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

Attilio Olivieri, Michele Cimminiello, Paolo Corradini, Nicola Mordini, Roberta Fedele, Carmine Selleri, Francesco Onida, Francesca Patriarca, Enzo Pavone, Silvia Svegliati, Armando Gabrielli, Paola Bresciani, Roberta Nuccorini, Sara Pascale, Sabrina Coluzzi, Fabrizio Pane, Antonella Poloni, Jacopo Olivieri, Pietro Leoni, Andrea Bacigalupo

Research output: Contribution to journalArticle

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 languageEnglish
Pages (from-to)4111-4118
Number of pages8
JournalBlood
Volume122
Issue number25
DOIs
Publication statusPublished - Dec 12 2013

ASJC Scopus subject areas

  • Hematology
  • Biochemistry
  • Cell Biology
  • Immunology

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    Olivieri, A., Cimminiello, M., Corradini, P., Mordini, N., Fedele, R., Selleri, C., Onida, F., Patriarca, F., Pavone, E., Svegliati, S., Gabrielli, A., Bresciani, P., Nuccorini, R., Pascale, S., Coluzzi, S., Pane, F., Poloni, A., Olivieri, J., Leoni, P., & Bacigalupo, A. (2013). Long-term outcome and prospective validation of NIH response criteria in 39 patients receiving imatinib for steroid-refractory chronic GVHD. Blood, 122(25), 4111-4118. https://doi.org/10.1182/blood-2013-05-494278