Skip to main page content
U.S. flag

An official website of the United States government

Dot gov

The .gov means it’s official.
Federal government websites often end in .gov or .mil. Before sharing sensitive information, make sure you’re on a federal government site.

Https

The site is secure.
The https:// ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely.

Access keys NCBI Homepage MyNCBI Homepage Main Content Main Navigation
. 2014 Jun 5;123(23):3574-7.
doi: 10.1182/blood-2014-02-555607. Epub 2014 Mar 31.

Patients with myeloid malignancies bearing PDGFRB fusion genes achieve durable long-term remissions with imatinib

Chan Y Cheah  1 , Kate Burbury  1 , Jane F Apperley  2 , Francoise Huguet  3 , Vincenzo Pitini  4 , Martine Gardembas  5 , David M Ross  6 , Donna Forrest  7 , Philippe Genet  8 , Philippe Rousselot  9 , Nigel Patton  10 , Graeme Smith  11 , Cynthia E Dunbar  12 , Sawa Ito  12 , Ricardo C T Aguiar  13 , Olatoyosi Odenike  14 , Alla Gimelfarb  15 , Nicholas C P Cross  16 , John F Seymour  1
Affiliations

Patients with myeloid malignancies bearing PDGFRB fusion genes achieve durable long-term remissions with imatinib

Chan Y Cheah et al. Blood. .

Abstract

Myeloid neoplasms and eosinophilia with rearrangements of PDGFRB are uncommon Philadelphia-negative myeloproliferative neoplasms. Patients are typically male, with morphologic features of a Philadelphia-negative chronic myeloproliferative syndrome or chronic myelomonocytic leukemia with eosinophilia. Reciprocal translocations involving PDGFRB result in fusion genes with constitutively activated receptor tyrosine kinase sensitive to inhibition with imatinib. We present an updated and expanded analysis of a cohort of 26 such patients treated with imatinib. After a median follow-up of 10.2 years (range, 1.8-17 years), the 10-year overall survival rate was 90% (95% confidence interval, 64%-97%); after median imatinib duration of 6.6 years (range, 0.1-12 years), the 6-year progression-free survival rate was 88% (95% confidence interval, 65%-96%). Of the patients, 96% responded; no patients who achieved a complete cytogenetic (n = 13) or molecular (n = 8) remission lost their response or progressed to blast crisis. Imatinib is well-tolerated and achieves excellent long-term responses in patients with PDGFRB rearrangements.

PubMed Disclaimer

Figures

Figure 1
Figure 1
Progression-free survival of patients with PDGFRB gene rearrangements receiving imatinib.
See this image and copyright information in PMC

Comment in

References

    1. Havelange V, Demoulin JB. Review of current classification, molecular alterations, and tyrosine kinase inhibitor therapies in myeloproliferative disorders with hypereosinophilia. J Blood Med. 2013;4:111–121. - PMC - PubMed
    1. Arefi M, García JL, Peñarrubia MJ, et al. Incidence and clinical characteristics of myeloproliferative neoplasms displaying a PDGFRB rearrangement. Eur J Haematol. 2012;89(1):37–41. - PubMed
    1. Lengline E, Beldjord K, Dombret H, Soulier J, Boissel N, Clappier E. Successful tyrosine kinase inhibitor therapy in a refractory B-cell precursor acute lymphoblastic leukemia with EBF1-PDGFRB fusion. Haematologica. 2013;98(11):e146–e148. - PMC - PubMed
    1. Tokita K, Maki K, Tadokoro J, et al. Chronic idiopathic myelofibrosis expressing a novel type of TEL-PDGFRB chimaera responded to imatinib mesylate therapy. Leukemia. 2007;21(1):190–192. - PubMed
    1. Golub TR, Barker GF, Lovett M, Gilliland DG. Fusion of PDGF receptor beta to a novel ets-like gene, tel, in chronic myelomonocytic leukemia with t(5;12) chromosomal translocation. Cell. 1994;77(2):307–316. - PubMed

Publication types

MeSH terms