Phase Ib study of PRT543, an oral protein arginine methyltransferase 5 (PRMT5) inhibitor, in patients with advanced splicing factor-mutant myeloid malignancies Research Letter

   


Authors: Bewersdorf, J. P.; Mi, X.; Lu, B.; Kuykendall, A.; Sallman, D.; Patel, M.; Stevens, D.; Philipovskiy, A.; Sutamtewagul, G.; Masarova, L.; Keiffer, G.; Verma, A.; Bhagwat, N.; Wang, M.; Moore, A.; Rager, J.; Heiser, D.; Ro, S.; Hong, W. J.; Abdel-Wahab, O.; Stein, E. M.
Title: Phase Ib study of PRT543, an oral protein arginine methyltransferase 5 (PRMT5) inhibitor, in patients with advanced splicing factor-mutant myeloid malignancies
Abstract: (Figure presented.) © The Author(s) 2025.
Keywords: adult; clinical article; controlled study; human tissue; unclassified drug; gene mutation; clinical trial; constipation; fatigue; neutropenia; diarrhea; drug dose reduction; drug efficacy; drug safety; hypertension; hypophosphatemia; monotherapy; side effect; alpha interferon; letter; gene; dna repair; edema; multiple cycle treatment; anemia; nausea; thrombocytopenia; incidence; cohort analysis; mutational analysis; backache; coughing; dizziness; dyspnea; fever; pneumonia; myelodysplastic syndrome; bone marrow biopsy; multicenter study; diagnosis; headache; phase 1 clinical trial; spliceosome; drug therapy; ubiquitin protein ligase e3; epistaxis; therapy; myeloproliferative neoplasm; thrombocyte transfusion; oral drug administration; protein arginine methyltransferase; protein arginine methyltransferase 5; clinical trial (topic); petechia; peptides and proteins; acute myeloid leukemia; contusion; agents affecting metabolism; human; article; rna sequencing; long untranslated rna; sf3b1 gene; loss of appetite; rna splicing factor; gene set enrichment analysis; srsf2 gene; u2af1 gene; ademetionine; edema peripheral; c1d25 antigen; protein arginine methyltransferase 5 inhibitor; prt543; mib2 gene; sdma gene; snhg12 gene
Journal Title: Leukemia
Volume: 39
Issue: 3
ISSN: 0887-6924
Publisher: Nature Publishing Group  
Date Published: 2025-03-01
Start Page: 765
End Page: 769
Language: English
DOI: 10.1038/s41375-025-02515-8
PUBMED: 39856223
PROVIDER: scopus
PMCID: PMC11879867
DOI/URL:

https://www.scopus.com/inward/record.uri?eid=2-s2.0-85217267956&doi=10.1038%2fs41375-025-02515-8&partnerID=40&md5=fcff2f3246fed658a3e19e8cdc211f6c
Notes: Letter -- MSK Cancer Center Support Grant (P30 CA008748) acknowledged in PubMed and PDF -- MSK corresponding authors are Omar Abdel-Wahab and Eytan Stein -- Source: Scopus
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MSK Authors
  1. Eytan Moshe Stein
    342 Stein
  2. Omar Ibrahim Abdel-Wahab
    573 Abdel-Wahab
  3. Xiaoli Mi
    16 Mi
  4. Bin Lu
    6 Lu
  5. Jan Philipp Bewersdorf
    96 Bewersdorf
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