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

Frederick Thielen; Hedwig Blommestein; LEM Oosten; FG Calkoen; AC Lankester; JJ Zwaginga; K Le Blanc; A Redondo; F Sánchez?Guijo; M Algeri; F (Franco) Locatelli; WE Fibbe; Carin Uyl - de Groot

doi:10.1111/ejh.13158

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

Frederick Thielen, Hedwig Blommestein, LEM Oosten, FG Calkoen, AC Lankester, JJ Zwaginga, K Le Blanc, A Redondo, F Sánchez?Guijo, M Algeri, F (Franco) Locatelli, WE Fibbe, Carin Uyl - de Groot

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Abstract

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 language	English
Pages (from-to)	676-683
Number of pages	8
Journal	European Journal of Haematology
Volume	101
Issue number	5
DOIs	https://doi.org/10.1111/ejh.13158
Publication status	Published - 11 Jul 2018

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Thielen, F., Blommestein, H., 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, C. (2018). Second?line treatment for acute graft?versus?host disease with mesenchymal stromal cells: A decision model. European Journal of Haematology, 101(5), 676-683. https://doi.org/10.1111/ejh.13158

Thielen, Frederick ; Blommestein, Hedwig ; Oosten, LEM ; Calkoen, FG ; Lankester, AC ; Zwaginga, JJ ; Le Blanc, K ; Redondo, A ; Sánchez?Guijo, F ; Algeri, M ; Locatelli, F (Franco) ; Fibbe, WE ; Uyl - de Groot, Carin. / Second?line treatment for acute graft?versus?host disease with mesenchymal stromal cells: A decision model. In: European Journal of Haematology. 2018 ; Vol. 101, No. 5. pp. 676-683.

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title = "Second?line treatment for acute graft?versus?host disease with mesenchymal stromal cells: A decision model.",

abstract = "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.",

author = "Frederick Thielen and Hedwig Blommestein and LEM Oosten and FG Calkoen and AC Lankester and JJ Zwaginga and {Le Blanc}, K and A Redondo and F S{\'a}nchez?Guijo and M Algeri and Locatelli, {F (Franco)} and WE Fibbe and {Uyl - de Groot}, Carin",

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month = jul,

day = "11",

doi = "10.1111/ejh.13158",

language = "English",

volume = "101",

pages = "676--683",

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Thielen, F , Blommestein, H, 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, C 2018, 'Second?line treatment for acute graft?versus?host disease with mesenchymal stromal cells: A decision model.', European Journal of Haematology, vol. 101, no. 5, pp. 676-683. 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 ; Blommestein, Hedwig; Oosten, LEM; Calkoen, FG; Lankester, AC; Zwaginga, JJ; Le Blanc, K; Redondo, A; Sánchez?Guijo, F; Algeri, M; Locatelli, F (Franco); Fibbe, WE; Uyl - de Groot, Carin.

In: European Journal of Haematology, Vol. 101, No. 5, 11.07.2018, p. 676-683.

Research output: Contribution to journal › Article › Academic › peer-review

TY - JOUR

T1 - Second?line treatment for acute graft?versus?host disease with mesenchymal stromal cells: A decision model.

AU - Thielen, Frederick

AU - Blommestein, Hedwig

AU - Oosten, LEM

AU - Calkoen, FG

AU - Lankester, AC

AU - Zwaginga, JJ

AU - Le Blanc, K

AU - Redondo, A

AU - Sánchez?Guijo, F

AU - Algeri, M

AU - Locatelli, F (Franco)

AU - Fibbe, WE

AU - Uyl - de Groot, Carin

PY - 2018/7/11

Y1 - 2018/7/11

N2 - 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.

AB - 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.

UR - https://onlinelibrary.wiley.com/doi/full/10.1111/ejh.13158

U2 - 10.1111/ejh.13158

DO - 10.1111/ejh.13158

M3 - Article

VL - 101

SP - 676

EP - 683

JO - European Journal of Haematology

JF - European Journal of Haematology

SN - 0902-4441

IS - 5

ER -

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

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