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

Monique C. Minnema, Kazem Nasserinejad, Bouke Hazenberg, Ute Hegenbart, Philip Vlummens, Paula F. Ypma, Nicolaus Kröger, Ka Lung Wu, Marie Jose Kersten, M. Ron Schaafsma, Sandra Croockewit, Esther de Waal, Sonja Zweegman, Lidwien Tick, Annemieke Broijl, Harry Koene, Gerard Bos, Pieter Sonneveld, Stefan Schönland

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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.
Original languageEnglish
Pages (from-to)2274-2282
Issue number11
Publication statusPublished - 2019

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