Details

Autor(en) / Beteiligte

• Jaccard, Arnaud
• Bridoux, Frank
• Roeloffzen, Wilfried
• Minnema, Monique C.
• Bergantim, Rui
• Hájek, Roman
• João, Cristina
• Cibeira, M. Teresa
• Palladini, Giovanni
• Schönland, Stefan
• Merlini, Giampaolo
• Milani, Paolo
• Dimopoulos, Meletios A.
• Ravichandran, Sriram
• Hegenbart, Ute
• Agis, Hermine
• Gros, Blanca
• Asra, Aisha
• Magarotto, Valeria
• Cheliotis, Giorgos
• Psarros, Giorgos
• Sonneveld, Pieter
• Wechalekar, Ashutosh
• Kastritis, Efstathios

Titel

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

Ist Teil von

• Clinical lymphoma, myeloma and leukemia, 2024-05, Vol.24 (5), p.e205-e216

Ort / Verlag

United States: Elsevier Inc

Erscheinungsjahr

2024

Quelle

MEDLINE

Beschreibungen/Notizen

• •What is already known about the topic?Real-world evidence of healthcare resource utilization (HCRU) and treatment costs for patients initiating treatment for systemic light chain (AL) amyloidosis in Europe are sparse.•What does the paper add to existing knowledge?This is the first European study to assess the burden of AL-Amyloidosis on patients and healthcare systems over two periods (2004-2010 and 2011-2018) marked by the introduction of proteasome inhibitors in 2011. Our data can inform AL treating physicians and hospital administrators about the burden of the disease and resources required for the patient's treatment according to disease prognostic factors and provides for the first time, a European cost-of-illness estimate.•What insights does the paper provide for informing healthcare-related decision making?The annual cost per patient is 10-12 K mainly driven by dialyses, hospitalizations and treatment-related costs. Interventions towards early AL amyloidosis diagnosis (i.e., before irreversible organ damage, especially of the kidneys) and more effective treatments targeting the underlying disease pathology are needed to alleviate the patient burden and reduce the costs incurred by the healthcare systems. To report healthcare resource utilization (HCRU) and safety outcomes in systemic light chain (AL) amyloidosis from the EMN23 study. 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. 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. EMN23 showed that the burden of AL amyloidosis is substantial, highlighting the need for early disease diagnosis and effective treatments targeting the underlying pathology. The EMN23 study assessed the HCRU data for 674 European patients with systemic light chain (AL) amyloidosis, who initiated treatment between 2004 and 2018. We reported that AL amyloidosis has a substantial burden on healthcare systems and patients alike. Early disease diagnosis and availability of effective treatments are needed.