How we treat lower risk myelodysplastic syndromes. | Aplastic Anemia and MDS International Foundation

How we treat lower risk myelodysplastic syndromes.

Journal Title: 
Blood
Author(s): 
Fenaux P, Adès L.
Primary Author: 
Fenaux P
Original Publication Date: 
Wednesday, April 10, 2013

Lower risk MDS are defined as having low or intermediate 1 risk by the IPSS, and are mainly characterized by anemia in most cases. Supportive care, mainly red blood cell transfusions, remains an important component of their treatment, but expose patients to insufficient correction of anemia , alloimmunization, and organ iron overload (for which the role of iron chelation remains debated). Treatment aimed at preventing anemia recurrence should therefore be used whenever possible. ESAs remain the first line treatment of anemia in most lower risk MDS without del(5q), while anemia of low risk MDS with del 5q responds to Lenalidomide in two thirds of the cases, but this drug should be used cautiously because profound cytopenias may occur initially. Treatment after failure of those first line therapies are overall disappointing, many patients eventually requiring long-term transfusions, but encouraging results have been reported with hypomethylating agents and lenalidomide. Selected patients respond to antithymocyte globulins, while thrombopoeitin receptor agonists are under investigation in lower risk MDS with thrombocytopenia. Some patients, while remaining "lower risk" MDS, have severe cytopenias and/or poor prognostic factors using newer prognostic parameters, and/or resistance to treatment, making them rapidly candidates for more intensive approaches, including allogeneic stem cell transplantation (SCT).

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