Decision analysis of allogeneic hematopoietic stem cell transplantation for patients with myelodysplastic syndrome stratified according to the revised International Prognostic Scoring System.
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.
Cancer Center, Humanitas Research Hospital & Humanitas University, Rozzano, Milan, Italy.
M G
MG
Della Porta
MRC Biostatistics Unit, Institute of Public Health, Cambridge, UK.
C H
CH
Jackson
Department of Hematology Oncology, Fondazione IRCCS Policlinico San Matteo, Pavia, Italy.
E P
EP
Alessandrino
Cancer Center, Humanitas Research Hospital & Humanitas University, Rozzano, Milan, Italy.
M
M
Rossi
Division of Hematology and Bone Marrow Transplant, Catholic University of the Sacred Heart, Fondazione Policlinico Universitario Gemelli, Rome, Italy.
A
A
Bacigalupo
Department of Hematology, San Martino Hospital, Genova, Italy.
M T
MT
van Lint
Hematology and Bone Marrow Transplantation Unit, Department of Oncology, San Raffaele Scientific Institute, Milan, Italy.
M
M
Bernardi
Division of Hematology & Department of Molecular Biotechnology and Health Sciences, University of Torino, Torino, Italy.
B
B
Allione
Hematology & Bone Marrow Transplantation, Azienda Ospedaliera Universitaria Careggi, Florence, Italy.
A
A
Bosi
Hematology & Bone Marrow Transplantation, Azienda Ospedaliera Universitaria Careggi, Florence, Italy.
S
S
Guidi
Hematology & Bone Marrow Transplantation, Azienda Ospedaliera Universitaria Careggi, Florence, Italy.
V
V
Santini
Department of Hematology Oncology, Fondazione IRCCS Policlinico San Matteo, Pavia, Italy.|Department of Molecular Medicine, University of Pavia, Pavia, Italy.
L
L
Malcovati
Department of Hematology Oncology, Fondazione IRCCS Policlinico San Matteo, Pavia, Italy.
M
M
Ubezio
Cancer Center, Humanitas Research Hospital & Humanitas University, Rozzano, Milan, Italy.
C
C
Milanesi
Cancer Center, Humanitas Research Hospital & Humanitas University, Rozzano, Milan, Italy.
E
E
Todisco
Department of Biomedicine and Prevention, University of Rome 'Tor Vergata', Rome, Italy.
M T
MT
Voso
Scientific Direction, IRCCS-CROB, Referral Cancer Center of Basilicata, Rionero in Vulture, Italy.
P
P
Musto
Hematology and Bone Marrow Transplantation Center, Fondazione IRCCS Ospedale Maggiore Policlinico, University of Milan, Milan, Italy.
F
0000-0002-2991-3474
F
Onida
Department of Cellular Biotechnologies and Hematology, Sapienza University, Rome, Italy.
A P
AP
Iori
Department of Hematology, Rome Transplant Network, Stem Cell Transplant Unit, Policlinico Tor Vergata, Rome, Italy.
R
R
Cerretti
Department of Hematology Oncology, Ospedale Niguarda Ca' Granda, Milan, Italy.
G
G
Grillo
Department of Hematology Oncology, Ospedale Niguarda Ca' Granda, Milan, Italy.
A
A
Molteni
Division of Hematology & Transplant Unit, Ospedale San Gerardo, Monza, Italy.
P
P
Pioltelli
Division of Hematology & Transplant Unit, Ospedale San Gerardo, Monza, Italy.
L
L
Borin
Hematology and Bone Marrow Transplantation Unit, Ospedale Oncologico di Riferimento Regionale A Businco, Cagliari, Italy.
E
0000-0002-6512-6080
E
Angelucci
Hematology and Bone Marrow Transplantation Unit, Azienda Ospedaliera Papa Giovanni XXIII, Bergamo, Italy.
E
E
Oldani
Department of Hematology, San Martino Hospital, Genova, Italy.
S
S
Sica
Department of Hematology Oncology, Fondazione IRCCS Policlinico San Matteo, Pavia, Italy.
C
C
Pascutto
Department of Hematology Oncology, Fondazione IRCCS Policlinico San Matteo, Pavia, Italy.
V
V
Ferretti
Cancer Center, Humanitas Research Hospital & Humanitas University, Rozzano, Milan, Italy.
A
A
Santoro
Institute of Hematology 'L. and A. Seràgnoli', University of Bologna, St Orsola-Malpighi University Hospital, Bologna, Italy.
F
F
Bonifazi
Department of Hematology Oncology, Fondazione IRCCS Policlinico San Matteo, Pavia, Italy.|Department of Molecular Medicine, University of Pavia, Pavia, Italy.
M
M
Cazzola
Hematology and Bone Marrow Transplantation Unit, Azienda Ospedaliera Papa Giovanni XXIII, Bergamo, Italy.|University of Milan, Milan, Italy.
A
A
Rambaldi
D003661:Decision Support Techniques; D005260:Female; D018380:Hematopoietic Stem Cell Transplantation; D006801:Humans; D008297:Male; D008875:Middle Aged; D011379:Prognosis; D019057:Quality-Adjusted Life Years