Prognostic Impact of Transfusion Dependency in Patients with Lower-Risk Myelodysplastic Syndrome.

Background: This study retrospectively analyzed the prognostic impact of transfusion burden in patients with lower-risk myelodysplastic syndrome (LR-MDS) and the outcomes of each treatment option.

Methods: Data on 168 patients with LR-MDS between July 2011 and April 2020 were retrospectively reviewed. Non-transfusion dependent (NTD) was defined as no transfusion history in a period of 16 weeks, low transfusion burden (LTB) as receiving 3 - 7 red blood cell (RBC) units in a period of 16 weeks, and high transfusion burden (HTB) as receiving ≥ 8 RBC units in a period of 16 weeks.

Results: The treatment response was observed over 4 - 6 months after treatment. Among the 168 patients, 105 were treated with anabolic steroids (n = 65), erythroid stimulating agents (n = 12), or hypomethylating agents (n = 28). The overall response rate was 53.3% (56/105), with 53 patients showing hematologic improvement (50.5%). The clinical benefit rate was 78.1% (82/105). The 5-year overall survival (OS) rates were 75.5%, 45.8%, and 33.3% for NTD, LTB, and HTB, respectively (p = 0.001). The 5-year incidences of acute myeloid leukemia were 0%, 9.9%, and 32.5% in NTD, LTB, and HTB, respectively (p < 0.001). In the multivariate analysis, age (hazard ratio [HR] 1.04, p = 0.009), LTB (HR 3.77, p = 0.002), HTB (HR 4.59, p < 0.001), and hemoglobin response (HR 0.45, p = 0.036) were significant factors for OS.

Conclusions: Our findings show transfusion dependency is an adverse prognostic factor in LR-MDS. HTB presented a higher risk of leukemic transformation.