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.
Myeloid/Lymphoid Neoplasms with FGFR1 Rearrangement and Pemigatinib / Vannucchi, Alessandro M.; Patel, Jay L; Kiladjian, Jean-Jacques. - In: BLOOD. - ISSN 0006-4971. - ELETTRONICO. - (2026), pp. 1-18. [10.1182/blood.2025031016]
Myeloid/Lymphoid Neoplasms with FGFR1 Rearrangement and Pemigatinib
Vannucchi, Alessandro M.;
2026
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.
| File | Dimensione | Formato | |
|---|---|---|---|
|
1-s2.0-S0006497126005276-main.pdf
Accesso chiuso
Tipologia:
Pdf editoriale (Version of record)
Licenza:
Tutti i diritti riservati
Dimensione
1.51 MB
Formato
Adobe PDF
|
1.51 MB | Adobe PDF | Richiedi una copia |
I documenti in FLORE sono protetti da copyright e tutti i diritti sono riservati, salvo diversa indicazione.
