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Observational Study
. 2023 Jan 25;13(1):19.
doi: 10.1038/s41408-023-00789-8.

The management of light chain (AL) amyloidosis in Europe: clinical characteristics, treatment patterns, and efficacy outcomes between 2004 and 2018

Giovanni Palladini  1 , Stefan Schönland  2 , Giampaolo Merlini  1 , Paolo Milani  1 , Arnaud Jaccard  3 , Frank Bridoux  4 , Meletios A Dimopoulos  5 , Sriram Ravichandran  6 , Ute Hegenbart  2 , Wilfried Roeloffzen  7 , M Teresa Cibeira  8 , Hermine Agis  9 , Monique C Minnema  10 , Rui Bergantim  11 , Roman Hájek  12 , Cristina João  13 , Alexandros Leonidakis  14 , Giorgos Cheliotis  14 , Pieter Sonneveld  15 , Efstathios Kastritis  16 , Ashutosh Wechalekar  6
Affiliations
Observational Study

The management of light chain (AL) amyloidosis in Europe: clinical characteristics, treatment patterns, and efficacy outcomes between 2004 and 2018

Giovanni Palladini et al. Blood Cancer J. .

Abstract

Systemic light-chain (AL) amyloidosis is a rare and debilitating disease. Advances have been made in new treatments in recent years, yet real-world data on the management of the disease are scarce. EMN23 is a retrospective, observational study of patients who initiated first-line treatment in 2004-2018 in Europe, presenting the demographics, clinical characteristics, treatment patterns, and outcomes, from 4480 patients. Regimens based on bortezomib were the most frequently used as first-line therapy; only 6.2% of the patients received autologous stem cell transplant. Hematologic responses improved post-2010 (67.1% vs 55.6% pre-2010). The median overall survival (OS) was 48.8 (45.2-51.7) months; 51.4 (47.3-57.7) months pre-2010 and 46.7 (41.3-52.2) months post-2010. Early mortality was 13.4% and did not improve (11.4% vs 14.4% pre- and post-2010); furthermore, it remained high in patients with advanced cardiac disease (over 39% for stage IIIb). There was a significant improvement for stage IIIa (14.2 vs 30.7 months, p = 0.0170) but no improvement for stage IIIb patients (5.0 vs 4.5 months). This European real-world study of AL-amyloidosis emphasizes the unmet needs of early diagnosis, and the lack of improvement in survival outcomes of the frail stage IIIb population, despite the introduction of new therapies in recent years.

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Conflict of interest statement

GP reports participating in advisory boards for Alexion, Argobio, Janssen, and Protego; honoraria from Alexion, Argobio, Janssen, Protego, The Binding Site, Pfizer, Prothena, Sebia, and Siemens; research funding from Gate bioscience and The Binding Site. SS reports research support from Janssen, Prothena, and Sanofi; participating in advisory boards for Janssen, Telix, and Prothena; honoraria from Janssen, Takeda, Pfizer, and Prothena; travel and congress participation grants from Janssen, Prothena, Celgene, Binding Site, and Jazz. PM reports speaker honoraria from Janssen and Pfizer; participating in advisory board for Janssen. AJ reports honoraria from Servier, Amgen, Abbvie, and Pfizer; participating in advisory committees for Janssen; research funding from Sanofi and Janssen. FB reports lecture fees from Janssen, Astra Zeneca, GSK, and Novartis; consultancy for Janssen and Astra Zeneca. MAD reports honoraria from participation in advisory boards from Amgen, Takeda, Beigene, BMS, and Janssen. UH reports travel grants from Janssen, Prothena, and Pfizer; participating in advisory boards for Pfizer, Janssen, and Prothena; honoraria from Janssen, Pfizer, Alnylam, and Akcea; research funding from Janssen and Prothena. WR reports honoraria/advisory board from Janssen, MSD, Amgen, and Sanofi. MTC reports honoraria for lectures/advisory board for Janssen, Amgen, Akcea, and Sanofi. MCC reports honoraria from BMS; consultancy for Janssen-Cilag and Gilead; speaker’s bureau for Medscaoe. RH reports consultancy/advisory relationship with Janssen, Amgen, Celgene, Abbvie, BMS, Novartis, PharmaMar, and Takeda; honoraria from Janssen, Amgen, Celgene, BMS, PharmaMar, and Takeda; research funding from Janssen, Amgen, Celgene, BMS, Novartis, and Takeda. CJ reports scientific advisory for Takeda, BMS, Janssen, GSK, and Amgen; research funding from Takeda. AL reports current employment with Health Data Specialists. GC reports current employment with Health Data Specialists. PS reports research support from Janssen, BMS, Sanofi, and Amgen, all for EMN trials; participating in advisory boards for Janssen, BMS, Sanofi, Amgen, Cargen, and Pfizer. EK reports honoraria/advisory board from Genesis Pharma, Janssen, GSK, and Pfizer; research support from Amgen, Janssen, and Pfizer. AW reports honoraria/advisory board from Janssen, Alexion, Attralus, Celgene/BMS, Takeda, and GSK. All remaining authors have declared no conflicts of interest.

Figures

Fig. 1
Fig. 1. Kaplan–Meier graph of overall survival by first-line treatment initiation period (pre- and post-2010) and Mayo2004/European stage at diagnosis.
Stage I, Stage II, Stage IIIa and Stage IIIb represent the comparison of survival curves in the pre- and post-2010, separately for the respective patient subgroups defined by Mayo 2004/European stage at diagnosis.
Fig. 2
Fig. 2. Kaplan–Meier graph of OS by hematologic response at 3 months after first-line treatment initiation, 2004–2018.
CR complete response, PD progressive disease, PFS progression-free survival, PR partial response, SD stable disease, VGPR very good partial response.
See this image and copyright information in PMC

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