Forty adults aged 28-73 years were entered into a prospective trial of imatinib for the treatment of steroid-refractory chronic graft-versus-host disease (SR-cGVHD). After 6 months, intention-to-treat (ITT) analysis of 39 patients who received the drug, regardless of the duration of treatment, revealed 14 partial responses (PR), 4 minor responses (MR) with relevant steroid sparing (46%) according to Couriel criteria, and 20 ≥PR (51.3%), as per the NIH criteria and NIH severity score changes. The best responses were seen in the lungs, guts, and skin (35%, 50%, and 32%, respectively). After a median follow-up of 40 (range, 12-54) months, 28 patients were alive, with 3-year overall survival (OS) and event-free survival of 72% and 46%, respectively. The 3-year OS was 94% for patients responding at 6 months and 58% for non-responders according to NIH response, suggesting that these criteria represent a reliable tool for predicting OS after second-line treatment. Monitoring of anti-platelet-derived growth factor receptor (PDGF-R) antibodies showed a significant decrease in PDGF-R stimulatory activity in 7 responders, while it remained high in 4 non-responders. In conclusion, this study confirms the efficacy of imatinib against SR-cGVHD and suggests that the response at 6 months significantly predicts long-term survival. This study is registered at https://eudract.ema.europa.eu/, EUDRACT number: 2009-012927-27.

Long-term outcome and prospective validation of NIH response criteria in 39 patients receiving imatinib for steroid-refractory chronic GVHD.

SELLERI, Carmine;
2013-01-01

Abstract

Forty adults aged 28-73 years were entered into a prospective trial of imatinib for the treatment of steroid-refractory chronic graft-versus-host disease (SR-cGVHD). After 6 months, intention-to-treat (ITT) analysis of 39 patients who received the drug, regardless of the duration of treatment, revealed 14 partial responses (PR), 4 minor responses (MR) with relevant steroid sparing (46%) according to Couriel criteria, and 20 ≥PR (51.3%), as per the NIH criteria and NIH severity score changes. The best responses were seen in the lungs, guts, and skin (35%, 50%, and 32%, respectively). After a median follow-up of 40 (range, 12-54) months, 28 patients were alive, with 3-year overall survival (OS) and event-free survival of 72% and 46%, respectively. The 3-year OS was 94% for patients responding at 6 months and 58% for non-responders according to NIH response, suggesting that these criteria represent a reliable tool for predicting OS after second-line treatment. Monitoring of anti-platelet-derived growth factor receptor (PDGF-R) antibodies showed a significant decrease in PDGF-R stimulatory activity in 7 responders, while it remained high in 4 non-responders. In conclusion, this study confirms the efficacy of imatinib against SR-cGVHD and suggests that the response at 6 months significantly predicts long-term survival. This study is registered at https://eudract.ema.europa.eu/, EUDRACT number: 2009-012927-27.
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Utilizza questo identificativo per citare o creare un link a questo documento: https://hdl.handle.net/11386/4185453
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