Etanercept as Treatment of Steroid-Refractory Acute Graft-versus-Host Disease in Pediatric Patients

Maura Faraci, Maria Grazia Calevo, Stefano Giardino, Massimiliano Leoni, Erica Ricci, Elio Castagnola, Edoardo Lanino

Research output: Contribution to journalArticlepeer-review


Corticosteroids are the standard of care for first-line treatment of patients who develop grade II-IV of acute graft-versus-host disease (aGVHD), but the optimal second-line treatment has not yet been determined. We prospectively evaluated the use of the anti-TNFα monoclonal antibody etanercept (ET) as second-line treatment in children with steroid-refractory (SR) aGVHD. Twenty-five children with either malignant or nonmalignant diseases experiencing grade II-IV SR aGVHD received ET as second-line treatment. ET was administered after a median of 14days (range, 5 to 135 days) from the onset of aGVHD. Seventeen out of 25 patients (68%) developed a complete response (CR) or partial response (PR) to ET. The overall response rate (CR plus PR) was 78% in patients with cutaneous SR aGVHD, 78% in those with gastrointestinal aGVHD, and 57% in those with hepatic aGVHD. On day +100 after the start of ET, 52% of the children were in CR, 16% were in PR, and the remaining 32% failed to respond. Overall survival was 76.5% in responders and 16.7% in nonresponders (P = .004). Transplantation-related mortality at 5years was 34.1% (95% confidence interval, 18.6% to 57.1%). In our experience, ET has proven to be effective as second-line treatment in children with SR aGVHD.

Original languageEnglish
Pages (from-to)743-748
Number of pages6
JournalBiology of Blood and Marrow Transplantation
Issue number4
Publication statusPublished - Apr 2019


  • Acute Disease
  • Adolescent
  • Child
  • Child, Preschool
  • Etanercept/pharmacology
  • Female
  • Gastrointestinal Agents/pharmacology
  • Graft vs Host Disease/drug therapy
  • Humans
  • Infant
  • Infant, Newborn
  • Male


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