TY - JOUR
T1 - Myeloid/lymphoid neoplasms with FGFR1 rearrangement
AU - Strati, Paolo
AU - Tang, Guilin
AU - Duose, Dzifa Y.
AU - Mallampati, Saradhi
AU - Luthra, Rajyalakshmi
AU - Patel, Keyur P.
AU - Hussaini, Mohammad
AU - Mirza, Abu Sayeef
AU - Komrokji, Rami S.
AU - Oh, Stephen
AU - Mascarenhas, John
AU - Najfeld, Vesna
AU - Subbiah, Vivek
AU - Kantarjian, Hagop
AU - Garcia-Manero, Guillermo
AU - Verstovsek, Srdan
AU - Daver, Naval
N1 - Publisher Copyright:
© 2017, © 2017 Informa UK Limited, trading as Taylor & Francis Group.
PY - 2018/7/3
Y1 - 2018/7/3
N2 - Myeloid/lymphoid neoplasms with FGFR1 rearrangement are a rare entity. We present a multicenter experience of 17 patients with FISH-confirmed FGFR1 rearrangement. The clinical presentation at diagnosis included myeloproliferative neoplasm (MPN) in 4 (24%) patients, acute leukemia (AL) in 7 (41%), and concomitant MPN with AL in 6 (35%). The two most frequently observed cytogenetic abnormalities were t(8;13)(p11.2;q12)(partner gene ZMYM2) and t(8;22)(p11.2; q11.2)(BCR). Seventy-eight percent of tested patients had a RUNX1 mutation, of whom all had AL. Overall response rate to frontline therapy was 69%, and 76% of patients subsequently received allogeneic stem cell transplant (ASCT). After a median follow-up of 11 months, median progression-free survival was 15 months and median overall survival was not reached. In conclusion, FGFR1-rearranged hematologic malignancies present with features of MPN and/or AL. FGFR1 and RUNX1 are therapeutic targets for ongoing and future clinical trials.
AB - Myeloid/lymphoid neoplasms with FGFR1 rearrangement are a rare entity. We present a multicenter experience of 17 patients with FISH-confirmed FGFR1 rearrangement. The clinical presentation at diagnosis included myeloproliferative neoplasm (MPN) in 4 (24%) patients, acute leukemia (AL) in 7 (41%), and concomitant MPN with AL in 6 (35%). The two most frequently observed cytogenetic abnormalities were t(8;13)(p11.2;q12)(partner gene ZMYM2) and t(8;22)(p11.2; q11.2)(BCR). Seventy-eight percent of tested patients had a RUNX1 mutation, of whom all had AL. Overall response rate to frontline therapy was 69%, and 76% of patients subsequently received allogeneic stem cell transplant (ASCT). After a median follow-up of 11 months, median progression-free survival was 15 months and median overall survival was not reached. In conclusion, FGFR1-rearranged hematologic malignancies present with features of MPN and/or AL. FGFR1 and RUNX1 are therapeutic targets for ongoing and future clinical trials.
KW - FGFR1 rearrangement
KW - Myeloproliferative neoplasm
KW - RUNX1
KW - acute leukemia
UR - http://www.scopus.com/inward/record.url?scp=85033693826&partnerID=8YFLogxK
U2 - 10.1080/10428194.2017.1397663
DO - 10.1080/10428194.2017.1397663
M3 - Article
C2 - 29119847
AN - SCOPUS:85033693826
SN - 1042-8194
VL - 59
SP - 1672
EP - 1676
JO - Leukemia and Lymphoma
JF - Leukemia and Lymphoma
IS - 7
ER -