Abstract Topic: 10. Myelodysplastic syndromes - Clinical
Background: The transfusion burden differs among patients with lower-risk myelodysplastic syndrome (LR-MDS), and those receiving red blood cell (RBC) transfusions are at risk of iron overload. In such cases, the binding capacity of transferrin for iron is surpassed, resulting in non–transferrin-bound iron circulating in the blood and subsequent deposition of free iron in tissues, which can cause significant organ damage and is an important cause of morbidity and mortality. Although new agent such as luspatercept showed a notable response in transfusion-dependent patients with LR-MDS, many patients still need RBC transfusions. Therefore, determining the clinical outcomes of transfusion burden and optimizing treatment for patients with MDS with transfusion dependencies are important.
Recently, the MDS International Working Group (IWG) has established criteria for transfusion dependence and defined three categories: non-transfusion-dependent (NTD), low transfusion burden (LTB), and high transfusion burden (HTB).
Aims: This study retrospectively analyzed the prognostic impact of transfusion burden in patients with 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. The treatment response was observed over 4–6 months after treatment.
Results: 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 incidence 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.
Summary/Conclusion: Our findings show that transfusion dependency is an adverse prognostic factor in LR-MDS. Furthermore, HTB presented a higher risk of leukemic transformation.
Keywords: Myelodysplastic syndrome, Transfusion, Prognostic factor