Imatinib for refractory chronic graft-versus-host disease with fibrotic features

Blood - Tập 114 - Trang 709-718 - 2009
Attilio Olivieri1, Franco Locatelli2, Marco Zecca2, Adele Sanna3, Michele Cimminiello1, Roberto Raimondi4, Guido Gini5, Nicola Mordini6, Adriana Balduzzi7, Pietro Leoni5, Armando Gabrielli8, Andrea Bacigalupo9
1Department of Hematology, San Carlo Hospital, Potenza
2Pediatric Hematology/Oncology, Fondazione Instituto Di Ricerca e Cura a Caratiere Scientifica, Policlinico San Matteo, University of Pavia, Pavia
3Unit of Bone Marrow Transplantation, Ospedale Regionale per le Microcitemie, Cagliari
4Department of Hematology, St Bortolo Hospital, Vicenza
5Department of Hematology, Università Politecnica delle Marche, Ancona
6Department of Hematology, Santa Croce e Carle Hospital, Cuneo
7Department of Pediatric Hematology, San Gerardo Hospital, Monza
8Department of Internal Medicine, Università Politecnica delle Marche, Ancona
9Hematology Unit and Stem Cell Transplant Unit San Martino Hospital, Genova, Italy

Tóm tắt

AbstractWe 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.

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