Conclusion

An allogeneic stem cell transplant remains the only therapy with a curative potential in the management of a patient with a diagnosis of MDS. However, the risks associated with an allogeneic transplant in this older population have to be weighed against the benefits. Statistical models predict that delaying an allo-geneic stem cell transplant for patients in the low-risk MDS group is associated with maximal life expectancy. Myeloablative regimens are associated with a lower risk of relapse in patients with high-risk MDS. Recent data using myeloablative regimens with targeted buslfan levels hold promise in both reducing the RRT

Table 42.3 Summary of data of patients with MDS who underwent an HLA-identical (related and unrelated) allogeneic SCT using a nonmyeloablative conditioning regimen

No. of Age High-risk MUD Preparative NRM DFS (%)

Study patients (median) MDS (%) (%) regimen (%) at 2 years OS (%)

Giralt11 26 57 100 33 FM or FAI 43 27 31

Paker et al.29 23 48 78 70 FB-Campath 26 39 48

Stuart et al.32 77 59 44 50 F-TBI NS NS 24(high risk)

40(low risk)

Martino et al.30 194 54 NS 0 various 30 46 at 1 year 55 at 1 year

De Lima et al.31 26 NS NS NS FM or FAI 30 Relapse risk 61%

with FAI vs 30% with FM*

DFS - disease-free survival; NRM - nonrelapse mortality; NS - not stated; OS - overall survival; TBI - total body irradiation; FAI - fludarabine, cytarabine, and idarubincin; FM - fludarabine and melphalan; FB - fludarabine and busulfan.

* Combined data of patients with diagnosis of MDS and acute myeloid leukemia.

and the risk of relapse. Preliminary data with non-myeloablative regimens show a definite reduction in NRM, though the high risk of relapse is of some concern, especially in patients in the high-risk group of MDS. Ongoing clinical trials may help identify an optimal nonmyeloablative regimen. Cytokine mobilized PBSC transplants appear to be superior to marrow transplants in this setting. Published data suggest that outcomes with MUD stem cell transplant are comparable to that with an HLA-identical related donor. Preliminary data with mismatched cord blood transplants are exciting, but remain to be validated.

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