Raras
Buscar doenças, sintomas, genes...
Neoplasias endócrinas múltiplas
ORPHA:100094CID-10 · D44.8CID-11 · 2F7A.0DOENÇA RARA

A seguir, uma lista dos tipos de câncer. O câncer é um grupo de doenças que envolvem aumentos anormais no número de células, com potencial para invadir ou se espalhar para outras partes do corpo. Nem todos os tumores ou nódulos são cancerosos; os tumores benignos não são classificados como câncer porque não se espalham para outras partes do corpo. Existem mais de 100 tipos diferentes de cânceres conhecidos que afetam os seres humanos.

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Introdução

O que você precisa saber de cara

📋

Doença rara caracterizada por múltiplos tumores em glândulas endócrinas, associada a pectus excavatum, infiltrados pulmonares, náuseas, vômitos, anemia, fadiga e alterações cardíacas. Pode envolver mutações em genes como SDHD e NTRK1.

🏥
SUS: Sem cobertura SUSScore: 0%
CID-10: D44.8
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Sinais e sintomas

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

Partes do corpo afetadas

📏
Crescimento
30 sintomas
🫃
Digestivo
23 sintomas
🦴
Ossos e articulações
13 sintomas
🫘
Rins
11 sintomas
🧠
Neurológico
10 sintomas
🩸
Sangue
4 sintomas

+ 68 sintomas em outras categorias

Características mais comuns

Pectus excavatum
Infiltrados pulmonares
Náusea e vômito
Anemia
Linfadenopatia mediastinal
Leiomiossarcoma
178sintomas
Sem dados (178)

Os sintomas variam de pessoa para pessoa. Abaixo estão as 178 características clínicas mais associadas, ordenadas por frequência.

Pectus excavatum
Infiltrados pulmonaresPulmonary infiltrates
Náusea e vômitoNausea and vomiting
Anemia
Linfadenopatia mediastinalMediastinal lymphadenopathy

Linha do tempo da pesquisa

Publicações por ano — veja quando o interesse científico cresceu
Anos de pesquisa11
Últimos 10 anos6publicações
Pico20252 papers
Linha do tempo
20202015Hoje · 2026📈 2025Ano de pico
Publicações por ano (últimos 10 anos)

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

10 genes identificados com associação a esta condição.

SDHDSuccinate dehydrogenase [ubiquinone] cytochrome b small subunit, mitochondrialDisease-causing germline mutation(s) inRestrito
FUNÇÃO

Membrane-anchoring subunit of succinate dehydrogenase (SDH) that is involved in complex II of the mitochondrial electron transport chain and is responsible for transferring electrons from succinate to ubiquinone (coenzyme Q) (PubMed:10482792, PubMed:9533030). SDH also oxidizes malate to the non-canonical enol form of oxaloacetate, enol-oxaloacetate (By similarity). Enol-oxaloacetate, which is a potent inhibitor of the succinate dehydrogenase activity, is further isomerized into keto-oxaloacetate

LOCALIZAÇÃO

Mitochondrion inner membrane

VIAS BIOLÓGICAS (3)
Maturation of TCA enzymes and regulation of TCA cycleCitric acid cycle (TCA cycle)Respiratory electron transport
MECANISMO DE DOENÇA

Pheochromocytoma/paraganglioma syndrome 1

A form of pheochromocytoma/paraganglioma syndrome, a tumor predisposition syndrome characterized by the development of neuroendocrine tumors, usually in adulthood. Pheochromocytomas are catecholamine-producing tumors that arise from chromaffin cells in the adrenal medulla. Paragangliomas develop from sympathetic paraganglia in the thorax, abdomen, and pelvis, as well as from parasympathetic paraganglia in the head and neck. PPGL1 inheritance is autosomal dominant.

EXPRESSÃO TECIDUAL(Ubíquo)
Rim - Medula
91.4 TPM
Músculo esquelético
85.3 TPM
Cólon transverso
81.0 TPM
Cólon sigmoide
78.9 TPM
Linfócitos
75.5 TPM
OUTRAS DOENÇAS (8)
Carney-Stratakis syndromepheochromocytoma/paraganglioma syndrome 1mitochondrial complex 2 deficiency, nuclear type 3sporadic pheochromocytoma/secreting paraganglioma
HGNC:10683UniProt:O14521
SDHASuccinate dehydrogenase [ubiquinone] flavoprotein subunit, mitochondrialCandidate gene tested inTolerante
FUNÇÃO

Flavoprotein (FP) subunit of succinate dehydrogenase (SDH) that is involved in complex II of the mitochondrial electron transport chain and is responsible for transferring electrons from succinate to ubiquinone (coenzyme Q) (PubMed:10746566, PubMed:24781757). SDH also oxidizes malate to the non-canonical enol form of oxaloacetate, enol-oxaloacetate (By similarity). Enol-oxaloacetate, which is a potent inhibitor of the succinate dehydrogenase activity, is further isomerized into keto-oxaloacetate

LOCALIZAÇÃO

Mitochondrion inner membrane

VIAS BIOLÓGICAS (1)
Maturation of TCA enzymes and regulation of TCA cycle
MECANISMO DE DOENÇA

Mitochondrial complex II deficiency, nuclear type 1

A disorder of the mitochondrial respiratory chain with heterogeneous clinical manifestations. Clinical features include psychomotor regression in infants, poor growth with lack of speech development, severe spastic quadriplegia, dystonia, progressive leukoencephalopathy, muscle weakness, exercise intolerance, cardiomyopathy. Some patients manifest Leigh syndrome or Kearns-Sayre syndrome. MC2DN1 inheritance is autosomal recessive.

EXPRESSÃO TECIDUAL(Ubíquo)
Coração - Ventrículo esquerdo
302.0 TPM
Músculo esquelético
237.6 TPM
Coração - Átrio
206.8 TPM
Glândula adrenal
172.2 TPM
Linfócitos
168.3 TPM
OUTRAS DOENÇAS (9)
mitochondrial complex II deficiency, nuclear type 1pheochromocytoma/paraganglioma syndrome 5neurodegeneration with ataxia and late-onset optic atrophydilated cardiomyopathy 1GG
HGNC:10680UniProt:P31040
NTRK1High affinity nerve growth factor receptorCandidate gene tested inTolerante
FUNÇÃO

Receptor tyrosine kinase involved in the development and the maturation of the central and peripheral nervous systems through regulation of proliferation, differentiation and survival of sympathetic and nervous neurons. High affinity receptor for NGF which is its primary ligand (PubMed:1281417, PubMed:15488758, PubMed:17196528, PubMed:1849459, PubMed:1850821, PubMed:22649032, PubMed:27445338, PubMed:8325889). Can also bind and be activated by NTF3/neurotrophin-3. However, NTF3 only supports axon

LOCALIZAÇÃO

Cell membraneEarly endosome membraneLate endosome membraneRecycling endosome membrane

VIAS BIOLÓGICAS (1)
TRKA activation by NGF
MECANISMO DE DOENÇA

Congenital insensitivity to pain with anhidrosis

Characterized by a congenital insensitivity to pain, anhidrosis (absence of sweating), absence of reaction to noxious stimuli, self-mutilating behavior, and intellectual disability. This rare autosomal recessive disorder is also known as congenital sensory neuropathy with anhidrosis or hereditary sensory and autonomic neuropathy type IV or familial dysautonomia type II.

EXPRESSÃO TECIDUAL(Tecido-específico)
Próstata
5.7 TPM
Testículo
4.9 TPM
Cervix Endocervix
4.3 TPM
Útero
3.7 TPM
Fallopian Tube
3.5 TPM
OUTRAS DOENÇAS (4)
hereditary sensory and autonomic neuropathy type 4hereditary sensory and autonomic neuropathy type 5familial medullary thyroid carcinomadifferentiated thyroid carcinoma
HGNC:8031UniProt:P04629
ESR2Estrogen receptor betaCandidate gene tested inTolerante
FUNÇÃO

Nuclear hormone receptor. Binds estrogens with an affinity similar to that of ESR1/ER-alpha, and activates expression of reporter genes containing estrogen response elements (ERE) in an estrogen-dependent manner (PubMed:20074560) Lacks ligand binding ability and has no or only very low ERE binding activity resulting in the loss of ligand-dependent transactivation ability

LOCALIZAÇÃO

Nucleus

VIAS BIOLÓGICAS (4)
PI5P, PP2A and IER3 Regulate PI3K/AKT SignalingPIP3 activates AKT signalingConstitutive Signaling by Aberrant PI3K in CancerExtra-nuclear estrogen signaling
MECANISMO DE DOENÇA

Ovarian dysgenesis 8

An autosomal dominant form of ovarian dysgenesis, a disorder characterized by lack of spontaneous pubertal development, primary amenorrhea, uterine hypoplasia, and hypergonadotropic hypogonadism as a result of streak gonads.

EXPRESSÃO TECIDUAL(Baixa expressão)
Ovário
4.6 TPM
Testículo
3.9 TPM
Glândula adrenal
3.6 TPM
Linfócitos
2.3 TPM
Cervix Endocervix
1.4 TPM
OUTRAS DOENÇAS (2)
ovarian dysgenesis 8familial medullary thyroid carcinoma
HGNC:3468UniProt:Q92731
CDKN2BCyclin-dependent kinase 4 inhibitor BCandidate gene tested inTolerante
FUNÇÃO

Interacts strongly with CDK4 and CDK6. Potent inhibitor. Potential effector of TGF-beta induced cell cycle arrest

LOCALIZAÇÃO

Cytoplasm

VIAS BIOLÓGICAS (5)
Cyclin D associated events in G1Senescence-Associated Secretory Phenotype (SASP)Oncogene Induced SenescenceOxidative Stress Induced SenescenceSMAD2/SMAD3:SMAD4 heterotrimer regulates transcription
OUTRAS DOENÇAS (2)
familial melanomamultiple endocrine neoplasia type 1
HGNC:1788UniProt:P42772
SDHCSuccinate dehydrogenase cytochrome b560 subunit, mitochondrialDisease-causing germline mutation(s) inTolerante
FUNÇÃO

Membrane-anchoring subunit of succinate dehydrogenase (SDH) that is involved in complex II of the mitochondrial electron transport chain and is responsible for transferring electrons from succinate to ubiquinone (coenzyme Q) (PubMed:9533030). SDH also oxidizes malate to the non-canonical enol form of oxaloacetate, enol-oxaloacetate (By similarity). Enol-oxaloacetate, which is a potent inhibitor of the succinate dehydrogenase activity, is further isomerized into keto-oxaloacetate (By similarity)

LOCALIZAÇÃO

Mitochondrion inner membrane

VIAS BIOLÓGICAS (3)
Maturation of TCA enzymes and regulation of TCA cycleCitric acid cycle (TCA cycle)Respiratory electron transport
MECANISMO DE DOENÇA

Pheochromocytoma/paraganglioma syndrome 3

A form of pheochromocytoma/paraganglioma syndrome, a tumor predisposition syndrome characterized by the development of neuroendocrine tumors, usually in adulthood. Pheochromocytomas are catecholamine-producing tumors that arise from chromaffin cells in the adrenal medulla. Paragangliomas develop from sympathetic paraganglia in the thorax, abdomen, and pelvis, as well as from parasympathetic paraganglia in the head and neck. PPGL3 inheritance is autosomal dominant.

EXPRESSÃO TECIDUAL(Ubíquo)
Fibroblastos
21.2 TPM
Glândula adrenal
19.6 TPM
Músculo esquelético
18.4 TPM
Cérebro - Hemisfério cerebelar
16.9 TPM
Linfócitos
16.5 TPM
OUTRAS DOENÇAS (6)
gastrointestinal stromal tumorCarney-Stratakis syndromepheochromocytoma/paraganglioma syndrome 3hereditary pheochromocytoma-paraganglioma
HGNC:10682UniProt:Q99643
SDHBSuccinate dehydrogenase [ubiquinone] iron-sulfur subunit, mitochondrialDisease-causing germline mutation(s) inTolerante
FUNÇÃO

Iron-sulfur protein (IP) subunit of the succinate dehydrogenase complex (mitochondrial respiratory chain complex II), responsible for transferring electrons from succinate to ubiquinone (coenzyme Q) (PubMed:26925370, PubMed:27604842). SDH also oxidizes malate to the non-canonical enol form of oxaloacetate, enol-oxaloacetate (By similarity). Enol-oxaloacetate, which is a potent inhibitor of the succinate dehydrogenase activity, is further isomerized into keto-oxaloacetate (By similarity)

LOCALIZAÇÃO

Mitochondrion inner membrane

VIAS BIOLÓGICAS (1)
Maturation of TCA enzymes and regulation of TCA cycle
MECANISMO DE DOENÇA

Pheochromocytoma/paraganglioma syndrome 4

A form of pheochromocytoma/paraganglioma syndrome, a tumor predisposition syndrome characterized by the development of neuroendocrine tumors, usually in adulthood. Pheochromocytomas are catecholamine-producing tumors that arise from chromaffin cells in the adrenal medulla. Paragangliomas develop from sympathetic paraganglia in the thorax, abdomen, and pelvis, as well as from parasympathetic paraganglia in the head and neck. PPGL4 inheritance is autosomal dominant.

EXPRESSÃO TECIDUAL(Ubíquo)
Músculo esquelético
177.6 TPM
Coração - Ventrículo esquerdo
168.2 TPM
Linfócitos
144.5 TPM
Fígado
124.9 TPM
Coração - Átrio
122.8 TPM
OUTRAS DOENÇAS (9)
Carney-Stratakis syndromepheochromocytoma/paraganglioma syndrome 4gastrointestinal stromal tumormitochondrial complex 2 deficiency, nuclear type 4
HGNC:10681UniProt:P21912
RETProto-oncogene tyrosine-protein kinase receptor RetDisease-causing germline mutation(s) inAltamente restrito
FUNÇÃO

Receptor tyrosine-protein kinase involved in numerous cellular mechanisms including cell proliferation, neuronal navigation, cell migration, and cell differentiation in response to glia cell line-derived growth family factors (GDNF, NRTN, ARTN, PSPN and GDF15) (PubMed:20064382, PubMed:20616503, PubMed:20702524, PubMed:21357690, PubMed:21454698, PubMed:24560924, PubMed:28846097, PubMed:28846099, PubMed:28953886, PubMed:31118272). In contrast to most receptor tyrosine kinases, RET requires not onl

LOCALIZAÇÃO

Cell membraneEndosome membrane

VIAS BIOLÓGICAS (4)
RET signalingFormation of the ureteric budFormation of the nephric ductNPAS4 regulates expression of target genes
MECANISMO DE DOENÇA

Hirschsprung disease 1

A disorder of neural crest development characterized by absence of enteric ganglia along a variable length of the intestine. It is the most common cause of congenital intestinal obstruction. Early symptoms range from complete acute neonatal obstruction, characterized by vomiting, abdominal distention and failure to pass stool, to chronic constipation in the older child.

EXPRESSÃO TECIDUAL(Tecido-específico)
Substância negra
6.3 TPM
Pituitária
4.8 TPM
Cerebelo
4.0 TPM
Cólon sigmoide
4.0 TPM
Brain Frontal Cortex BA9
3.8 TPM
OUTRAS DOENÇAS (12)
multiple endocrine neoplasia type 2Bpheochromocytomafamilial medullary thyroid carcinomamultiple endocrine neoplasia type 2A
HGNC:9967UniProt:P07949
CDKN1BCyclin-dependent kinase inhibitor 1BDisease-causing germline mutation(s) inModerado
FUNÇÃO

Important regulator of cell cycle progression. Inhibits the kinase activity of CDK2 bound to cyclin A, but has little inhibitory activity on CDK2 bound to SPDYA (PubMed:28666995). Involved in G1 arrest. Potent inhibitor of cyclin E- and cyclin A-CDK2 complexes. Forms a complex with cyclin type D-CDK4 complexes and is involved in the assembly, stability, and modulation of CCND1-CDK4 complex activation. Acts either as an inhibitor or an activator of cyclin type D-CDK4 complexes depending on its ph

LOCALIZAÇÃO

NucleusCytoplasmEndosome

VIAS BIOLÓGICAS (10)
Cyclin D associated events in G1Defective binding of RB1 mutants to E2F1,(E2F2, E2F3)SCF(Skp2)-mediated degradation of p27/p21TP53 Regulates Transcription of Genes Involved in G1 Cell Cycle ArrestDNA Damage/Telomere Stress Induced Senescence
MECANISMO DE DOENÇA

Multiple endocrine neoplasia 4

Multiple endocrine neoplasia (MEN) syndromes are inherited cancer syndromes of the thyroid. MEN4 is a MEN-like syndrome with a phenotypic overlap of both MEN1 and MEN2.

OUTRAS DOENÇAS (2)
multiple endocrine neoplasia type 4multiple endocrine neoplasia type 1
HGNC:1785UniProt:P46527
MEN1MeninDisease-causing germline mutation(s) inAltamente restrito
FUNÇÃO

Essential component of a MLL/SET1 histone methyltransferase (HMT) complex, a complex that specifically methylates 'Lys-4' of histone H3 (H3K4). Functions as a transcriptional regulator. Binds to the TERT promoter and represses telomerase expression. Plays a role in TGFB1-mediated inhibition of cell-proliferation, possibly regulating SMAD3 transcriptional activity. Represses JUND-mediated transcriptional activation on AP1 sites, as well as that mediated by NFKB subunit RELA. Positively regulates

LOCALIZAÇÃO

Nucleus

VIAS BIOLÓGICAS (4)
SMAD2/SMAD3:SMAD4 heterotrimer regulates transcriptionDeactivation of the beta-catenin transactivating complexFormation of the beta-catenin:TCF transactivating complexFormation of WDR5-containing histone-modifying complexes
MECANISMO DE DOENÇA

Familial multiple endocrine neoplasia type I

Autosomal dominant disorder characterized by tumors of the parathyroid glands, gastro-intestinal endocrine tissue, the anterior pituitary and other tissues. Cutaneous lesions and nervous-tissue tumors can exist. Prognosis in MEN1 patients is related to hormonal hypersecretion by tumors, such as hypergastrinemia causing severe peptic ulcer disease (Zollinger-Ellison syndrome, ZES), primary hyperparathyroidism, and acute forms of hyperinsulinemia.

EXPRESSÃO TECIDUAL(Ubíquo)
Cerebelo
45.1 TPM
Tireoide
43.2 TPM
Cérebro - Hemisfério cerebelar
40.2 TPM
Fibroblastos
37.9 TPM
Baço
35.0 TPM
OUTRAS DOENÇAS (7)
multiple endocrine neoplasia type 1pituitary gigantismnull pituitary adenomaprolactin-producing pituitary gland adenoma
HGNC:7010UniProt:O00255

Variantes genéticas (ClinVar)

1,239 variantes patogênicas registradas no ClinVar.

🧬 SDHD: NM_003002.4(SDHD):c.174del (p.Ser59fs) ()
🧬 SDHD: NM_003002.4(SDHD):c.196del (p.Trp66fs) ()
🧬 SDHD: NM_003002.4(SDHD):c.334dup (p.Thr112fs) ()
🧬 SDHD: NM_003002.4(SDHD):c.233del (p.Gly78fs) ()
🧬 SDHD: NM_003002.4(SDHD):c.170-1G>C ()
Ver todas no ClinVar

Vias biológicas (Reactome)

49 vias biológicas associadas aos genes desta condição.

Respiratory electron transport Citric acid cycle (TCA cycle) Maturation of TCA enzymes and regulation of TCA cycle PLC-gamma1 signalling Signalling to RAS Frs2-mediated activation ARMS-mediated activation Retrograde neurotrophin signalling NGF-independant TRKA activation TRKA activation by NGF Signalling to p38 via RIT and RIN PI3K/AKT activation Signalling to STAT3 PIP3 activates AKT signaling Constitutive Signaling by Aberrant PI3K in Cancer Nuclear Receptor transcription pathway PI5P, PP2A and IER3 Regulate PI3K/AKT Signaling ESR-mediated signaling Extra-nuclear estrogen signaling SMAD2/SMAD3:SMAD4 heterotrimer regulates transcription Oxidative Stress Induced Senescence Senescence-Associated Secretory Phenotype (SASP) Oncogene Induced Senescence Cyclin D associated events in G1 RAF/MAP kinase cascade RET signaling NPAS4 regulates expression of target genes Formation of the nephric duct Formation of the ureteric bud SCF(Skp2)-mediated degradation of p27/p21 AKT phosphorylates targets in the cytosol DNA Damage/Telomere Stress Induced Senescence RHO GTPases activate CIT Constitutive Signaling by AKT1 E17K in Cancer TP53 Regulates Transcription of Genes Involved in G1 Cell Cycle Arrest Cyclin E associated events during G1/S transition p53-Dependent G1 DNA Damage Response Cyclin A:Cdk2-associated events at S phase entry PTK6 Regulates Cell Cycle FLT3 Signaling FOXO-mediated transcription of cell cycle genes Estrogen-dependent nuclear events downstream of ESR-membrane signaling Defective binding of RB1 mutants to E2F1,(E2F2, E2F3) Formation of the beta-catenin:TCF transactivating complex Deactivation of the beta-catenin transactivating complex Regulation of Insulin-like Growth Factor (IGF) transport and uptake by Insulin-like Growth Factor Binding Proteins (IGFBPs) RHO GTPases activate IQGAPs Post-translational protein phosphorylation Formation of WDR5-containing histone-modifying complexes

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Publicações mais relevantes

Timeline de publicações
0 papers (10 anos)
#1

Case Report: Novel likely pathogenic MEN1 mosaic mutation in the family with MEN-1 syndrome.

Frontiers in endocrinology2025

Multiple endocrine neoplasia type 1 (MEN-1; OMIM 131100) is a rare, autosomal dominant syndrome caused by heterozygous inactivating mutations in the MEN1 tumor suppressor gene (11q13; OMIM 613733). MEN-1 is characterized by polyglandular pathology, which typically involves the parathyroid glands (90%), pancreas (30-80%) and anterior pituitary (15-50%). To date, over 1,600 pathogenic MEN1 variants have been documented, including nonsense, frameshift, and splice-site mutations, as well as rare large deletions. While germline mutation detection rates reach 70-90% in clinically diagnosed probands, approximately 10-30% of phenotypically confirmed MEN-1 families test negative by conventional sequencing, suggesting possible regulatory region defects, deep intronic mutations, or mosaic variants. In cases where MEN1 germline testing is negative despite a clinical MEN-1 phenotype, somatic mosaicism should be considered. We investigated a familial cohort presenting with primary hyperparathyroidism, multifocal pancreatic and pituitary neuroendocrine neoplasms - a triad strongly suggestive of MEN-1. Using a multi-tissue sequencing approach, we analyzed DNA extracted from peripheral blood leukocytes and parathyroid adenomas tissue via both Sanger sequencing and next-generation sequencing (NGS) with high coverage. While conventional Sanger analysis failed to detect a mutation, targeted NGS revealed a novel, likely pathogenic MEN1 variant present at low allele frequency (5-15%), consistent with postzygotic mosaicism. The variant was classified as pathogenic per ACMG/AMP guidelines and correlated with disease manifestations in affected tissues. These findings demonstrate that high-coverage NGS of multiple tissues is critical for identifying low-level mosaic MEN1 mutations missed by standard testing. Alternative screening methods are required for patients with strong clinical indications of MEN-1 and/or a family history, but negative germline test results, one such method is NGS with high coverage.

#2

Increase in serum parathyroid hormone level intraoperatively after parathyroidectomy for primary hyperparathyroidism.

American journal of otolaryngology2025

To evaluate the significance of increased of intraoperative parathyroid hormone(IOPTH) 10 min after parathyroidectomy in primary hyperparathyroidism. All patients underwent parathyroidectomy were retrospectively included. Following the results of IOPTH, three groups were defined: Group 1: increased of IOPTH, Group 2: <50 % decreased of IOPTH, and Group 3: >50 % decreased of IOPTH. Unilateral approach was performed and shifted to bilateral neck exploration(BNE) when indicated. There were single adenoma, double adenomas, and hyperplasia in 84 %, 5 %, and 11 % of cases respectively. We noted that 100 %, 80 %, and 4 % of patients had a polyglandular diseases in Groups 1, 2, and 3 respectively. Double adenoma and hyperplasia were present in 55 %, and 45 %, 22 %, and 58 %, 1 %, and 3 % in Groups 1, 2, and 3 respectively. Female patients represented 55 %, 71 %, and 81 % of patients in Groups 1, 2, and 3 respectively. Cure rate was 99 %. Patients with increased of IOPTH level 10 min after parathyroidectomy had a polyglandular diseases in 100 % of cases and needed BNE.

#3

Paradoxical cerebral embolism caused by patent foramen ovale in a patient with multiple endocrine neoplasia type 1 and severe primary hyperparathyroidism.

BMJ case reports2024 Nov 24

A man in his late 30s with gait difficulty, dysarthria, impaired consciousness and polyuria was diagnosed with left thalamic infarction. Hypercalcaemia (3.52 mmol/L (2.15-2.52)), high intact-parathyroid hormone (i-PTH) levels (88.8 pmol/L (1.1-6.9)) and high D-dimer levels (14.7 mg/L (<1.0)) were identified, followed by a positive microbubble test on transesophageal echocardiogram, suggesting high-risk patent foramen ovale (PFO) for ischaemic stroke. Paradoxical cerebral embolism via PFO, complicated by a hypercoagulable state and hypercalcemic dehydration, was considered. Polyglandular parathyroid hyperplasia, plus radiolucent mandibular tumours, suggested multiple endocrine neoplasia type 1 (MEN1) or hyperparathyroidism-jaw tumour syndrome. Genetic testing confirmed MEN1. Treatment was 24 mg of oral evocalcet and total parathyroidectomy with forearm autotransplantation, resulting in improved serum calcium and i-PTH levels. Finally, he underwent transcatheter PFO closure. We emphasise careful, etiological pursuit in young-onset stroke and the usefulness of genetic testing in differentiating hyperparathyroidism associated with mandibular tumours.

#4

First proof of association between autoimmune polyglandular syndrome and multiple endocrine neoplasia in humans.

Endocrine journal2020 Sep 28

Autoimmune Addison's disease (AAD) is a rare condition occurring either in isolation or associated with other autoimmune diseases as part of an autoimmune polyglandular syndrome (APS) type 1, 2 or 4. Multiple endocrine neoplasia (MEN) type 1, 2 or 4 is a hereditary autosomal dominant cancer syndrome. Medullary thyroid carcinoma and pheochromocytoma are neoplasms common to MEN-2a and MEN-2b. We describe a unique, complex case of a man resulted affected by both APS-2 and MEN-2a. The patient developed Hashimoto's thyroiditis, diabetes mellitus type 1 and AAD, despite testing negative for adrenal cortex autoantibodies (ACA) and steroid 21-hydroxylase autoantibodies (21-OHAb). Moreover, he had also a family history for MEN-2a and he first developed medullay thyroid cancer, then bilateral pheochromocytoma on the adrenal substrate of an AAD. On adrenal histology we found complete bilateral cortical atrophy in the presence of a lymphocytic infiltration and fibrosis, confirming an ACA and 21-OHAb-negative AAD. This datum is the first documented in a living individual and confirms that the absence of autoantibodies is not incompatible with an autoimmune disease and confirms that AAD is a cell-mediated autoimmune disease limited to the adrenal cortex and sparing medullary. In the light of a literature review concerning the association between APS and MEN, this is the first proven case to be reported in humans. Finally, our findings suggest that adrenal medullary tumor can develop even on an adrenal gland with cortical atrophy due to autoimmune adrenalitis.

#5

Isolated adrenocorticotropic hormone deficiency and thyroiditis associated with nivolumab therapy in a patient with advanced lung adenocarcinoma: a case report and review of the literature.

Journal of medical case reports2019 Mar 26

Immune checkpoint inhibitors are a promising class of anticancer drugs. The clinical benefits afforded by immune checkpoint inhibitors can be accompanied by immune-related adverse events that affect multiple organs, and endocrine immune-related adverse events include thyroiditis and hypophysitis. Hypophysitis is less frequent and has a less severe clinical presentation in patients treated with other immune checkpoint inhibitors, such as nivolumab, pembrolizumab, and atezolizumab, than in those treated with ipilimumab. However, studies have described isolated adrenocorticotropic hormone deficiency cases associated with nivolumab, pembrolizumab, and atezolizumab therapy, most of which occurred during the course of immune checkpoint inhibitor therapy. We report a rare case of patient with isolated adrenocorticotropic hormone deficiency that occurred after nivolumab therapy. A 69-year-old Japanese woman with advanced lung adenocarcinoma developed painless thyroiditis with transient elevations of serum thyroid hormones during 3 months of cancer treatment with nivolumab and began thyroid hormone replacement therapy for subsequent primary hypothyroidism. Four months after nivolumab therapy was discontinued, she developed isolated adrenocorticotropic hormone deficiency; corticosteroid replacement therapy relieved her secondary adrenal insufficiency symptoms, such as anorexia and fatigue. Human leukocyte antigen typing revealed the presence of DRB1*04:05-DQB1*04:01-DQA1*03:03 and DRB1*09:01-DQB1*03:03-DQA1*03:02 haplotypes, which increase susceptibility to autoimmune polyendocrine syndrome associated with thyroid and pituitary disorders in the Japanese population. Our patient developed thyroiditis during cancer treatment with nivolumab and subsequently exhibited isolated adrenocorticotropic hormone deficiency 4 months after discontinuing the drug. Administration of nivolumab in combination with a genetic predisposition to polyglandular autoimmunity probably caused both the thyroiditis and hypophysitis, resulting in primary hypothyroidism and isolated adrenocorticotropic hormone deficiency, respectively, in our patient. The present case highlights the need for physicians to be aware that endocrine immune-related adverse events, including hypophysitis, can occur more than several months after discontinuing a drug.

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

  1. Case Report: Novel likely pathogenic MEN1 mosaic mutation in the family with MEN-1 syndrome.
    Frontiers in endocrinology· 2025· PMID 41323978mais citado
  2. Increase in serum parathyroid hormone level intraoperatively after parathyroidectomy for primary hyperparathyroidism.
    American journal of otolaryngology· 2025· PMID 39826328mais citado
  3. Paradoxical cerebral embolism caused by patent foramen ovale in a patient with multiple endocrine neoplasia type 1 and severe primary hyperparathyroidism.
    BMJ case reports· 2024· PMID 39581682mais citado
  4. First proof of association between autoimmune polyglandular syndrome and multiple endocrine neoplasia in humans.
    Endocrine journal· 2020· PMID 32475862mais citado
  5. Isolated adrenocorticotropic hormone deficiency and thyroiditis associated with nivolumab therapy in a patient with advanced lung adenocarcinoma: a case report and review of the&#xa0;literature.
    Journal of medical case reports· 2019· PMID 30909965mais citado

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  1. ORPHA:100094(Orphanet)
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  3. GARD:19766(GARD (NIH))
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  5. Busca completa no PubMed(PubMed)
  6. Q55785235(Wikidata)

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