Attitudes about when and how to treat patients with AL amyloidosis

an international survey

Paolo Milani, Morie Gertz, Giampaolo Merlini, Angela Dispenzieri

Research output: Contribution to journalArticle

11 Citations (Scopus)

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)1-4
Number of pages4
JournalAmyloid
DOIs
StateAccepted/In press - Aug 28 2017

Fingerprint

Amyloidosis
Physicians
Stem Cells
Therapeutics
Clone Cells
Transplants
Aptitude
Amyloid
Cyclophosphamide
Dexamethasone
Surveys and Questionnaires
Consensus
Decision Making
Kidney

Keywords

  • Amyloidosis
  • prognosis
  • relapse
  • treatment

ASJC Scopus subject areas

  • Internal Medicine

Cite this

Attitudes about when and how to treat patients with AL amyloidosis : an international survey. / Milani, Paolo; Gertz, Morie; Merlini, Giampaolo; Dispenzieri, Angela.

In: Amyloid, 28.08.2017, p. 1-4.

Research output: Contribution to journalArticle

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