Attitudes about when and how to treat patients with AL amyloidosis: an international survey

Paolo Milani, Morie A. Gertz, Giampaolo Merlini, Angela Dispenzieri

Research output: Contribution to journalArticle

21 Scopus citations

Abstract

The aim of this survey was to describe the treatment decision making of expert physicians in when and how to treat patients with AL amyloidosis. Fifty amyloid expert physicians completed the survey. Autologous stem cell transplant (ASCT) was considered the first line therapy, if medically feasible, by 73% of the physicians. Excluding ASCT, cyclophosphamide-bortezomib-dexamethasone regimen was the preferred strategy by 72%. Depending on organ involvement, the goal for treatment was CR for 27–35% and very good partial response (VGPR) for 65–72%. In the absence of organ progression but rising FLC, the factors that most influenced when to reinstitute therapy included: dFLC at diagnosis (35.2%); how sick the patient was at diagnosis (24.1%); and time to FLC rise (18.5%). For patients who achieved CR after first-line, in the presence of cardiac/renal progression, 37/42% of providers would consider starting clone directed therapy without evidence of a clone. These data would imply that the current definitions of hematologic progression do not match clinical judgment, clinical experience and a comprehensive evaluation of patient status. These disparities challenge the ability to design therapeutic trials for patients with relapsed/refractory disease. A consensus statement with the definition and validation of new hematologic progression criteria is required.

Original languageEnglish (US)
Pages (from-to)213-216
Number of pages4
JournalAmyloid
Volume24
Issue number4
DOIs
StatePublished - Oct 2 2017

Keywords

  • Amyloidosis
  • prognosis
  • relapse
  • treatment

ASJC Scopus subject areas

  • Internal Medicine

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