Decision analysis of allogeneic hematopoietic stem cell transplantation for patients with myelodysplastic syndrome stratified according to the revised international prognostic scoring system (IPSS-R)

M G Della Porta, Christopher H. Jackson, E P Alessandrino, M Rossi, A Bacigalupo, M T van Lint, M Bernardi, B. Allione, A. Bosi, S. Guidi, V. Santini, L Malcovati, M. C. Ubezio, C Milanesi, E Todisco, M. T. Voso, P Musto, F Onida, A. P. Iori, Raffaella CerrettiG. Grillo, A. Molteni, P. Pioltelli, L. Borin, E. Angelucci, Elisa Oldani, Giuseppe S. Sica, Cristiana Pascutto, V Ferretti, A Santoro, F. Bonifazi, M Cazzola, Alessandro Rambaldi

Research output: Contribution to journalArticle

Abstract

Allogeneic hematopoietic stem cell transplantation (allo-SCT) represents the only curative treatment for patients with myelodysplastic syndrome (MDS), but involves non-negligible morbidity and mortality. Crucial questions in clinical decision making include the definition of optimal timing of the procedure and the benefit of cytoreduction before transplant in high risk patients. We carried out a decision analysis on 1728 MDS who received supportive care, transplantation or hypomethylating agents (HMAs). Risk assessment was based on the revised International Prognostic Scoring System (IPSS-R). We used a continuous-time multistate Markov model to describe the natural history of disease and evaluate the effect of different treatment policies on survival. Life expectancy increased when transplantation was delayed from the initial stages to intermediate IPSS-R risk (gain of life expectancy 5.3, 4.7 and 2.8 years for patients aged ⩽55, 60 and 65 years, respectively), and then decreased for higher risks. Modelling decision analysis on IPSS-R vs original IPSS changed transplantation policy in 29% of patients, resulting in a 2-year gain in life expectancy. In advanced stages, HMAs given before transplant is associated with a 2-year gain of life expectancy, especially in older patients. These results provide a preliminary evidence to maximize the effectiveness of allo-SCT in MDS.Leukemia accepted article preview online, 21 March 2017. doi:10.1038/leu.2017.88.
Original languageEnglish
JournalLeukemia
DOIs
Publication statusPublished - Mar 21 2017

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Decision Support Techniques
Hematopoietic Stem Cell Transplantation
Myelodysplastic Syndromes
Life Expectancy
Transplantation
Transplants
Leukemia
Morbidity
Survival
Mortality
Therapeutics

Keywords

  • Journal Article

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Decision analysis of allogeneic hematopoietic stem cell transplantation for patients with myelodysplastic syndrome stratified according to the revised international prognostic scoring system (IPSS-R). / Della Porta, M G; Jackson, Christopher H.; Alessandrino, E P; Rossi, M; Bacigalupo, A; van Lint, M T; Bernardi, M; Allione, B.; Bosi, A.; Guidi, S.; Santini, V.; Malcovati, L; Ubezio, M. C.; Milanesi, C; Todisco, E; Voso, M. T.; Musto, P; Onida, F; Iori, A. P.; Cerretti, Raffaella; Grillo, G.; Molteni, A.; Pioltelli, P.; Borin, L.; Angelucci, E.; Oldani, Elisa; Sica, Giuseppe S.; Pascutto, Cristiana; Ferretti, V; Santoro, A; Bonifazi, F.; Cazzola, M; Rambaldi, Alessandro.

In: Leukemia, 21.03.2017.

Research output: Contribution to journalArticle

Della Porta, M G ; Jackson, Christopher H. ; Alessandrino, E P ; Rossi, M ; Bacigalupo, A ; van Lint, M T ; Bernardi, M ; Allione, B. ; Bosi, A. ; Guidi, S. ; Santini, V. ; Malcovati, L ; Ubezio, M. C. ; Milanesi, C ; Todisco, E ; Voso, M. T. ; Musto, P ; Onida, F ; Iori, A. P. ; Cerretti, Raffaella ; Grillo, G. ; Molteni, A. ; Pioltelli, P. ; Borin, L. ; Angelucci, E. ; Oldani, Elisa ; Sica, Giuseppe S. ; Pascutto, Cristiana ; Ferretti, V ; Santoro, A ; Bonifazi, F. ; Cazzola, M ; Rambaldi, Alessandro. / Decision analysis of allogeneic hematopoietic stem cell transplantation for patients with myelodysplastic syndrome stratified according to the revised international prognostic scoring system (IPSS-R). In: Leukemia. 2017.
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abstract = "Allogeneic hematopoietic stem cell transplantation (allo-SCT) represents the only curative treatment for patients with myelodysplastic syndrome (MDS), but involves non-negligible morbidity and mortality. Crucial questions in clinical decision making include the definition of optimal timing of the procedure and the benefit of cytoreduction before transplant in high risk patients. We carried out a decision analysis on 1728 MDS who received supportive care, transplantation or hypomethylating agents (HMAs). Risk assessment was based on the revised International Prognostic Scoring System (IPSS-R). We used a continuous-time multistate Markov model to describe the natural history of disease and evaluate the effect of different treatment policies on survival. Life expectancy increased when transplantation was delayed from the initial stages to intermediate IPSS-R risk (gain of life expectancy 5.3, 4.7 and 2.8 years for patients aged ⩽55, 60 and 65 years, respectively), and then decreased for higher risks. Modelling decision analysis on IPSS-R vs original IPSS changed transplantation policy in 29{\%} of patients, resulting in a 2-year gain in life expectancy. In advanced stages, HMAs given before transplant is associated with a 2-year gain of life expectancy, especially in older patients. These results provide a preliminary evidence to maximize the effectiveness of allo-SCT in MDS.Leukemia accepted article preview online, 21 March 2017. doi:10.1038/leu.2017.88.",
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T1 - Decision analysis of allogeneic hematopoietic stem cell transplantation for patients with myelodysplastic syndrome stratified according to the revised international prognostic scoring system (IPSS-R)

AU - Della Porta, M G

AU - Jackson, Christopher H.

AU - Alessandrino, E P

AU - Rossi, M

AU - Bacigalupo, A

AU - van Lint, M T

AU - Bernardi, M

AU - Allione, B.

AU - Bosi, A.

AU - Guidi, S.

AU - Santini, V.

AU - Malcovati, L

AU - Ubezio, M. C.

AU - Milanesi, C

AU - Todisco, E

AU - Voso, M. T.

AU - Musto, P

AU - Onida, F

AU - Iori, A. P.

AU - Cerretti, Raffaella

AU - Grillo, G.

AU - Molteni, A.

AU - Pioltelli, P.

AU - Borin, L.

AU - Angelucci, E.

AU - Oldani, Elisa

AU - Sica, Giuseppe S.

AU - Pascutto, Cristiana

AU - Ferretti, V

AU - Santoro, A

AU - Bonifazi, F.

AU - Cazzola, M

AU - Rambaldi, Alessandro

PY - 2017/3/21

Y1 - 2017/3/21

N2 - Allogeneic hematopoietic stem cell transplantation (allo-SCT) represents the only curative treatment for patients with myelodysplastic syndrome (MDS), but involves non-negligible morbidity and mortality. Crucial questions in clinical decision making include the definition of optimal timing of the procedure and the benefit of cytoreduction before transplant in high risk patients. We carried out a decision analysis on 1728 MDS who received supportive care, transplantation or hypomethylating agents (HMAs). Risk assessment was based on the revised International Prognostic Scoring System (IPSS-R). We used a continuous-time multistate Markov model to describe the natural history of disease and evaluate the effect of different treatment policies on survival. Life expectancy increased when transplantation was delayed from the initial stages to intermediate IPSS-R risk (gain of life expectancy 5.3, 4.7 and 2.8 years for patients aged ⩽55, 60 and 65 years, respectively), and then decreased for higher risks. Modelling decision analysis on IPSS-R vs original IPSS changed transplantation policy in 29% of patients, resulting in a 2-year gain in life expectancy. In advanced stages, HMAs given before transplant is associated with a 2-year gain of life expectancy, especially in older patients. These results provide a preliminary evidence to maximize the effectiveness of allo-SCT in MDS.Leukemia accepted article preview online, 21 March 2017. doi:10.1038/leu.2017.88.

AB - Allogeneic hematopoietic stem cell transplantation (allo-SCT) represents the only curative treatment for patients with myelodysplastic syndrome (MDS), but involves non-negligible morbidity and mortality. Crucial questions in clinical decision making include the definition of optimal timing of the procedure and the benefit of cytoreduction before transplant in high risk patients. We carried out a decision analysis on 1728 MDS who received supportive care, transplantation or hypomethylating agents (HMAs). Risk assessment was based on the revised International Prognostic Scoring System (IPSS-R). We used a continuous-time multistate Markov model to describe the natural history of disease and evaluate the effect of different treatment policies on survival. Life expectancy increased when transplantation was delayed from the initial stages to intermediate IPSS-R risk (gain of life expectancy 5.3, 4.7 and 2.8 years for patients aged ⩽55, 60 and 65 years, respectively), and then decreased for higher risks. Modelling decision analysis on IPSS-R vs original IPSS changed transplantation policy in 29% of patients, resulting in a 2-year gain in life expectancy. In advanced stages, HMAs given before transplant is associated with a 2-year gain of life expectancy, especially in older patients. These results provide a preliminary evidence to maximize the effectiveness of allo-SCT in MDS.Leukemia accepted article preview online, 21 March 2017. doi:10.1038/leu.2017.88.

KW - Journal Article

U2 - 10.1038/leu.2017.88

DO - 10.1038/leu.2017.88

M3 - Article

JO - Leukemia

JF - Leukemia

SN - 0887-6924

ER -