New and developing therapies for AL amyloidosis
Giulia Zumbo1, Omid Sadeghi-Alavijeh2, Philip N Hawkins1
1a National Amyloidosis Centre , University College London , London , UK.
Expert Opinion on Pharmacotherapy
|December 23, 2016
Summary
Systemic light-chain amyloidosis (AL) treatment focuses on suppressing the underlying plasma cell disorder. New therapies targeting amyloid deposits directly show promise for improved survival and organ function.
Area of Science:
- Hematology
- Oncology
- Immunology
Background:
- Systemic light-chain (AL) amyloidosis involves abnormal immunoglobulin light chain accumulation.
- Cardiac involvement significantly impacts prognosis and survival in AL amyloidosis.
- Diagnosis requires confirming amyloid deposition and fibril protein type.
Purpose of the Study:
- To review current treatments for systemic AL amyloidosis.
- To explore emerging drug targets and therapeutic strategies.
- To provide expert opinion on future directions in AL amyloidosis management.
Main Methods:
- Review of current treatment modalities including autologous stem cell transplantation (ASCT) and combination chemotherapy.
- Analysis of chemotherapy agents used: dexamethasone, melphalan, cyclophosphamide, thalidomide, bortezomib, lenalidomide, bendamustine.
- Discussion of treatment influenced by multiple myeloma clinical practice due to limited randomized controlled trials.
Main Results:
- Optimal outcomes depend on near-complete suppression of the underlying hematological disorder.
- Current treatments include ASCT and various chemotherapy combinations.
- Emerging therapies, particularly anti-amyloid antibodies, demonstrate significant potential.
Conclusions:
- Effective AL amyloidosis management necessitates aggressive treatment of the underlying plasma cell dyscrasia.
- Future therapeutic strategies are focused on directly targeting amyloid deposits.
- Advancements in anti-amyloid antibody development offer hope for improved patient survival and organ function.
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