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Primary Systemic Amyloidosis Treatment & Management

  • Author: Judit Nyirady, MD, MBA; Chief Editor: Dirk M Elston, MD  more...
Updated: May 19, 2016

Medical Care

The treatment of primary systemic amyloidosis (PSA) is directed toward the affected organ and the specific type of the disease. In studies of different regimens of intermittent oral melphalan and prednisone, Skinner et al and Kyle et al reported that the response rates were low, with an increased survival from a median of approximately 7-9 months in patients who did not receive chemotherapy to approximately 12-18 months in those receiving chemotherapy.[12, 22]

Shimojima et al reported a patient with primary systemic amyloidosis who achieved partial hematological response after 2 courses of the VAD (vincristine, doxorubicin [Adriamycin], and dexamethasone) chemotherapy regimen and subsequent high-dose melphalan followed by autologous peripheral blood stem cell transplantation despite involvement of multiple organs, including the heart.[23] When amyloidosis-related dysfunction is seen in multiple organs, intensive chemotherapy might be a possible therapeutic option, although several modifications in the regimen and careful management are necessary.

The nephrotic syndrome requires supportive therapy and diuretics, and renal failure can be successfully treated with dialysis.

Congestive heart failure may respond to diuretics, but larger doses are often required as the disease progresses. The use of calcium channel blockers, beta-blockers, and digoxin are contraindicated in cardiac amyloidosis, because they may cause toxicity at therapeutic levels.

Gastrointestinal involvement and neuropathy are treated symptomatically.



The presence of diffuse skin infiltrates and the loss of scalp hair can be distressing.

Macroglossia may be associated with painful dysphagia.

Cardiac involvement may cause angina, infarction, arrhythmias, or orthostatic hypotension.[24, 25]

Congestive heart failure or arrhythmias account for death in about 40% of the cases of primary systemic amyloidosis.

Blood vessel infiltration may lead to claudication of the legs or jaw. A case of temporal arteritis also has been described.[26]

Amyloid infiltration of the gastrointestinal tract may result in hemorrhage that can cause malabsorption or even death.[27]

Contributor Information and Disclosures

Judit Nyirady, MD, MBA Adjunct Assistant Professor, Department of Dermatology, Rutgers New Jersey Medical School

Judit Nyirady, MD, MBA is a member of the following medical societies: American Academy of Dermatology, International Society of Dermatology, European Academy of Dermatology and Venereology, Women's Dermatologic Society, Society for Investigative Dermatology

Disclosure: Received salary from Novartis Pharmaceuticals Corporation for employment.


Robert A Schwartz, MD, MPH Professor and Head of Dermatology, Professor of Pathology, Pediatrics, Medicine, and Preventive Medicine and Community Health, Rutgers New Jersey Medical School; Visiting Professor, Rutgers University School of Public Affairs and Administration

Robert A Schwartz, MD, MPH is a member of the following medical societies: Alpha Omega Alpha, New York Academy of Medicine, American Academy of Dermatology, American College of Physicians, Sigma Xi

Disclosure: Nothing to disclose.

Specialty Editor Board

David F Butler, MD Section Chief of Dermatology, Central Texas Veterans Healthcare System; Professor of Dermatology, Texas A&M University College of Medicine; Founding Chair, Department of Dermatology, Scott and White Clinic

David F Butler, MD is a member of the following medical societies: American Medical Association, Alpha Omega Alpha, Association of Military Dermatologists, American Academy of Dermatology, American Society for Dermatologic Surgery, American Society for MOHS Surgery, Phi Beta Kappa

Disclosure: Nothing to disclose.

Edward F Chan, MD Clinical Assistant Professor, Department of Dermatology, University of Pennsylvania School of Medicine

Edward F Chan, MD is a member of the following medical societies: American Academy of Dermatology, American Society of Dermatopathology, Society for Investigative Dermatology

Disclosure: Nothing to disclose.

Chief Editor

Dirk M Elston, MD Professor and Chairman, Department of Dermatology and Dermatologic Surgery, Medical University of South Carolina College of Medicine

Dirk M Elston, MD is a member of the following medical societies: American Academy of Dermatology

Disclosure: Nothing to disclose.

Additional Contributors

Takeji Nishikawa, MD Emeritus Professor, Department of Dermatology, Keio University School of Medicine; Director, Samoncho Dermatology Clinic; Managing Director, The Waksman Foundation of Japan Inc

Disclosure: Nothing to disclose.

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Amorphous eosinophilic interstitial amyloid observed on a renal biopsy.
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