An analysis of 246 myelodysplastic syndrome (MDS) patients with low or intermediate-1 disease according to the International Prognostic Scoring System (IPSS) found that the majority of these patients (76%) actually had intermediate or higher-risk disease according to the revised IPSS. The researchers called these patients “false low-risk” patients, and determined which disease and patient factors allowed these patients to achieve better-than-expected hematopoietic cell transplant (HCT) outcomes: 3-year overall survival (OS) and progression-free survival (PFS) of 58% and 54%, respectively.
A multivariate analysis found 3 adverse risk factors for PFS: marrow blast percentage (hazard ratio (HR): 1.77, p=0.037), donor/recipient cytomegalovirus (CMV) serostatus (donor−/recipient+: HR: 2.02, p=0.011), and source of stem cells (marrow and HCT in non-complete remission (CR): HR: 5.72, p<0.0001, marrow and HCT in CR: HR: 3.17, p=0.027).
Independent risk factors for OS were disease status at time of transplant and the use of in vivo T-cell depletion (TCD). Patients who did not receive TCD and were transplanted from an unrelated donor had worse OS (HR: 4.08, p<0.0001).
Although historically low-risk patients have not benefitted from HCT, the researchers concluded that “lower-risk" MDS patients have better outcomes after HCT than those with “higher risk” and that “selecting the right source of stem cells, a CMV-positive donor for CMV-positive patients, and using in vivo TCD results in the best outcome in these patients.”