March 6, 2026
View Full PaperMyeloid/Lymphoid Neoplasms with FGFR1 Rearrangement and Pemigatinib
Key Points
- This research aims to evaluate the efficacy of pemigatinib in treating myeloid/lymphoid neoplasms with FGFR1 rearrangement.
- Reviewed clinical data on pemigatinib's effects on M/LN-FGFR1 neoplasms
- Assessed survival rates with pemigatinib compared to conventional therapies
- Summarized outcomes leading to pemigatinib's approval for treatment
- Pemigatinib showed unprecedented efficacy in treating M/LN-FGFR1 neoplasms
- Survival rates improved significantly compared to conventional therapies
- Efficacy observed in various presentations of the disease, including acute leukemia and lymphoma
Abstract
Myeloid/Lymphoid neoplasms with FGFR1 rearrangement (M/LN-FGFR1) are rare, heterogenous diseases due to fusion transcripts originated by translocations of FGFR1 with different partners, resulting in constitutive FGFR1-mediated signaling. Presentation varies from chronic myeloid neoplasms to acute leukemia or lymphoma and extramedullary localizations are common. Outside allogeneic stem cell transplantation (ASCT), survival with conventional therapy is dismal, representing an unmet clinical need. We summarize here the data that led to approval of pemigatinib, a FGFR1 inhibitor, showing unprecedented efficacy in M/LN-FGFR1.