Light-chain amyloidosis: SCT, novel agents and beyond
M Rosenzweig1, S Giralt, H Landau
1Department of Hematology and HCT, City of Hope National Cancer Center, Duarte, CA, USA.
Bone Marrow Transplantation
|October 30, 2012
Summary
Light-chain amyloidosis, a plasma cell disorder, requires specialized diagnosis and treatment. Novel therapies and clinical trials offer improved outcomes by targeting abnormal plasma cells and amyloid deposits.
Area of Science:
- Hematology
- Oncology
- Nephrology
Background:
- Light-chain amyloidosis is a plasma cell dyscrasia causing organ damage due to monoclonal light chain deposition.
- Diagnosis is complex, often necessitating biopsies and specialized tests for systemic disease confirmation.
Purpose of the Study:
- To review current and emerging treatment strategies for light-chain amyloidosis.
- To emphasize the importance of specialized centers and clinical trials for improving patient outcomes.
Main Methods:
- Review of standard treatments: high-dose melphalan with autologous hematopoietic stem cell transplant (SCT) or oral melphalan with dexamethasone.
- Evaluation of novel agents (thalidomide, lenalidomide, bortezomib) in combination therapies.
- Assessment of risk-adapted SCT followed by novel agent consolidation.
Main Results:
- Novel agents demonstrate efficacy in treating light-chain amyloidosis.
- Risk-adapted SCT with novel consolidation reduces treatment-related mortality.
- Immunotherapy targeting plasma cells and amyloid fibrils is under development.
Conclusions:
- Eradicating the monoclonal plasma cell population and suppressing pathologic light chains are key treatment goals.
- A risk-adapted approach and novel agents improve efficacy and reduce mortality.
- Further research into amyloidogenic light-chain biology and clinical trials is crucial.
![Dynamic Imaging of Chimeric Antigen Receptor T Cells with [18F]Tetrafluoroborate Positron Emission Tomography/Computed Tomography](/_next/image?url=https%3A%2F%2Fcloudfront.jove.com%2FCDNSource%2Fteasers%2F62334.jpg&w=3840&q=50)
Related Concept Videos
03:03Amyloid Fibrils
Amyloid fibrils are aggregates of misfolded proteins. Under most circumstances, misfolded proteins are either refolded by chaperone proteins or degraded by the proteasome. However, in the case of a mutation or a disease, these proteins can accumulate to form large clusters and often further assemble to form elongated fibers, called fibrils.
Amyloid deposits were observed as early as 1639 in the liver and the spleen. In 1854, Rudolph Virchow performed iodine staining, normally used to...
Amyloid deposits were observed as early as 1639 in the liver and the spleen. In 1854, Rudolph Virchow performed iodine staining, normally used to...
03:03Amyloid Fibrils
Amyloid fibrils are aggregates of misfolded proteins. Under most circumstances, misfolded proteins are either refolded by chaperone proteins or degraded by the proteasome. However, in the case of a mutation or a disease, these proteins can accumulate to form large clusters and often further assemble to form elongated fibers, called fibrils.
Amyloid deposits were observed as early as 1639 in the liver and the spleen. In 1854, Rudolph Virchow performed iodine staining, normally used to...
Amyloid deposits were observed as early as 1639 in the liver and the spleen. In 1854, Rudolph Virchow performed iodine staining, normally used to...
02:57Targeted Cancer Therapies
The targeted cancer therapies, also known as “molecular targeted therapies,” take advantage of the molecular and genetic differences between the cancer cells and the normal cells. It needs a thorough understanding of the cancer cells to develop drugs that can target specific molecular aspects that drive the growth, progression, and spread of cancer cells without affecting the growth and survival of other normal cells in the body.
There are several types of targeted therapies against specific...
There are several types of targeted therapies against specific...
01:22Alzheimer's Disease: Treatment
Alzheimer's Disease (AD), a neurodegenerative disorder, is pathologically identified by amyloid plaques and neurofibrillary tangles composed of tau protein. AD pharmacotherapy aims to manage cognitive symptoms, delay disease progression, and treat behavioral symptoms. The treatment is primarily symptomatic and palliative, with no definitive disease-modifying therapy available. Cholinesterase inhibitors, including donepezil (Aricept), rivastigmine (Exelon), and galantamine (Razadyne), are...
01:19Cushing Syndrome II: Pathophysiology
Cortisol production is normally governed by the hypothalamic–pituitary–adrenal (HPA) axis, which maintains hormonal balance through tightly regulated feedback mechanisms. Disruption of this regulatory system is central to the development of Cushing syndrome, whether the excess cortisol originates from external medications or internal pathology. Persistent cortisol elevation alters metabolism, immune function, and endocrine signaling, producing the characteristic clinical features of the...