Ex Vivo Drug Sensitivity Evaluation of a Rare ZMYM2::FGFR1+ Leukemia Patient

This study focused on a 36-year-old male patient with 8p11 myeloproliferative syndrome and a ZMYM2::FGFR1 fusion, who was treated with various drug regimens including Pemigatinib, Ponatinib, and Bortezomib. Ex vivo drug sensitivity assays revealed that Pemigatinib was effective, even as the patient developed resistance to Ponatinib, potentially due to an identified FGFR1 kinase domain mutation (FGFR1F686L; ZMYM2::FGFR1F1171L). Other FGFR inhibitors also showed high efficacy. The findings suggest Pemigatinib's potential as a treatment for ZMYM2::FGFR1 fusion-positive leukemia, though further research is needed to confirm its standalone effectiveness.