Novel therapies in low- and high-risk myelodysplastic syndrome.
Introduction: Myelodysplastic syndromes (MDS) comprise a heterogeneous group of myeloid neoplasms with diverse clinical courses. The revised version of the international prognostic scoring system (IPSS-R) provides risk stratification into 5 different groups. Areas covered: For lower-risk patients, red blood cell transfusions and iron chelation are the backbone of supportive care. In addition, erythropoiesis-stimulating agents (ESA) are used to ameliorate anemia. Lenalidomide is approved for the treatment of lower-risk patients with del(5q) who are transfusion-dependent. Patients with higher-risk disease should be offered allogeneic stem cell transplantation whenever possible. If they are unfit for transplantation or an appropriate donor cannot be found, hypomethylating agents may be used. Expert opinion: New therapeutic options for lower-risk patients include thrombopoietin analogues, the TGF-beta family ligand trapping drug Luspatercept, and the telomerase inhibitor Imetelstat. Combinations of hypomethylating agents (HMA) with other compounds, and inhibitors of bcl2, such as venetoclax are being developed for higher-risk patients. Finally, hypomethylating agents in combination with donor lymphocytes may lead to long-term remission following molecular or hematological relapse after allogeneic SCT.
['Germing|Ulrich|U|', 'Schroeder|Thomas|T|', 'Kaivers|Jennifer|J|', 'Kündgen|Andrea|A|', 'Kobbe|Guido|G|', 'Gattermann|Norbert|N|']
D030301:Activin Receptors, Type II D000740:Anemia D000970:Antineoplastic Agents D001374:Azacitidine D019086:Bridged Bicyclo Compounds, Heterocyclic D000077209:Decitabine D017707:Erythrocyte Transfusion D006397:Hematinics D006801:Humans D007141:Immunoglobulin Fc Fragments D007502:Iron Chelating Agents D000077269:Lenalidomide D009190:Myelodysplastic Syndromes D009841:Oligonucleotides D011379:Prognosis D011993:Recombinant Fusion Proteins D018570:Risk Assessment D033581:Stem Cell Transplantation D013449:Sulfonamides D013792:Thalidomide D013926:Thrombopoietin D014184:Transplantation, Homologous
Myelodysplastic syndromes hypomethylating agents MRD monitoring TGF-beta ligand trapping bcl2 inhibition erythropoiesis stimulating agents immunomodulating drugs investigational drugs risk-adapted therapy thrombopoiesis stimulating agents