Journal
BLOOD REVIEWS
Volume 60, Issue -, Pages -Publisher
CHURCHILL LIVINGSTONE
DOI: 10.1016/j.blre.2023.101072
Keywords
Myelodysplastic syndrome; MDS; Genetics; Animal models; Immunology; Novel therapies
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Biological events in the pathogenesis of MDS are being characterized and translated into therapeutic strategies. Updates from the iwMDS provide advances in understanding the genetic landscape of MDS, as well as the development of novel therapies targeting specific molecular alterations and the innate immune system. However, more work is needed to develop individualized care for MDS patients.
Biological events that contribute to the pathogenesis of myelodysplastic syndromes/neoplasms (MDS) are becoming increasingly characterized and are being translated into rationally designed therapeutic strategies. Herein, we provide updates from the first International Workshop on MDS (iwMDS) of the International Consortium for MDS (icMDS) detailing recent advances in understanding the genetic landscape of MDS, including germline predisposition, epigenetic and immune dysregulation, the complexities of clonal hematopoiesis progression to MDS, as well as novel animal models of the disease. Connected to this progress is the development of novel therapies targeting specific molecular alterations, the innate immune system, and immune checkpoint anti-CD47 and anti-TIM3 antibodies, and cellular therapies), none have been approved for MDS. Additional preclinical and clinical work is needed to develop a truly individualized approach to the care of MDS patients.
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