What is the role of medications in the treatment of cardiac amyloidosis?

Updated: Mar 25, 2020
  • Author: Gyanendra K Sharma, MD, FACC, FASE; Chief Editor: Terrence X O'Brien, MD, MS, FACC  more...
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The goals of pharmacotherapy include symptom relief, improved cardiac output, shortened hospital stay, fewer emergency department visits, and decreased mortality.

Because of their negative inotropic effects, rate-limiting calcium channel blocking agents (ie, verapamil, diltiazem) are contraindicated, as they can precipitate congestive heart failure.

Melphalan and prednisone have been used with limited benefit in cardiac amyloidosis. Better results may be obtained with the combination of melphalan and dexamethasone. Cyclophosphamide and thalidomide have also been used as an alternative therapy. Colchicine has also been used sometimes. Autologous stem cell transplantation and high-dose chemotherapy have shown promise in patients with limited cardiac involvement, with increased survival and better quality of life.

In a study that used a combination of cyclophosphamide/bortezomib/dexamethasone (CyBorD) to treat 230 patients with light chain (AL) amyloidosis, investigators found that the overall hematologic response rate was 60%. [77] Of the 201 patients with measurable disease, the overall hematologic response rate was 62%, with 43% achieving at least very good partial response (VGPR). [77]  In another study, use of triple therapy (bortezomib, dexamethasone, and an alkylating agent) in the treatment of patients with AL who presented with symptomatic heart failure resulted in improved survival after adjustment of clinical variables. [78]

Cardiac transplantation is not a good choice in patients with cardiac involvement because of increased mortality. [79, 80, 81, 82, 83, 84, 85]  A 2016 Mayo Clinic study showed a median overall survival of 3.5 years in 23 patients (median age, 53 years) with AL who received orthotopic heart transplantation. [86] Seven patients who achieved a complete hematologic response to either chemotherapy or autologous stem cell transplantation had a median survival of 10.8 years. [86]

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