The Impact of Transfusion-Dependency on the Outcome of Patients with Myelodysplastic Syndromes (MDS) Receiving Allogeneic Peripheral Blood Stem Cell Transplantation after Myeloablative Conditioning.

Introduction: The majority of MDS patients become red blood cell transfusion-dependent during their clinical course. In fact, the presence or absence as well the extent of red blood cell transfusion-dependency (RBC-TD) has been recently shown to add significant prognostic information for an individual MDS patient treated with supportive care (BSC) only. Given the fact that allogeneic hematopoietic cell transplantation (HCT) is the only curative option for MDS patients, the aim of this study was to elucidate the role of RBC-TD on patient outcome.

Methods: We report results of a retrospective multicenter German-Austrian study investigating 100 patients with MDS (RA(RS) n=33, RAEB n=33, RAEB-t n=19, CMMOL n=11, MDS/AML n=4) with either IPPS LOW (n=2), INT-1 (n=31), INT-2 (n=37) or HIGH (n=30) undergoing allogeneic myeloablative conditioning followed by peripheral blood stem cells (PBSC) from related (n=39) or unrelated donors (n=61). The median age of the patients was 50 years (range 18–68).

Results: With a median follow-up of 32 months the 2-year overall survival (OS) was 48% for all patients and 64%, 50% and 34% for patients according to IPSS INT-1, INT-2 or HIGH, respectively (p=0.03 for INT-1 vs. HIGH). The 2-year OS of all patients was not different when comparing 68 patients displaying vs. 24 not displaying RBC-TD prior to PBSCT (46% vs. 55%, p=0.67). This holds also true when analyzing INT-1/INT-2 (n=62) as well as HIGH (n=27) patients separately. In a multivariate analysis only FAB classification (p=0.03) but not prior induction chemotherapy (n=18), presence of fibrosis (n=18), IPSS LOW/INT-1 vs. INT-2/HIGH, RBC-TD as well as ferritin level lower or above the median 973 μg/l (range 19–7000) had an impact on OS.

Conclusion: These data suggest that the negative prognostic impact of RBC-TD in MDS patients receiving BSC can be overcome by myeloablative allogeneic PBSCT.