referência em Doenças Raras ou Serviços de Atenção Especializada. O atendimento é pelo SUS, com encaminhamento da rede de atenção básica.

Dados de DATASUS/CNES, SBGM, ABNeuro e Ministério da Saúde. Sempre confirme a disponibilidade diretamente com o estabelecimento.

Pesquisa ativa

Ensaios clínicos abertos e novidades científicas recentes

🟢 Recrutando agora

10 pesquisas recrutando participantes. Converse com seu médico sobre a possibilidade de participar.

Outros ensaios clínicos

185 ensaios clínicos encontrados, 12 ativos.

Distribuição por fase
Ver todos no ClinicalTrials.gov
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Publicações mais relevantes

🥈Melhor nível de evidência: Observacional
Timeline de publicações
2.483 papers (10 anos)

Mostrando amostra de 200 publicações de um total de 2.483

#1

Diagnostic dilemma in a neonate: Pseudo Hypoaldosteronism mimicking congenital adrenal hyperplasia.

Pakistan journal of medical sciences2026 Feb

Pseudo hypoaldosteronism (PHA) and congenital adrenal hyperplasia (CAH) can present with similar biochemical abnormalities in neonates, making early diagnosis challenging. We report a neonate who presented with hyponatremia, hyperkalemia, and metabolic acidosis and was initially managed as CAH with steroid therapy. Despite treatment, the electrolyte abnormalities persisted, prompting further evaluation. Genetic testing revealed a homozygous pathogenic mutation in SCNN1B, confirming autosomal recessive PHA Type I-B. Persistent electrolyte derangements despite appropriate steroid therapy should raise suspicion for PHA. Early genetic testing is essential for accurate diagnosis and targeted management in neonates with overlapping presentations of CAH and PHA.

#2

Newborn screening in an Eastern Indian province: prevalence and trends from a burgeoning newborn screening unit.

Journal of tropical pediatrics2026 Jan 02

Neonatal screening has not been demanded by pediatricians nor parents in India because of limited resources, inadequate health information, early hospital discharges, a large rate of deliveries at home, and challenges with follow-up of results. This study was conducted based on the laboratory e-records from the Newborn Screening Unit, where Dried Blood Spot-based analysis was performed for G6PD deficiency, congenital adrenal hyperplasia, neonatal hypothyroidism, biotinidase deficiency, and galactosemia. The total study population comprised 3210 neonates. Overall, 329 children (10.2%) had some form of inherited disorder, and 12 (0.4%) suffered from two disorders concomitantly. The highest number of children were suffering from partial biotinidase deficiency (4.5%), followed by G6PD deficiency (2.7%). There was no significant difference in the levels of measured analytes in males and females, except for 17-OHP, which was significantly higher in males. Compared with females, males were more likely to have G6PD and biotinidase deficiency. Our study provides a snapshot of five inherited disorders previously unexamined by neonatal screening in Eastern India. We found a high prevalence of G6PD deficiency and other substantive neonatal conditions. Now that the significant prevalence of these disorders is known, stakeholders should work with policy-makers to institute national-level screening to determine their distribution in the broader population and address them in affected groups. Further studies are needed to identify effective implementation strategies, overcome financial and logistical barriers to an NBS program, and assess whether such a program can reach and benefit all communities, especially the most vulnerable.

#3

Genetic diagnosis of CYP21A2-related CAH: adaptive sampling long-read sequencing is an accurate and scalable solution.

European journal of human genetics : EJHG2026 Jan 22

Congenital adrenal hyperplasia (CAH) is an autosomal recessive disorder, commonly caused by variants in CYP21A2 (chr6p21.33), which encodes the 21-hydroxylase enzyme. Genetic diagnosis is challenging due to the high homology between CYP21A2 and its nearby pseudogene CYP21A1P. The current gold standard, PCR-based Sanger sequencing combined with multiplex ligation-dependent probe amplification (MLPA), is labor-intensive, costly, and amenable to PCR bias. Furthermore, it is not reliable in detecting complex structural variants, and it provides no information on whether variants are located on the same allele or not. The purpose of this study was to develop a method based on long-read sequencing (LRS) for accurate diagnostics of CYP21A2 variants and their phasing. Adaptive sampling (AS-)-LRS with chromosome 6 as region-of-interest was applied to DNA from 34 patients clinically diagnosed with CAH. To overcome mapping challenges in the highly homologous regions, we developed NanoCAH, a custom bioinformatic tool that accurately distinguishes between CYP21A2 and CYP21A1P reads. Using AS-LRS and NanoCAH, we genetically confirmed CYP21A2-associated CAH in 32 (94%) of the patients, including reliable phasing of the variants without the need for parental testing. AS-LRS clarified previously ambiguous findings, including the detection of chimeric genes, deletions, and missed variants. Compared to current gold standard methods, AS-LRS proved to be faster and more scalable, while providing greater accuracy in detecting variants within the CYP21A2 region. This makes AS-LRS a promising tool not only for CAH diagnosis but also for genetic testing in other regions with complex genomic architecture.

#4

Approach to Disorders of Sex Development in the Genomic Era.

Indian journal of pediatrics2026 Jan 20

Disorders of or differences in sex development (DSD) are congenital conditions characterized by a discrepancy between chromosomal, gonadal and/or anatomic sex. They are broadly classified on the basis of karyotype into 46,XX, 46,XY and sex chromosomal DSDs. While the most common cause of 46,XX DSD is congenital adrenal hyperplasia (CAH) due to 21-hydroxylase deficiency, the genetics of 46,XY DSD is more complex with over 100 genes implicated. A precise genetic diagnosis of DSD ends the diagnostic odyssey and helps predict the future course with respect to gender development, puberty and extragenital features. With advancements in genetic testing including next-generation sequencing (NGS) and chromosomal microarray (CMA), it is possible to get a precise genetic diagnosis in up to 40-50% patients with 46,XY DSD. The choice of genetic tests depends on the expected pathophysiology inferred from the clinical phenotype, aiming to maximize diagnostic yields while minimizing costs. However, genetic investigations are not without their own shortcomings, some of which include high cost and complex data analysis, identification of 'variants of uncertain significance (VUS)', and negative results despite exhaustive testing. Therefore, genetic testing should always be undertaken following pre-test counseling, and in consultation with a pediatric endocrinologist and a geneticist. This article reviews the genetic mechanisms underlying DSD, the various genetic testing modalities along with their indications, prerequisites and pitfalls; and outlines a broad algorithmic approach to genetic diagnosis in DSD.

#5

Targeted editing of the 21-hydroxylase locus confers durable therapeutic effect in a murine model of congenital adrenal hyperplasia.

Molecular therapy : the journal of the American Society of Gene Therapy2026 Feb 04

Contemporary treatment for congenital adrenal hyperplasia is lifesaving; however, long-term deleterious effects from the disease and complications from the inadequacy of available treatment remain problematic. Locus-specific correction of the defective 21-hydroxylase gene through genomic editing has the potential to address this unmet need by simultaneously restoring gene function and physiological control. Editing is necessitated by the constant cellular turnover in the adrenal cortex which limits the durability of conventional gene addition strategies using recombinant adeno-associated virus. We have developed a homology-independent targeted integration genomic editing strategy that conferred phenotypic benefit in a mouse model using a dual recombinant adeno-associated virus approach. Reconstitution of 21-hydroxylase expression in the adrenal gland increased corticosteroid production, reduced adrenal gland hyperplasia, and reduced expression of renin and aldosterone synthase with these effects maintained to 15 weeks without diminution. This is beyond the adrenocortical cellular turnover time, providing initial evidence of adrenocortical progenitor cell targeting. Importantly, the editing strategy used is potentially applicable to the majority of causative mutations in classical congenital adrenal hyperplasia. Collectively these data provide strong impetus for addressing the challenges of clinical translation, with development and evaluation of strategies for efficient targeting of human adrenocortical progenitor cells being the most formidable.

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2026

Reduced cortical thickness in individuals with congenital adrenal hyperplasia (CAH).

Scientific reports
2026

Twelve Brazilian families with X-linked Congenital Adrenal Hypoplasia: new rearrangements and new variants in the NR0B1 gene.

Jornal de pediatria
2026

Author Correction to: "Assessment of hormone measurement methods in girls with premature adrenarche, polycystic ovary syndrome, and non-classical congenital adrenal hyperplasia." [Turk J Pediatr 2025; 67: 692-699.].

The Turkish journal of pediatrics
2026

Perspectives on "Assessment of hormone measurement methods in girls with premature adrenarche, polycystic ovary syndrome, and non-classical congenital adrenal hyperplasia".

The Turkish journal of pediatrics
2026

How has genetics changed the diagnosis and the management of differences of sex development?

Annales d'endocrinologie
2026

Novel CYP17A1 variants and functional validation in a large Chinese cohort of complete 17α-hydroxylase deficiency.

The Journal of clinical endocrinology and metabolism
2026

Uncontrolled hypertension in siblings: an unsuspected diagnosis.

AJOG global reports
2026

Male sex assignment in severely virilized 46,XX children with congenital adrenal hyperplasia.

Frontiers in endocrinology
2026

Bone age advancement in non-obese children with premature adrenarche: relationship to growth acceleration and predicted adult height outcomes.

Journal of pediatric endocrinology &amp; metabolism : JPEM
2026

Bilateral adrenal lesions as a manifestation of prolonged glucocorticoid withdrawal in classical adrenal hyperplasia.

Endokrynologia Polska
2026

Optimization of Triptorelin Administration in Children With Central Precocious Puberty and Short Stature.

Clinical endocrinology
2026

Diagnostic dilemma in a neonate: Pseudo Hypoaldosteronism mimicking congenital adrenal hyperplasia.

Pakistan journal of medical sciences
2026

Birth Prevalence of Endocrine-Metabolic Disorders Detected by Newborn Screening Test in Pune (India) Population.

Indian journal of clinical biochemistry : IJCB
2026

Adiposity rebound and height velocity in patients with Congenital Adrenal Hyperplasia.

European journal of endocrinology
2026

Clinical Challenges in Transition to Adult Care for Young People With Endocrinopathies.

Clinical endocrinology
2026

Muscle pain in a woman with congenital adrenal hyperplasia due to 21-hydroxylase deficiency resolved with testosterone therapy. A case report with 10 years of follow-up.

Frontiers in endocrinology
2026

Efficacy of liquid-chromatography and radioimmunoassay in false-positives' drop-off in CAH newborn screening.

The Journal of clinical endocrinology and metabolism
2026

Live Births Following IVF-FET in Two Adult Sisters with Nonclassic P450 Oxidoreductase Deficiency: A Case Report Identifying a Novel POR Variant.

International journal of women's health
2026

Unveiling Salt-Wasting Congenital Adrenal Hyperplasia in an Infant: A Diagnostic Challenge.

Cureus
2026

An isotope dilution-liquid chromatography-tandem mass spectrometry-based candidate reference measurement procedure for the quantification of 17-hydroxyprogesterone in human serum and plasma.

Clinical chemistry and laboratory medicine
2026

Congenital adrenal hyperplasia in Saudi Arabia: Epidemiology, genetic mutations, and evolving management strategies.

Journal of the National Medical Association
2026

Dexamethasone therapy in adolescents with inadequately controlled congenital adrenal hyperplasia: effects on hormonal Suppression, Puberty, and gonadal outcomes.

Endocrine
2026

Four Consecutive False Negative Newborn Screens in a Patient with Classical Congenital Adrenal Hyperplasia: A Case Report.

Journal of clinical research in pediatric endocrinology
2026

Simple virilizing form of 21-hydroxylase deficiency presenting with renal Insufficiency and polycythemia: a case report.

Frontiers in endocrinology
2026

Glucocorticoid Prescribing Trends in Congenital Adrenal Hyperplasia, 2017 to 2023.

Endocrine connections
2026

Current insights into monitoring of congenital adrenal hyperplasia.

Frontiers in endocrinology
2026

Clitoroplasty with corporoplasty in feminizing genitoplasty: Multicenter evaluation of sensory and cosmetic outcomes.

Journal of pediatric urology
2026

Study on the reference intervals of six androgen "backdoor pathway" steroids in healthy Chinese adults.

Clinica chimica acta; international journal of clinical chemistry
2026

A Rare Cause of False-Positive 18 F FDG Uptake in Testicular Adrenal Rest Tumors Mimicking Malignancy in a Patient With Congenital Adrenal Hyperplasia : A Complete Metabolic Response on FDG PET/CT Following Treatment.

Clinical nuclear medicine
2026

Clinical tools for evaluating congenital adrenal hyperplasia in resource-limited hospitals: a study at a tertiary hospital in Saudi Arabia.

Frontiers in endocrinology
2026

Automated bone age assessment in rare pediatric growth disorders: a comparative study using Deeplasia.

Frontiers in endocrinology
2026

A Decade-Long Diagnostic Challenge: A Case of Nonclassical 21-Hydroxylase Deficiency Mistaken for Polycystic Ovary Syndrome.

The journal of obstetrics and gynaecology research
2026

Clinical and genetic characteristics of rare congenital adrenal hyperplasia: a retrospective analysis in a Chinese population.

Frontiers in genetics
2026

Quality of Life in Children With Congenital Adrenal Hyperplasia: A Tertiary-Center Case-Control Study.

Clinical endocrinology
2026

Decreased Gross Motor, Working Memory, and Adaptive Behavior in Children with Congenital Adrenal Hyperplasia.

Hormone research in paediatrics
2026

ACTH and renin in 529 healthy youths: associations to sex, puberty and contraceptives.

Endocrine connections
2026

Hemodynamic and microvascular abnormalities in P450 oxidoreductase deficiency: evidence for COX-dependent dysfunction.

European journal of endocrinology
2026

Clinical Application of Steroid Profiles and Their Interpretation in Adrenal Disorders.

Diagnostics (Basel, Switzerland)
2026

Congenital adrenal hyperplasia with bilateral testicular adrenal rest tumors: a case description emphasizing early ultrasound detection.

Quantitative imaging in medicine and surgery
2026

Clitoroplasty and urogenital sinus mobilization in females with congenital adrenal hyperplasia: A case report with focused literature discussion.

Journal of gynecology obstetrics and human reproduction
2026

Two Distinct Endocrine Conditions in a Single Pediatric Patient: Congenital Adrenal Hyperplasia and Type 1 Diabetes Mellitus.

Cureus
2026

[Non-classic lipoid adrenal hyperplasia: clinical cases report].

Problemy endokrinologii
2025

[Salivary steroid profile investigation by high performance liquid chromatography-tandem mass spectrometry in children with congenital adrenal hyperplasia].

Problemy endokrinologii
2026

Newborn screening in an Eastern Indian province: prevalence and trends from a burgeoning newborn screening unit.

Journal of tropical pediatrics
2026

Testicular adrenal rest tumors and congenital adrenal hyperplasia.

Medicina clinica
2026

Adrenal Venous Sampling Aids in Distinguishing 17-Hydroxyprogesterone Hypersecreting Adrenal Cortical Adenomas from Non-Classical 21-Hydroxylase Deficiency.

Diagnostics (Basel, Switzerland)
2026

Molecular Landscape of Congenital Adrenal Hyperplasia Due to Steroid 21-Hydroxylase Deficiency in India.

Indian journal of pediatrics
2026

Improving diagnosis-specific knowledge of people with differences of sex development (DSD): Evaluation of the two-day Empower-DSD training course in Germany.

Patient education and counseling
2026

Early androgens and development of social personality traits: Evidence from classical congenital adrenal hyperplasia.

Hormones and behavior
2026

Prostatic development and verumontanum in a 46,XX karyotype : An embryological paradox redefining diagnostic algorithm for testicular DSD.

Urology case reports
2026

Genetic diagnosis of CYP21A2-related CAH: adaptive sampling long-read sequencing is an accurate and scalable solution.

European journal of human genetics : EJHG
2026

An isotope dilution-liquid chromatography tandem mass spectrometry-based candidate reference measurement procedure for the quantification of dehydroepiandrosterone in human serum and plasma.

Clinical chemistry and laboratory medicine
2026

Approach to Disorders of Sex Development in the Genomic Era.

Indian journal of pediatrics
2026

Adrenocortical Carcinoma With Right Atrial Extension in a Three-Year-Old Child: A Case Report.

Cureus
2026

Androgen production in adrenocortical H295R cells is regulated by thyroid hormone T3 without reciprocal thyroid axis modulation in pediatric CAH.

The Journal of steroid biochemistry and molecular biology
2026

Contemporary global management of 21-hydroxylase deficiency congenital adrenal hyperplasia in early infancy: a multi-national registry study.

European journal of endocrinology
2026

A novel 2D and 3D model for primary adrenocortical carcinoma of advanced and metastasized stage co-secreting cortisol, aldosterone, testosterone, 18-oxocortisol and 18-hydroxycortisol.

Endocrine-related cancer
2026

Fetal Ovarian Cyst Associated With Disorders of Sex Development: A Case Report.

Cureus
2025

Late-Onset NR0B1-Related Adrenal Hypoplasia Congenita Presenting With Primary Adrenal Insufficiency and Pubertal Delay.

Cureus
2026

StAR Protein Deficiency in Clinical Practice: A Case Series From Saudi Arabia.

Case reports in endocrinology
2026

Continuous subcutaneous hydrocortisone infusion in pediatric primary adrenal insufficiency: a cohort study.

Endocrine connections
2025

Case Report: Challenges of an extremely delayed diagnosis of classic congenital adrenal hyperplasia in a completely virilized 46,XX patient.

Frontiers in endocrinology
2026

Robinow Syndrome Mimicking Congenital Adrenal Hyperplasia.

JCEM case reports
2025

Prevalence of Adrenal Pathology in Children Referred for Premature Pubarche: A 10-Year Retrospective Review in an Indiana Cohort.

Hormone research in paediatrics
2025

Genetic characterization and screening of congenital adrenal hyperplasia by long-read sequencing in a cohort of 21,239 newborns.

Genome medicine
2025

Rare forms of congenital adrenal hyperplasia: pathogenesis, clinical, treatment and management.

Journal of endocrinological investigation
2026

Case Report: Compound Heterozygous SCNN1B Mutations Causing Pseudohypoaldosteronism Type 1B2 in Neonatal Twins.

Molecular genetics &amp; genomic medicine
2025

[Prenatal genetic analysis of a fetus with 21-hydroxylase deficiency due to compound heterozygous variants of CYP21A2 gene].

Zhonghua yi xue yi chuan xue za zhi = Zhonghua yixue yichuanxue zazhi = Chinese journal of medical genetics
2025

Exploration of the potential of genomic editing in the treatment of congenital adrenal hyperplasia.

Frontiers in endocrinology
2025

Neonatal Screening for Congenital Adrenal Hyperplasia in Guangzhou: 7 Years of Experience.

International journal of neonatal screening
2025

Severe Clinical Manifestation of a Salt Wasting Form of Congenital Adrenal Hyperplasia Harboring a Complex Genotype.

Case reports in endocrinology
2025

Significance of a Three-Missense Pathogenic Variant in the Substrate-Binding Lesion in a Subject With 21-Hydroxylase Deficiency: A Case Report.

Cureus
2025

Carrier screening for multiple complex monogenic diseases using long-read sequencing: a population-based study of premarital couples in Shanghai.

Human genomics
2026

Mild Autonomous Cortisol Secretion in Congenital Adrenal Hyperplasia Managed With Mini Back Scope Adrenalectomy.

JCEM case reports
2025

Bilateral giant adrenal myelolipomas: a rare complication of classical congenital adrenal hyperplasia.

BMJ case reports
2026

Improved Performance of Newborn Screening for Congenital Adrenal Hyperplasia Using 21-deoxycortisol Measurement.

Journal of the Endocrine Society
2025

Fertility in congenital adrenal hyperplasia due to 21-hydroxylase deficiency: a review.

Frontiers in endocrinology
2025

Beyond endocrine features in non-classical congenital adrenal hyperplasia: a narrative review of psychoneuro-social perspectives in pediatric and adolescent patients.

European journal of pediatrics
2025

Concomitant Mosaic Turner Syndrome and Congenital Adrenal Hyperplasia in 1 of 3 Patients of USP9X Variant-Associated Autism Spectrum Disorder.

Molecular syndromology
2025

Hyperkalemia in the neonatal intensive care unit: actual or pseudohyperkalemia?

Postgraduate medicine
2025

Impact of Growth-promoting Therapies on Puberty, Growth, and Final Height in Classical 21-hydroxylase Deficiency.

The Journal of clinical endocrinology and metabolism
2025

Bone Health Index (BoneXpert) and parameters of peripheral quantitative computed tomography indicate overall adequate bone health in adolescents with chronic endocrine diseases at time of transition.

PloS one
2025

Assessment of hormone measurement methods in girls with premature adrenarche, polycystic ovary syndrome, and non-classical congenital adrenal hyperplasia.

The Turkish journal of pediatrics
2025

Crinecerfont: CRF1R Antagonist Approved for Treatment of Congenital Adrenal Hyperplasia.

The Annals of pharmacotherapy
2026

Rhabdomyolysis: Initial Presentation of Congenital Adrenal Hyperplasia.

Indian journal of pediatrics
2025

Final Heights in Patients with Congenital Adrenal Hyperplasia: a Retrospective Cohort Study.

Journal of preventive medicine and hygiene
2025

High-Resolution Genetic Analysis in Congenital Adrenal Hyperplasia Due to 21-Hydroxylase Deficiency.

The Journal of clinical endocrinology and metabolism
2025

When Hormones Shape the Mind: Neuropsychiatric Manifestations in a Patient With Congenital Adrenal Hyperplasia and Genital Ambiguity.

Cureus
2025

Partial Androgen Insensitivity Syndrome and Congenital Adrenal Hyperplasia-A Case Report of the Coexistence of Two Rare Diseases in One Patient.

Reports (MDPI)
2025

Newborn genetic screening of congenital adrenal hyperplasia using long-read sequencing.

Orphanet journal of rare diseases
2025

What is the need for adrenalectomy in patients with congenital adrenal hyperplasia in the era of CRF1/ACTH inhibitors?

Frontiers in endocrinology
2026

The Use of Routine Laboratory 17-Hydroxyprogesterone for Identification of Cases of 21-Hydroxylase Deficiency Congenital Adrenal Hyperplasia.

Clinical endocrinology
2025

Silent Threat: A Complex Presentation of Testicular Adrenal Rest Tumors in a Male With Congenital Adrenal Hyperplasia.

Clinical case reports
2025

Single-Center Experience in Five Patients Diagnosed with Lipoid Congenital Adrenal Hyperplasia Due to Steroidogenic Acute Regulatory Protein (STAR) Gene Variants: A Rare Cause of Adrenal Insufficiency.

Journal of clinical research in pediatric endocrinology
2025

A novel POR G88S mutation causes severe PORD and establishes a critical pharmacogenomic risk profile.

The Journal of clinical endocrinology and metabolism
2025

[Clinical and genetic characteristics of congenital adrenal hyperplasia: a retrospective analysis].

Zhongguo dang dai er ke za zhi = Chinese journal of contemporary pediatrics
2025

Spontaneous Pregnancy in Genetically Confirmed 11-Beta Hydroxylase Deficiency: A Case Series and Literature Review.

Cureus
2025

Depressive and anxiety disorders in children and adolescents with selected endocrine diseases.

Pediatric endocrinology, diabetes, and metabolism
2026

Targeted editing of the 21-hydroxylase locus confers durable therapeutic effect in a murine model of congenital adrenal hyperplasia.

Molecular therapy : the journal of the American Society of Gene Therapy
2025

Long-read sequencing analysis of non-classical congenital adrenal hyperplasia prevalence and carrier frequency in Chinese polycystic ovarian syndrome patients.

Journal of ovarian research
2025

A practical approach to diagnosis and treatment in children with primary adrenal insufficiency.

European journal of endocrinology
2026

Pubertal characteristics, final height, and associated factors in patients with nonclassical congenital adrenal hyperplasia: a single center experience.

Journal of pediatric endocrinology &amp; metabolism : JPEM
2025

Metabolic complications in a large cohort of Portuguese women with hirsutism-related disorders.

Journal of endocrinological investigation
2025

The Complex Spectrum of 11β-Hydroxylase Deficiency: A Case of Precocious Puberty, Hypertension, and Testicular Adrenal Rest Tumors (TARTs).

Cureus
2026

An UPLC-MS/MS method for the simultaneous quantification of 6 steroids from the androgen "backdoor pathway" in human plasma.

Clinica chimica acta; international journal of clinical chemistry
2025

Enduring prenatal androgen effects on the female brain.

Brain communications
2025

Caring for Girls With Salt-Wasting Congenital Adrenal Hyperplasia: A Qualitative Study From Central Java, Indonesia.

Journal of pediatric health care : official publication of National Association of Pediatric Nurse Associates &amp; Practitioners
2025

Assessing cortisol levels in non-classical congenital adrenal hyperplasia: focus on the V281L variant.

Therapeutic advances in endocrinology and metabolism
2025

Comprehensive characterization of 21-hydroxylase deficiency in a Chinese pediatric cohort: phenotype, steroid profiles and genetics.

Frontiers in endocrinology
2025

Neonatal presentation of congenital adrenal hyperplasia accompanied by isolated cleft palate: case report.

Oxford medical case reports
2025

CAH due to 11β-hydroxylase deficiency - same same but different!

The Journal of clinical endocrinology and metabolism
2026

Perception of women with classic congenital adrenal hyperplasia and their parents on genital surgery and a diagnosis of differences of sex development: a retrospective survey.

Journal of endocrinological investigation
2025

Measurement of twenty-one serum steroid profiles by UPLC-MS/MS for the diagnosis and monitoring of congenital adrenal hyperplasia.

Journal of mass spectrometry and advances in the clinical lab
2025

17α-hydroxylase deficiency with a novel CYP17A1 mutation: a case report.

Translational pediatrics
2025

Challenges in Sex Assignment in 46,XX Congenital Adrenal Hyperplasia due to 21-hydroxylase Deficiency and 11β-hydroxylase Deficiency in Developing Countries: Insights from an Expert Center in Indonesia.

Journal of clinical research in pediatric endocrinology
2025

Novel Algorithm for Monogenic Noninvasive Prenatal Testing With Highly Similar Parental Pathogenic Haplotypes: A Representative Case of Congenital Adrenal Hyperplasia Pedigree.

Human mutation
2025

Feminizing genitoplasty-management strategy in a case of congenital adrenal hyperplasia with ambiguous genitalia.

Indian journal of urology : IJU : journal of the Urological Society of India
2026

Positive neonatal screening test for congenital adrenal hyperplasia in a case with 3β-hydroxysteroid dehydrogenase type 2 deficiency.

Journal of pediatric endocrinology &amp; metabolism : JPEM
2025

Accurate diagnosis of congenital adrenal hyperplasia due to CYP21A2 variants requires promoter analysis.

European journal of endocrinology
2025

The Steroidogenic Acute Regulatory (STAR) Gene Anatomy, Expression, and Roles.

Development &amp; reproduction
2025

Delayed Diagnosis of Congenital Adrenal Hyperplasia Due to 3β-Hydroxysteroid Dehydrogenase Type 2 Deficiency.

JCEM case reports
2025

Diagnostic and therapeutic pitfalls in the management of pediatric patients with 3β-hydroxysteroid dehydrogenase type 2 (3β-HSD2) deficiency - a single center experience.

Frontiers in endocrinology
2025

Rare causes of pediatric primary adrenal insufficiency: Data from a large nationwide Tunisian cohort.

Annales d'endocrinologie
2025

Advances in congenital adrenal hyperplasia newborn screening: 11-ketotestosterone and 21-deoxycortisone as additional discriminatory biomarkers.

European journal of endocrinology
2025

Human Sexual Polymorphism and Predicted Ranges of Morphological Variation in Human Skeletal Sex Indicators.

American journal of biological anthropology
2025

Male Pattern Androgenetic Alopecia Linked to an Adrenal Tumor: A Case Report.

Skin appendage disorders
2025

STAR/STARD1: A mitochondrial intermembrane space cholesterol shuttle degraded through mitophagy.

Proceedings of the National Academy of Sciences of the United States of America
2026

The Importance of Disease-specific Growth Charts for Children with Congenital Adrenal Hyperplasia.

The Journal of clinical endocrinology and metabolism
2026

Clinical and Biochemical Phenotype Across the Genotypic Spectrum of 21-hydroxylase Deficiency in 457 Individuals.

The Journal of clinical endocrinology and metabolism
2025

The Current Treatment Landscape for Congenital Adrenal Hyperplasia.

Drugs
2025

Unveiling the hidden burden: challenges and spectrum of inborn errors of metabolism in LMICs.

Pediatric research
2025

Bone Health in Youth with Congenital Adrenal Hyperplasia: Abdominal and Total Adiposity Is Associated with Bone Mineral Density.

Hormone research in paediatrics
2025

Pharmacological Characterization of the Novel CRF1 Receptor Antagonist, Thiazolo[4,5-d] Pyrimidine Analog, M43.

Biomolecules
2025

Social Cognition in Adolescents With Gender Dysphoria and Congenital Adrenal Hyperplasia: A Preliminary Investigation of Biological vs. Experiential Gender Effects.

Journal of clinical research in pediatric endocrinology
2025

Optimizing glucocorticoid therapy in congenital adrenal hyperplasia and analog conditions: the intersection of dose reduction, patient care, and coverage in the US.

Frontiers in endocrinology
2025

Urethral stricture in a 46,XX male with congenital adrenal hyperplasia (CAH) due to 21-hydroxylase deficiency: A literature review and case report.

Urology case reports
2025

Increased mortality in primary adrenal insufficiency: a systematic review and meta-analysis.

European journal of endocrinology
2025

Newborn screening for congenital adrenal hyperplasia due to 21 hydroxylase deficiency: the Italian experience 2006-2019.

Journal of endocrinological investigation
2025

Gender Dysphoria in Disorders of Sexual Development: Approach and Prevalence in a Single Center.

Journal of Indian Association of Pediatric Surgeons
2025

Labial Adhesions Secondary to Congenital Adrenal Hyperplasia.

Urology
2025

Adolescent hyperandrogenism: diagnostic challenges and therapeutic approaches.

World journal of pediatrics : WJP
2026

Under the hood: vulvar anatomy and pathology with a focus on MRI.

Abdominal radiology (New York)
2026

Enhancing Accuracy of Newborn Screening for 21OHD: Strategic Use of 21-Deoxycortisol in a Large-Scale Tokyo Cohort.

The Journal of clinical endocrinology and metabolism
2026

Longitudinal Characterization and Sonographic Staging of Testicular Adrenal Rest Tumors.

The Journal of clinical endocrinology and metabolism
2025

Arterial stiffness and shortened QTc interval are associated with androgen and ACTH levels in classic congenital adrenal hyperplasia.

Frontiers in endocrinology
2025

Inherited, Non-CAH Primary Adrenal Insufficiency in Children: A Genetic and Clinical Profile from a Tertiary Care Centre.

Indian journal of endocrinology and metabolism
2025

Combination of Si@UiO-66-NH2 paper-based thin film microextraction with direct solid-state spectrofluorimetry for extraction and determination of estradiol in urine.

Analytica chimica acta
2025

Mineralocorticoid effects of fludrocortisone and hydrocortisone in primary adrenal insufficiency: EU-AIR patient data.

Journal of endocrinological investigation
2025

Apparent Clitoromegaly in a Newborn: A Case of Congenital Adrenal Hyperplasia.

Cureus
2025

Navigating Cervical Cancer Risk in a Patient With Classic Congenital Adrenal Hyperplasia (CAH): A Case Report.

Cureus
2025

Life-Threatening Hyponatremia, Hyperkalemia, and Shock in Infancy: Not Always Congenital Adrenal Hyperplasia.

Cureus
2025

Clinical outcomes and treatment adequacy assessment in congenital adrenal hyperplasia: A Single-Center experience with Long-Term Follow-Up.

Endocrine
2025

Orthodontic Management of a Pediatric Patient Affected by Congenital Adrenal Hyperplasia: Case Report.

Special care in dentistry : official publication of the American Association of Hospital Dentists, the Academy of Dentistry for the Handicapped, and the American Society for Geriatric Dentistry
2025

Phenotypic spectrum and diagnostic challenges in non-21-alpha-hydroxylase deficiency congenital adrenal hyperplasia: a case series from a tertiary care center.

Endocrine regulations
2025

Nonclassical Congenital Adrenal Hyperplasia Presenting With Isolated Hirsutism in a Young Adult: A Case Report.

Cureus
2025

Newborn Screening of the Endocrine System: Best Practices for Evaluation of Hypothyroidism and Congenital Adrenal Hyperplasia.

Clinics in perinatology
2025

Endocrine Disorders and COVID-19 Severity in Pediatric Populations: A Systematic Review.

Cureus
2026

A Recurrent Splice Variant Sheds Light on 11β-Hydroxylase Deficiency in a Unique Large Cohort.

The Journal of clinical endocrinology and metabolism
2025

First intragenic inversion of CYP11B1 gene causing 11β-hydroxylase deficiency: a molecular diagnosis easily overlooked.

Journal of medical genetics
2025

Leptin and adiponectin in children and young persons with congenital adrenal hyperplasia.

European journal of endocrinology
2025

Hepatic metabolism of 11-oxygenated androgens in humans: an integrated in vivo and ex vivo approach.

European journal of endocrinology
2025

Steroid 21-hydroxylase deficiency dysregulates essential molecular pathways of metabolism and energy provision.

Biology open
2025

21-deoxycortisol as a second-tier test in congenital adrenal hyperplasia newborn screening in The Netherlands: two-year evaluation.

Archives of disease in childhood
2025

Elevation of corticosterone and 17OH progesterone in extremely preterm infants and clinical implications.

Pediatric research
2025

A Scoping Review of Literature Exploring the Healthcare Transition of Individuals with Congenital Adrenal Hyperplasia.

Hormone research in paediatrics
2025

The options for delayed surgery - Is there evidence available for delayed genitoplasty in differences/disorders of sex development?

Best practice &amp; research. Clinical endocrinology &amp; metabolism
2025

Management of Giant Bilateral Adrenal Myelolipomas in Congenital Adrenal Hyperplasia.

JCEM case reports
2025

Metabolic phenotype in non-aldosterone producing adrenal adenomas with co-existent polycystic ovary syndrome: a joint Ens@t project.

Endocrine
2025

How should glucocorticoid and mineralocorticoid replacement be optimised in a young patient with classic 21-hydroxylase congenital adrenal hyperplasia?

Internal medicine journal
2025

Three misdiagnoses before a final diagnosis of 17α-hydroxylase/17,20-lyase deficiency: A case report.

Medicine
2025

A Case of Salt-Wasting Congenital Adrenal Hyperplasia Caused by a Rare Intronic Variant in the CYP21A2 Gene.

International journal of molecular sciences
2025

Integration of Adjunctive Therapy for Congenital Adrenal Hyperplasia.

Children (Basel, Switzerland)
2025

Predicting Residual 21-Hydroxylase Enzymatic Activity in Pediatric and Adult Congenital Adrenal Hyperplasia Patients: Towards Individualized Therapy.

CPT: pharmacometrics &amp; systems pharmacology
2025

Alteration of Cytokine/Chemokine Transcript Levels in the Placenta of Humanized Mouse Models Treated Prenatally With Dexamethasone.

Birth defects research
2025

Non-classical congenital adrenal hyperplasia: current insights into clinical implications, diagnosis and treatment.

Endocrine
2025

Longitudinal 12-Month Follow-Up of a Male Infant with CYP21A2 Compound Heterozygous Genotype in China: A Case Report.

AJP reports
2025

Elective Reconstruction for Children With Congenital Adrenal Hyperplasia: Evaluating Association of Familial Characteristics.

The Journal of surgical research
2026

Enhancing primary newborn screening efficiency for congenital adrenal hyperplasia with LC-MS/MS/MS.

Clinica chimica acta; international journal of clinical chemistry
2026

Expanded Steroid Profiling Identifies Novel Newborn Screening Markers for Congenital Adrenal Hyperplasia.

The Journal of clinical endocrinology and metabolism
2025

Long-read sequencing transforms the diagnosis of congenital adrenal hyperplasia: resolving pseudogene interference and structural variations.

Frontiers in pediatrics
2024

A Neonate Presenting with Severe Dehydration: A Rare Case of Congenital Adrenal Hyperplasia with Salt Losing Crisis.

JNMA; journal of the Nepal Medical Association
2025

Adrenocortical organoids: A promising tool for modelling human physiology and translational research.

Presse medicale (Paris, France : 1983)
2025

A Case of Secondary Pseudohypoaldosteronism in a Neonate not Due to Urinary Tract Issues.

Journal of clinical research in pediatric endocrinology
2025

High precision characterization of RCCX rearrangements in a 21-hydroxylase deficiency Latin American cohort using oxford nanopore long read sequencing.

Scientific reports
2025

Testicular adrenal rest tumors in Indonesian boys with congenital adrenal hyperplasia.

Journal of pediatric endocrinology &amp; metabolism : JPEM
2025

Case Report: Type II Bartter syndrome with a novel KCNJ1 variant in a premature neonate presenting with features of salt-wasting congenital adrenal crisis and pseudo-hypoaldosteronism.

Frontiers in pediatrics
2025

The Role of MRI Findings in the Treatment of Testicular Adrenal Rest Tumors in a Child With Salt-Wasting Congenital Adrenal Hyperplasia: A Case Report.

Cureus
2025

Histopathological Features of the Testes of a Patient With Congenital Lipoid Adrenal Hyperplasia.

IJU case reports
2025

Impact of dual-release hydrocortisone on disease control and metabolism in congenital adrenal hyperplasia: a retrospective cohort study.

Endocrine
2025

Approach to the Patient With a Difference of Sexual Development.

The Journal of clinical endocrinology and metabolism
2025

Prevalence of Differences of Sex Development Among Pediatric Endocrine Care Centers in Switzerland From 2000 to 2019.

Journal of the Endocrine Society
2025

PGT-M as A Family Planning Tool for A Couple in Which The Woman Carries A Novel NCAH Variant and A CYP21A2 Variant also Present in Her Husband: A Case Report.

International journal of fertility &amp; sterility
2025

Long-term outcomes in patients with congenital adrenal hyperplasia treated with hydrocortisone modified-release hard capsules.

European journal of endocrinology
2025

Exploring Hirsutism: Epidemiology, Associated Endocrinal Abnormalities, and Societal Challenges in GCC-A Narrative Review.

International journal of molecular sciences
2025

Growth Assessment and Nutritional Status in Children with Congenital Adrenal Hyperplasia-A Cross-Sectional Study from a Vietnamese Tertiary Pediatric Center.

Diagnostics (Basel, Switzerland)
2025

Experience with pediatric and adult cases of ambiguous genitalia reconstructed with a single stage feminizing genitoplasty procedure.

Acta chirurgiae plasticae
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Doenças relacionadas

Doenças com sintomas parecidos — ajudam quem ainda está buscando diagnóstico

Ordenadas pelo número de sintomas em comum.

Referências e fontes

Bases de dados externas citadas neste artigo

Publicações científicas

Artigos indexados no PubMed ligados a esta doença no grafo RarasNet — título, periódico e PMID direto da fonte, sem intermediação de IA.

  1. Diagnostic dilemma in a neonate: Pseudo Hypoaldosteronism mimicking congenital adrenal hyperplasia.
    Pakistan journal of medical sciences· 2026· PMID 41836828mais citado
  2. Newborn screening in an Eastern Indian province: prevalence and trends from a burgeoning newborn screening unit.
    Journal of tropical pediatrics· 2026· PMID 41620979mais citado
  3. Genetic diagnosis of CYP21A2-related CAH: adaptive sampling long-read sequencing is an accurate and scalable solution.
    European journal of human genetics : EJHG· 2026· PMID 41571809mais citado
  4. Approach to Disorders of Sex Development in the Genomic Era.
    Indian journal of pediatrics· 2026· PMID 41555119mais citado
  5. Targeted editing of the 21-hydroxylase locus confers durable therapeutic effect in a murine model of congenital adrenal hyperplasia.
    Molecular therapy : the journal of the American Society of Gene Therapy· 2026· PMID 41220178mais citado
  6. Predictors of quality of life in parents of children with rare diseases: a tertiary care center cross-sectional study in Saudi Arabia.
    Front Public Health· 2026· PMID 41994538recente
  7. Hair cortisol as a marker of glucocorticoid replacement adequacy in adrenal insufficiency.
    Front Endocrinol (Lausanne)· 2026· PMID 41993980recente
  8. Case Report: Compound heterozygous mutation comprising p.Pro31Leu and exons 1-3 ins/del variants in CYP21A2 causes non-classical congenital adrenal hyperplasia in a Chinese girl.
    Front Pediatr· 2026· PMID 41988147recente
  9. Exogenous glucocorticoid dose impacts circulating microRNA expression in patients with adrenal insufficiency due to 21-hydroxylase deficiency.
    Front Endocrinol (Lausanne)· 2026· PMID 41987891recente
  10. Early Risk Stratification in Non-Classical Congenital Adrenal Hyperplasia Based on Newborn 17-OHP Screening Values, Hormonal Findings, and Genotype.
    J Clin Med· 2026· PMID 41976932recente

Bases de dados e fontes oficiais

Identificadores e referências canônicas usadas para montar este verbete.

  1. ORPHA:418(Orphanet)
  2. MONDO:0018479(MONDO)
  3. Hiperplasia Adrenal Congenita(PCDT · Ministério da Saúde)
  4. GARD:1467(GARD (NIH))
  5. Variantes catalogadas(ClinVar)
  6. Busca completa no PubMed(PubMed)
  7. Artigo Wikipedia(Wikipedia)
  8. Q366868(Wikidata)

Dados compilados pelo RarasNet a partir de fontes abertas (Orphanet, OMIM, MONDO, PubMed/EuropePMC, ClinicalTrials.gov, DATASUS, PCDT/MS). Este conteúdo é informativo e não substitui avaliação médica.

Conteúdo mantido por Agente Raras · Médicos e pesquisadores podem colaborar

Hiperplasia suprarrenal congênita
Compêndio · Raras BR

Hiperplasia suprarrenal congênita

ORPHA:418 · MONDO:0018479
🇧🇷 Brasil SUS
Triagem
17-OH-Progesterona em sangue seco
PNTN
Fase 1 · Nacional
Incidência BR
1:15.000
Internações
890/ano
Prevalência BR
1:15000
Custo SUS
R$ 3.210/internação
Dados
DATASUS 2024
Geral
Prevalência
1-9 / 100 000
Herança
Autosomal recessive
CID-10
E25.0 · Transtornos adrenogenitais congênitos associados à deficiência enzimática
CID-11
Ensaios
12 ativos
Medicamentos
12 registrados
Prevalência
10.0 (Europe)
MedGen
UMLS
C0001627
Repurposing
3 candidatos
methylprednisolone-aceponateanti-inflammatory agent|glucocorticoid receptor agonist
prednisolone-sodium-phosphateglucocorticoid receptor agonist
prednisone
EuropePMC
Wikidata
Wikipedia
Papers 10a
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