There is no consensus on the optimal treatment of
steroid-refractory acute
graft-versus-host disease (SR-aGVHD) after allogeneic
hematopoietic stem cell transplantation. In our center, the treatment policy has changed over time with
mycophenolate mofetil (MMF) being used from 1999 to 2003, and
etanercept or
inolimomab after 2004. An observational study compared survival and
infection rates in all consecutive patients receiving 1 of these 3 treatments. Ninety-three patients were included. The main end point was overall survival (OS). Median age was 37 years. Acute GVHD developed at a median of 15 days after
transplantation. Second-line treatment was initiated a median of 12 days after aGVHD diagnosis.
Therapies were MMF in 56%,
inolimomab in 22%, and
etanercept in 23% of the patients. Overall, second-line treatment response rate was 45% (complete response: 28%), MMF: 55%,
inolimomab: 35%, and
etanercept: 28%. With 74 months median follow-up, the 2-year survival was 30% (95% confidence interval: 22-41). Risk factors significantly associated with OS in multivariate analysis were disease status at
transplantation; grade III-IV aGVHD at second-line treatment institution; and liver involvement. None of the second-line
therapy influenced this poor outcome. Viral and
fungal infections were not statistically different among the 3 treatment options; however,
bacterial infections were more frequent in patients treated with anticytokines. Over an 11-year period, 3 treatment strategies, including 2 anticytokines, give similar results in patients with SR-aGVHD.