Treatment of MDS: something old, something new, something borrowed...
As opposed to the treatment landscape for myelodysplastic syndromes (MDS) two decades ago, potential therapies now abound for the treatment of lower-risk and higher-risk populations. In lower-risk patients, decision tools can be used to determine the likelihood of response to erythropoiesis stimulating agents (ESAs), which have demonstrated survival advantages in retrospective studies in patients with MDS, and whether these patients should be treated initially with ESAs or non-growth factor ("active") therapies. Lenalidomide has shown good activity in transfusion-dependent patients with the del(5q) cytogenetic abnormality and modest activity in other lower-risk patients. In higher-risk patients, the DNA methyltransferase inhibitors produce complete and partial responses in 20% to 30% of patients, and for the first time, the MDS drug azacitidine has demonstrated a survival advantage when compared with conventional therapies. Newer therapies stimulate platelet production and target novel pathways, while a panoply of combination studies are underway or recently completed and that likely represent the next frontier in MDS therapy.
Sekeres
Mikkael A
MA
Department of Hematologic Oncology and Blood Disorders, Cleveland Clinic Taussig Cancer Institute, Cleveland, OH 44195, USA. sekerem@ccf.org
D001374:Azacitidine / Q000031:analogs & derivatives / Q000627:therapeutic use; D001565:Benzoates / Q000627:therapeutic use; D001803:Blood Transfusion; D001855:Bone Marrow Diseases / Q000139:chemically induced; D002872:Chromosome Deletion; D002895:Chromosomes, Human, Pair 5 / Q000648:ultrastructure; D002986:Clinical Trials as Topic / Q000706:statistics & numerical data; D003131:Combined Modality Therapy; D015257:DNA-Cytosine Methylases / Q000037:antagonists & inhibitors; D000077209:Decitabine; D005978:Glutathione / Q000031:analogs & derivatives / Q000627:therapeutic use; D006397:Hematinics / Q000009:adverse effects / Q000627:therapeutic use; D006801:Humans; D006834:Hydrazines / Q000627:therapeutic use; D007155:Immunologic Factors / Q000627:therapeutic use; D000077269:Lenalidomide; D009190:Myelodysplastic Syndromes / Q000145:classification / Q000188:drug therapy / Q000191:economics / Q000235:genetics / Q000628:therapy*; D011720:Pyrazoles / Q000627:therapeutic use; D016032:Randomized Controlled Trials as Topic / Q000706:statistics & numerical data; D011961:Receptors, Fc / Q000627:therapeutic use; D011993:Recombinant Fusion Proteins / Q000627:therapeutic use; D012306:Risk; D013792:Thalidomide / Q000009:adverse effects / Q000031:analogs & derivatives / Q000627:therapeutic use; D013926:Thrombopoietin / Q000627:therapeutic use
D001565:Benzoates; D006397:Hematinics; D006834:Hydrazines; D007155:Immunologic Factors; D011720:Pyrazoles; D011961:Receptors, Fc; D011993:Recombinant Fusion Proteins; C096329:gamma-Glu-S-BzCys-PhGly diethyl ester; D013792:Thalidomide; D000077209:Decitabine; D013926:Thrombopoietin; D015257:DNA-Cytosine Methylases; D000077269:Lenalidomide; D005978:Glutathione; C488777:romiplostim; D001374:Azacitidine; C520809:eltrombopag