High-dose melphalan and ASCT, with its higher likelihood of inducing a hematologic response compared with conventional therapy, may be the treatment of choice in highly selected patients with good organ function. However, no randomized study has demonstrated that this results in improved survival. Two cycles of oral MP therapy do not significantly impact subsequent stem cell collection. Older age is not a contraindication to ASCT, but performance status should be carefully considered. Patients with cardiac dysfunction from amyloidosis have inferior survival, regardless of treatment with ASCT or conventional regimens.4
Treatment-associated mortality in patients undergoing ASCT for amyloidosis (even at the most experienced centers) is 10% to 15%, far in excess of what is reported for myeloma patients undergoing ASCT utilizing the same conditioning regimen.
- There have been no randomized trials demonstrating improved survival following ASCT versus conventional nontransplant therapy.
4. Lachmann HJ, Booth DR, Booth SE, et al. Misdiagnosis of hereditary amyloidosis as AL (primary) amyloidosis.
N Engl J Med 2002;346:1786-91.
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