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Learn about a targeted therapy approved for lower- and higher-risk mIDH1 R/R MDS
Promote the growth and maturation of specific hematopoietic stem/progenitor cell (HSPC) lineages to address cytopenias.2
Target dysfunctional HSPCs to improve cytopenias and slow progression to AML.2,5
HSCT is the only curative option for eligible patients with high-risk MDS.7
Research in therapies targeting actionable mutations is ongoing.
Clinical trials evaluating various therapies are currently ongoing in previously untreated, R/R, high-risk MDS, and low-risk MDS, aiming to fulfill the unmet clinical needs of the disease.7
Discover active clinical trials for patients with mIDH1 MDS ›
AML, acute myeloid leukemia; IPSS-M, International Prognostic Scoring System-Molecular; MDS, myelodysplastic syndromes; RBC, red blood cell; R/R, relapsed or refractory.
References: 1. Sekeres M. Patient education: myelodysplastic syndromes (mds) in adults (beyond the basics). UpToDate. Updated November 2, 2022. Accessed July 19, 2023. 2. Sekeres MA, Taylor J. Diagnosis and treatment of myelodysplastic syndromes: a review. JAMA. 2022;328(9):872-880. doi:10.1001/jama.2022.14578 3. Kubasch AS, Platzbecker U. Setting fire to ESA and EMA resistance: new targeted treatment options in lower risk myelodysplastic syndromes. Int J Mol Sci. 2019;20(16):3853. doi:10.3390/ijms20163853 4. Referenced with permission from the NCCN Clinical Practice Guidelines in Oncology (NCCN Guidelines®) for Myelodysplastic Syndromes V.1.2023. © National Comprehensive Cancer Network, Inc. 2023. All rights reserved. Accessed August 10, 2023. To view the most recent and complete version of the guideline, go online to NCCN.org. 5. Platzbecker U. Treatment of MDS. Blood. 2019;133(10):1096-1107. doi:10.1182/blood-2018-10-844696 6. Steensma DP. Myelodysplastic syndromes current treatment algorithm 2018. Blood Cancer J. 2018;8(5):47. doi:https://doi.org/10.1038/s41408-018-0085-4 7. Pagliuca S, Gurnari C, Visconte V. Molecular targeted therapy in myelodysplastic syndromes: new options for tailored treatments. Cancers (Basel). 2021;13(4):784. doi:10.3390/cancers13040784 8. What is targeted therapy? Cancer.Net. Approved May 2022. Accessed July 19, 2023. https://www.cancer.net/navigating-cancer-care/how-cancer-treated/personalized-and-targeted-therapies/what-targeted-therapy 9. Nazha A, Sekeres MA, Gore SD, Zeidan AM. Molecular testing in myelodysplastic syndromes for the practicing oncologist: Will the progress fulfill the promise? Oncologist. 2015;20(9):1069-1076. doi:10.1634/theoncologist.2015-0067 10. Platzbecker U, Kubasch AS, Homer-Bouthiette C, Prebet T. Current challenges and unmet medical needs in myelodysplastic syndromes. Leukemia. 2021;35(8):2182-2198. doi:10.1038/s41375-021-01265-7