Daclizumab therapy for children with corticosteroid-resistant acute graft-vs.-host disease

MAbs such as daclizumab have shown promising results in the treatment of corticosteroid-resistant aGVHD. Data describing the efficacy of MAbs in children are limited. We describe the efficacy of daclizumab in children with corticosteroid-resistant aGVHD. Seventeen children (0.416.2 yr) received daclizumab for aGVHD. Safety was evaluated in all 17. Response was evaluated in the 15 children who received a full course of daclizumab. A PR was defined as improvement of aGVHD symptoms in at least one organ without worsening in other organs. We observed an overall response rate (CR plus PR) of 40% (6/15). Four children had a CR and two had a PR. aGVHD involving the gut had the highest response rate (five of 10; 50%). Adverse effects attributed to daclizumab were limited to an episode of reactive arthritis. Mortality was high (47%; eight of 17) though five of the six responders to daclizumab survived. The mean length of follow-up in the surviving nine patients was 32 months (range seven to 48). We conclude that daclizumab is effective in the treatment of children with corticosteroidresistant aGVHD, especially in patients with gut involvement.