title: Randomized trial of hydroxychloroquine for newly diagnosed chronic graft-versus-host disease in children: a Children's Oncology Group study.
abstract: The Children's Oncology Group conducted a multicenter Phase III trial for chronic graft-versus-host disease (cGVHD). The double-blind, placebo-controlled, randomized study evaluated hydroxychloroquine added to standard therapy for children with newly diagnosed cGVHD. The study also used a novel grading and response scoring system and evaluated clinical laboratory correlates of cGVHD. The primary endpoint was complete response (CR) after 9 months of therapy. Fifty-four patients (27 on each arm) were enrolled before closure because of slow accrual. The CR rate was 28% in the hydroxychloroquine arm versus 33% in the placebo arm (odds ratio [OR] = 0.77, 95% confidence interval [CI]: 0.20-2.93, P = .75) for 42 evaluable patients. For 41 patients with severity assessment at enrollment, 20 (49%) were severe and 18 (44%) moderate according to the National Institutes of Health Consensus Conference global scoring system. The CR rate was 15% for severe cGVHD and 44% for moderate cGVHD (OR = 0.24, 95% CI: 0.05-1.06, P = .07). Although the study could not resolve the primary question, it provided important information for future cGVHD study design in this population.
authors.affiliation: Levine Children's Hospital, Charlotte, North Carolina 28232, USA. andrew.gilman@carolinashealthcare.org
authors.forename: Andrew L
authors.initials: AL
authors.lastname: Gilman
authors.forename: Kirk R
authors.initials: KR
authors.lastname: Schultz
authors.forename: Frederick D
authors.initials: FD
authors.lastname: Goldman
authors.forename: George E
authors.initials: GE
authors.lastname: Sale
authors.forename: Mark D
authors.initials: MD
authors.lastname: Krailo
authors.forename: Zhengjia
authors.initials: Z
authors.lastname: Chen
authors.forename: Bryan
authors.initials: B
authors.lastname: Langholz
authors.forename: David A
authors.initials: DA
authors.lastname: Jacobsohn
authors.forename: Ka-Wah
authors.initials: KW
authors.lastname: Chan
authors.forename: Robin E
authors.initials: RE
authors.lastname: Ryan
authors.forename: Michael
authors.initials: M
authors.lastname: Kellick
authors.forename: Steven M
authors.initials: SM
authors.lastname: Neudorf
authors.forename: Kamar
authors.initials: K
authors.lastname: Godder
authors.forename: Eric S
authors.initials: ES
authors.lastname: Sandler
authors.forename: Indira
authors.initials: I
authors.lastname: Sahdev
authors.forename: Stephan A
authors.initials: SA
authors.lastname: Grupp
authors.forename: Jean E
authors.initials: JE
authors.lastname: Sanders
authors.forename: Donna A
authors.initials: DA
authors.lastname: Wall
mesh_terms: D000293:Adolescent; D000328:Adult; D002648:Child; D002675:Child, Preschool; D002908:Chronic Disease; D004311:Double-Blind Method; D005260:Female; D006086:Graft vs Host Disease / Q000175:diagnosis / Q000188:drug therapy*; D006801:Humans; D006886:Hydroxychloroquine / Q000627:therapeutic use*; D007223:Infant; D008297:Male; D016896:Treatment Outcome; D055815:Young Adult
chemical_list: D006886:Hydroxychloroquine