title: Imatinib for refractory chronic graft-versus-host disease with fibrotic features.
abstract: We previously reported that patients with fibrotic, chronic graft-versus-host disease (cGVHD) have antibodies activating the platelet-derived growth factor receptor pathway. Because this pathway can be inhibited by imatinib, we performed a pilot study including 19 patients with refractory cGVHD, given imatinib at a starting dose of 100 mg per day. All patients had active cGVHD with measurable involvement of skin or other districts and had previously failed at least 2 treatment lines. Patient median age was 29 years (range, 10-62 years), and median duration of cGvHD was 37 months (range, 4-107 months). The organs involved were skin (n = 17), lung (n = 11), and bowel (n = 5); 15 patients had sicca syndrome. Imatinib-related, grade 3 to 4 toxicity included fluid retention, infections, and anemia. Imatinib was discontinued in 8 patients: in 3 because of toxicity and in 5 because of lack of response (n = 3) or relapse of malignancy (n = 2). Overall response rate at 6 months was 79%, with 7 complete remissions (CRs) and 8 partial remissions (PRs). With a median follow-up of 17 months, 16 patients are alive, 14 still in CR or PR. The 18-month probability of overall survival is 84%. This study suggests that imatinib is a promising treatment for patients with refractory fibrotic cGVHD.
authors.affiliation: Department of Hematology, San Carlo Hospital, via Potito Petrone 1, Potenza, Italy. attilio.olivieri@ospedalesancarlo.it
authors.forename: Attilio
authors.initials: A
authors.lastname: Olivieri
authors.forename: Franco
authors.initials: F
authors.lastname: Locatelli
authors.forename: Marco
authors.initials: M
authors.lastname: Zecca
authors.forename: Adele
authors.initials: A
authors.lastname: Sanna
authors.forename: Michele
authors.initials: M
authors.lastname: Cimminiello
authors.forename: Roberto
authors.initials: R
authors.lastname: Raimondi
authors.forename: Guido
authors.initials: G
authors.lastname: Gini
authors.forename: Nicola
authors.initials: N
authors.lastname: Mordini
authors.forename: Adriana
authors.initials: A
authors.lastname: Balduzzi
authors.forename: Pietro
authors.initials: P
authors.lastname: Leoni
authors.forename: Armando
authors.initials: A
authors.lastname: Gabrielli
authors.forename: Andrea
authors.initials: A
authors.lastname: Bacigalupo
mesh_terms: D000293:Adolescent; D000328:Adult; D001549:Benzamides; D002648:Child; D005260:Female; D005355:Fibrosis / Q000473:pathology*; D006086:Graft vs Host Disease / Q000150:complications / Q000188:drug therapy* / Q000401:mortality / Q000473:pathology; D006801:Humans; D000068877:Imatinib Mesylate; D007410:Intestinal Diseases; D008171:Lung Diseases; D008297:Male; D008875:Middle Aged; D010865:Pilot Projects; D010879:Piperazines / Q000008:administration & dosage* / Q000633:toxicity; D011743:Pyrimidines / Q000008:administration & dosage* / Q000633:toxicity; D012074:Remission Induction; D016879:Salvage Therapy / Q000379:methods*; D012871:Skin Diseases; D016019:Survival Analysis; D016896:Treatment Outcome
chemical_list: D001549:Benzamides; D010879:Piperazines; D011743:Pyrimidines; D000068877:Imatinib Mesylate