Bortezomib-based induction followed by stem cell transplantation in light chain amyloidosis: results of the multicenter HOVON 104 trial
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Bortezomib-based induction followed by stem cell transplantation in light chain amyloidosis: results of the multicenter HOVON 104 trial. / Minnema, Monique C; Nasserinejad, Kazem; Hazenberg, Bouke; Hegenbart, Ute; Vlummens, Philip; Ypma, Paula F; Kröger, Nicolaus; Wu, Ka Lung; Kersten, Marie Jose; Schaafsma, M Ron; Croockewit, Sandra; de Waal, Esther; Zweegman, Sonja; Tick, Lidwien; Broijl, Annemieke; Koene, Harry; Bos, Gerard; Sonneveld, Pieter; Schönland, Stefan.
in: HAEMATOLOGICA, Jahrgang 104, Nr. 11, 11.2019, S. 2274-2282.Publikationen: SCORING: Beitrag in Fachzeitschrift/Zeitung › SCORING: Zeitschriftenaufsatz › Forschung › Begutachtung
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T1 - Bortezomib-based induction followed by stem cell transplantation in light chain amyloidosis: results of the multicenter HOVON 104 trial
AU - Minnema, Monique C
AU - Nasserinejad, Kazem
AU - Hazenberg, Bouke
AU - Hegenbart, Ute
AU - Vlummens, Philip
AU - Ypma, Paula F
AU - Kröger, Nicolaus
AU - Wu, Ka Lung
AU - Kersten, Marie Jose
AU - Schaafsma, M Ron
AU - Croockewit, Sandra
AU - de Waal, Esther
AU - Zweegman, Sonja
AU - Tick, Lidwien
AU - Broijl, Annemieke
AU - Koene, Harry
AU - Bos, Gerard
AU - Sonneveld, Pieter
AU - Schönland, Stefan
N1 - Copyright© 2019 Ferrata Storti Foundation.
PY - 2019/11
Y1 - 2019/11
N2 - 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).
AB - 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).
U2 - 10.3324/haematol.2018.213900
DO - 10.3324/haematol.2018.213900
M3 - SCORING: Journal article
C2 - 30923094
VL - 104
SP - 2274
EP - 2282
JO - HAEMATOLOGICA
JF - HAEMATOLOGICA
SN - 0390-6078
IS - 11
ER -
