Daclizumab, a humanized MoAB to IL-2Ra, has been found to be safe and effective in adults with refractory GvHD; however, data in children are limited. The aim of this prospective study was to evaluate the long-term safety and efficacy of daclizumab in children with steroid-refractory GI aGvHD. This study included 13 children who developed steroid-refractory GI GvHD between 2007 and 2009. When first-line treatment failed, daclizumab was given in a regimen of 1 mg/kg intravenously and then repeated on a 10- to 14-day interval for maximum five doses if necessary. Daclizumab was well tolerated, but infections were common. Ten patients responded to daclizumab completely, one patient responded partially, and two patients failed to respond. With a median follow-up of 630 days, 10 patients were alive and free of severe infections, but among them, four patients were suffering from cGvHD. Of the three remaining patients, one died because of bacterial meningitis, and the other two patients died because of severe refractory GI GvHD. This long-term evaluation showed that daclizumab could be an effective and relatively safe treatment in most of the pediatric patients with severe steroid-refractory GI GvHD.
© 2012 John Wiley & Sons A/S.