Transfusion avoidance in myelodysplastic neoplasms

Abstract

Purpose of review Myelodysplastic neoplasms (MDS) are diseases of stem cell aging associated with complications from inadequate hematopoiesis (red cells, neutrophils and platelets) and variable risk for transformation to acute myeloid leukemia. Those with low-risk disease also suffer and die from MDS-related complications. Among the most challenging is development of anemia and transfusion dependence, which impacts quality of life and is associated with reduced survival. Appreciating and measuring the quality-of-life impact, preventing (if possible), treating, and managing the complications from anemia in MDS are of critical importance. Recent findings Recent developments in basic science highlight the potential deleterious impact of iron overload within the developing red cell niche. Iron overload can compromise red cell maturation from healthy as well as malignant clones and produces an environment favoring expansion of mutant clonal cells, potentially driving disease progression. Observational studies in nontransfusion dependent MDS highlight that iron overload occurs even in the nontransfusion dependent. The newly approved (and established) therapies for management of MDS-related anemia work best when begun before patients become heavily transfusion-dependent. Summary Iron overload is detrimental to hematopoiesis. Understanding the benefit afforded by transfusion is critical to optimal application and patient reported outcomes can inform this. Recently developed therapies are active and optimized application may improve response.

Latest Insights and Therapeutic Advances in Myelodysplastic Neoplasms

“…Particularly attention, in clinical practice as well as in counselling of family members, must be given to MDS patients with germline mutations [20][21][22]. Regardless of the MDS subtype and prognostic allocation, anaemia is prevalent in 80% of MDS patients, and 50% of them receive red blood cell (RBC) transfusions during their disease [71][72][73]. Of note, anaemia necessitates focused management given its significant impact on QoL [64] and organ damage, which may be secondary to demand ischaemia, increased cardiac output, and left ventricular remodelling [74,75].…”

“…The clinical management of LR-MDS focuses on treating symptoms and cytopenias, among which anaemia is the hallmark of the disease and the most common indication for treatment [43]; however, isolated thrombocytopenia and neutropenia are encountered less often [62,82]. Therefore, the main therapeutic efforts are aimed at correcting chronic anaemia [71][72][73] and thrombocytopenia [83][84][85], reducing recurrent infections [82], and improving or maintaining QoL [58,64,86]. Figure 2 shows an updated algorithm proposed for the treatment of LR-MDS [62].…”