The present invention belongs to the field of
medicine and discloses the use of a CSF1R inhibitor in preparing a
drug for treating or alleviating
myelofibrosis. At the same time, the present invention also discloses a
drug for treating or alleviating
myelofibrosis. ‑ / ‑ miR‑146a ‑ / ‑ A del(5q) MDS mouse model constructed by DKO mice was established. Treatment with the CSF1R inhibitor PLX3397 demonstrated the following positive effects: PLX3397 alleviated
anemia in DKO mice, significantly reduced the number of fibrocytes in their
peripheral blood and
spleen, and decreased reticular and
collagen fiber deposition in the
bone marrow, effectively improving
myelofibrosis. Furthermore, PLX3397 reduced the clonogenic capacity of DKO mice in their
peripheral blood and
spleen and prolonged their survival. Flow cytometric analysis demonstrated that PLX3397 treatment increased the number of early erythroid
progenitor cells and LSK cells in the
bone marrow of DKO mice, partially ameliorating their hematopoietic defects. Overall, this study reveals the potential
efficacy of PLX3397 in treating MDS mouse models and provides a new approach for the
clinical treatment and alleviation of myelofibrosis caused by MDS.