Randomized trial of hydroxychloroquine for newly diagnosed chronic graft-versus-host disease in children: A Children's Oncology Group study Journal Article
| Authors: | Gilman, A. L.; Schultz, K. R.; Goldman, F. D.; Sale, G. E.; Krailo, M. D.; Chen, Z.; Langholz, B.; Jacobsohn, D. A.; Chan, K. W.; Ryan, R. E.; Kellick, M.; Neudorf, S. M.; Godder, K.; Sandler, E. S.; Sahdev, I.; Grupp, S. A.; Sanders, J. E.; Wall, D. A. |
| Article 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. © 2012 American Society for Blood and Marrow Transplantation. |
| Keywords: | adolescent; adult; child; clinical article; controlled study; preschool child; school child; treatment outcome; treatment response; child, preschool; young adult; placebo; hypertension; infection; randomized controlled trial; hyperglycemia; chronic disease; chronic graft versus host disease; disease severity; infant; scoring system; hydroxychloroquine; gvhd; graft vs host disease; double blind procedure; double-blind method; eye toxicity; randomized; avascular necrosis; hypertransaminasemia; chronic graft-versus-host disease |
| Journal Title: | Biology of Blood and Marrow Transplantation |
| Volume: | 18 |
| Issue: | 1 |
| ISSN: | 1083-8791 |
| Publisher: | Elsevier Inc. |
| Date Published: | 2012-01-01 |
| Start Page: | 84 |
| End Page: | 91 |
| Language: | English |
| DOI: | 10.1016/j.bbmt.2011.05.016 |
| PROVIDER: | scopus |
| PUBMED: | 21689773 |
| PMCID: | PMC4627789 |
| DOI/URL: | |
| Notes: | --- - "Export Date: 1 March 2012" - "CODEN: BBMTF" - "Source: Scopus" |
