• 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, and Stefan Schönland.
• Department of Hematology, UMC Utrecht Cancer Center, Utrecht, the Netherlands m.c.minnema@umcutrecht.nl.
• Haematologica. 2019 Nov 1; 104 (11): 2274-2282.

AbstractThis 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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