A randomized double-blind trial of hydroxychloroquine for the prevention of chronic graft-versus-host disease after allogeneic peripheral blood stem cell transplantation BIOLOGY OF BLOOD AND MARROW TRANSPLANTATION Fong, T., Trinkaus, A., Adkins, D., Vij, R., Devine, S. M., Tomasson, M., Goodnough, L. T., Lopez, S., Graubert, T., Shenoy, S., DiPersio, J. F., Khoury, H. Y. 2007; 13 (10): 1201-1206


Hydroxychloroquine (HCQ) is an immunosuppressive lysosomotropic amine that has activity against graft-versus-host disease (GVHD). In a single-institution phase III trial, 95 recipients of allogeneic peripheral blood stem cell (PBSC) transplantation were randomized to receive, in a double-blind fashion, and in addition to prophylactic cyclosporine A (CSA), HCQ, or placebo starting 21 days pretransplant and continued until day +365. HCQ was very well tolerated and not associated with side effects. Overall, the incidence of acute GVHD (aGVHD) was 59% in both arms, and severe aGVHD occurred in 11% (HCQ) and 14% (placebo) (P = .76). Sixty percent and 78% of patients developed chronic GVHD (cGVHD) in the HCQ and the placebo arms, respectively (P = .15). With a median follow-up of 18 months, relapse-free and overall survivals (OS) were comparable in both groups. In summary, in this randomized trial, the addition of HCQ to single-agent CSA had no effects on aGVHD or cGVHD or survival.

View details for DOI 10.1016/j.bbmt.2007.06.012

View details for Web of Science ID 000250103400010

View details for PubMedID 17889357