A randomized double-blind trial of hydroxychloroquine for the prevention of chronic graft-versus-host disease after allogeneic peripheral blood stem cell transplantation.
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.
Fong
Thomas
T
Division of Oncology, Section of Leukemia & Bone Marrow Transplantation, Siteman Cancer Center, Washington University School of Medicine, St. Louis, Missouri, USA.
Trinkaus
Kathryn
K
Adkins
Douglas
D
Vij
Ravi
R
Devine
Steven M
SM
Tomasson
Michael
M
Goodnough
Lawrence T
LT
Lopez
Sandra
S
Graubert
Timothy
T
Shenoy
Shalini
S
Dipersio
John F
JF
Khoury
Hanna J
HJ
D000328:Adult; D002908:Chronic Disease; D018572:Disease-Free Survival; D004311:Double-Blind Method; D005260:Female; D006086:Graft vs Host Disease / Q000145:classification / Q000517:prevention & control*; D019337:Hematologic Neoplasms / Q000628:therapy; D006801:Humans; D006886:Hydroxychloroquine / Q000627:therapeutic use*; D007166:Immunosuppressive Agents / Q000627:therapeutic use*; D053208:Kaplan-Meier Estimate; D008297:Male; D008875:Middle Aged; D036102:Peripheral Blood Stem Cell Transplantation / Q000379:methods*; D019172:Transplantation Conditioning / Q000379:methods; D014184:Transplantation, Homologous / Q000379:methods; D016896:Treatment Outcome
D007166:Immunosuppressive Agents; D006886:Hydroxychloroquine