Phase I First-in-Human Dose Escalation Study of the oral SF3B1 modulator H3B-8800 in myeloid neoplasms

We conducted a phase I clinical trial of H3B-8800, an oral small molecule that binds Splicing Factor 3B1 (SF3B1), in patients with MDS, CMML, or AML. Among 84 enrolled patients (42 MDS, 4 CMML and 38 AML), 62 were red blood cell (RBC) transfusion dependent at study entry. Dose escalation cohorts exa...

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Detalles Bibliográficos
Autores principales: Steensma, David P., Wermke, Martin, Klimek, Virginia M., Greenberg, Peter L., Font, Patricia, Komrokji, Rami S., Yang, Jay, Brunner, Andrew M., Carraway, Hetty E., Ades, Lionel, Al-Kali, Aref, Alonso-Dominguez, Juan M., Alfonso-Piérola, Ana, Coombs, Catherine C., Deeg, H. Joachim, Flinn, Ian, Foran, James M., Garcia-Manero, Guillermo, Maris, Michael B., McMasters, Malgorzata, Micol, Jean-Baptiste, De Oteyza, Jaime Perez, Thol, Felicitas, Wang, Eunice S., Watts, Justin M., Taylor, Justin, Stone, Richard, Gourineni, Vikram, Marino, Alyssa J., Yao, Huilan, Destenaves, Benoit, Yuan, Xiaobin, Yu, Kun, Dar, Sara, Ohanjanian, Lernik, Kuida, Keisuke, Xiao, Jianjun, Scholz, Catherine, Gualberto, Antonio, Platzbecker, Uwe
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group UK 2021
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8632688/
https://www.ncbi.nlm.nih.gov/pubmed/34172893
http://dx.doi.org/10.1038/s41375-021-01328-9

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