open access

Vol 53, No 3 (2022)
Original research article
Submitted: 2021-12-01
Accepted: 2022-03-21
Published online: 2022-06-05
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Treosulfan-based conditioning vs. low-dose busulfan-based conditioning for allogeneic hematopoietic stem cell transplantation: a cost-utility analysis in Poland

Paweł Kawalec1, Przemysław Holko1, George Bungey2, Owen Moseley2, Magdalena Żegleń3, Mirosław Markiewicz4
DOI: 10.5603/AHP.a2022.0022
·
Acta Haematol Pol 2022;53(3):191-200.
Affiliations
  1. Department of Nutrition and Drug Research, Institute of Public Health, Jagiellonian University Medical College, Kraków, Poland
  2. DRG (Part of Clarivate), London, United Kingdom
  3. Pain Research Group, Institute of Psychology, Jagiellonian University, Kraków, Poland
  4. Department of Hematology, Institute of Medical Sciences, College of Medical Sciences, University of Rzeszow, Rzeszów, Poland

open access

Vol 53, No 3 (2022)
ORIGINAL RESEARCH ARTICLE
Submitted: 2021-12-01
Accepted: 2022-03-21
Published online: 2022-06-05

Abstract

Introduction: In Poland, busulfan conditioning is used for allogeneic hematopoietic stem cell transplantation (allo-HSCT). Cost-utility analyses comparing alternative conditioning regimens in patients undergoing allo-HSCT have not been conducted so far. Material and methods: A United Kingdom-based partitioned survival model was adapted to the Polish setting to compare treosulfan to low-dose busulfan conditioning regimen from the public payer’s perspective in Poland. Patient characteristics, overall survival (OS), event-free survival (EFS), and the rate of adverse events were obtained from the randomized MC-FludT.14/L trial. Parametric survival models of up to 5 years (the cure threshold), with subsequent mortality defined using survival of the general population of Poland adjusted for cancer survivors, were used to extrapolate OS and EFS beyond the trial duration. Published utilities were adjusted for age using age-dependent general population utilities. The costs of treatment, adverse events, and inpatient/outpatient care were assessed via official remuneration schemes. Results: Treosulfan-based conditioning outperformed low-dose busulfan, i.e. it was more effective with incremental quality-adjusted life years (QALY) of 0.78 and less expensive by 1,139 PLN per patient over the lifetime horizon. Deterministic sensitivity analyses revealed treosulfan was highly cost-effective (i.e. incremental cost-utility ratio was lower than the gross domestic product per capita in Poland) compared to low-dose busulfan, if most uncertain parameters are changed or alternative scenarios are implemented. The probability of treosulfan being cost-effective with a threshold of 155,514 PLN was 99.6%. Conclusions: Compared to low-dose busulfan, treosulfan is a highly cost-effective conditioning regimen for allo-HSCT patients ineligible for standard conditioning regimens.

Abstract

Introduction: In Poland, busulfan conditioning is used for allogeneic hematopoietic stem cell transplantation (allo-HSCT). Cost-utility analyses comparing alternative conditioning regimens in patients undergoing allo-HSCT have not been conducted so far. Material and methods: A United Kingdom-based partitioned survival model was adapted to the Polish setting to compare treosulfan to low-dose busulfan conditioning regimen from the public payer’s perspective in Poland. Patient characteristics, overall survival (OS), event-free survival (EFS), and the rate of adverse events were obtained from the randomized MC-FludT.14/L trial. Parametric survival models of up to 5 years (the cure threshold), with subsequent mortality defined using survival of the general population of Poland adjusted for cancer survivors, were used to extrapolate OS and EFS beyond the trial duration. Published utilities were adjusted for age using age-dependent general population utilities. The costs of treatment, adverse events, and inpatient/outpatient care were assessed via official remuneration schemes. Results: Treosulfan-based conditioning outperformed low-dose busulfan, i.e. it was more effective with incremental quality-adjusted life years (QALY) of 0.78 and less expensive by 1,139 PLN per patient over the lifetime horizon. Deterministic sensitivity analyses revealed treosulfan was highly cost-effective (i.e. incremental cost-utility ratio was lower than the gross domestic product per capita in Poland) compared to low-dose busulfan, if most uncertain parameters are changed or alternative scenarios are implemented. The probability of treosulfan being cost-effective with a threshold of 155,514 PLN was 99.6%. Conclusions: Compared to low-dose busulfan, treosulfan is a highly cost-effective conditioning regimen for allo-HSCT patients ineligible for standard conditioning regimens.

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Keywords

cost-effectiveness, cost-utility, treosulfan, busulfan, hematopoietic stem cell transplantation

About this article
Title

Treosulfan-based conditioning vs. low-dose busulfan-based conditioning for allogeneic hematopoietic stem cell transplantation: a cost-utility analysis in Poland

Journal

Acta Haematologica Polonica

Issue

Vol 53, No 3 (2022)

Article type

Original research article

Pages

191-200

Published online

2022-06-05

Page views

1969

Article views/downloads

167

DOI

10.5603/AHP.a2022.0022

Bibliographic record

Acta Haematol Pol 2022;53(3):191-200.

Keywords

cost-effectiveness
cost-utility
treosulfan
busulfan
hematopoietic stem cell transplantation

Authors

Paweł Kawalec
Przemysław Holko
George Bungey
Owen Moseley
Magdalena Żegleń
Mirosław Markiewicz

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