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Healthcare Resource Utilization and Cost-of-Illness in Systemic Light Chain (AL) Amyloidosis in Europe: Results From the Real-World, Retrospective EMN23 Study

A. Jaccard, F. Bridoux, W. Roeloffzen, MC. Minnema, R. Bergantim, R. Hájek, C. João, MT. Cibeira, G. Palladini, S. Schönland, G. Merlini, P. Milani, MA. Dimopoulos, S. Ravichandran, U. Hegenbart, H. Agis, B. Gros, A. Asra, V. Magarotto, G....

. 2024 ; 24 (5) : e205-e216. [pub] 20240201

Jazyk angličtina Země Spojené státy americké

Typ dokumentu časopisecké články, práce podpořená grantem, pozorovací studie, multicentrická studie

Perzistentní odkaz   https://www.medvik.cz/link/bmc24014127

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.

Citace poskytuje Crossref.org

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$a OBJECTIVES: To report healthcare resource utilization (HCRU) and safety outcomes in systemic light chain (AL) amyloidosis from the EMN23 study. MATERIALS AND METHODS: The retrospective, observational, multinational EMN23 study included 4,480 patients initiating first-line treatment for AL amyloidosis in 2004-2018 and assessed, among other objectives, HCRU and safety outcomes. HCRU included hospitalizations, examinations, and dialysis; safety included serious adverse events (SAEs) and adverse events of special interest (AESIs). Data were descriptively analyzed by select prognostic factors (e.g., cardiac staging by Mayo2004/European) for 2004-2010 and 2011-2018. A cost-of-illness analysis was conducted for the UK and Spain. RESULTS: HCRU/safety and dialysis data were extracted for 674 and 774 patients, respectively. Of patients with assessed cardiac stage (2004-2010: 159; 2011-2018: 387), 67.9% and 61.0% had ≥ 1 hospitalization, 56.0% and 51.4% had ≥ 1 SAE, and 31.4% and 28.9% had ≥ 1 AESI across all cardiac stages in 2004-2010 and 2011-2018, respectively. The per-patient-per-year length of hospitalization increased with disease severity (cardiac stage). Of patients with dialysis data (2004-2010: 176; 2011-2018: 453), 23.9% and 14.8% had ≥ 1 dialysis session across all cardiac stages in 2004-2010 and 2011-2018, respectively. The annual cost-of-illness was estimated at €40,961,066 and €31,904,386 for the UK and Spain, respectively; dialysis accounted for ∼28% (UK) and ∼35% (Spain) of the total AL amyloidosis costs. CONCLUSIONS: EMN23 showed that the burden of AL amyloidosis is substantial, highlighting the need for early disease diagnosis and effective treatments targeting the underlying pathology.
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$a Bridoux, Frank $u CHU Poitiers, Nephrology Unit, Poitiers, France
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$a Roeloffzen, Wilfried $u Amyloidosis Centre of Expertise Department of Internal Medicine, Faculty of Medical Sciences, University Medical Center Groningen, Groningen, Netherlands
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$a Minnema, Monique C $u Department of Hematology, University Medical Center Utrecht, Utrecht, Netherlands
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$a Bergantim, Rui $u Department of Hematology, Hospital São João, Porto, Portugal
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$a Hájek, Roman $u Department of Haematooncology, University Hospital Ostrava, Ostrava, Czech Republic
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$a João, Cristina $u Department of Hematology, Hospital Clinic, IDIBAPS, Champalimaud Center for the Unknown, Lisbon, Portugal
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$a Cibeira, M Teresa $u Amyloidosis and Myeloma Unit, Department of Hematology, Hospital Clinic, IDIBAPS, Barcelona, Spain
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$a Palladini, Giovanni $u Department of Molecular Medicine, University of Pavia, Pavia, Italy; Amyloidosis Research and Treatment Center, Fondazione IRCCS Policlinico San Matteo, Pavia, Italy
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$a Schönland, Stefan $u Medical Department V, Amyloidosis Center Heidelberg, University of Heidelberg, Heidelberg, Germany
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$a Merlini, Giampaolo $u Department of Molecular Medicine, University of Pavia, Pavia, Italy; Amyloidosis Research and Treatment Center, Fondazione IRCCS Policlinico San Matteo, Pavia, Italy
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$a Milani, Paolo $u Department of Molecular Medicine, University of Pavia, Pavia, Italy; Amyloidosis Research and Treatment Center, Fondazione IRCCS Policlinico San Matteo, Pavia, Italy
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$a Dimopoulos, Meletios A $u Department of Clinical Therapeutics, National and Kapodistrian University of Athens, School of Medicine, Athens, Greece
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$a Ravichandran, Sriram $u National Amyloidosis Centre, University College London, London, United Kingdom
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$a Hegenbart, Ute $u CHU Poitiers, Nephrology Unit, Poitiers, France
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$a Agis, Hermine $u Department of Internal Medicine I, Division of Hematology & Hemostaseology, Medical University Vienna, Vienna, Austria
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$a Gros, Blanca $u Janssen-Cilag S.A., Madrid, Spain
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$a Asra, Aisha $u Janssen-Cilag, London, United Kingdom
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$a Magarotto, Valeria $u MD Janssen-Cilag, Rome, Italy
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$a Cheliotis, Giorgos $u Health Data Specialists, Dublin, Ireland
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$a Psarros, Giorgos $u Health Data Specialists, Dublin, Ireland
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$a Sonneveld, Pieter $u Erasmus University Medical Center, Rotterdam, Netherlands
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$a Wechalekar, Ashutosh $u National Amyloidosis Centre, University College London, London, United Kingdom
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$a Kastritis, Efstathios $u Department of Clinical Therapeutics, National and Kapodistrian University of Athens, School of Medicine, Athens, Greece. Electronic address: ekastritis@gmail.com
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