Promising Outcomes of FGFR1 Inhibitor Pemigatinib for Myeloid Lymphoid Neoplasms

Daniel DeAngelo, MD, PhD, Dana-Farber Cancer Institute, Boston, Massachusetts, presents data on the promising outcomes of pemigatinib, a fibroblast growth factor receptor (FGFR1) inhibitor, among patients with myeloid lymphoid neoplasms, which he presented at the 2023 Great Debates & Updates in Hematologic Malignancies meeting in Boston, Massachusetts. 

Transcript:

Hi, my name is Dan DeAngelo. I'm chief of the division of leukemia at the Dana-Farber Cancer Institute in Boston. I'm currently at the Great Debates and Updates [in Hematologic Malignancies] in Boston. I'm going to be presenting data on pemigatinib, which is a first-in-class FGFR1 inhibitor. 

FGFR1 is a tyrosine kinase [inhibitor] that has many partners, and it induces an unusual lymphoid myeloid neoplasm where it has features of both myeloid as well as lymphoid. Patients can present both in a chronic phase, that is without a lot of blasts, or in a blast phase—very commonly lymphoid blast, but not always. These can be very difficult to treat [in] both chronic phase and blast phase. Without stem cell transplantation, patients have a very shortened survival.

Pemigatinib, which is an FGFR1 inhibitor, has been used to treat both the chronic as well as the advanced phase forms of these myeloid lymphoid neoplasms with FGFR1 rearrangements. Again, all types of rearrangements have been treated. Robust responses, both histologic responses and cytogenetic responses, have been seen in patients with chronic phase FGFR1 translocations. 

But even so, in patients with blast phase or patients with extramedullary disease, responses have also been seen—and responses durable enough to allow patients to go to a potentially curative stem cell transplant. This has opened up [a] really nice option for patients with a rare disease. Again, FGFR1 [for] myeloid lymphoid neoplasms.

Source: 

DeAngelo, D. Orphan Hematologic Malignancies-Systemic Mastocytosis and Myeloid-Lymphoid Neoplasms: Classification, Diagnosis, Genetics and Targeted Therapeutic Options. Presented at the Great Debates and Updates in Hematologic Malignancies Meeting; August 17-19, 2023; Boston, Massachusetts.