# Amiloidose sistêmica primária

> Página oficial: https://raras.org/doenca/amiloidose-sistemica-primaria
> Fonte: Raras.org — Banco de Dados de Doenças Raras em Português (CC BY-NC-SA 4.0)
> Última atualização: 2026-05-07

## Identificadores

- **ORPHA**: 314701 — https://www.orpha.net/en/disease/detail/314701
- **CID-10**: E85.0

## Descrição clínica

Primary systemic amyloidosis (PSA) is a form of AL amyloidosis caused by the aggregation and deposition of insoluble amyloid fibrils derived from misfolded monoclonal immunoglobulin light chains usually produced by a plasma cell tumor and characterized by multiple organ involvement.

## Epidemiologia e herança

- **Prevalência**: 1-5 / 10 000
- **Padrão de herança**: Not applicable

## Medicamentos em desenvolvimento (10)

- MELPHALAN — Fase PHASE_3
- DEXAMETHASONE — Fase PHASE_3 (Glucocorticoid receptor agonist)
- ETANERCEPT — Fase PHASE_2 (TNF-alpha inhibitor)
- MELPHALAN HYDROCHLORIDE — Fase PHASE_2
- THALIDOMIDE — Fase PHASE_2 (CRL4(CRBN) E3 ubiquitin ligase inhibitor)
- LENALIDOMIDE — Fase PHASE_2 (CRL4(CRBN) E3 ubiquitin ligase modulator)
- DOXYCYCLINE — Fase PHASE_2 (Matrix metalloproteinase 8 inhibitor)
- DEZAMIZUMAB — Fase PHASE_1 (Serum amyloid P-component inhibitor)
- MIRIDESAP — Fase PHASE_1 (Serum amyloid P-component cross-linking agent)
- DOXORUBICIN — Fase PHASE_1 (DNA topoisomerase II alpha inhibitor)
- Fonte: https://platform.opentargets.org/disease/MONDO_0017816

## Ensaios clínicos ativos (17)

- **NCT05758493** [RECRUITING]: Characterizing Iodine-124 Evuzumitide (AT-01) in Systemic Amyloidosis — https://clinicaltrials.gov/study/NCT05758493
- **NCT07151690** [RECRUITING]: BCMA/CD3 Bispecific Antibody Treatment for Newly Diagnosed Amyloidosis — https://clinicaltrials.gov/study/NCT07151690
- **NCT07081646** [RECRUITING]: A Phase 1b/2 Study of CAR T Cell Therapy Targeting CD19 and BCMA in Participants With Relapsed or Refractory AL Amyloidosis. — https://clinicaltrials.gov/study/NCT07081646
- **NCT06192979** [RECRUITING]: Optimize First-line Treatment for AL Amyloidosis With t (11; 14) — https://clinicaltrials.gov/study/NCT06192979
- **NCT06627309** [RECRUITING]: Rapid dFLC Response Predict CHR in AL Amyloidosis — https://clinicaltrials.gov/study/NCT06627309
- **NCT07172243** [RECRUITING]: BE.Amycon Biobank & Data Registry UZ Leuven — https://clinicaltrials.gov/study/NCT07172243
- **NCT05145816** [RECRUITING]: Phase 1/2a Study of Belantamab Mafodotin in Relapsed or Refractory AL Amyloidosis — https://clinicaltrials.gov/study/NCT05145816
- **NCT07388602** [RECRUITING]: A Study to Evaluate the Safety and Efficacy of SCTC21C in Combination With Bortezomib, Cyclophosphamide, and Dexamethasone in Patients With Newly Diagnosed Systemic Light-Chain Amyloidosis (NDSLCA) — https://clinicaltrials.gov/study/NCT07388602
- **NCT07266116** [RECRUITING]: Assessment of the Efficacy and Safety of Injectable TQB2934 (Subcutaneous Injection) in Systemic Light Chain Amyloidosis Patients — https://clinicaltrials.gov/study/NCT07266116
- **NCT07039578** [RECRUITING]: Study Evaluating the Efficacy and Safety of CM336 Injection in the Treatment of Light-Chain Amyloidosis — https://clinicaltrials.gov/study/NCT07039578

## Importante

O Raras **não diagnostica e não prescreve**. Esta página é educativa e informativa.
Pacientes devem consultar profissionais de saúde qualificados para decisões clínicas.

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