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.

Amyloidosis and Myeloma Unit Department of Hematology Hospital Clinic de Barcelona IDIBAPS Barcelona Spain

Amyloidosis Centre of Expertise Department of Internal Medicine Faculty of Medical Sciences University Medical Center Groningen Groningen Netherlands

Amyloidosis Research and Treatment Center Foundation Instituto di Ricovero e Cura a Carattere Scientifico Policlinico San Matteo Department of Molecular Medicine University of Pavia Pavia Italy

Clinical Hematology Centro Hospitalar São João Porto Portugal; i3S Instituto de Investigação e Inovação em Saúde University of Porto Porto Portugal; Cancer Drug Resistance Group IPATIMUP Institute of Molecular Pathology and Immunology of the University of Porto Porto Portugal; Clinical Hematology FMUP Faculty of Medicine University of Porto Porto Portugal

Department of Clinical Therapeutics National and Kapodistrian University of Athens School of Medicine Athens Greece

Department of Haematooncology University Hospital Ostrava and Department of Haematooncology Faculty of Medicine University of Ostrava Ostrava Czech Republic

Department of Hematology University Medical Center Utrecht Utrecht Netherlands

Department of Internal Medicine 1 Division of Oncology Medical University Vienna Vienna Austria

Erasmus MC Cancer Institute Rotterdam Netherlands

Health Data Specialists Dublin Ireland

Hematology Department Champalimaud Center for the Unknown Lisbon Portugal

Medical Department 5 Amyloidosis Center Heidelberg University of Heidelberg Heidelberg Germany

National Amyloidosis Center and Hematology Unit CHU Limoges Limoges France

National Amyloidosis Centre University College London London UK

Nephrology Unit CHU Poitiers Poitiers France

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