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Abstract

Prognostic Impact of Transfusion Dependency in Patients with Lower-Risk Myelodysplastic Syndrome by Jungmin Lee, Hee Jeong Cho, Juhyung Kim, Dong Won Baek, Hyukjin Choi, Ji Yeon Ham, Soon Hee Chang, Jong Gwang Kim, Sang Kyun Sohn, Joon Ho Moon

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.

DOI: 10.7754/Clin.Lab.2024.240820