RESUMO
High-dose methotrexate (MTX) given in four hospitalizations during interim maintenance for high-risk pediatric B-lineage acute lymphocytic leukemia significantly improves survival but increases resource utilization. Children remain hospitalized for intravenous hydration and blood or urine monitoring until MTX clearance parameters are reached. Improved supportive care, extended infusion center hours, and pediatric home health expertise afford alternatives to prolonged hospital admissions, potentially offering quality, cost-effective approaches that positively impact the delivery of care.
RESUMO
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.
