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Amiloidose sistêmica primária
ORPHA:314701CID-10 · E85.0DOENÇA RARA

A proteína de Bence Jones é uma proteína monoclonal ou imunoglobulina de cadeia leve encontrado na urina, com um peso molecular de 22 a 24 kDa. A presença da proteína de Bence Jones na urina pode ser sugestiva de mieloma múltiplo ou Macroglobulinemia de Waldenstrom.

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Introdução

O que você precisa saber de cara

📋

Doença rara caracterizada pelo acúmulo de amiloide (proteína anormal) em órgãos e tecidos, levando a disfunção progressiva. Afeta múltiplos sistemas, como coração, rins e nervos, com sintomas variados.

Pesquisas ativas
17 ensaios
287 total registrados no ClinicalTrials.gov
Publicações científicas
521 artigos
Último publicado: 2026 Jan 13
Medicamentos
10 registrados
MELPHALAN, DEXAMETHASONE, ETANERCEPT

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10 medicamentos registrados
Ver detalhes, fases e interações →
MELPHALANDEXAMETHASONEETANERCEPTMELPHALAN HYDROCHLORIDETHALIDOMIDELENALIDOMIDEDOXYCYCLINEDEZAMIZUMABMIRIDESAPDOXORUBICIN

Escala de raridade

CLASSIFICAÇÃO ORPHANET · BRASIL 2024
1-5 / 10 000
Ultra-rara
<1/50k
Muito rara
1/20k
Rara
1/10k
Pouco freq.
1/5k
Incomum
1/2k
Prevalência
30.0
Europe
Herança
Not applicable
🏥
SUS: Cobertura mínimaScore: 20%
1 medicamentos CEAFCID-10: E85.0
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Sinais e sintomas

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

Linha do tempo da pesquisa

Publicações por ano — veja quando o interesse científico cresceu
Anos de pesquisa1desde 2026
Total histórico521PubMed
Últimos 10 anos66publicações
Pico202110 papers
Linha do tempo
2026Hoje · 2026🧪 1979Primeiro ensaio clínico📈 2021Ano 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

🧬

Nenhum gene associado encontrado

Os dados genéticos desta condição ainda estão sendo catalogados.

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.
3Fase 34
2Fase 214
1Fase 14
·Pré-clínico8
Medicamentos catalogadosEnsaios clínicos· 10 medicamentos · 20 ensaios
Carregando informações de tratamento...

Onde tratar no SUS

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

🇧🇷 Atendimento SUS — Amiloidose sistêmica primária

🗺️

Selecione um estado ou use sua localização para ver resultados.

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

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

Outros ensaios clínicos

287 ensaios clínicos encontrados, 17 ativos.

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

Timeline de publicações
65 papers (10 anos)
#1

Cutaneous Amyloidosis: An Updated Approach Focusing on Macular Amyloidosis.

Dermatology (Basel, Switzerland)2026 Jan 13

Amyloidosis is formed following deposition of protein aggregates and is classified by systemic or cutaneous deposition. These aggregates can be distributed in different organs such as the heart, liver, lungs, kidneys, and skin. Primary cutaneous amyloidosis has been classified into three groups: macular, lichen, and nodular, the former two being one often overlapping process, and the latter a localized plasma dyscrasia with a small risk of representing a systemic disease. Historically, cutaneous amyloidosis has been misdiagnosed, and most treatment regimens have been ineffective or only provide supportive management, such as decreasing pruritus. The current standard of care, high-potency corticosteroids, can provide symptomatic relief. Newer therapies may decrease amyloid deposition and progression of disease.

#2

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.

#3

Amyloid associated alopecia: A case report and review of the literature.

Dermatology online journal2025 Feb 15

Primary systemic amyloidosis is a condition marked by the extracellular deposition of amyloid proteins within various organ systems in the body. Although cutaneous involvement is well-described, scalp involvement in the form of alopecia is rarely reported. We report a case of amyloid associated alopecia confirmed by histologic analysis to highlight this rare scalp manifestation associated with systemic amyloidosis.

#4

Primary Systemic Amyloidosis Characterized by Multiple Keratinous Cysts: A Case Report.

Journal of cutaneous pathology2025 Mar

A 67-year-old male presented with plaques around the orbit and ecchymosis on the neck and back of hands for 2 years. Physical examination showed seborrheic-keratosis-like plaques around the orbit, ecchymosis on the neck and back of hands, as well as nail dystrophy. Serum λ light chain was positive. Histopathological examination demonstrated multiple keratinous cysts in the dermis and eosinophilic homogeneous material in the dermis, which was positive for Congo red with apple-green birefringence on polarized light. Kidney biopsy showed deposition of acellular eosinophilic material in the arteriole wall. Congo red staining was positive. A bone marrow biopsy revealed multiple myeloma. The diagnosis of primary systemic amyloidosis with multiple myeloma was made.

#5

Primary systemic amyloidosis: A brief overview.

Presse medicale (Paris, France : 1983)2025 Mar

Primary systemic amyloidosis, or light chain (AL) amyloidosis, is a rare lymphoproliferative disorder in which aberrant light-chain immunoglobulins secreted into the bloodstream aggregate into fibrils and deposit into tissues, causing widespread organ damage and, if not treated, death. This review provides a comprehensive summary of the pathophysiology and manifestations of AL amyloidosis; standard-of-care diagnostic approach; typical treatment regimens; and areas of active investigation.

Publicações recentes

Ver todas no PubMed

📚 EuropePMC333 artigos no totalmostrando 65

2026

Cutaneous Amyloidosis: An Updated Approach Focusing on Macular Amyloidosis.

Dermatology (Basel, Switzerland)
2025

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

Kidney medicine
2025

Amyloid associated alopecia: A case report and review of the literature.

Dermatology online journal
2025

Pinch Purpura: A Clinical Clue to Primary Systemic Amyloidosis.

Indian dermatology online journal
2025

Primary Systemic Amyloidosis Characterized by Multiple Keratinous Cysts: A Case Report.

Journal of cutaneous pathology
2025

Primary systemic amyloidosis: A brief overview.

Presse medicale (Paris, France : 1983)
2024

[MOTOR NEUROPATHY PRECEDING PRIMARY SYSTEMIC AMYLOIDOSIS].

Harefuah
2024

Primary Systemic Amyloidosis with Multisystem Involvement: A Case Report.

Indian dermatology online journal
2023

Primary Systemic Amyloidosis: A Case Report.

JNMA; journal of the Nepal Medical Association
2024

Spontaneous ecchymoses and conjunctival deposits in primary systemic amyloidosis.

Indian journal of dermatology, venereology and leprology
2023

Hepatic Involvement as the Sole Presentation of Systemic Amyloid Light Chain (AL) Amyloidosis: A Diagnostic Challenge.

Cureus
2023

Pinch Purpura in Adult Colloid Milium-A Case Report.

Indian dermatology online journal
2023

A Case of Multiple Myeloma-Associated Systemic Amyloidosis with Multiple Skin Manifestations as the First Symptom.

Clinical, cosmetic and investigational dermatology
2023

Cutis Verticis Gyrata Heralding a Diagnosis of Primary Systemic Amyloidosis in a Patient with Cardioembolic Stroke.

Annals of Indian Academy of Neurology
2023

Impact of second primary malignancy post-autologous transplantation on outcomes of multiple myeloma: a CIBMTR analysis.

Blood advances
2023

Plasma cell neoplasms and related entities-evolution in diagnosis and classification.

Virchows Archiv : an international journal of pathology
2022

Myocardial Uptake of 68Ga-Pentixafor in a Patient With Systemic Amyloidosis.

Clinical nuclear medicine
2022

Persistent diarrhea with petechial rash - unusual pattern of light chain amyloidosis deposition on skin and gastrointestinal biopsies: A case report.

World journal of clinical cases
2022

Primary Systemic Amyloidosis With Cardiac and Renal Involvement.

Cureus
2022

Macroglossia Associated With Primary Systemic Amyloidosis - Case Report With Unusual Clinical Presentation.

Journal of Ayub Medical College, Abbottabad : JAMC
2022

Primary Systemic Amyloidosis.

Journal of cutaneous medicine and surgery
2021

[Dermatological manifestations of monoclonal gammopathies].

Revista medica de Chile
2021

[Daratumumab for the treatment of primary systemic amyloidosis: a multicenter retrospective analysis].

Zhonghua nei ke za zhi
2021

Early Dermoscopic Signs Leading to Primary Systemic Amyloidosis' Diagnosis: A Case Report.

Dermatology practical &amp; conceptual
2021

Autopsy findings in a patient with primary systemic AL (kappa light chain) amyloidosis.

Autopsy &amp; case reports
2021

Non-variceal upper gastrointestinal bleed as first presentation of primary systemic amyloidosis - A case report.

International journal of health sciences
2020

Drooping shoulders: A rare manifestation of an uncommon disease.

The National medical journal of India
2021

Localized Immunoglobulin Light-Chain Amyloidosis of the Ulnar Nerve.

Case reports in neurology
2021

Hepatocyte Growth Factor and Primary Systemic Amyloidosis.

Journal of UOEH
2021

Usefulness of dermoscopy in identifying amyloid purpura.

The Journal of dermatology
2021

Spontaneous bilateral peri-orbital purpura: an important clinical sign of primary systemic amyloidosis.

BMJ case reports
2020

Peripheral Nervous System Involvement.

Hematology/oncology clinics of North America
2020

Traumatic Avulsion of Upper Eyelid Skin Following Surgery in a Patient With Multiple Myeloma and Amyloid Light-chain Amyloidosis.

Wound management &amp; prevention
2020

Cutaneous amyloidosis as the first presentation of Waldenstrom macroglobulinemia.

Caspian journal of internal medicine
2021

A Case of Primary Systemic Amyloidosis Involving the Sinonasal Tract.

Ear, nose, &amp; throat journal
2020

Primary Localized Conjunctival Amyloidosis Presenting Corneal Whorl-like Opacity Patterns.

Cornea
2020

Multiple myeloma with primary amyloidosis presenting with digestive symptoms: A case report and literature review.

Arab journal of gastroenterology : the official publication of the Pan-Arab Association of Gastroenterology
2019

Classic features of primary systemic amyloidosis (AL amyloidosis) leading to diagnosis of plasma cell myeloma.

Dermatology online journal
2019

Scientific Advances in and Clinical Approaches to Small-Fiber Polyneuropathy: A Review.

JAMA neurology
2020

Occult cardiac amyloidosis: the last chapter of a 2-year long story.

Forensic science, medicine, and pathology
2019

Primary Systemic Amyloidosis Mimicking Rhinophyma.

Indian dermatology online journal
2019

Amyloid-Associated Alopecia: A Reappraisal Including Its Pathophysiology.

The American Journal of dermatopathology
2018

Light chain cardiac amyloidosis - a rare cause of heart failure in a young adult.

Revista da Associacao Medica Brasileira (1992)
2019

Improved Treatment Related Mortality in Patients with Primary Systemic Amyloidosis (AL Amyloidosis) undergoing Autologous Hematopoietic Stem Cell Transplant (aHSCT).

Archives of hematology and blood diseases
2018

Thermoregulation in neuropathies.

Handbook of clinical neurology
2018

Suspected Pericardial Tuberculosis Revealed as an Amyloid Pericardial Mass.

Case reports in hematology
2019

Primary systemic amyloidosis in patients with Waldenström macroglobulinemia.

Leukemia
2018

Plasma ropivacaine concentration after TAP block in a patient with cardiac and renal failure.

Local and regional anesthesia
2018

Exceptional mucocutaneous manifestations with amyloid nephropathy: a case report.

Journal of medical case reports
2018

Cutis verticis gyrata - A rare presentation of Primary systemic Amyloidosis.

International journal of trichology
2018

Primary systemic amyloidosis with skin and cardiac involvement: a case report.

Acta dermatovenerologica Alpina, Pannonica, et Adriatica
2017

Multiple myeloma presenting with acquired cutis laxa and primary systemic amyloidosis.

European journal of dermatology : EJD
2017

Relapsing bullous amyloidosis of the oral mucosa and acquired cutis laxa in a patient with multiple myeloma: a rare triple association.

Clinical and experimental dermatology
2016

Primary systemic amyloidosis: imaging interpretation of this complex multisystemic disease.

BJR case reports
2016

[Hepatic amyloidosis as a rare differential diagnosis of progressive liver failure].

Deutsche medizinische Wochenschrift (1946)
2016

Primary Systemic Amyloidosis Involving the Lacrimal Sac.

Korean journal of ophthalmology : KJO
2016

Primary Systemic Amyloidosis with Unusual Dermatological Manifestations: A Rare Case Report.

Indian journal of dermatology
2016

Myeloma Associated Primary Systemic Amyloidosis.

Indian journal of dermatology
2016

[Primary localized cutaneous nodular amyloidosis: A diagnostic and therapeutic challenge].

Annales de dermatologie et de venereologie
2016

Oral purpura as the first manifestation of primary systemic amyloidosis.

The British journal of oral &amp; maxillofacial surgery
2015

Autologous Stem Cell Transplantation in Patients With Primary Systemic Amyloidosis: Experience of a Tertiary Hospital.

Transplantation proceedings
2015

Primary systemic amyloidosis initially presenting with digestive symptoms: a case report and review of the literature.

Diagnostic pathology
2016

Urinary retinol binding protein is a potential biomarker for renal function in primary systemic amyloidosis: A retrospective study.

Leukemia &amp; lymphoma
2015

Osteoprotegerin is a significant prognostic factor for overall survival in patients with primary systemic amyloidosis independent of the Mayo staging.

Blood cancer journal
2014

Bladder perforation secondary to primary systemic amyloidosis.

Case reports in urology
Ver todos os 333 no EuropePMC

Associações

Organizações que acompanham esta doença — pra ter apoio e orientação

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Comunidades

Grupos ativos de quem convive com esta doença aqui no Raras

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Doenças relacionadas

Doenças com sintomas parecidos — ajudam quem ainda está buscando diagnóstico

Ainda não achamos doenças com sintomas parecidos o suficiente.

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. Cutaneous Amyloidosis: An Updated Approach Focusing on Macular Amyloidosis.
    Dermatology (Basel, Switzerland)· 2026· PMID 41528921mais citado
  2. Heavy Chains, Heavy Consequences: A Case of Concomitant Heavy Chain Amyloidosis and Heavy Chain Deposition Disease.
    Kidney medicine· 2025· PMID 41209163mais citado
  3. Amyloid associated alopecia: A case report and review of the literature.
    Dermatology online journal· 2025· PMID 40526933mais citado
  4. Primary Systemic Amyloidosis Characterized by Multiple Keratinous Cysts: A Case Report.
    Journal of cutaneous pathology· 2025· PMID 39704222mais citado
  5. Primary systemic amyloidosis: A brief overview.
    Presse medicale (Paris, France : 1983)· 2025· PMID 39672504mais citado
  6. Pinch Purpura: A Clinical Clue to Primary Systemic Amyloidosis.
    Indian Dermatol Online J· 2025· PMID 39850707recente

Bases de dados e fontes oficiais

Identificadores e referências canônicas usadas para montar este verbete.

  1. ORPHA:314701(Orphanet)
  2. MONDO:0017816(MONDO)
  3. GARD:17431(GARD (NIH))
  4. Busca completa no PubMed(PubMed)
  5. Q7243179(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 sistêmica primária
Compêndio · Raras BR

Amiloidose sistêmica primária

ORPHA:314701 · MONDO:0017816
🇧🇷 Brasil SUS
CEAF
1ATafamidis
Geral
Prevalência
1-5 / 10 000
Herança
Not applicable
CID-10
E85.0 · Amiloidose heredofamiliar não-neuropática
Ensaios
17 ativos
Medicamentos
10 registrados
Prevalência
30.0 (Europe)
MedGen
UMLS
C0268380
EuropePMC
Wikidata
Papers 10a
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