European cost-effectiveness study of uPA/PAI-1 biomarkers to guide adjuvant chemotherapy decisions in breast cancer.
This study investigated the cost effectiveness of guideline-recommended (American Society of Clinical Oncology, European Society of Medical Oncology) urokinase plasminogen activator (uPA)/plasminogen activator inhibitor-1 (PAI-1) biomarkers to guide adjuvant chemotherapy decisions for hormone receptor-positive, node-negative early breast cancer patients at intermediate risk of relapse, in France, Germany, and The Netherlands.



uPA/PAI-1 testing was compared to chemotherapy for all patients and to no chemotherapy in two age-related subgroups (35-49 and 50-75 years). A partitioned survival analysis was performed using patient-level data for survival outcomes and secondary sources. Mean quality-adjusted life years (QALYs) and costs were estimated over a lifetime horizon to calculate the incremental net monetary benefit (INMB) at a willingness-to-pay of €50,000/QALY. Uncertainty was explored through bootstrap and probabilistic sensitivity analysis using 5000 replicates.



In the 35-49 year age group, INMBs were negative when uPA/PAI-1 testing was compared to chemotherapy for all patients but positive when it was compared to no chemotherapy for the three countries. In the 50-75 year age group, INMBs of uPA/PAI-1 testing compared to both reference strategies were positive in the three countries, with cost-effectiveness probabilities for the uPA/PAI-1 strategy of 65%, 70%, and 59% for France, Germany, and the Netherlands, respectively, compared with chemotherapy for all patients, and 64%, 58%, and 65%, respectively, compared with no chemotherapy.



uPA/PAI-1 testing could allow the selection of patients older than 50 years requiring chemotherapy in this population, but the cost effectiveness of this strategy is uncertain. Chemotherapy for all patients is the most cost-effective strategy for patients younger than 50 years.
Gustave Roussy, Université Paris-Saclay, Service de biostatistique et d'épidémiologie, F-94805, Villejuif, France. Electronic address: sophie.marguet@gustaveroussy.fr.
Sophie
S
Marguet
Gustave Roussy, Université Paris-Saclay, Département de chirugie, F-94805, Villejuif, France.
Chafika
C
Mazouni
Department of Clinical Epidemiology and Medical Technology Assessment (KEMTA), Maastricht University Medical Centre, Maastricht, The Netherlands.
Bram L T
BL
Ramaekers
Gustave Roussy, Université Paris-Saclay, Service de biostatistique et d'épidémiologie, F-94805, Villejuif, France.
Ariane
A
Dunant
REK Consulting, Otterfing, Germany.
Ronald
R
Kates
Frauenklinik (OB/GYN), Paracelsus Medical University (PMU), Salzburg, Austria; Frauenklinik (OB/GYN), Technical University Munich (TUM), Munich, Germany.
Volker R
VR
Jacobs
Department of Clinical Epidemiology and Medical Technology Assessment (KEMTA), Maastricht University Medical Centre, Maastricht, The Netherlands; Department of Health Services Research, CAPHRI - School for Public Health and Primary Care, Maastricht University, Maastricht, The Netherlands.
Manuela A
MA
Joore
Breast Center, University of Munich, Munich, Germany.
Nadia
N
Harbeck
Gustave Roussy, Université Paris-Saclay, Service de biostatistique et d'épidémiologie, F-94805, Villejuif, France; Université Paris-Saclay, Univ. Paris-Sud, UVSQ, CESP, INSERM, F-94805, Villejuif, France.
Julia
J
Bonastre
D000328:Adult; D000368:Aged; D000971:Antineoplastic Combined Chemotherapy Protocols / Q000627:therapeutic use*; D014408:Biomarkers, Tumor / Q000032:analysis* / Q000191:economics; D001943:Breast Neoplasms / Q000175:diagnosis* / Q000188:drug therapy* / Q000191:economics; D017024:Chemotherapy, Adjuvant; D003362:Cost-Benefit Analysis; D005260:Female; D005602:France; D005858:Germany; D017048:Health Care Costs; D006801:Humans; D008875:Middle Aged; D009426:Netherlands; D017395:Plasminogen Activator Inhibitor 1 / Q000032:analysis* / Q000191:economics; D011788:Quality of Life; D016019:Survival Analysis; D014568:Urokinase-Type Plasminogen Activator / Q000032:analysis* / Q000191:economics
D014408:Biomarkers, Tumor; D017395:Plasminogen Activator Inhibitor 1; D014568:Urokinase-Type Plasminogen Activator
Adjuvant chemotherapy; Breast cancer; Cost effectiveness; Quality of life; uPA/PAI-1