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Myelodysplastic syndrome

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

Leukemia volume 31, pages 2449–2457 (2017)Cite this article

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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. 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.

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Acknowledgements

The study was supported by AIRC (Associazione Italiana Per la Ricerca sul Cancro, IG_17554), Milan; Fondazione Veronesi, Milan, Fondazione Cariplo and Regione Lombardia, Milan (Grant ID 42916996); Beat Leukemia Foundation, Milan, Fondazione Banca del Monte di Lombardia, Pavia, Italy; Worldwide Cancer Research (Grant 15-1226), St Andrews – UK to MGDP; by UK Medical Research Council (Grant code U105260566) to CHJ; and by AIRC, Milan, Italy (Special Program Molecular Clinical Oncology 5x1000, project 1005) to MC.

Author contributions

MGDP, CHJ, MC and AR designed, performed and coordinated the research, collected, contributed, analyzed and interpreted the data, and wrote the manuscript; CHJ and CP performed the statistical analyses, produced the figures and edited the manuscript; EPA, MR, AB, MTvL, MB, BA, AB, SG, VS, LM, MU, CM, ET, MTV, PM, FO, API, RC, GG, AM, PP, LB, EA, EO, SS, VF, AS and FB collected and contributed data and critically reviewed the manuscript.

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Author notes

  1. M G Della Porta and C H Jackson: These authors are equal first authors.

  2. M Cazzola and A Rambaldi: These authors are equal senior authors.

Authors and Affiliations

  1. Cancer Center, Humanitas Research Hospital & Humanitas University, Rozzano, Milan, Italy

    M G Della Porta, M Rossi, C Milanesi, E Todisco & A Santoro

  2. MRC Biostatistics Unit, Institute of Public Health, Cambridge, UK

    C H Jackson

  3. Department of Hematology Oncology, Fondazione IRCCS Policlinico San Matteo, Pavia, Italy

    E P Alessandrino, L Malcovati, M Ubezio, C Pascutto, V Ferretti & M Cazzola

  4. Division of Hematology and Bone Marrow Transplant, Catholic University of the Sacred Heart, Fondazione Policlinico Universitario Gemelli, Rome, Italy

    A Bacigalupo

  5. Department of Hematology, San Martino Hospital, Genova, Italy

    M T van Lint & S Sica

  6. Department of Oncology, Hematology and Bone Marrow Transplantation Unit, San Raffaele Scientific Institute, Milan, Italy

    M Bernardi

  7. Division of Hematology & Department of Molecular Biotechnology and Health Sciences, University of Torino, Torino, Italy

    B Allione

  8. Hematology & Bone Marrow Transplantation, Azienda Ospedaliera Universitaria Careggi, Florence, Italy

    A Bosi, S Guidi & V Santini

  9. Department of Molecular Medicine, University of Pavia, Pavia, Italy

    L Malcovati & M Cazzola

  10. Department of Biomedicine and Prevention, University of Rome ‘Tor Vergata’, Rome, Italy

    M T Voso

  11. Scientific Direction, IRCCS-CROB, Referral Cancer Center of Basilicata, Rionero in Vulture, Italy

    P Musto

  12. Hematology and Bone Marrow Transplantation Center, Fondazione IRCCS Ospedale Maggiore Policlinico, University of Milan, Milan, Italy

    F Onida

  13. Department of Cellular Biotechnologies and Hematology, Sapienza University, Rome, Italy

    A P Iori

  14. Department of Hematology, Rome Transplant Network, Stem Cell Transplant Unit, Policlinico Tor Vergata, Rome, Italy

    R Cerretti

  15. Department of Hematology Oncology, Ospedale Niguarda Ca' Granda, Milan, Italy

    G Grillo & A Molteni

  16. Division of Hematology & Transplant Unit, Ospedale San Gerardo, Monza, Italy

    P Pioltelli & L Borin

  17. Hematology and Bone Marrow Transplantation Unit, Ospedale Oncologico di Riferimento Regionale A Businco, Cagliari, Italy

    E Angelucci

  18. Hematology and Bone Marrow Transplantation Unit, Azienda Ospedaliera Papa Giovanni XXIII, Bergamo, Italy

    E Oldani & A Rambaldi

  19. Institute of Hematology ‘L. and A. Seràgnoli’, University of Bologna, St Orsola-Malpighi University Hospital, Bologna, Italy

    F Bonifazi

  20. University of Milan, Milan, Italy

    A Rambaldi

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  1. M G Della Porta
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on behalf of the Gruppo Italiano Trapianto di Midollo Osseo (GITMO, www.gitmo.it)

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Correspondence to M G Della Porta.

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Della Porta, M., Jackson, C., Alessandrino, E. et al. Decision analysis of allogeneic hematopoietic stem cell transplantation for patients with myelodysplastic syndrome stratified according to the revised International Prognostic Scoring System. Leukemia 31, 2449–2457 (2017). https://doi.org/10.1038/leu.2017.88

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