Amyloidosis medical therapy
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Editor-In-Chief: C. Michael Gibson, M.S., M.D. [1] Shyam Patel [2]; Associate Editor(s)-in-Chief:
Overview
There are few available treatments for primary amyloidosis. Since the disease is typically discovered at an advanced stage, the initial treatment is aimed at preventing further organ damage and correcting the effects of organ failure.
Medical Therapy
Some patients with primary amyloidosis respond to chemotherapy focused on the abnormal plasma cells. A stem cell transplant may be done, as in multiple myeloma.
- The initial target in the treatment of this disorder is to correct the organ failure, as the disease is discovered at an advanced stage.
- Nephrotic syndrome is treated using supportive therapy and diuretics.
- Renal failure is treated with dialysis.
- Heart failure is treated using diuretics.
- Gastrointestinal and nerve involvement are treated symptomatically.
The most commonly used regimen for AL amyloidosis is CyBorD, which consists of cyclophosphamide, bortezomib, and dexamethasone.[1]
Therapy | Mechanism of Action | Dosing | Adverse Effects |
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Bortezomib |
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Peripheral neuropathy, VZV reactivation, hepatic impairment, asthenia, diarrhea, nausea, constipation, arthralgia, edema, dizziness |
Dexamethasone |
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Infections, immunosuppression, bone loss, cataract formation, glaucoma, muscular atrophy |
Melphalan |
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Myelosuppression, nausea, vomiting, pulmonary fibrosis, stomatitis |
Cyclophosphamide |
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Myelosuppression, nausea, vomiting, hemorrhagic cystitis, secondary malignancies |
Patisiran[2] |
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Dyspepsia, dyspnea, erythema, bronchitis, blurry vision |
Daratumumab[3] |
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Anemia, neutropenia, false positive indirect Coomb's test, infusion reaction, lymphopenia |
Treatment options with limited success include melphalan, prednisone, and colchicine.
In secondary amyloidosis, aggressively treating the disease that is causing the excess amyloid protein can improve symptoms and/or slow the disease from getting worse. Complications such as heart failure, renal failure, and other problems can sometimes be treated, when needed.
References
- ↑ Milani P, Merlini G, Palladini G (2018). "Novel Therapies in Light Chain Amyloidosis". Kidney Int Rep. 3 (3): 530–541. doi:10.1016/j.ekir.2017.11.017. PMC 5976806. PMID 29854961.
- ↑ Adams D, Suhr OB, Dyck PJ, Litchy WJ, Leahy RG, Chen J; et al. (2017). "Trial design and rationale for APOLLO, a Phase 3, placebo-controlled study of patisiran in patients with hereditary ATTR amyloidosis with polyneuropathy". BMC Neurol. 17 (1): 181. doi:10.1186/s12883-017-0948-5. PMC 5594468. PMID 28893208.
- ↑ van de Donk NW, Janmaat ML, Mutis T, Lammerts van Bueren JJ, Ahmadi T, Sasser AK; et al. (2016). "Monoclonal antibodies targeting CD38 in hematological malignancies and beyond". Immunol Rev. 270 (1): 95–112. doi:10.1111/imr.12389. PMC 4755228. PMID 26864107.