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Amiloidose AH
ORPHA:442582CID-10 · E85.9CID-11 · 5D00.YDOENÇA RARA

Amiloidose sistémica rara caracterizada pela agregação e deposição de fibrilhas amilóides compostas por fragmentos de cadeia pesada de imunoglobulina monoclonal, geralmente produzida por uma neoplasia de células plasmáticas. As fibrilas amilóides depositam-se em vários órgãos, em particular nos rins. Habitualmente afeta doentes mais idosos e o quadro clínico inclui sinais e sintomas de disfunção renal, progredindo por vezes para uma síndrome nefrótica e doença renal em estágio terminal. O envolvimento cardíaco, hepático e neuronal também foi descrito.

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Introdução

O que você precisa saber de cara

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Amiloidose sistêmica rara descrita pela agregação e deposição de fibrilhas amilóides compostas por fragmentos de cadeia pesada de imunoglobulina monoclonal, geralmente produzida por uma neoplasia de células plasmáticas. As fibrilas amilóides depositam-se em vários órgãos, em particular nos enxaguatórios. Habitualmente afeta pacientes mais idosos e o quadro clínico inclui sinais e sintomas de disfunção renal, progredindo por vezes para uma síndrome nefrótica e doença renal em estágio terminal. O envolvimento cardíaco, hepático e neuronal também foi descrito.

Pesquisas ativas
1 ensaio
1 total registrados no ClinicalTrials.gov
Publicações científicas
22 artigos
Último publicado: 2025 Nov

Escala de raridade

CLASSIFICAÇÃO ORPHANET · BRASIL 2024
<1 / 1 000 000
Ultra-rara
<1/50k
Muito rara
1/20k
Rara
1/10k
Pouco freq.
1/5k
Incomum
1/2k
Prevalência
0.0
Worldwide
Casos conhecidos
12
pacientes catalogados
Início
Neonatal
🏥
SUS: Cobertura mínimaScore: 20%
1 medicamentos CEAFCID-10: E85.9
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Entender a doença

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Sinais e sintomas

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

Partes do corpo afetadas

🫘
Rins
2 sintomas
❤️
Coração
1 sintomas
🧠
Neurológico
1 sintomas

+ 4 sintomas em outras categorias

Características mais comuns

90%prev.
Nefropatia
Muito frequente (99-80%)
55%prev.
Paraproteinemia
Frequente (79-30%)
55%prev.
Amiloidose cardíaca
Frequente (79-30%)
55%prev.
Bócio
Frequente (79-30%)
55%prev.
Aumento da concentração circulante de NT-proBNP
Frequente (79-30%)
55%prev.
Amiloidose de nervos periféricos
Frequente (79-30%)
8sintomas
Muito frequente (1)
Frequente (6)
Ocasional (1)

Os sintomas variam de pessoa para pessoa. Abaixo estão as 8 características clínicas mais associadas, ordenadas por frequência.

NefropatiaNephropathy
Muito frequente (99-80%)90%
Paraproteinemia
Frequente (79-30%)55%
Amiloidose cardíacaCardiac amyloidosis
Frequente (79-30%)55%
BócioGoiter
Frequente (79-30%)55%
Aumento da concentração circulante de NT-proBNPIncreased circulating NT-proBNP concentration
Frequente (79-30%)55%

Linha do tempo da pesquisa

Publicações por ano — veja quando o interesse científico cresceu
Anos de pesquisa1desde 2025
Total histórico22PubMed
Últimos 10 anos13publicações
Pico20253 papers
Linha do tempo
2025Hoje · 2026🧪 2009Primeiro ensaio clínico
Publicações por ano (últimos 10 anos)

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Genética e causas

O que está alterado no DNA e como passa nas famílias

🧬

Nenhum gene associado encontrado

Os dados genéticos desta condição ainda estão sendo catalogados.

Diagnóstico

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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

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Pesquisa ativa

Ensaios clínicos abertos e novidades científicas recentes

🟢 Recrutando agora

1 pesquisa recrutando participantes. Converse com seu médico sobre a possibilidade de participar.

Outros ensaios clínicos

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Publicações mais relevantes

Timeline de publicações
13 papers (10 anos)
#1

Heavy Chains, Heavy Consequences: A Case of Concomitant Heavy Chain Amyloidosis and Heavy Chain Deposition Disease.

Kidney medicine2025 Nov

Heavy chain (AH) amyloidosis is a rare form of primary systemic amyloidosis that predominantly affects the kidneys and can lead to nephrotic syndrome. It is marked by the deposition of amyloid fibrils derived from immunoglobulin (Ig)-heavy chains. Monoclonal immunoglobulin deposition disease is a similarly rare disorder involving deposition of nonfibrillar and Congo red-negative monotypic Ig molecules in basement membranes. It can be derived from Ig light chains, Ig heavy chains, or Ig light and heavy chains. Cases of combined amyloidosis and monoclonal immunoglobulin deposition disease are exceedingly rare. Only a handful of concomitant amyloidosis and heavy chain deposition disease have been previously reported, and the spectrum of such diagnoses is poorly described. We describe a case of concomitant IgG4-type AH amyloidosis and heavy chain deposition disease in an 83-year-old man with a history of Agent Orange exposure who developed nephrotic syndrome resulting in diuretic-resistant anasarca. A subsequent bone marrow biopsy demonstrated a λ-restricted plasma cell population. The patient was initiated on hemodialysis and chemotherapy, resulting in clinical stabilization. This case report also contrasts AH amyloidosis with other forms of amyloidosis, emphasizing its unique clinical features, diagnostic challenges, and management considerations.

#2

An atypical cause of amyloidosis: a case of combined heavy and light chain amyloidosis.

BMC nephrology2025 Jul 01

The International Society of Amyloidosis recognizes 15 types of kidney-related amyloidosis, with most studies identifying immunoglobulin light chain (AL) amyloidosis as the leading cause in over half of these cases. Heavy chain (AH) amyloidosis has been reported in 0.1-2.3% of kidney amyloidosis cases. This case is particularly novel due to the extremely rare occurrence of combined heavy and light chain (AHL) amyloidosis and its uncommon outcome. A 78-year-old man with hypertension and hyperlipidemia presented with swelling in both the upper and lower extremities, accompanied by dyspnea. On examination, he had crackles in both lung fields on auscultation. His abdomen was distended, and his upper and lower extremities were noted with + 3 edema. Workup revealed a serum creatinine of 1.25 mg/dL, and serum albumin of 2.7 g/dL, 22 g of proteinuria in a 24-hour collection, high lambda light chains at 3,800 mg/L with kappa/lambda ratio of 0.01, and elevated IgM levels at 3,500 mg/L. A kidney biopsy showed mesangial expansion with negative periodic-acid-Schiff and positive Congo Red Stain with apple-green birefringence under polarized light. Immunofluorescence revealed diffuse mesangial staining for IgM (3+), C3 (2+), and lambda light chains (2+). Mass spectrometry confirmed lambda light chain and mu heavy chain amyloidosis. The patient was diagnosed with AHL amyloidosis with a precursor low-grade B-cell lymphoma with plasmacytic differentiation. Based on these findings, rituximab and bendamustine were started. The patient's condition continued to deteriorate, ultimately progressing to dialysis dependance. Two months after starting dialysis, he chose to transition to hospice care. Kidney AHL amyloidosis is extremely rare. Diagnosis requires a kidney biopsy and mass spectrometry to identify the amyloidogenic protein, given the highest sensitivity and specificity of this technique and the limitations of immunofluorescence. Compared to AL, AHL patients have a higher prevalence of IgM monoclonal gammopathy, longer overall survival (5-year survival rate of 66%), and less advanced disease. Although data are limited, our case underscores the potential rapid progression to dialysis in kidney AHL amyloidosis patients with high proteinuria at diagnosis.

#3

A case of IgG-type heavy chain amyloidosis with membranous nephropathy-like changes with long-term survival.

CEN case reports2025 Jun

We performed a kidney biopsy on a 68-year-old man with 2.6 g/day proteinuria. Immunofluorescence (IF) study showed a positive finding of IgG (IgG1) along the glomerular capillary wall, suggesting membranous nephropathy. However, electron microscopy showed no subepithelial electron dense deposits and amorphous deposits of 8-12 nm fibrillar structure, and positive DFS/Congo red staining confirmed the diagnosis of amyloidosis. Amyloid deposits were localized only in the glomeruli. No overt extrarenal amyloid lesions were identified. Serum and urine were positive for monoclonal IgG-lambda, but IF was negative for kappa and lambda, ruling out AL-amyloidosis. At 75 years of age, he underwent a second kidney biopsy, and the IgG-positive findings at IF became more pronounced, with progressive glomerular sclerosing lesions. A proteomic analysis was performed focusing on the positive IgG finding in IF. The amyloid was proven to be composed of fragments of a heavy chain variable region sequence. AH-amyloidosis was the final diagnosis. He was treated according to the treatment for AL-amyloidosis, but hemodialysis was started at the age of 81, and died at the age of 86. We report a valuable case of AH-amyloidosis with an observed long-term prognosis.

#4

A Case of Amyloid Goitre in Heavy Chain Amyloidosis: Diagnostic Challenges and Clinical Implications.

European journal of case reports in internal medicine2024

Immunoglobulin heavy chain amyloidosis (AH amyloidosis) is an extremely rare subtype of immunoglobulin-derived amyloidosis and there is limited literature on how to diagnose and manage this disorder. We describe a rare case of AH amyloidosis with amyloid goitre and the importance of mass spectrometry in the identification of the different types of amyloids. While additional studies are needed, several observations suggest important practical implications, including differences in clinical picture, prognosis, and pathologic diagnosis. Immunoglobulin heavy chain amyloidosis is an extremely rare subtype of immunoglobulin-derived amyloidosis and amyloid goitre is even rarer.There is limited literature on how to diagnose and manage this disorder.This case portrays one of these cases - one of the few existing in the literature - and reinforces the diagnostic complexity of this entity.

#5

A unique case of AH-dominant type nodular pulmonary amyloidosis presenting as a spontaneous pneumothorax: a case report and review of the literature.

Pathology oncology research : POR2023

Amyloidosis is a rare metabolic disorder primarily brought on by misfolding of an autologous protein, which causes its local or systemic deposition in an aberrant fibrillar form. It is quite rare for pulmonary tissue to be impacted by amyloidosis; of the three forms it can take when involving pulmonary tissue, nodular pulmonary amyloidosis is the most uncommon. Nodular pulmonary amyloidosis rarely induces clinical symptoms, and most often, it is discovered accidentally during an autopsy or via imaging techniques. Only one case of nodular pulmonary amyloidosis, which manifested as a spontaneous pneumothorax, was found in the literature. In terms of more precise subtyping, nodular amyloidosis is typically AL or mixed AL/AH type. No publications on AH-dominant type of nodular amyloidosis were found in the literature. We present a case of an 81 years-old male with nodular pulmonary AH-dominant type amyloidosis who presented with spontaneous pneumothorax. For a deeper understanding of the subject, this study also provides a review of the literature on cases with nodular pulmonary amyloidosis in relation to precise amyloid fibril subtyping. Since it is often a difficult process, accurate amyloid type identification is rarely accomplished. However, this information is very helpful for identifying the underlying disease process (if any) and outlining the subsequent diagnostic and treatment steps. Even so, it is crucial to be aware of this unit and make sure it is taken into consideration when making a differential diagnosis of pulmonary lesions.

Publicações recentes

Ver todas no PubMed

📚 EuropePMC8 artigos no totalmostrando 13

2025

Heavy Chains, Heavy Consequences: A Case of Concomitant Heavy Chain Amyloidosis and Heavy Chain Deposition Disease.

Kidney medicine
2025

An atypical cause of amyloidosis: a case of combined heavy and light chain amyloidosis.

BMC nephrology
2025

A case of IgG-type heavy chain amyloidosis with membranous nephropathy-like changes with long-term survival.

CEN case reports
2024

A Case of Amyloid Goitre in Heavy Chain Amyloidosis: Diagnostic Challenges and Clinical Implications.

European journal of case reports in internal medicine
2023

A unique case of AH-dominant type nodular pulmonary amyloidosis presenting as a spontaneous pneumothorax: a case report and review of the literature.

Pathology oncology research : POR
2021

Combined renal proximal tubulopathy and crystal storing histiocytosis in a patient with κ light chain multiple myeloma.

Pathologica
2021

Development of diagnostic antibodies against immunoglobulin heavy chain variable region for heavy chain amyloidosis (AH amyloidosis).

Pathology international
2020

Renal Involvement in Systemic Amyloidosis Caused by Monoclonal Immunoglobulins.

Hematology/oncology clinics of North America
2020

How We Manage Systemic Immunoglobulin Heavy Chain Amyloidosis (AH Amyloidosis) and Immunoglobulin Heavy-and-Light-Chain Amyloidosis (AH/AL Amyloidosis).

Clinical lymphoma, myeloma &amp; leukemia
2019

Monoclonal Immunoglobulin Deposition Disease and Related Diseases.

Journal of Nippon Medical School = Nippon Ika Daigaku zasshi
2019

A Case of T-Cell Large Granular Lymphocytic Leukemia and Renal Immunoglobulin Heavy Chain Amyloidosis.

The American journal of case reports
2018

Usefulness of gastroduodenal biopsy in the differential diagnosis of systemic AH amyloidosis from systemic AL amyloidosis.

Histopathology
2015

Renal AH Amyloidosis Associated With a Truncated Immunoglobulin Heavy Chain Undetectable by Immunostaining.

American journal of kidney diseases : the official journal of the National Kidney Foundation

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Doenças relacionadas

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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. Heavy Chains, Heavy Consequences: A Case of Concomitant Heavy Chain Amyloidosis and Heavy Chain Deposition Disease.
    Kidney medicine· 2025· PMID 41209163mais citado
  2. An atypical cause of amyloidosis: a case of combined heavy and light chain amyloidosis.
    BMC nephrology· 2025· PMID 40597878mais citado
  3. A case of IgG-type heavy chain amyloidosis with membranous nephropathy-like changes with long-term survival.
    CEN case reports· 2025· PMID 40021557mais citado
  4. A Case of Amyloid Goitre in Heavy Chain Amyloidosis: Diagnostic Challenges and Clinical Implications.
    European journal of case reports in internal medicine· 2024· PMID 38455688mais citado
  5. A unique case of AH-dominant type nodular pulmonary amyloidosis presenting as a spontaneous pneumothorax: a case report and review of the literature.
    Pathology oncology research : POR· 2023· PMID 37808084mais citado

Bases de dados e fontes oficiais

Identificadores e referências canônicas usadas para montar este verbete.

  1. ORPHA:442582(Orphanet)
  2. MONDO:0018613(MONDO)
  3. GARD:21847(GARD (NIH))
  4. Busca completa no PubMed(PubMed)
  5. Q55788217(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

Amiloidose AH
Compêndio · Raras BR

Amiloidose AH

ORPHA:442582 · MONDO:0018613
🇧🇷 Brasil SUS
CEAF
1ATafamidis
Geral
Prevalência
<1 / 1 000 000
Casos
12 casos conhecidos
Herança
Not applicable
CID-10
E85.9 · Amiloidose não especificada
CID-11
Ensaios
1 ativos
Início
Neonatal
Prevalência
0.0 (Worldwide)
MedGen
UMLS
C5204115
EuropePMC
Wikidata
Papers 10a
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