Economic evaluation of treosulfan in patients undergoing allogeneic haematopoietic stem cell transplantation

Authors

  • Chiara Bini Faculty of Economics, Economic Evaluation and HTA (EEHTA), CEIS, University of Rome “Tor Vergata”, Rome - Italy https://orcid.org/0000-0001-5704-6542
  • Martina Paoletti Faculty of Economics, Economic Evaluation and HTA (EEHTA), CEIS, University of Rome “Tor Vergata”, Rome - Italy https://orcid.org/0000-0002-2564-6698
  • Andrea Marcellusi Faculty of Economics, Economic Evaluation and HTA (EEHTA), CEIS, University of Rome “Tor Vergata”, Rome - Italy https://orcid.org/0000-0002-3974-3798
  • Carlo Tomino Scientific Direction, IRCCS San Raffaele Roma, Rome - Italy https://orcid.org/0000-0003-1823-2195
  • Francesco Saverio Mennini Faculty of Economics, Economic Evaluation and HTA (EEHTA), CEIS, University of Rome “Tor Vergata”, Rome - Italy and Institute for Leadership and Management in Health – Kingston University London, London - UK

DOI:

https://doi.org/10.33393/grhta.2022.2412

Keywords:

Acute Myeloid Leukemia, Myelodysplastic Syndrome, alloHSCT, Treosulfan

Abstract

Objective. To assess the cost-effectiveness and economic sustainability of treosulfan plus fludarabine compared with busulfan plus fludarabine as a conditioning treatment for malignant disease prior to allogeneic haematopoietic stem cell transplantation (alloHSCT) in adult patients in Italy.

Method. The two theoretical cohorts of patients aged ≥ 60 years with acute myeloid leukaemia (AML) and myelodysplastic syndrome (MDS) were pooled and followed over time using a partitioned survival model with cycles of 28 days. Patients can transition between a post-HSCT recovery/remission state (Event-Free Survival state, EFS state), a relapsed/progressed disease state, and a death state. A lifetime horizon for cost-effectiveness analysis and a 5-years’ time horizon for budget impact analysis were used. The perspective of the Italian National Health Service was adopted. Utility values were obtained from published sources. Costs included: drug acquisition, HSCT procedure, management and treatment of adverse reactions, graft-versus-host disease (GvHD) and health states, end of life treatment. Discounting of 3% per year was applied for both costs and outcomes according to Italian guidelines. Sensitivity was tested through both one-way and probabilistic analyses.

Results. Cost-effectiveness analysis showed that treosulfan is both more effective and less expensive compared with busulfan (+1.11 life-years, +0.96 quality-adjusted life-years per patient and -€ 41,784 per patient). On the side of economic sustainability, the introduction of treosulfan in the market could generate a cumulative decrement of the expense incurred by NHS of about -€ 179,174 over five years.

Conclusion. Treosulfan could represent a cost-effective and sustainable treatment alternative from the perspective of the NHS.

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Additional Files

Published

2022-10-05

How to Cite

1.
Bini C, Paoletti M, Marcellusi A, Tomino C, Mennini FS. Economic evaluation of treosulfan in patients undergoing allogeneic haematopoietic stem cell transplantation. Grhta [Internet]. 2022 Oct. 5 [cited 2022 Dec. 6];9(1):105-16. Available from: https://journals.aboutscience.eu/index.php/grhta/article/view/2412

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