Abstract
Forty adults aged 28 to 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 National Institutes of Health (NIH) criteria and NIH severity score changes. The best responses were seen in the lungs, gut, and skin (35%, 50%, and 32%, respectively). After a median follow-up of 40 months, 28 patients were alive, with a 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 nonresponders 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, whereas it remained high in 4 nonresponders. This study confirms the efficacy of imatinib against SR-cGVHD and suggests that the response at 6 months significantly predicts long-term survival.
| Original language | English |
|---|---|
| Pages (from-to) | 4111-4118 |
| Number of pages | 8 |
| Journal | Blood |
| Volume | 122 |
| Issue number | 25 |
| DOIs | |
| Publication status | Published - Dec 12 2013 |
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ASJC Scopus subject areas
- Hematology
- Biochemistry
- Cell Biology
- Immunology
Cite this
Long-term outcome and prospective validation of NIH response criteria in 39 patients receiving imatinib for steroid-refractory chronic GVHD. / Olivieri, Attilio; Cimminiello, Michele; Corradini, Paolo; Mordini, Nicola; Fedele, Roberta; Selleri, Carmine; Onida, Francesco; Patriarca, Francesca; Pavone, Enzo; Svegliati, Silvia; Gabrielli, Armando; Bresciani, Paola; Nuccorini, Roberta; Pascale, Sara; Coluzzi, Sabrina; Pane, Fabrizio; Poloni, Antonella; Olivieri, Jacopo; Leoni, Pietro; Bacigalupo, Andrea.
In: Blood, Vol. 122, No. 25, 12.12.2013, p. 4111-4118.Research output: Contribution to journal › Article
}
TY - JOUR
T1 - Long-term outcome and prospective validation of NIH response criteria in 39 patients receiving imatinib for steroid-refractory chronic GVHD
AU - Olivieri, Attilio
AU - Cimminiello, Michele
AU - Corradini, Paolo
AU - Mordini, Nicola
AU - Fedele, Roberta
AU - Selleri, Carmine
AU - Onida, Francesco
AU - Patriarca, Francesca
AU - Pavone, Enzo
AU - Svegliati, Silvia
AU - Gabrielli, Armando
AU - Bresciani, Paola
AU - Nuccorini, Roberta
AU - Pascale, Sara
AU - Coluzzi, Sabrina
AU - Pane, Fabrizio
AU - Poloni, Antonella
AU - Olivieri, Jacopo
AU - Leoni, Pietro
AU - Bacigalupo, Andrea
PY - 2013/12/12
Y1 - 2013/12/12
N2 - Forty adults aged 28 to 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 National Institutes of Health (NIH) criteria and NIH severity score changes. The best responses were seen in the lungs, gut, and skin (35%, 50%, and 32%, respectively). After a median follow-up of 40 months, 28 patients were alive, with a 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 nonresponders 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, whereas it remained high in 4 nonresponders. This study confirms the efficacy of imatinib against SR-cGVHD and suggests that the response at 6 months significantly predicts long-term survival.
AB - Forty adults aged 28 to 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 National Institutes of Health (NIH) criteria and NIH severity score changes. The best responses were seen in the lungs, gut, and skin (35%, 50%, and 32%, respectively). After a median follow-up of 40 months, 28 patients were alive, with a 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 nonresponders 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, whereas it remained high in 4 nonresponders. This study confirms the efficacy of imatinib against SR-cGVHD and suggests that the response at 6 months significantly predicts long-term survival.
UR - http://www.scopus.com/inward/record.url?scp=84891318859&partnerID=8YFLogxK
UR - http://www.scopus.com/inward/citedby.url?scp=84891318859&partnerID=8YFLogxK
U2 - 10.1182/blood-2013-05-494278
DO - 10.1182/blood-2013-05-494278
M3 - Article
VL - 122
SP - 4111
EP - 4118
JO - Blood
JF - Blood
SN - 0006-4971
IS - 25
ER -