title: Optimal timing of allogeneic hematopoietic stem cell transplantation in patients with myelodysplastic syndrome.
abstract: Allogeneic hematopoietic stem cell transplantation (HSCT) represents the only curative treatment for patients with myelodysplastic syndrome (MDS), but involves non-negligible morbidity and mortality. Registry studies have shown that advanced disease stage at transplantation is associated with inferior overall survival. To define the optimal timing of allogeneic HSCT, we carried out a decision analysis by studying 660 patients who received best supportive care and 449 subjects who underwent transplantation. Risk assessment was based on both the International Prognostic Scoring System (IPSS) and the World Health Organization classification-based Prognostic Scoring System (WPSS). We used a continuous-time multistate Markov model to describe the natural history of disease and evaluate the effect of allogeneic HSCT on survival. This model estimated life expectancy from diagnosis according to treatment policy at different risk stages. Relative to supportive care, estimated life expectancy increased when transplantation was delayed from the initial stages until progression to intermediate-1 IPSS-risk or to intermediate WPSS-risk stage, and then decreased for higher risks. Modeling decision analysis on WPSS versus IPSS allowed better estimation of the optimal timing of transplantation. These observations indicate that allogeneic HSCT offers optimal survival benefits when the procedure is performed before MDS patients progress to advanced disease stages.
authors.affiliation: Department of Hematology Oncology, Fondazione IRCCS Policlinico San Matteo, Pavia, Italy; Department of Molecular Medicine, University of Pavia, Pavia, Italy.
authors.forename: Emilio Paolo
authors.initials: EP
authors.lastname: Alessandrino
authors.forename: Matteo G Della
authors.initials: MG
authors.lastname: Porta
authors.forename: Luca
authors.initials: L
authors.lastname: Malcovati
authors.forename: Christopher H
authors.initials: CH
authors.lastname: Jackson
authors.forename: Cristiana
authors.initials: C
authors.lastname: Pascutto
authors.forename: Andrea
authors.initials: A
authors.lastname: Bacigalupo
authors.forename: Maria
authors.initials: M
authors.lastname: Teresa van Lint
authors.forename: Michele
authors.initials: M
authors.lastname: Falda
authors.forename: Massimo
authors.initials: M
authors.lastname: Bernardi
authors.forename: Francesco
authors.initials: F
authors.lastname: Onida
authors.forename: Stefano
authors.initials: S
authors.lastname: Guidi
authors.forename: Anna Paola
authors.initials: AP
authors.lastname: Iori
authors.forename: Raffaella
authors.initials: R
authors.lastname: Cerretti
authors.forename: Paola
authors.initials: P
authors.lastname: Marenco
authors.forename: Pietro
authors.initials: P
authors.lastname: Pioltelli
authors.forename: Emanuele
authors.initials: E
authors.lastname: Angelucci
authors.forename: Rosi
authors.initials: R
authors.lastname: Oneto
authors.forename: Francesco
authors.initials: F
authors.lastname: Ripamonti
authors.forename: Alessandro
authors.initials: A
authors.lastname: Rambaldi
authors.forename: Alberto
authors.initials: A
authors.lastname: Bosi
authors.forename: Mario
authors.initials: M
authors.lastname: Cazzola
mesh_terms: D000293:Adolescent; D000328:Adult; D000368:Aged; D000369:Aged, 80 and over; D002648:Child; D015331:Cohort Studies; D005260:Female; D018380:Hematopoietic Stem Cell Transplantation; D006801:Humans; D008297:Male; D008390:Markov Chains; D008875:Middle Aged; D009190:Myelodysplastic Syndromes / Q000175:diagnosis / Q000401:mortality / Q000628:therapy*; D012306:Risk; D016019:Survival Analysis; D013997:Time Factors; D014184:Transplantation, Homologous