Melphalan and dexamethasone with or without bortezomib in newly diagnosed AL amyloidosis: A matched case-control study on 174 patients

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Abstract

Oral melphalan and dexamethasone (MDex) is a standard treatment for patients with AL amyloidosis who are not eligible for stem cell transplantation at many referral centers. However, following encouraging reports on the activity of bortezomib combined with alkylators and dexamethasone, these combinations are being moved to frontline therapy. We compared the outcome of 87 patients treated with bortezomib plus MDex (BMDex) with that of 87 controls treated with MDex. Patients and controls were matched for age, cardiac and renal function and free light chain burden. A higher rate of complete responses was observed with BMDex (42 vs 19%), but this did not result in a survival improvement in the overall population. However, a significant survival advantage for BMDex was observed in patients without severe (New York Heart Association class III or IV) heart failure and with N-terminal pro-natriuretic peptide type-B

Original languageEnglish
Pages (from-to)2311-2316
Number of pages6
JournalLeukemia
Volume28
Issue number12
DOIs
Publication statusPublished - Dec 11 2014

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Melphalan
Amyloidosis
Dexamethasone
Case-Control Studies
Survival
Brain Natriuretic Peptide
Alkylating Agents
Stem Cell Transplantation
Referral and Consultation
Heart Failure
Kidney
Light
Bortezomib
Therapeutics
Population

ASJC Scopus subject areas

  • Hematology
  • Cancer Research
  • Anesthesiology and Pain Medicine
  • Medicine(all)

Cite this

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title = "Melphalan and dexamethasone with or without bortezomib in newly diagnosed AL amyloidosis: A matched case-control study on 174 patients",
abstract = "Oral melphalan and dexamethasone (MDex) is a standard treatment for patients with AL amyloidosis who are not eligible for stem cell transplantation at many referral centers. However, following encouraging reports on the activity of bortezomib combined with alkylators and dexamethasone, these combinations are being moved to frontline therapy. We compared the outcome of 87 patients treated with bortezomib plus MDex (BMDex) with that of 87 controls treated with MDex. Patients and controls were matched for age, cardiac and renal function and free light chain burden. A higher rate of complete responses was observed with BMDex (42 vs 19{\%}), but this did not result in a survival improvement in the overall population. However, a significant survival advantage for BMDex was observed in patients without severe (New York Heart Association class III or IV) heart failure and with N-terminal pro-natriuretic peptide type-B",
author = "G. Palladini and P. Milani and A. Foli and Rosin, {M. Vidus} and M. Basset and F. Lavatelli and M. Nuvolone and L. Obici and S. Perlini and G. Merlini",
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T2 - A matched case-control study on 174 patients

AU - Palladini, G.

AU - Milani, P.

AU - Foli, A.

AU - Rosin, M. Vidus

AU - Basset, M.

AU - Lavatelli, F.

AU - Nuvolone, M.

AU - Obici, L.

AU - Perlini, S.

AU - Merlini, G.

PY - 2014/12/11

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AB - Oral melphalan and dexamethasone (MDex) is a standard treatment for patients with AL amyloidosis who are not eligible for stem cell transplantation at many referral centers. However, following encouraging reports on the activity of bortezomib combined with alkylators and dexamethasone, these combinations are being moved to frontline therapy. We compared the outcome of 87 patients treated with bortezomib plus MDex (BMDex) with that of 87 controls treated with MDex. Patients and controls were matched for age, cardiac and renal function and free light chain burden. A higher rate of complete responses was observed with BMDex (42 vs 19%), but this did not result in a survival improvement in the overall population. However, a significant survival advantage for BMDex was observed in patients without severe (New York Heart Association class III or IV) heart failure and with N-terminal pro-natriuretic peptide type-B

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