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Bortezomib-based induction followed by stem cell transplantation in light chain amyloidosis: results of the multicenter HOVON 104 trial.

Authors
  • Minnema, Monique C1
  • Nasserinejad, Kazem2
  • Hazenberg, Bouke3
  • Hegenbart, Ute4
  • Vlummens, Philip5
  • Ypma, Paula F6
  • Kröger, Nicolaus7
  • Wu, Ka Lung8
  • Kersten, Marie Jose9
  • Schaafsma, M Ron10
  • Croockewit, Sandra11
  • de Waal, Esther12
  • Zweegman, Sonja13
  • Tick, Lidwien14
  • Broijl, Annemieke15
  • Koene, Harry16
  • Bos, Gerard17
  • Sonneveld, Pieter15
  • Schönland, Stefan4
  • 1 Department of Hematology, UMC Utrecht Cancer Center, Utrecht, the Netherlands [email protected] , (Netherlands)
  • 2 HOVON Data Center, Department of Hematology, Erasmus MC Cancer Institute, Rotterdam, the Netherlands. , (Netherlands)
  • 3 Department of Rheumatology & Clinical Immunology, University of Groningen Medical Center, Groningen, the Netherlands. , (Netherlands)
  • 4 Department of Hematology, Oncology and Rheumatology, Heidelberg University, Amyloidosis Center, Heidelberg, Germany. , (Germany)
  • 5 Department of Haematology, Ghent University, Gent, Belgium. , (Belgium)
  • 6 Department of Hematology, HagaZiekenhuis, Den Haag, the Netherlands. , (Netherlands)
  • 7 Department of Stem Cell Transplantation, University Medical Center Hamburg-Eppendorf, Hamburg, Germany. , (Germany)
  • 8 Department of Hematology, ZNA Stuivenberg, Antwerp, Belgium. , (Belgium)
  • 9 Department of Hematology, Academic Medical Center, Lymphoma and Myeloma Center, Amsterdam, the Netherlands. , (Netherlands)
  • 10 Department of Hematology, Medisch Spectrum Twente, Enschede, the Netherlands. , (Netherlands)
  • 11 Department of Hematology, Radboud University Medical Center, Nijmegen, the Netherlands. , (Netherlands)
  • 12 Department of Hematology, University of Groningen, Medical Center, Groningen, the Netherlands. , (Netherlands)
  • 13 Department of Hematology, VU University Medical Center, Amsterdam Cancer Center, Amsterdam, the Netherlands. , (Netherlands)
  • 14 Department of Internal Medicine, Maxima Medisch Centrum, Eindhoven, the Netherlands. , (Netherlands)
  • 15 Department of Hematology, Erasmus MC Cancer Institute, Rotterdam, the Netherlands. , (Netherlands)
  • 16 Department of Hematology, St. Antonius Hospital, Nieuwegein, the Netherlands. , (Netherlands)
  • 17 Department of Hematology, Maastricht University Medical Center, Maastricht, the Netherlands. , (Netherlands)
Type
Published Article
Journal
Haematologica
Publisher
Ferrata Storti Foundation
Publication Date
Nov 01, 2019
Volume
104
Issue
11
Pages
2274–2282
Identifiers
DOI: 10.3324/haematol.2018.213900
PMID: 30923094
Source
Medline
Language
English
License
Unknown

Abstract

This prospective, multicenter, phase II study investigated the use of four cycles of bortezomib-dexamethasone induction treatment, followed by high-dose melphalan and autologous stem cell transplantation (SCT) in patients with newly diagnosed light chain amyloidosis. The aim of the study was to improve the hematologic complete remission (CR) rate 6 months after SCT from 30% to 50%. Fifty patients were enrolled and 72% had two or more organs involved. The overall hematologic response rate after induction treatment was 80% including 20% CR and 38% very good partial remissions (VGPR). Fifteen patients did not proceed to SCT for various reasons but mostly treatment-related toxicity and disease-related organ damage and death (2 patients). Thirty-one patients received melphalan 200 mg/m2 and four patients a reduced dose because of renal function impairment. There were no deaths related to the transplantation procedure. Hematologic responses improved at 6 months after SCT to 86% with 46% CR and 26% VGPR. However, due to the high treatment discontinuation rate before transplantation the primary endpoint of the study was not met and the CR rate in the intention-to-treat analysis was 32%. Organ responses continued to improve after SCT. We confirm the high efficacy of bortezomib-dexamethasone treatment in patients with AL amyloidosis. However, because of both treatment-related toxicity and disease characteristics, 30% of the patients could not proceed to SCT after induction treatment. (Trial registered at Dutch Trial Register identifier NTR3220). Copyright© 2019 Ferrata Storti Foundation.

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