Red blood cell transfusion burden in myelodysplastic syndromes (MDS) with ring Sideroblasts (RS): A retrospective multicenter study by the Groupe Francophone des Myélodysplasies (GFM)

Abstract

Background MDS‐RS patients are characterized by chronic anemia and a low risk of Acute Myeloid Leukemia (AML) progression and they generally become Red Blood Cell (RBC) transfusion dependent (TD). Study design and methods We performed a retrospective “real‐life” observational study of 6 months in 100 MDS‐RS TD patients, recruited in 12 French centers, to describe transfusion characteristics, and evaluate the frequency and causes of hospitalizations, health costs, and morbidity, associated with transfusion dependency, in a French population of RBC transfusion‐dependent MDS‐RS patients. Results 79% of the patients had high transfusion burden (HTB) and 21% low transfusion burden (LTB). HTB patients had a longer disease duration (6 vs. 3.7 years, p = 0.0078), more frequent iron chelation (82% vs. 50%, p = 0.0052) and higher serum ferritin (p = 0.03). During the 6‐month study period, 22% of the patients required inpatient hospitalization, 36% of them for symptomatic anemia requiring emergency RBC transfusion. The 6‐month median transfusion costs, including the cost of the day care facility, transportation to and from the hospital, iron chelation, and lab tests, was 16,188€/patient. Discussion MDS‐RS represents the archetypal type of chronically transfused lower‐risk MDS. Most of those patients have a high transfusion burden and thus frequently need visits to the hospital's day care facility, and frequent hospitalizations, with an overall high median treatment cost. Those costs should be compared with costs of new treatments potentially able to avoid RBC transfusion dependence and to reduce the complications of chronic anemia in MDS‐RS patients.

When Ring Sideroblasts on Bone Marrow Smears Are Inconsistent with the Diagnosis of Myelodysplastic Neoplasms

“…Thus, sideroblastic anemias combine various disorders characterized by iron overload and impaired heme biosynthesis. The occurrence of new therapeutic strategies using Luspacercept for the treatment argues for a rigorous assessment of RS [ 39 , 40 , 41 ].…”

Transfusion avoidance in myelodysplastic neoplasms

“…However, recent studies demonstrate the occurrence of iron loading from ineffective erythropoiesis prior to the advent of transfusion dependence in patients with MDS, and these data suggest that pretransfusional iron loading may not be as benign as previously believed. This seems especially true in those with MDS-RS [13][14][15].…”

“…However, recent studies demonstrate the occurrence of iron loading from ineffective erythropoiesis prior to the advent of transfusion dependence in patients with MDS, and these data suggest that pretransfusional iron loading may not be as benign as previously believed. This seems especially true in those with MDS-RS [13–15]. Improved understanding of the impact of iron overload (both due to ineffective erythropoiesis and from transfusions) on both healthy and MDS-clonal erythropoiesis and the potential impact of nontransferrin bound iron as a driver of disease progression for patients with lower risk MDS calls into question the benign nature of pretransfusional iron overload and raises the possibility that earlier iron chelation approaches might be disease modifying in MDS [16,17].…”