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Hipoaldosteronismo familiar
ORPHA:427CID-10 · E27.4CID-11 · 5A73DOENÇA RARA

A deficiência da aldosterona sintase é um defeito hereditário raro da etapa final da biossíntese da aldosterona (conversão de desoxicorticosterona em aldosterona).

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Introdução

O que você precisa saber de cara

📋

A deficiência da aldosterona sintase é um defeito hereditário raro da etapa final da biossíntese da aldosterona (conversão de desoxicorticosterona em aldosterona).

Publicações científicas
65 artigos
Último publicado: 2026 Mar 27

Escala de raridade

CLASSIFICAÇÃO ORPHANET · BRASIL 2024
Unknown
Ultra-rara
<1/50k
Muito rara
1/20k
Rara
1/10k
Pouco freq.
1/5k
Incomum
1/2k
Prevalência
0.0
Worldwide
Início
Adolescent
+ adult, infancy, neonatal
🏥
SUS: Sem cobertura SUSScore: 0%
CID-10: E27.4
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Entender a doença

Do básico ao detalhe, leia no seu ritmo

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Sinais e sintomas

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

Partes do corpo afetadas

🫘
Rins
5 sintomas
📏
Crescimento
5 sintomas
🫃
Digestivo
3 sintomas
🧠
Neurológico
1 sintomas

+ 17 sintomas em outras categorias

Características mais comuns

100%prev.
Perda renal de sal
100%prev.
Aumento do nível circulante de renina
90%prev.
Insuficiência adrenal
Muito frequente (99-80%)
90%prev.
Hipovolemia
Muito frequente (99-80%)
90%prev.
Hiponatremia
Muito frequente (99-80%)
90%prev.
Nível diminuído de aldosterona circulante
Muito frequente (99-80%)
31sintomas
Muito frequente (9)
Frequente (10)
Sem dados (12)

Os sintomas variam de pessoa para pessoa. Abaixo estão as 31 características clínicas mais associadas, ordenadas por frequência.

Perda renal de salRenal salt wasting
Muito frequente100%
Aumento do nível circulante de reninaIncreased circulating renin level
Muito frequente100%
Insuficiência adrenalAdrenal insufficiency
Muito frequente (99-80%)90%
HipovolemiaHypovolemia
Muito frequente (99-80%)90%
HiponatremiaHyponatremia
Muito frequente (99-80%)90%

Linha do tempo da pesquisa

Publicações por ano — veja quando o interesse científico cresceu
Anos de pesquisa1desde 2025
Total histórico65PubMed
Últimos 10 anos28publicações
Pico20225 papers
Linha do tempo
2025Hoje · 2026🧪 2007Primeiro ensaio clínico📈 2022Ano de pico
Publicações por ano (últimos 10 anos)

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Genética e causas

O que está alterado no DNA e como passa nas famílias

Genes associados

1 gene identificado com associação a esta condição. Padrão de herança: Autosomal recessive.

CYP11B2Cytochrome P450 11B2, mitochondrialDisease-causing germline mutation(s) inTolerante
FUNÇÃO

A cytochrome P450 monooxygenase that catalyzes the biosynthesis of aldosterone, the main mineralocorticoid in the human body responsible for salt and water homeostasis, thus involved in blood pressure regulation, arterial hypertension, and the development of heart failure (PubMed:11856349, PubMed:12530636, PubMed:1518866, PubMed:15356073, PubMed:1594605, PubMed:1775135, PubMed:22446688, PubMed:23322723, PubMed:9814482, PubMed:9814506). Catalyzes three sequential oxidative reactions of 11-deoxyco

LOCALIZAÇÃO

Mitochondrion inner membrane

VIAS BIOLÓGICAS (3)
Endogenous sterolsGlucocorticoid biosynthesisMineralocorticoid biosynthesis
MECANISMO DE DOENÇA

Corticosterone methyloxidase 1 deficiency

Autosomal recessive disorder of aldosterone biosynthesis. There are two biochemically different forms of selective aldosterone deficiency be termed corticosterone methyloxidase (CMO) deficiency type 1 and type 2. In CMO-1 deficiency, aldosterone is undetectable in plasma, while its immediate precursor, 18-hydroxycorticosterone, is low or normal.

EXPRESSÃO TECIDUAL(Tecido-específico)
Glândula adrenal
89.4 TPM
Testículo
0.2 TPM
Cervix Ectocervix
0.0 TPM
Baço
0.0 TPM
Fígado
0.0 TPM
OUTRAS DOENÇAS (4)
corticosterone methyloxidase type 1 deficiencycorticosterone methyloxidase type 2 deficiencyglucocorticoid-remediable aldosteronismearly-onset familial hypoaldosteronism
HGNC:2592UniProt:P19099

Variantes genéticas (ClinVar)

194 variantes patogênicas registradas no ClinVar.

🧬 CYP11B2: NM_000498.3(CYP11B2):c.1398+1G>C ()
🧬 CYP11B2: NM_000498.3(CYP11B2):c.799+1G>A ()
🧬 CYP11B2: GRCh37/hg19 8q24.13-24.3(chr8:126446968-146295771)x3 ()
🧬 CYP11B2: GRCh37/hg19 8q24.3(chr8:142893048-144990940)x1 ()
🧬 CYP11B2: NM_000498.3(CYP11B2):c.955-1G>A ()
Ver todas no ClinVar

Classificação de variantes (ClinVar)

Distribuição de 1 variantes classificadas pelo ClinVar.

1
Patogênica (100.0%)
VARIANTES MAIS SIGNIFICATIVAS
CYP11B2: NM_000498.3(CYP11B2):c.945C>G (p.Ser315Arg) [Likely pathogenic]

Vias biológicas (Reactome)

4 vias biológicas associadas aos genes desta condição.

Diagnóstico

Os sinais que médicos procuram e os exames que confirmam

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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.
·Pré-clínico1
Medicamentos catalogadosEnsaios clínicos· 0 medicamentos · 1 ensaio
Carregando informações de tratamento...

Onde tratar no SUS

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

🇧🇷 Atendimento SUS — Hipoaldosteronismo familiar

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Pesquisa ativa

Ensaios clínicos abertos e novidades científicas recentes

Pesquisa e ensaios clínicos

0 ensaios clínicos encontrados.

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

📖Melhor nível de evidência: Revisão
Timeline de publicações
30 papers (10 anos)
#1

A Rare Case of Apparent Mineralocorticoid Excess Presenting as Endocrine Hypertension.

Indian journal of nephrology2025

A 3-year-old boy presented with polyuria and polydipsia for 18 months, along with growth failure. He was born prematurely, at 34 weeks of gestation, with a low birth weight. On examination, the child was severely underweight and hypertensive. Clinical history and evaluation could not identify any secondary causes of hypertension. There was no significant family history. An endocrine workup was planned, considering hypokalemia and metabolic alkalosis. This demonstrated hyporeninemic hypoaldosteronism and raised the possibility of apparent mineralocorticoid excess (AME) and Liddle syndrome. Clinical exome sequence analysis of HSD11B2 revealed a homozygous mutation in exon 5 (911A>G; p.His304Arg), which resulted in a missense mutation that confirmed the diagnosis of AME. A novel homozygous variant was found in the HSD11B2 gene in a subject with early onset hypertension associated with hypokalemic metabolic alkalosis, establishing the diagnosis of AME.

#2

Transient secondary pseudo-hypoaldosteronism in infants with urinary tract infections: systematic literature review.

European journal of pediatrics2024 Oct

Infants with a congenital anomaly of the kidney and urinary tract sometimes present with hyponatremia, hyperkalemia, and metabolic acidosis due to under-responsiveness to aldosterone, hereafter referred to as secondary pseudo-hypoaldosteronism. The purpose of this report is to investigate pseudo-hypoaldosteronism in infant urinary tract infection. A systematic review was conducted following PRISMA guidelines after PROSPERO (CRD42022364210) registration. The National Library of Medicine, Excerpta Medica, Web of Science, and Google Scholar without limitations were used. Inclusion criteria involved pediatric cases with documented overt pseudo-hypoaldosteronism linked to urinary tract infection. Data extraction included demographics, clinical features, laboratory parameters, management, and course. Fifty-seven reports were selected, detailing 124 cases: 95 boys and 29 girls, 10 months or less of age (80% of cases were 4 months or less of age). The cases exhibited hyponatremia, hyperkalemia, acidosis, and activated renin-angiotensin II-aldosterone system. An impaired kidney function was found in approximately every third case. Management included antibiotics, fluids, and, occasionally, emergency treatment of hyperkalemia, hyponatremia, or acidosis. The recovery time averaged 1 week for electrolyte, acid-base imbalance, and kidney function. Notably, anomalies of the kidney and urinary tract were identified in 105 (85%) cases. This review expands the understanding of overt transient pseudo-hypoaldosteronism complicating urinary tract infection. Management involves antimicrobials, fluid replacement, and consideration of electrolyte imbalances. Raising awareness of this condition within pediatric hospitalists is desirable. • Infants affected by a congenital anomaly of the kidney and urinary tract may present with clinical and laboratory features resembling primary pseudo-hypoaldosteronism. • Identical features occasionally occur in infant urinary tract infection. • Most cases of secondary pseudo-hypoaldosteronism associated with a urinary tract infection are concurrently affected by a congenital anomaly of the kidney and urinary tract. • Treatment with antibiotics and parenteral fluids typically results in the normalization of sodium, potassium, bicarbonate, and creatinine within approximately 1 week.

#3

Non-canonical Wnt signaling triggered by WNT2B drives adrenal aldosterone production.

bioRxiv : the preprint server for biology2024 Aug 24

The steroid hormone aldosterone, produced by the zona glomerulosa (zG) of the adrenal gland, is a master regulator of plasma electrolytes and blood pressure. While aldosterone control by the renin-angiotensin system is well understood, other key regulatory factors have remained elusive. Here, we replicated a prior association between a non-coding variant in WNT2B and an increased risk of primary aldosteronism, a prevalent and debilitating disease caused by excessive aldosterone production. We further show that in both mice and humans, WNT2B is expressed in the mesenchymal capsule surrounding the adrenal cortex, in close proximity to the zG. Global loss of Wnt2b in the mouse results in a dysmorphic and hypocellular zG, with impaired aldosterone production. Similarly, humans harboring WNT2B loss-of-function mutations develop a novel form of Familial Hyperreninemic Hypoaldosteronism, designated here as Type 4. Additionally, we demonstrate that WNT2B signals by activating the non-canonical Wnt/planar cell polarity pathway. Our findings identify WNT2B as a key regulator of zG function and aldosterone production with important clinical implications.

#4

Hyporeninemic hypoaldosteronism in RMND1-related mitochondrial disease.

Pediatric nephrology (Berlin, Germany)2024 Jan

RMND1 is a nuclear gene needed for proper function of mitochondria. A pathogenic gene will cause multiple oxidative phosphorylation defects. A renal phenotype consisting of hyponatremia, hyperkalemia, and acidosis is frequently reported, previously considered to be due to aldosterone insensitivity. Clinical features and pathophysiology of three patients will be reported. DNA of these patients was subjected to exome screening. In the first family, one pathogenic heterozygous and one highly probable heterozygous mutation were detected. In the second family, a homozygous pathogenic mutation was present. The electrolyte disbalance was not due to aldosterone insensitivity but to low plasma aldosterone concentration, a consequence of low plasma renin activity. This disbalance can be treated. In all three patients, the kidney function declined. In the first family, both children suffered from an unexplained arterial thrombosis with dire consequences. Hyporeninemic hypoaldosteronism is the mechanism causing the electrolyte disbalance reported in patients with RMND1 mutations, and can be treated.

#5

Identification of a Novel CYP11B2 Variant in a Family with Varying Degrees of Aldosterone Synthase Deficiency.

Journal of clinical research in pediatric endocrinology2024 Mar 11

Isolated aldosterone synthase deficiency is a rare autosomal recessive disorder caused by pathogenic variants in CYP11B2, resulting in impaired aldosterone synthesis. We report on a neonate with isolated aldosterone synthase deficiency caused by a novel homozygous CYP11B2 variant Chr8:NM_000498.3:c.400G>A p.(Gly134Arg). The patient presented shortly after birth with severe signs of aldosterone deficiency. Interestingly, segregation analysis revealed that the patient’s asymptomatic father was also homozygous for the CYP11B2 variant. Biochemical evaluation of the father indicated subclinical enzyme impairment, characterized by elevated aldosterone precursors. Apparently, this homozygous variant led to different clinical phenotypes in two affected relatives. In this manuscript we elaborate on the biochemical and genetic work-up performed and describe potential pitfalls in CYP11B2 sequencing due to its homology to CYP11B1.

Publicações recentes

Ver todas no PubMed

📚 EuropePMC1 artigos no totalmostrando 28

2025

A Rare Case of Apparent Mineralocorticoid Excess Presenting as Endocrine Hypertension.

Indian journal of nephrology
2024

Non-canonical Wnt signaling triggered by WNT2B drives adrenal aldosterone production.

bioRxiv : the preprint server for biology
2024

Transient secondary pseudo-hypoaldosteronism in infants with urinary tract infections: systematic literature review.

European journal of pediatrics
2023

Familial Glucocorticoid Deficiency: the changing landscape of an eponymous syndrome.

Frontiers in endocrinology
2023

Role of NCC in the pathophysiology of hypertension in primary aldosteronism.

Current opinion in nephrology and hypertension
2024

Hyporeninemic hypoaldosteronism in RMND1-related mitochondrial disease.

Pediatric nephrology (Berlin, Germany)
2023

Classification of pseudohypoaldosteronism type II as type IV renal tubular acidosis: results of a literature review.

Endocrine journal
2023

Loss of LGR4/GPR48 causes severe neonatal salt wasting due to disrupted WNT signaling altering adrenal zonation.

The Journal of clinical investigation
2024

Identification of a Novel CYP11B2 Variant in a Family with Varying Degrees of Aldosterone Synthase Deficiency.

Journal of clinical research in pediatric endocrinology
2022

A Novel Frame-Shift Mutation in SCNN1B Identified in a Chinese Family Characterized by Early-Onset Hypertension.

Frontiers in cardiovascular medicine
2022

A 3-Year-Old Boy with an Xp21 Deletion Syndrome: A Case Report.

Endocrine, metabolic &amp; immune disorders drug targets
2022

Pseudo-hypoaldosteronism secondary to infantile urinary tract infections: role of ultrasound.

Italian journal of pediatrics
2022

Familial hyperkalemic hypertension: hyperkalemia not hypertension defines dominant KLHL3 disease and may permit earlier recognition and tailored therapy.

Journal of nephrology
2022

Catch-up Growth and Discontinuation of Fludrocortisone Treatment in Aldosterone Synthase Deficiency.

The Journal of clinical endocrinology and metabolism
2021

A rare case of persistent hyperkalaemia.

Annals of clinical biochemistry
2021

Liddle Syndrome due to a Novel c.1713 Deletion in the Epithelial Sodium Channel β-Subunit in a Normotensive Adolescent.

AACE clinical case reports
2020

Metabolic Acidosis, Hyperkalemia, and Renal Unresponsiveness to Aldosterone Syndrome: Response to Treatment with Low-Potassium Diet.

Saudi journal of kidney diseases and transplantation : an official publication of the Saudi Center for Organ Transplantation, Saudi Arabia
2020

Aldosterone deficiency with a hormone profile mimicking pseudohypoaldosteronism.

Journal of pediatric endocrinology &amp; metabolism : JPEM
2021

Clinical and Genetic Characteristics of Patients with Corticosterone Methyloxidase Deficiency Type 2: Novel Mutations in CYP11B2.

Journal of clinical research in pediatric endocrinology
2019

Influence of Genotype and Hyperandrogenism on Sexual Function in Women With Congenital Adrenal Hyperplasia.

The journal of sexual medicine
2019

Isolated hypoaldosteronism as first sign of X-linked adrenal hypoplasia congenita caused by a novel mutation in NR0B1/DAX-1 gene: a case report.

BMC medical genetics
2019

Systemic Pseudohypoaldosteronism Type 1 due to 3 Novel Mutations in SCNN1Aand SCNN1BGenes.

Hormone research in paediatrics
2019

Adrenal hypoplasia congenita in identical twins.

Saudi medical journal
2018

Liddle's-like syndrome associated with nephrotic syndrome secondary to membranous nephropathy: the first case report.

BMC nephrology
2018

Molecular genetic studies in a case series of isolated hypoaldosteronism due to biosynthesis defects or aldosterone resistance.

Clinical endocrinology
2016

Corticosterone Methyl Oxidase Deficiency Type 1 with Normokalemia in an Infant.

Journal of clinical research in pediatric endocrinology
2017

Apparent mineralocorticoid excess and the long term treatment of genetic hypertension.

The Journal of steroid biochemistry and molecular biology
2015

Presence of 21-Hydroxylase Antibodies in a Boy with X-Linked Adrenal Hypoplasia Congenita.

Hormone research in paediatrics

Associações

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Comunidades

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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. A Rare Case of Apparent Mineralocorticoid Excess Presenting as Endocrine Hypertension.
    Indian journal of nephrology· 2025· PMID 40352883mais citado
  2. Transient secondary pseudo-hypoaldosteronism in infants with urinary tract infections: systematic literature review.
    European journal of pediatrics· 2024· PMID 38985174mais citado
  3. Non-canonical Wnt signaling triggered by WNT2B drives adrenal aldosterone production.
    bioRxiv : the preprint server for biology· 2024· PMID 39229119mais citado
  4. Hyporeninemic hypoaldosteronism in RMND1-related mitochondrial disease.
    Pediatric nephrology (Berlin, Germany)· 2024· PMID 37450011mais citado
  5. Identification of a Novel CYP11B2 Variant in a Family with Varying Degrees of Aldosterone Synthase Deficiency.
    Journal of clinical research in pediatric endocrinology· 2024· PMID 35848593mais citado
  6. Congenital aldosterone deficiency and its resistance.
    Endocr J· 2026· PMID 41905953recente
  7. A novel compound heterozygous mutation in CYP11B2 (p.A153P and p.Q337*) associated with primary hypoaldosteronism.
    Front Med (Lausanne)· 2026· PMID 41889508recente
  8. Clinical and genetic characteristics of rare congenital adrenal hyperplasia: a retrospective analysis in a Chinese population.
    Front Genet· 2026· PMID 41709876recente
  9. Genetic Insights and Lifelong Management of Aldosterone Synthase Deficiency: A Case of Hyperreninemic Hypoaldosteronism.
    Clin Case Rep· 2026· PMID 41479431recente
  10. Aldosterone Synthase Deficiency Type I in a Neonate: Diagnostic, Genetic, and Therapeutic Insights From a Novel CYP11B2 Variant.
    Cureus· 2025· PMID 41426917recente

Bases de dados e fontes oficiais

Identificadores e referências canônicas usadas para montar este verbete.

  1. ORPHA:427(Orphanet)
  2. MONDO:0018541(MONDO)
  3. GARD:16532(GARD (NIH))
  4. Variantes catalogadas(ClinVar)
  5. Busca completa no PubMed(PubMed)
  6. Q56014164(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

Compêndio · Raras BR

Hipoaldosteronismo familiar

ORPHA:427 · MONDO:0018541
Prevalência
Unknown
Herança
Autosomal recessive
CID-10
E27.4 · Outras insuficiências adrenocorticais e as não especificadas
CID-11
Início
Adolescent, Adult, Infancy, Neonatal
Prevalência
0.0 (Worldwide)
MedGen
UMLS
C4275180
EuropePMC
Wikidata
Papers 10a
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