title: Patients with myeloid malignancies bearing PDGFRB fusion genes achieve durable long-term remissions with imatinib.
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.
authors.affiliation: Department of Haematology, Peter MacCallum Cancer Centre, East Melbourne, VIC, Australia; University of Melbourne, Parkville, VIC, Australia;
authors.forename: Chan Y
authors.initials: CY
authors.lastname: Cheah
authors.affiliation: Department of Haematology, Peter MacCallum Cancer Centre, East Melbourne, VIC, Australia; University of Melbourne, Parkville, VIC, Australia;
authors.forename: Kate
authors.initials: K
authors.lastname: Burbury
authors.affiliation: Department of Haematology, Faculty of Medicine, Imperial College London, London, United Kingdom;
authors.forename: Jane F
authors.initials: JF
authors.lastname: Apperley
authors.affiliation: Department of Haematology, Centre Hospitalier Universitaire, Toulouse, France;
authors.forename: Francoise
authors.initials: F
authors.lastname: Huguet
authors.affiliation: Oncologia Medica e Trapianto di Midollo Osseo, Messina, Italy;
authors.forename: Vincenzo
authors.initials: V
authors.lastname: Pitini
authors.affiliation: Department of Haematology, Centre Hospitalier Universitaire, Angers, France;
authors.forename: Martine
authors.initials: M
authors.lastname: Gardembas
authors.affiliation: Haematology, South Australia Pathology, Adelaide, SA, Australia; School of Medicine, University of Adelaide, Adelaide, SA, Australia; School of Medicine, Flinders University, Adelaide, SA, Australia;
authors.forename: David M
authors.initials: DM
authors.lastname: Ross
authors.affiliation: British Columbia Cancer Agency, Vancouver, BC, Canada;
authors.forename: Donna
authors.initials: D
authors.lastname: Forrest
authors.affiliation: Department of Haematology, Hôpital Victor Dupouy, Argenteuil, France;
authors.forename: Philippe
authors.initials: P
authors.lastname: Genet
authors.affiliation: Department of Haematology, Hôpital de Versailles and Université de Versailles Saint-Quentin, Le Chesnay, France;
authors.forename: Philippe
authors.initials: P
authors.lastname: Rousselot
authors.affiliation: Department of Haematology, Auckland City Hospital, Auckland, New Zealand;
authors.forename: Nigel
authors.initials: N
authors.lastname: Patton
authors.affiliation: Department of Haematology, St. James's University Hospital, Leeds, United Kingdom;
authors.forename: Graeme
authors.initials: G
authors.lastname: Smith
authors.affiliation: Hematology Branch, National Heart, Lung and Blood Institute, National Institutes of Health, Bethesda, MD;
authors.forename: Cynthia E
authors.initials: CE
authors.lastname: Dunbar
authors.affiliation: Hematology Branch, National Heart, Lung and Blood Institute, National Institutes of Health, Bethesda, MD;
authors.forename: Sawa
authors.initials: S
authors.lastname: Ito
authors.affiliation: Department of Medicine, University of Texas Health Science Center, San Antonio, TX; Audie Murphy Veterans Affairs Hospital, San Antonio, TX;
authors.forename: Ricardo C T
authors.initials: RC
authors.lastname: Aguiar
authors.affiliation: Department of Medicine, Section of Hematology/Oncology, University of Chicago, Chicago, IL;
authors.forename: Olatoyosi
authors.initials: O
authors.lastname: Odenike
authors.affiliation: Northshore University Health Systems, Chicago, IL;
authors.forename: Alla
authors.initials: A
authors.lastname: Gimelfarb
authors.affiliation: Faculty of Medicine, University of Southampton, Southampton, United Kingdom.
authors.forename: Nicholas C P
authors.initials: NC
authors.lastname: Cross
authors.affiliation: Department of Haematology, Peter MacCallum Cancer Centre, East Melbourne, VIC, Australia; University of Melbourne, Parkville, VIC, Australia;
authors.forename: John F
authors.initials: JF
authors.lastname: Seymour
mesh_terms: D000293:Adolescent; D000328:Adult; D000368:Aged; D000970:Antineoplastic Agents / Q000627:therapeutic use*; D001549:Benzamides / Q000627:therapeutic use*; D002648:Child; D002675:Child, Preschool; D004802:Eosinophilia / Q000188:drug therapy / Q000453:epidemiology / Q000235:genetics; D005260:Female; D006801:Humans; D000068877:Imatinib Mesylate; D007223:Infant; D007951:Leukemia, Myeloid / Q000188:drug therapy* / Q000235:genetics*; D008297:Male; D008875:Middle Aged; D015514:Oncogene Proteins, Fusion / Q000235:genetics*; D010879:Piperazines / Q000627:therapeutic use*; D011743:Pyrimidines / Q000627:therapeutic use*; D020797:Receptor, Platelet-Derived Growth Factor beta / Q000235:genetics*; D012074:Remission Induction; D014178:Translocation, Genetic; D055815:Young Adult
chemical_list: D000970:Antineoplastic Agents; D001549:Benzamides; D015514:Oncogene Proteins, Fusion; D010879:Piperazines; D011743:Pyrimidines; D000068877:Imatinib Mesylate; D020797:Receptor, Platelet-Derived Growth Factor beta