Myeloid/lymphoid neoplasms with <i>FGFR1</i> 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.
Article Details
Authors (3)
Alessandro M. Vannucchi
3Center for Research and Innovation of Myeloproliferative Neoplasms, AOU Careggi, University of Florence, Florence, Italy
Jay L. Patel
2Huntsman Cancer Institute and ARUP Laboratories, The University of Utah, Salt Lake City, UT
Jean-Jacques Kiladjian