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Bortezomib-based induction followed by stem cell transplantation in light chain amyloidosis: results of the multicenter HOVON 104 trial.
- Source :
-
Haematologica [Haematologica] 2019 Nov; Vol. 104 (11), pp. 2274-2282. Date of Electronic Publication: 2019 Mar 28. - Publication Year :
- 2019
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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/m <superscript>2</superscript> 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 ).<br /> (Copyright© 2019 Ferrata Storti Foundation.)
- Subjects :
- Aged
Biomarkers
Bortezomib administration & dosage
Bortezomib adverse effects
Combined Modality Therapy
Disease Progression
Female
Humans
Immunoglobulin Light-chain Amyloidosis diagnosis
Immunoglobulin Light-chain Amyloidosis mortality
Immunophenotyping
Male
Middle Aged
Severity of Illness Index
Transplantation, Autologous
Treatment Outcome
Bortezomib therapeutic use
Hematopoietic Stem Cell Transplantation adverse effects
Hematopoietic Stem Cell Transplantation methods
Immunoglobulin Light-chain Amyloidosis therapy
Subjects
Details
- Language :
- English
- ISSN :
- 1592-8721
- Volume :
- 104
- Issue :
- 11
- Database :
- MEDLINE
- Journal :
- Haematologica
- Publication Type :
- Academic Journal
- Accession number :
- 30923094
- Full Text :
- https://doi.org/10.3324/haematol.2018.213900