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Eligibility for Hematopoietic Stem-Cell Transplantation for Primary Systemic Amyloidosis Is a Favorable Prognostic Factor for Survival

From the Division of Hematology and Internal Medicine, and Section of Biostatistics, Mayo Clinic, Rochester, MN.

Address reprint requests to Angela Dispenzieri, MD, Mayo Clinic, 200 First St SW, Rochester, MN 55905; email: dispenzieri.angela@ mayo.edu.

PURPOSE: Based on the success of hematopoietic stem-cell transplantation (HSCT) for multiple myeloma, HSCT is being used to treat patients with primary systemic amyloidosis (AL). This article addresses the extent to which eligibility to undergo HSCT is a favorable prognostic feature and explores prognostic factors within the subset of eligible patients.

PATIENTS AND METHODS: The Mayo Clinic amyloid database was queried for all patients with AL seen at the Mayo Clinic from 1983 through 1997 who would have been eligible for peripheral-blood stem-cell transplantation. Inclusion criteria included biopsy-proven amyloid, symptomatic disease, absence of a clinical diagnosis of multiple myeloma, age 70 years, cardiac interventricular septal thickness 15 mm, cardiac ejection fraction more than 55%, serum creatinine 2 mg/dL, and direct bilirubin 2.0 mg/dL.

RESULTS: Median age was 56 years (range, 25 to 70) with 79 (34%) older than 60 years. One hundred patients had early cardiac involvement; 41, hepatic involvement; 167, renal involvement; and 39, nerve involvement. The 229 patients have had a median follow-up of 52 months, and 151 have died. The median survival was 42 months with 5- and 10-year survival rates of 36% and 15%, respectively. Important predictors of survival were size of M-component in 24-hour urine, number of involved organs, alkaline phosphatase, performance score, and weight loss.

CONCLUSION: The same patients who are eligible for HSCT are a good-risk population who do relatively well with chemotherapy (median survival, 42 months), substantially better than the expected median survival of 18 months for all patients with AL. A randomized trial is needed to assess the true effect of HSCT.

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