Pemigatinib for the treatment of myeloid/lymphoid neoplasms with FGFR1 rearrangement

ABSTRACT

Introduction

Myeloid/lymphoid neoplasms with fibroblast growth factor receptor-1 (FGFR1) rearrangements (MLN^FGFR1) are rare entities with aggressive features and poor prognosis. Presentation is heterogeneous, ranging from myeloproliferative neoplasms (with or without eosinophilia) to T-cell lymphoma and acute leukemia. Historical treatments have been guided by the presenting phenotype with induction chemotherapy frequently used. Pemigatinib is a FGFR1–3 tyrosine kinase inhibitor that has demonstrated high complete hematologic and cytogenetic response rates in MLN^FGFR1.

Areas covered

We discuss the pathogenesis, presentation, and historical treatments for MLN^FGFR1, in addition to clinical data using pemigatinib and other targeted therapies. Discussion of the mechanism of action and adverse events is also included.

Expert opinion

Pemigatinib represents a significant advance in the management of MLN^FGFR1. High rates of complete hematologic and cytogenetic response have been observed. While direct comparative data are unavailable, outcomes appear favorable compared to conventional approaches. Long-term efficacy and tolerability are not yet known, and allogeneic hematopoietic stem cell transplant (alloHSCT) continues to be the treatment with the highest chance of long-term disease free survival in responding patients. Combinations of pemigatinib and chemotherapy, particularly for more aggressive phenotypes, warrant future investigation as does the use of pemigatinib maintenance following alloHSCT.

KEYWORDS:

• Fibroblast growth factor receptor
• FGFR1 rearrangement
• myeloid/lymphoid neoplasm
• eosinophilia
• myeloproliferative neoplasm
• pemigatinib

Declaration of interest

The authors have no relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript. This includes employment, consultancies, honoraria, stock ownership or options, expert testimony, grants or patents received or pending, or royalties.

Reviewer disclosures

Peer reviewers on this manuscript have no relevant financial or other relationships to disclose.

Supplementary material

Supplemental data for this article can be accessed online at https://doi.org/10.1080/14737140.2023.2192930

Additional information

Funding

The authors have no funding to report.