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Risk-adjusted manipulation of melphalan dose before stem cell transplantation in patients with amyloidosis is associated with a lower response rate.

Authors
  • Gertz, M A
  • Lacy, M Q
  • Dispenzieri, A
  • Ansell, S M
  • Elliott, M A
  • Gastineau, D A
  • Inwards, D J
  • Micallef, I N M
  • Porrata, L F
  • Tefferi, A
  • Litzow, M R
Type
Published Article
Journal
Bone Marrow Transplantation
Publisher
Springer Nature
Publication Date
Dec 01, 2004
Volume
34
Issue
12
Pages
1025–1031
Identifiers
PMID: 15516945
Source
Medline
License
Unknown

Abstract

High-dose chemotherapy and autologous stem cell transplantation are used increasingly to treat patients with light-chain-related amyloidosis (AL). Treatment-related mortality is approximately 15%. To enable more patients to undergo stem cell transplantation, a risk-adapted strategy has been developed to treat with lower chemotherapy doses those patients who are at excessive risk. It is unclear whether reducing the chemotherapy dose in patients at excessive risk of treatment toxicity reduces the overall response. We retrospectively reviewed 171 AL patients who underwent conditioning chemotherapy with stem cell transplantation. The patients comprised two groups: those receiving standard high-dose melphalan and those receiving intermediate-dose melphalan. Responses were categorized as hematologic response, which used criteria for myeloma response. The two groups showed statistically significant differences; the overall response rates were 75% in the high-dose group and 53% in the intermediate-dose group although treatment-related mortality was the same in both groups. Reducing the melphalan dose appeared to render more AL patients eligible for stem cell transplantation but sacrificed an element of response. Methods are needed to reduce treatment-related toxicity so that more patients can receive full-dose conditioning chemotherapy.

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