Second-line treatment for acute graft-versus-host disease with mesenchymal stromal cells: A decision model

Frederick W Thielen, Hedwig M Blommestein, Liesbeth E M Oosten, Friso G Calkoen, Arjan C Lankester, Jaap J Zwaginga, Katarina Le Blanc, Alba Redondo, Fermin Sánchez-Guijo, Mattia Algeri, Franco Locatelli, Wim E Fibbe, Carin A Uyl-de Groot

Research output: Contribution to journalArticle

Abstract

OBJECTIVE: No standard second-line treatment exists for acute graft-versus-host disease steroid-refractory (SR-aGvHD), and long-term outcomes remain poor. Mesenchymal stromal cells (MSCs) have been evaluated as treatment, but no disease model (DM) exists that integrates and extrapolates currently available evidence. The aim of this study was to develop such a DM to describe the natural history of SR-aGvHD and to predict long-term outcomes.

METHOD: The DM was developed in collaboration with experts in haematology-oncology. Subsequently, a model simulation was run. Input parameters for transition and survival estimates were informed by published data of clinical trials on MSC treatment for SR-aGvHD. Parametric distributions were used to estimate long-term survival rates after MSCs.

RESULTS: The newly developed DM is a cohort model that consists of eight health states. For the model simulation, we obtained data on 327 patients from 14 published phase II trials. Due to limited evidence, DM structure was simplified and several assumptions had to be made. Median overall survival was 3.2 years for complete response and 0.5 years for no complete response.

CONCLUSION: The DM provides a comprehensive overview on the second-line treatment pathway for aGvHD and enables long-term predictions that can be used to perform a cost-effectiveness analysis comparing any treatment for SR-aGvHD.

Original languageEnglish
JournalEuropean Journal of Haematology
DOIs
Publication statusE-pub ahead of print - Aug 6 2018

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Graft vs Host Disease
Mesenchymal Stromal Cells
Therapeutics
Survival
Hematology
Natural History
Cost-Benefit Analysis
Survival Rate
Steroids
Clinical Trials
Health

Cite this

Thielen, F. W., Blommestein, H. M., Oosten, L. E. M., Calkoen, F. G., Lankester, A. C., Zwaginga, J. J., ... Uyl-de Groot, C. A. (2018). Second-line treatment for acute graft-versus-host disease with mesenchymal stromal cells: A decision model. European Journal of Haematology. https://doi.org/10.1111/ejh.13158

Second-line treatment for acute graft-versus-host disease with mesenchymal stromal cells : A decision model. / Thielen, Frederick W; Blommestein, Hedwig M; Oosten, Liesbeth E M; Calkoen, Friso G; Lankester, Arjan C; Zwaginga, Jaap J; Le Blanc, Katarina; Redondo, Alba; Sánchez-Guijo, Fermin; Algeri, Mattia; Locatelli, Franco; Fibbe, Wim E; Uyl-de Groot, Carin A.

In: European Journal of Haematology, 06.08.2018.

Research output: Contribution to journalArticle

Thielen, FW, Blommestein, HM, Oosten, LEM, Calkoen, FG, Lankester, AC, Zwaginga, JJ, Le Blanc, K, Redondo, A, Sánchez-Guijo, F, Algeri, M, Locatelli, F, Fibbe, WE & Uyl-de Groot, CA 2018, 'Second-line treatment for acute graft-versus-host disease with mesenchymal stromal cells: A decision model', European Journal of Haematology. https://doi.org/10.1111/ejh.13158
Thielen, Frederick W ; Blommestein, Hedwig M ; Oosten, Liesbeth E M ; Calkoen, Friso G ; Lankester, Arjan C ; Zwaginga, Jaap J ; Le Blanc, Katarina ; Redondo, Alba ; Sánchez-Guijo, Fermin ; Algeri, Mattia ; Locatelli, Franco ; Fibbe, Wim E ; Uyl-de Groot, Carin A. / Second-line treatment for acute graft-versus-host disease with mesenchymal stromal cells : A decision model. In: European Journal of Haematology. 2018.
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abstract = "OBJECTIVE: No standard second-line treatment exists for acute graft-versus-host disease steroid-refractory (SR-aGvHD), and long-term outcomes remain poor. Mesenchymal stromal cells (MSCs) have been evaluated as treatment, but no disease model (DM) exists that integrates and extrapolates currently available evidence. The aim of this study was to develop such a DM to describe the natural history of SR-aGvHD and to predict long-term outcomes.METHOD: The DM was developed in collaboration with experts in haematology-oncology. Subsequently, a model simulation was run. Input parameters for transition and survival estimates were informed by published data of clinical trials on MSC treatment for SR-aGvHD. Parametric distributions were used to estimate long-term survival rates after MSCs.RESULTS: The newly developed DM is a cohort model that consists of eight health states. For the model simulation, we obtained data on 327 patients from 14 published phase II trials. Due to limited evidence, DM structure was simplified and several assumptions had to be made. Median overall survival was 3.2 years for complete response and 0.5 years for no complete response.CONCLUSION: The DM provides a comprehensive overview on the second-line treatment pathway for aGvHD and enables long-term predictions that can be used to perform a cost-effectiveness analysis comparing any treatment for SR-aGvHD.",
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T1 - Second-line treatment for acute graft-versus-host disease with mesenchymal stromal cells

T2 - A decision model

AU - Thielen, Frederick W

AU - Blommestein, Hedwig M

AU - Oosten, Liesbeth E M

AU - Calkoen, Friso G

AU - Lankester, Arjan C

AU - Zwaginga, Jaap J

AU - Le Blanc, Katarina

AU - Redondo, Alba

AU - Sánchez-Guijo, Fermin

AU - Algeri, Mattia

AU - Locatelli, Franco

AU - Fibbe, Wim E

AU - Uyl-de Groot, Carin A

N1 - © 2018 The Authors. European Journal of Haematology Published by John Wiley & Sons Ltd.

PY - 2018/8/6

Y1 - 2018/8/6

N2 - OBJECTIVE: No standard second-line treatment exists for acute graft-versus-host disease steroid-refractory (SR-aGvHD), and long-term outcomes remain poor. Mesenchymal stromal cells (MSCs) have been evaluated as treatment, but no disease model (DM) exists that integrates and extrapolates currently available evidence. The aim of this study was to develop such a DM to describe the natural history of SR-aGvHD and to predict long-term outcomes.METHOD: The DM was developed in collaboration with experts in haematology-oncology. Subsequently, a model simulation was run. Input parameters for transition and survival estimates were informed by published data of clinical trials on MSC treatment for SR-aGvHD. Parametric distributions were used to estimate long-term survival rates after MSCs.RESULTS: The newly developed DM is a cohort model that consists of eight health states. For the model simulation, we obtained data on 327 patients from 14 published phase II trials. Due to limited evidence, DM structure was simplified and several assumptions had to be made. Median overall survival was 3.2 years for complete response and 0.5 years for no complete response.CONCLUSION: The DM provides a comprehensive overview on the second-line treatment pathway for aGvHD and enables long-term predictions that can be used to perform a cost-effectiveness analysis comparing any treatment for SR-aGvHD.

AB - OBJECTIVE: No standard second-line treatment exists for acute graft-versus-host disease steroid-refractory (SR-aGvHD), and long-term outcomes remain poor. Mesenchymal stromal cells (MSCs) have been evaluated as treatment, but no disease model (DM) exists that integrates and extrapolates currently available evidence. The aim of this study was to develop such a DM to describe the natural history of SR-aGvHD and to predict long-term outcomes.METHOD: The DM was developed in collaboration with experts in haematology-oncology. Subsequently, a model simulation was run. Input parameters for transition and survival estimates were informed by published data of clinical trials on MSC treatment for SR-aGvHD. Parametric distributions were used to estimate long-term survival rates after MSCs.RESULTS: The newly developed DM is a cohort model that consists of eight health states. For the model simulation, we obtained data on 327 patients from 14 published phase II trials. Due to limited evidence, DM structure was simplified and several assumptions had to be made. Median overall survival was 3.2 years for complete response and 0.5 years for no complete response.CONCLUSION: The DM provides a comprehensive overview on the second-line treatment pathway for aGvHD and enables long-term predictions that can be used to perform a cost-effectiveness analysis comparing any treatment for SR-aGvHD.

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