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, 31 (11), 2449-2457

Decision Analysis of Allogeneic Hematopoietic Stem Cell Transplantation for Patients With Myelodysplastic Syndrome Stratified According to the Revised International Prognostic Scoring System

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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

M G Della Porta et al. Leukemia.

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.

Conflict of interest statement

Conflict-of-interest disclosure: The authors declare no competing financial interests.

Figures

Figure 1
Figure 1
Markov continuous-time Multi-State Models of the MDS natural history. IPSS-R risk scores were adopted as time-dependent indicators of the natural course of MDS. Allo-SCT was modelled as a time-dependent covariate, and its effect on survival was estimated as a hazard ratio with respect to the “no allo-SCT” category. Solid arrows represent transitions according to the natural course of the disease, whereas the effect of allo-SCT on mortality (i.e. transition to death) in each state is represented by dot arrows.
Figure 2
Figure 2
Overall survival of MDS patients. A: overall survival in the Pavia cohort classified by time-dependent IPSS-R; B: overall survival in the GITMO cohort classified into IPSS-R groups evaluated at the time of allo-SCT.
Figure 3
Figure 3
Gain in expected survival under different transplant policies with respect to a nontransplantation policy. We assumed that the MDS patient was classified as very low IPSS-R risk at the time of diagnosis. Each policy was then evaluated for a set of different ages at diagnosis (as shown in the box) and for different waiting times t (between 0 and 5 years since entering any disease state).
Figure 4
Figure 4
Expected additional survival (compared to no-intervention), by transplant policy, age and treatment with HMA.

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