TY - JOUR AU - Basood, May AU - Oster, Howard S. AU - Mittelman, Moshe PY - 2018/07/01 Y2 - 2024/03/28 TI - THROMBOCYTOPENIA IN PATIENTS WITH MYELODYSPLASTIC SYNDROMES - STILL AN UNSOLVED PROBLEM JF - Mediterranean Journal of Hematology and Infectious Diseases JA - Mediterr J Hematol Infect Dis VL - 10 IS - 1 SE - Review Articles DO - 10.4084/mjhid.2018.046 UR - https://www.mjhid.org/mjhid/article/view/2018.046 SP - e2018046 AB - <p>The myelodysplastic syndromes (MDS) are a group of clonal bone marrow (BM) stem cell disorders, characterized by ineffective hematopoiesis, peripheral cytopenias and hematologic cellular dysfunction, as well as potential transformation to acute leukemia.</p><p>Thrombocytopenia is common in MDS and is associated with bleeding complications, occasionally life threatening. Low platelet count (PLT), as well declining PLT also serves as a prognostic marker. Understanding thrombopoiesis led to the cloning of thrombopoietin, resulting in the development of platelet stimulating agents, thrombomimetics, romiplostim and eltrombopag.</p><p>Both agents have been shown to increase PLT, decrease the need for platelet transfusions and reduce the number of bleeding episodes, with a reasonable tolerance. They are already approved for immune thrombocytopenia and thrombocytopenia related to liver disease.</p><p>Romiplostim and eltrombopag have proven efficacy in lower- and higher-risk MDS with thrombocytopenia, as monotherapy, as well as a part of a combination, either with lenalidomide, and mainly combined with hypomethylating agents. However, safety concerns have been raised: while several trials have been completed with no evidence of disease progression, others have been early terminated due to increased number of BM blasts and possible leukemic transformation in treated-patients. The jury is still out regarding this safety concern, although recent publications are more encouraging. </p><p>Keywords: Myelodysplastic syndrome, Thrombocytopenia, Thrombomimetics,</p><p>Romiplostim, Eltrombopag.</p> ER -