Topical thalidomide gel in oral chronic GVHD and role of in situ cytokine expression in monitoring biological activity

Chronic graft-versus-host disease (cGVHD) is a major complication for nearly half of allogeneic haematopoietic stem cell transplantation (HSCT) survivors.1 Chronic GVHD derives from and is primarily mediated by grafted-immunocompetent T cells that proliferate in the recipients and reject host cells as foreign protein, resulting in multi-organ dysfunction. Approximately 80% of patients with cGVHD have oral involvement characterized by erythema, lichen planus, hyperkeratotic plaques, pseudomembranes and ulcers.2 Oral cGVHD-related pain is reported frequently, and oral dryness has been associated with inferior health-related quality of life. Thalidomide has shown efficacy and safety with minimal systemic absorption for treatment of aphthous stomatitis in HIV (+) patients.3 Here we report preliminary data for topical thalidomide gel in the management of oral ulcerative cGVHD.