Myeloid/lymphoid neoplasms with FGFR1 rearrangement and pemigatinib
Abstract
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, 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.
Fields of Study & Subjects
Eosinophilic Disorders and Syndromes
Fibroblast Growth Factor Research
Chronic Myeloid Leukemia Treatments
Publication details
- Journal / source
- Blood
- Publisher
- American Society of Hematology
- Volume
- 147
- Issue
- 19
- Pages
- 2171–2175
- Publication date
- 4 March 2026
- Language
- EN