Journal
BLOOD
Volume 114, Issue 3, Pages 709-718Publisher
AMER SOC HEMATOLOGY
DOI: 10.1182/blood-2009-02-204156
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Funding
- AIRC (Associazione Italiana Ricerca sul Cancro)
- CNR (Consiglio Nazionale delle Ricerche)
- MURST (Ministero dell'Universita e della Ricerca Scientifica e Tecnologica)
- European Union
- Fondazione IRCCS Policlinico San Matteo
- Azienda Ospedaliera San Carlo
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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.(Blood. 2009;114:709-718)
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