Valutazione economica di treosulfan in pazienti sottoposti a trapianto allogenico di cellule staminali ematopoietiche

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 (+1.08 life-years, +0.95 quality-adjusted life-years per patient and –€ 41,388 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 –€ 212,063 over five years.

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

Fig. 1 -

Regimi di condizionamento comunemente usati in LMA e SMD sottoposti a TCSE. Dati CIBMTR 2007-2017 (4). Abbreviazioni: allo: allogeneic; bu: busulfan; cy: cyclophosphamide; flu: fludarabine; HSCT: haematopoietic stem cell transplantation; MAC: myeloablative conditioning; Mel: melphalan; RIC: reduced-intensity conditioning; TBI: total body irradiation.

Fig. 2 -

Schema di partitioned survival model per la coorte teorica di pazienti di età ≥ 60 anni con LMA e SMD post TCSE (13).

Fig. 3 -

Impatti cumulati sul budget per voce di costo.

Fig. 4 -

Impatti cumulati totali sul budget.

Fig. 5 -

Grafico tornado: Analisi di costo-efficacia.

Fig. 6 -

Curva di accettabilità di costo-efficacia.

Fig. 7 -

Grafico tornado: Analisi di impatto sul budget.