Decision‐Making on First‐Line Treatment Alternatives in Chronic Lymphocytic Leukemia: A Multi‐Criteria Decision Analysis to Elicit Stakeholders' Preferences in Italy
8 affiliations
- 1Dipartmento di Medicina e Chirugia Centro di Ricerca Emato‐Oncologica (CREO) Universitá di Perugia Perugia Italy
- 2AIL Patient Groups Torino Italy
- 3BeOne Medicines Italy Srl Milan Italy
- 4IQVIA Solutions Italy Srl, Real World Solutions Milan Italy
- 5Department of Pharmaceutical Science University of Milan Milan Italy
- 6AxES IMT School for Advanced Studies Lucca Italy
- 7IUSS Pavia Italy
- 8Department of Medical Sciences University Hospital of Ferrara Ferrara Italy
Abstract
Chronic lymphocytic leukemia (CLL) is the most common form of leukemia in Western countries. The introduction of targeted therapies has expanded first‐line treatment options, which differ across multiple relevant dimensions, making therapeutic decisions increasingly challenging. A multi‐criteria decision analysis (MCDA) was conducted to identify and quantify the importance of key treatment selection criteria and to evaluate available first‐line alternatives for patients with mutated and unmutated immunoglobulin heavy‐chain variable region (IGHVmut/IGHVunmut) gene status, from the perspective of clinicians, patients, payers, and methodologists. The analysis focused on targeted therapies recommended by European Society for Medical Oncology (ESMO) guidelines and reimbursed in Italy, including ibrutinib, acalabrutinib, zanubrutinib, venetoclax + ibrutinib, and venetoclax+obinutuzumab. The MCDA was developed in accordance with International Society for Pharmacoeconomics and Outcomes Research (ISPOR) Good Practice recommendations, using the MACBETH (Measuring Attractiveness by a Categorical‐Based Evaluation Technique) method to elicit stakeholder preferences. Decision criteria were selected based on literature review, stakeholder input, and data availability. Preferences were collected from 20 stakeholders (nine clinicians, five patients, six payers/methodologists) via structured questionnaires. Six criteria were selected: efficacy, safety, quality of life (QoL), drug cost, convenience of administration, and treatment duration. Efficacy and safety emerged among the most important criteria across all groups, with efficacy particularly prioritized in the IGHVunmut population. Among the other criteria, notable differences emerged between stakeholder groups: clinicians assigned relatively greater importance to treatment duration, especially in the IGHVmut population; patients prioritized QoL more than other groups; payers and methodologists gave more weight to drug cost than clinicians and patients. In the IGHVmut population, venetoclax + ibrutinib, and zanubrutinib were the most valued options. In the IGHVunmut population, second‐generation Bruton tyrosine kinase inhibitors (zanubrutinib and acalabrutinib) ranked highest. Sensitivity analyses confirmed the robustness of these findings. This is the first MCDA study applied to CLL treatment in Italy. It highlights the value of structured, stakeholder‐informed approaches in supporting value‐based treatment decisions.
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BibTeX
@article{sportoletti2026decisionmaking,
title = {{Decision‐Making on First‐Line Treatment Alternatives in Chronic Lymphocytic Leukemia: A Multi‐Criteria Decision Analysis to Elicit Stakeholders' Preferences in Italy}},
author = {Paolo Sportoletti and Felice Bombaci and Chiara Cernetti and Alessandra Di Costanzo and Francesca Fiorentino and Sara Manurita and Andrea Marcellusi and Riccardo Mercati and Massimo Riccaboni and Chiara Vassallo and Antonio Cuneo},
journal = {Hematological Oncology},
year = {2026},
month = nov,
doi = {10.1002/hon.70274},
url = {https://doi.org/10.1002/hon.70274},
note = {CC BY 4.0},
}
RIS
TY - JOUR
TI - Decision‐Making on First‐Line Treatment Alternatives in Chronic Lymphocytic Leukemia: A Multi‐Criteria Decision Analysis to Elicit Stakeholders' Preferences in Italy
AU - Paolo Sportoletti
AU - Felice Bombaci
AU - Chiara Cernetti
AU - Alessandra Di Costanzo
AU - Francesca Fiorentino
AU - Sara Manurita
AU - Andrea Marcellusi
AU - Riccardo Mercati
AU - Massimo Riccaboni
AU - Chiara Vassallo
AU - Antonio Cuneo
PY - 2026
DA - 2026/11/05
JO - Hematological Oncology
DO - 10.1002/hon.70274
UR - https://doi.org/10.1002/hon.70274
AB - Chronic lymphocytic leukemia (CLL) is the most common form of leukemia in Western countries. The introduction of targeted therapies has expanded first‐line treatment options, which differ across multiple relevant dimensions, making therapeutic decisions increasingly challenging. A multi‐criteria decision analysis (MCDA) was conducted to identify and quantify the importance of key treatment selection criteria and to evaluate available first‐line alternatives for patients with mutated and unmutated immunoglobulin heavy‐chain variable region (IGHVmut/IGHVunmut) gene status, from the perspective of clinicians, patients, payers, and methodologists. The analysis focused on targeted therapies recommended by European Society for Medical Oncology (ESMO) guidelines and reimbursed in Italy, including ibrutinib, acalabrutinib, zanubrutinib, venetoclax + ibrutinib, and venetoclax+obinutuzumab. The MCDA was developed in accordance with International Society for Pharmacoeconomics and Outcomes Research (ISPOR) Good Practice recommendations, using the MACBETH (Measuring Attractiveness by a Categorical‐Based Evaluation Technique) method to elicit stakeholder preferences. Decision criteria were selected based on literature review, stakeholder input, and data availability. Preferences were collected from 20 stakeholders (nine clinicians, five patients, six payers/methodologists) via structured questionnaires. Six criteria were selected: efficacy, safety, quality of life (QoL), drug cost, convenience of administration, and treatment duration. Efficacy and safety emerged among the most important criteria across all groups, with efficacy particularly prioritized in the IGHVunmut population. Among the other criteria, notable differences emerged between stakeholder groups: clinicians assigned relatively greater importance to treatment duration, especially in the IGHVmut population; patients prioritized QoL more than other groups; payers and methodologists gave more weight to drug cost than clinicians and patients. In the IGHVmut population, venetoclax + ibrutinib, and zanubrutinib were the most valued options. In the IGHVunmut population, second‐generation Bruton tyrosine kinase inhibitors (zanubrutinib and acalabrutinib) ranked highest. Sensitivity analyses confirmed the robustness of these findings. This is the first MCDA study applied to CLL treatment in Italy. It highlights the value of structured, stakeholder‐informed approaches in supporting value‐based treatment decisions.
SN - 0278-0232
SN - 1099-1069
ER -