Amyloidosis medical therapy

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Classification • Primary amyloidosis • Secondary amyloidosis • Familial amyloidosis • Wild-type (senile) amyloidosis • Cardiac amyloidosis • Beta-2 microglobulin related amyloidosis • Gelsolin related amyloidosis • Lysozyme amyloid related amyloidosis • Leucocyte cell-derived chemotaxin 2 related amyloidosis • Fibrinogen A alpha-chain associated amyloidosis
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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 is CyBorD, which consists of cyclophosphamide, bortezomib, and dexamethasone.

Therapy	Mechanism of Action	Dosing	Adverse Effects
Bortezomib	Reversibly inhibits the 26S proteasome, preventing recycling of proteins and inducing cell cycle arrest and apoptosis	Cycles 1-4: 1.3mg/m2 IV/SC on days 1, 4, 8, 11, 22, 25, 29, 32 Cycles 5-9: 1.3mg/m2 IV/SC on days 1, 8, 22, 29	Peripheral neuropathy, VZV reactivation, hepatic impairment, asthenia, diarrhea, nausea, constipation, arthralgia, edema, dizziness
Dexamethasone	Suppresses polymorphonuclear leukocytes Inhibits prostaglandins and proinflammatory cytokines Suppresses lymphocyte proliferation	40mg PO weekly	Infections, immunosuppression, bone loss, cataract formation, glaucoma, muscular atrophy
Melphalan	Inhibits DNA and RNA synthesis Crosslinks DNA and causes DNA replication failure	6mg PO daily for 2-3 weeks, OR 10mg PO daily for 7-10 days, OR 0.15mg/kg daily PO for 7 days, THEN 1-3mg or 0.05mg/kg PO daily after counts recover	Myelosuppression, nausea, vomiting, pulmonary fibrosis, stomatitis
Cyclophosphamide	Alkylating agent Crosslinks DNA and causes DNA replication failure	40-50mg/kg weekly	Myelosuppression, nausea, vomiting, hemorrhagic cystitis, secondary malignancies

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.