The optimal dose and schedule of mycophenolate mofetil (MMF) in pediatric allogeneic stem cell transplant recipients remains to be determined. We previously reported safety and pharmacokinetics of MMF at 900 mg/m2 q6h dosing. This study was conducted to investigate the efficacy of tacrolimus plus q8h MMF dosing for acute graft versus host disease (GVHD) prophylaxis in a heterogeneous population of children, adolescent, young adult allogeneic stem cell transplant recipients, utilizing multiple allogeneic donor sources.Procedure
GVHD prophylaxis consisted of tacrolimus 0.03–0.04 mg/kg/day intravenous continuous infusion or 0.12–0.16 mg/kg/day orally divided q8–12h and MMF 900 mg/m2/dose (max. 1.5 g) or 15 mg/kg/dose intravenous/orally (age ≥18 years) q8h starting on Day +1. MMF was discontinued on Day +30 or Day +60 in the absence of acute GVHD. Thirty-five children, adolescents, and young adult allogeneic stem cell transplant recipients with malignant and nonmalignant disorders were enrolled.Results
Kaplan–Meier probability of grade II–IV and grade III–IV acute GVHD was 22.8% (CI95: 5.2–47.9 [where CI stands for confidence interval]) and 5.7% (CI95: 0–48.9), respectively. Probability of extensive and limited chronic GVHD was 22.6% (CI95: 3.4–52.2) and 12.2% (CI95: 0.3–45.7), respectively. Probability of 1 year overall survival was 82% (CI95: 64.1–99.8). Myeloablative conditioning was predictive of higher risk of acute GVHD in the univariate analysis (P = 0.01, hazard ratio = 6.6, CI95: 0.91–48).Conclusion
This study demonstrated a low probability of acute and chronic GVHD in a diverse cohort of childhood, adolescent, and young adult allogeneic stem cell transplant recipients following MMF q8h plus tacrolimus prophylaxis.