Myelodysplastic syndrome

Fit older adults with advanced myelodysplastic syndromes: who is most likely to benefit from transplant?

Abstract

We conducted a prospective observational study of fit adults aged 60–75 with advanced MDS, enrolled hierarchically for adverse MDS risk (intermediate-2 or high-risk international prognostic score [IPSS], low or intermediate-1 IPSS with poor-risk cytogenetics, or therapy-related MDS) or standard risk with severe cytopenia. A total of 290 patients enrolled at two centers: 175 for adverse risk and 115 for standard risk with severe cytopenia. 113 underwent HCT after a median of 5 months; median follow-up for all was 39.5 months. In univariable analyses, the hazard ratio (HR) for death comparing HCT with no HCT was 0.84 (p = 0.30). The HR for death was 0.64 (p = 0.04) for HCT ≤ 5 months after enrollment and 1.20 (p = 0.39) for HCT > 5 months. In multivariable analyses controlling for age, gender, ECOG performance status, cytogenetic risk, and IPSS risk group, HR for death was 0.75 (p = 0.13) for HCT compared to no HCT, 0.57 (p = 0.01) for adverse MDS risk and 1.33 (p = 0.36) for standard risk with severe cytopenia. In this large, prospective cohort of fit older adults with advanced MDS, we found that survival was significantly improved if HCT was performed early or for adverse risk disease but not for standard risk disease with severe cytopenia.

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Fig. 1: Simon-Makuch Curves for Overall Survival.
Fig. 2: Simon-Makuch Curves for Overall Survival: Subgroup Analysis by Eligibility Criteria and IPSS.
Fig. 3: Kaplan-Meier Curves for Overall Survival by Entry Criteria and IPSS.
Fig. 4: Which Patients Were Most likely to Benefit from HCT?

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Acknowledgements

This work was supported by a Leukemia and Lymphoma Society Research Scholar Grant (GAA), an American Cancer Society Clinical Scholar Award (GAA), an NCI P01 Award (1P01CA229092-01A1; RJS and HK), and an NCI/NIH T32 Grant (CA092203; AH). This work was presented in preliminary form at the American Society of Hematology 2018 Annual Meeting, Abstract #972.

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Correspondence to Gregory A. Abel.

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Abel, G.A., Kim, H.T., Hantel, A. et al. Fit older adults with advanced myelodysplastic syndromes: who is most likely to benefit from transplant?. Leukemia (2020). https://doi.org/10.1038/s41375-020-01092-2

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