Eligibility for hematopoietic stem-cell transplantation for primary systemic amyloidosis is a favorable prognostic factor for survival

Purpose: Based on the success of hematopoietic stem-cell transplantation (H SCT) for multiple myeloma, HSCT is being used to treat patients with primar y systemic amyloidosis (AL). This article addresses the extent to which eli gibility to undergo HSCT is a favorable prognostic feature and explores pro gnostic 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 h ave 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 less than or equal to 70 years , cardiac interventricular septal thickness less than or equal to 15 mm, ca rdiac ejection fraction more than 55%, serum creatinine less than or equal to 2 mg/dL, and direct bilirubin less than or equal to 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 pat ients have had a median follow-up of 52 months, and 151 have died. The medi an survival was 42 months with 5- and 10-year survival rates of 36% and 15% , respectively. Important predictors of survival were size of an-component in 24-hour urine, number of involved organs, alkaline phosphatase, performa nce scare, and weight loss. Conclusion: The same patients who are eligible for HSCT are a good-risk pop ulation who do relatively well with chemotherapy (median survival, 42 month s), substantially better than the expected median survival of 18 months for all patients with AL. A randomized trial is needed to assess the true effe ct of HSCT. (C) 2001 by American Society of Clinical Oncology.