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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 C.1
  • Nasserinejad, Kazem2
  • Hazenberg, Bouke3
  • Hegenbart, Ute4
  • Vlummens, Philip5
  • Ypma, Paula F.6
  • 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
  • 2 HOVON Data Center, Department of Hematology, Erasmus MC Cancer Institute, Rotterdam, the Netherlands
  • 3 Department of Rheumatology & Clinical Immunology, University of Groningen Medical Center, Groningen, the Netherlands
  • 4 Department of Hematology, Oncology and Rheumatology, Heidelberg University, Amyloidosis Center, Heidelberg, Germany
  • 5 Department of Haematology, Ghent University, Gent, Belgium
  • 6 Department of Hematology, HagaZiekenhuis, Den Haag, the Netherlands
  • 7 Department of Stem Cell Transplantation, University Medical Center Hamburg-Eppendorf, Hamburg, Germany
  • 8 Department of Hematology, ZNA Stuivenberg, Antwerp, Belgium
  • 9 Department of Hematology, Academic Medical Center, Lymphoma and Myeloma Center, Amsterdam, the Netherlands
  • 10 Department of Hematology, Medisch Spectrum Twente, Enschede, the Netherlands
  • 11 Department of Hematology, Radboud University Medical Center, Nijmegen, the Netherlands
  • 12 Department of Hematology, University of Groningen, Medical Center, Groningen, the Netherlands
  • 13 Department of Hematology, VU University Medical Center, Amsterdam Cancer Center, Amsterdam, the Netherlands
  • 14 Department of Internal Medicine, Maxima Medisch Centrum, Eindhoven, the Netherlands
  • 15 Department of Hematology, Erasmus MC Cancer Institute, Rotterdam, the Netherlands
  • 16 Department of Hematology, St. Antonius Hospital, Nieuwegein, the Netherlands
  • 17 Department of Hematology, Maastricht University Medical Center, Maastricht, the 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
PMCID: PMC6821610
Source
PubMed Central
License
Green

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 ).

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