Decision analysis of allogeneic hematopoietic stem cell transplantation for patients with myelodysplastic syndrome stratified according to the revised International Prognostic Scoring System

M. G. Della Porta, C. 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. Ubezio, C. Milanesi, E. Todisco, M. T. Voso, P. Musto, F. Onida, A. P. Iori, R. Cerretti & 13 others G. Grillo, A. Molteni, P. Pioltelli, L. Borin, E. Angelucci, E. Oldani, S. Sica, C. Pascutto, V. Ferretti, A. Santoro, F. Bonifazi, M. Cazzola, A. Rambaldi

Research output: Contribution to journalArticle

11 Citations (Scopus)

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. Modeling decision analysis on IPSS-R versus 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.
Original languageEnglish
Pages (from-to)2449-2457
Number of pages9
JournalLeukemia
Volume31
Issue number11
DOIs
Publication statusPublished - Nov 1 2017

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

Keywords

  • Decision Support Techniques
  • Female
  • Hematopoietic Stem Cell Transplantation
  • Humans
  • Male
  • Middle Aged
  • Prognosis
  • Quality-Adjusted Life Years

Cite this

Decision analysis of allogeneic hematopoietic stem cell transplantation for patients with myelodysplastic syndrome stratified according to the revised International Prognostic Scoring System. / Porta, M. G. Della; Jackson, C. H.; Alessandrino, E. P.; Rossi, M.; Bacigalupo, A.; Lint, M. T. van; Bernardi, M.; Allione, B.; Bosi, A.; Guidi, S.; Santini, V.; Malcovati, L.; Ubezio, M.; Milanesi, C.; Todisco, E.; Voso, M. T.; Musto, P.; Onida, F.; Iori, A. P.; Cerretti, R.; Grillo, G.; Molteni, A.; Pioltelli, P.; Borin, L.; Angelucci, E.; Oldani, E.; Sica, S.; Pascutto, C.; Ferretti, V.; Santoro, A.; Bonifazi, F.; Cazzola, M.; Rambaldi, A.

In: Leukemia, Vol. 31, No. 11, 01.11.2017, p. 2449-2457.

Research output: Contribution to journalArticle

Porta, MGD, Jackson, CH, Alessandrino, EP, Rossi, M, Bacigalupo, A, Lint, MTV, Bernardi, M, Allione, B, Bosi, A, Guidi, S, Santini, V, Malcovati, L, Ubezio, M, Milanesi, C, Todisco, E, Voso, MT, Musto, P, Onida, F, Iori, AP, Cerretti, R, Grillo, G, Molteni, A, Pioltelli, P, Borin, L, Angelucci, E, Oldani, E, Sica, S, Pascutto, C, Ferretti, V, Santoro, A, Bonifazi, F, Cazzola, M & Rambaldi, A 2017, 'Decision analysis of allogeneic hematopoietic stem cell transplantation for patients with myelodysplastic syndrome stratified according to the revised International Prognostic Scoring System', Leukemia, vol. 31, no. 11, pp. 2449-2457. https://doi.org/10.1038/leu.2017.88 [doi]
Porta, M. G. Della ; Jackson, C. H. ; Alessandrino, E. P. ; Rossi, M. ; Bacigalupo, A. ; Lint, M. T. van ; Bernardi, M. ; Allione, B. ; Bosi, A. ; Guidi, S. ; Santini, V. ; Malcovati, L. ; Ubezio, M. ; Milanesi, C. ; Todisco, E. ; Voso, M. T. ; Musto, P. ; Onida, F. ; Iori, A. P. ; Cerretti, R. ; Grillo, G. ; Molteni, A. ; Pioltelli, P. ; Borin, L. ; Angelucci, E. ; Oldani, E. ; Sica, S. ; Pascutto, C. ; Ferretti, V. ; Santoro, A. ; Bonifazi, F. ; Cazzola, M. ; Rambaldi, A. / Decision analysis of allogeneic hematopoietic stem cell transplantation for patients with myelodysplastic syndrome stratified according to the revised International Prognostic Scoring System. In: Leukemia. 2017 ; Vol. 31, No. 11. pp. 2449-2457.
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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

AU - Porta, M. G. Della

AU - Jackson, C. H.

AU - Alessandrino, E. P.

AU - Rossi, M.

AU - Bacigalupo, A.

AU - Lint, M. T. van

AU - Bernardi, M.

AU - Allione, B.

AU - Bosi, A.

AU - Guidi, S.

AU - Santini, V.

AU - Malcovati, L.

AU - Ubezio, M.

AU - Milanesi, C.

AU - Todisco, E.

AU - Voso, M. T.

AU - Musto, P.

AU - Onida, F.

AU - Iori, A. P.

AU - Cerretti, R.

AU - Grillo, G.

AU - Molteni, A.

AU - Pioltelli, P.

AU - Borin, L.

AU - Angelucci, E.

AU - Oldani, E.

AU - Sica, S.

AU - Pascutto, C.

AU - Ferretti, V.

AU - Santoro, A.

AU - Bonifazi, F.

AU - Cazzola, M.

AU - Rambaldi, A.

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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. Modeling decision analysis on IPSS-R versus 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.

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. Modeling decision analysis on IPSS-R versus 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.

KW - Decision Support Techniques

KW - Female

KW - Hematopoietic Stem Cell Transplantation

KW - Humans

KW - Male

KW - Middle Aged

KW - Prognosis

KW - Quality-Adjusted Life Years

U2 - 10.1038/leu.2017.88 [doi]

DO - 10.1038/leu.2017.88 [doi]

M3 - Article

VL - 31

SP - 2449

EP - 2457

JO - Leukemia

JF - Leukemia

SN - 0887-6924

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ER -