The optimal therapy for myelodysplastic syndrome (MDS) is allogeneic bone marrow (BM) or blood (BSC) stem cell transplantation (SCT), although outcomes are limited by nonrelapse mortality (NRM) and relapse. A retrospective review was performed of 156 patients who underwent SCT (114 BM, 42 BSC) for MDS or secondary acute myelogenous leukemia (sAML) at our institution. Fifty-five patients remain in continuous complete remission: 35 BM recipients and 20 BSC recipients (median follow-up 139 and 89 months, respectively). Estimated 7-year event-free survival (EFS), NRM, and risk of relapse (ROR) are 33% (95% confidence intervals [CI] 25%-43%), 42% (CI 33%-51%), and 25% (CI 17%-33%) for the BM cohort and 45% (CI 32%-64%, P = .07), 32% (CI 18%-47%, P = .15), and 23% (CI 11%-37%, P = .79) for the BSC cohort. Multivariate analysis showed IPSS poor-risk cytogenetics (P < .001), time from diagnosis to SCT (P < .001), FAB subgroup (P = .001), recipients not in complete remission (CR1) at SCT (P = .005), and the development of acute graft-versus-host disease (aGVHD) (P = .04) were all predictive of an inferior EFS. The FAB subgroup (P = .002), poor-risk karyotype (P = .004), and non-CR1 status also correlated with ROR in multivariate analysis. EFS for poor-risk karyotype patients was superior after receiving BSC compared to BM (39% versus 6%, P < .001). SCT outcomes in MDS/sAML are strongly associated with the IPSS cytogenetic risk group, although the use of BSC in poor-risk karyotype patients may lead to a more favorable long-term EFS. © 2009 American Society for Blood and Marrow Transplantation.
Nevill, T. J., Shepherd, J. D., Sutherland, H. J., Abou Mourad, Y. R., Lavoie, J. C., Barnett, M. J., … Smith, C. A. (2009). IPSS Poor-Risk Karyotype as a Predictor of Outcome for Patients with Myelodysplastic Syndrome following Myeloablative Stem Cell Transplantation. Biology of Blood and Marrow Transplantation, 15(2), 205–213. https://doi.org/10.1016/j.bbmt.2008.11.015