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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. [ABSTRACT FROM AUTHOR]

Objectives: To report healthcare resource utilization (HCRU) and safety outcomes in systemic light chain (AL) amyloidosis from the EMN23 study., Materials and Methods: The retrospective, observational, multinational EMN23 study included 4,480 patients initiating first-line treatment for AL amyloidosis in 2004-2018 and assessed, among other objectives, HCRU and safety outcomes. HCRU included hospitalizations, examinations, and dialysis; safety included serious adverse events (SAEs) and adverse events of special interest (AESIs). Data were descriptively analyzed by select prognostic factors (e.g., cardiac staging by Mayo2004/European) for 2004-2010 and 2011-2018. A cost-of-illness analysis was conducted for the UK and Spain., Results: HCRU/safety and dialysis data were extracted for 674 and 774 patients, respectively. Of patients with assessed cardiac stage (2004-2010: 159; 2011-2018: 387), 67.9% and 61.0% had ≥ 1 hospitalization, 56.0% and 51.4% had ≥ 1 SAE, and 31.4% and 28.9% had ≥ 1 AESI across all cardiac stages in 2004-2010 and 2011-2018, respectively. The per-patient-per-year length of hospitalization increased with disease severity (cardiac stage). Of patients with dialysis data (2004-2010: 176; 2011-2018: 453), 23.9% and 14.8% had ≥ 1 dialysis session across all cardiac stages in 2004-2010 and 2011-2018, respectively. The annual cost-of-illness was estimated at €40,961,066 and €31,904,386 for the UK and Spain, respectively; dialysis accounted for ∼28% (UK) and ∼35% (Spain) of the total AL amyloidosis costs., Conclusions: EMN23 showed that the burden of AL amyloidosis is substantial, highlighting the need for early disease diagnosis and effective treatments targeting the underlying pathology., Competing Interests: Disclosure Arnaud Jaccard, honoraria from Servier, Amgen, Abbvie, and Pfizer; participating in advisory committees for Janssen; research funding from Sanofi and Janssen; Frank Bridoux, lecture fees from Janssen, Astra Zeneca, GSK, and Novartis; consultancy for Janssen, Attralus and Astra Zeneca; Wilfried Roeloffzen, honoraria/advisory board from Janssen, MSD, Amgen, and Sanofi; Monique C. Minnema, honoraria from BMS; consultancy for Janssen-Cilag, GSK, CDR life and Gilead; speaker's bureau for Medscape. Research support from Beigene; Rui Bergantim, honoraria/advisory board from Janssen, BMS, Amgen, Takeda and Pfizer; research funding from BMS and Amgen; Roman Hájek, 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; Cristina João, scientific advisory for Takeda, BMS, Janssen, GSK, and Amgen, Abbvie, Pfizer; M. Teresa Cibeira, honoraria for lectures/advisory board for Janssen, Amgen, Akcea, and Sanofi; Giovanni Palladini; 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; Stefan Schönland, 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; Paolo Milani, speaker honoraria from Janssen and Pfizer; participating in advisory board for Janssen; Meletios A. Dimopoulos, honoraria from participation in advisory boards from Abbvie, Amgen, Bristol Myers Squibb, Beigene Inc, GSK, Janssen, Karyopharm, Pfizer, Regeneron, Sanofi and Takeda; Ute Hegenbart, 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; Blanca Gros, current employment with Janssen; Aisha Asra, current employment with Janssen; Valeria Magarotto, current employment with Janssen; Giorgos Cheliotis, current employment with Health Data Specialists; Giorgos Psarros, current employment with Health Data Specialists; Pieter Sonneveld, research support from Janssen, BMS, Sanofi, and Amgen, all for EMN trials; participating in advisory boards for Janssen, BMS, Sanofi, Amgen, Cargen, and Pfizer; Ashutosh Wechalekar, honoraria/advisory board from Janssen, Alexion, Attralus, Celgene/BMS, Takeda, and GSK; Efstathios Kastritis, honoraria/advisory board from Genesis Pharma, Janssen, GSK, and Pfizer; research support from Amgen, Janssen, and Pfizer., (Copyright © 2024 Elsevier Inc. All rights reserved.)

Background: Patients with hematological malignancies (HM) are at high risk of mortality from SARS-CoV-2 disease 2019 (COVID-19). A better understanding of risk factors for adverse outcomes may improve clinical management in these patients. We therefore studied baseline characteristics of HM patients developing COVID-19 and analyzed predictors of mortality., Methods: The survey was supported by the Scientific Working Group Infection in Hematology of the European Hematology Association (EHA). Eligible for the analysis were adult patients with HM and laboratory-confirmed COVID-19 observed between March and December 2020., Results: The study sample includes 3801 cases, represented by lymphoproliferative (mainly non-Hodgkin lymphoma n = 1084, myeloma n = 684 and chronic lymphoid leukemia n = 474) and myeloproliferative malignancies (mainly acute myeloid leukemia n = 497 and myelodysplastic syndromes n = 279). Severe/critical COVID-19 was observed in 63.8% of patients (n = 2425). Overall, 2778 (73.1%) of the patients were hospitalized, 689 (18.1%) of whom were admitted to intensive care units (ICUs). Overall, 1185 patients (31.2%) died. The primary cause of death was COVID-19 in 688 patients (58.1%), HM in 173 patients (14.6%), and a combination of both COVID-19 and progressing HM in 155 patients (13.1%). Highest mortality was observed in acute myeloid leukemia (199/497, 40%) and myelodysplastic syndromes (118/279, 42.3%). The mortality rate significantly decreased between the first COVID-19 wave (March-May 2020) and the second wave (October-December 2020) (581/1427, 40.7% vs. 439/1773, 24.8%, p value < 0.0001). In the multivariable analysis, age, active malignancy, chronic cardiac disease, liver disease, renal impairment, smoking history, and ICU stay correlated with mortality. Acute myeloid leukemia was a higher mortality risk than lymphoproliferative diseases., Conclusions: This survey confirms that COVID-19 patients with HM are at high risk of lethal complications. However, improved COVID-19 prevention has reduced mortality despite an increase in the number of reported cases., (© 2021. The Author(s).)