Background Allogeneic hematopoietic stem cell transplantation (HSCT) is the first-line treatment for many haematological diseases. Graft-versus-host disease (GVHD) is the major complication of HSCT.

Steroids are the mainstay treatment of GVHD for reducing the systemic pro-inflammatory response. In about half of the patients, steroid treatment fails and they have to be treated with other immunosuppressant drugs. Of these, treatment with anti-TNF drugs such as etanercept is becoming an option for digestive manifestations of GVHD.

Purpose To describe etanercept use and effectiveness in steroid-refractory acute graft-versus-host disease, after hematopoietic cell transplantation.

Material and methods Patients treated with etanercept, an off-label use for steroid-refractory acute graft-versus-host disease, were selected and each patient’s medical history was reviewed to assess the clinical response.

Results The study included 5 patients: 4 presented with digestive manifestations and one 1 presented pulmonary and liver manifestations. 4 patients showed a clinical response: 3 of them a partial response and 1 a total response.

In 4 cases, etanercept 25 mg was administered twice a week with variable duration of treatment, achieving no response in 1 case (3 weeks), partial response in 2 cases (4 weeks and 8 weeks) and a complete response in 1 case (8 week period). Only one patient was treated with etanercept 50 mg administered twice a week for 5 weeks with a partial treatment response.

Conclusion Previously published data estimated the response rates at about 60–62%, which was consistent with our results. These update the scarce bibliographic information about etanercept use in steroid-refractory acute graft-versus-host disease. Due to clinical design limitations and the small patient population, further clinical studies should be conducted to assess the efficacy and safety of etanercept in these patients

References and/or Acknowledgements

1. Busca A, Locatelli F, Marmont F, et al. Response to mycophenolate mofetil therapy in refractory chronic graft-versus-host disease. Haematologica 2003;88(7):837–9.

References and/or AcknowledgementsNo conflict of interest.