Novel Treatment of Biologic and Molecular Targets for MDS-related Anemias and Higher Risk ... PDF
Preview Novel Treatment of Biologic and Molecular Targets for MDS-related Anemias and Higher Risk ...
Novel Treatment of Biologic and Molecular Targets for MDS-related Anemias and Higher Risk Disease Uwe Platzbecker D·MDS Medizinische Klinik und Poliklinik I Deutsche MDS-Studiengruppe Universitätsklinikum „Carl Gustav Carus“ Dresden, Germany Disclosure I have the following financial relationships: • Consultant: Amgen, Celgene, Janssen, Novartis • Research support: Amgen, Celgene, Janssen, Novartis MDS as a disease model of leukemogenesis Normal Low-risk MDS High-risk MDS AML years MDS as a disease model of leukemogenesis Normal Low-risk MDS High-risk MDS AML Genetic „Hit“ years Classification quantitative Normal Low-risk MDS High-risk MDS AML % blasts Molecular Classification Targets? Normal Low-risk MDS High-risk MDS AML Papaemmanuil et al. Blood 2013 Therapeutic principles Normal Low-risk MDS High-risk MDS AML Stimulation Suppression Therapy lower-risk MDS Normal Low-risk MDS High-risk MDS AML Erythropoiesis-stimulating agents (ESA) ESA Registration Trials in MDS EPO-a 40-80.000 E qw vs PLACEBO • IPSS low/int-1 R • EPO<500 • <=4 RBCs/8 W. Darbepoetin 500 q3w vs PLACEBO Fenaux et al. EHA 2016, Platzbecker et al. EHA 2016 Darbepoetin-trial Transfusions p = 0.008 4 100 3 100 7 2 - - 59.2% 0 5 3 sk 80 (29 / 49) s 80 e k 50.8% e e e W 60 36.1% W 60 (64 / 126) d (35 / 97) d e e s u 40 s 40 u f sn fs n a a rT 20 r 20 T % % 0 0 D arbepoetin alfa Placebo D arbepoetin alfa 2 4 -w e e k D o u b le -b lin d 4 8 -w e e k O p e n -la b e l Platzbecker et al. EHA 2016
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