Mycophenolate mofetil for treatment of steroid‐refractory acute graft‐versus‐host disease after pediatric hematopoietic stem cell transplantation. (23rd June 2015)

• Record Type: Journal Article
• Authors: Inagaki, Jiro; Kodama, Yuichi; Fukano, Reiji; Noguchi, Maiko; Okamura, Jun
• Is Part Of: Pediatric transplantation. Volume 19:Number 6(2015:Sep.)
• Page Start: 652
• Page End: 658
• Publication Date: 2015-06-23
• Subjects: Transplantation of organs, tissues, etc. in children -- Periodicals; 617.95408305
• DOI: 10.1111/petr.12545
• Languages: English
• ISSNs: 1397-3142

Abstract

<abstract abstract-type="main" id="petr12545-abs-0001"> <title>Abstract</title> <p>A standard treatment is yet to be established for steroid‐refractory acute aGVHD following HSCT. The effects of MMF have not been well studied in children with aGVHD. We evaluated the effectiveness of oral MMF in 14 children with steroid‐refractory aGVHD (grade II in one patient, grade III to IV in 13 patients). The median initial dose of MMF was 40 mg/kg/day (range, 30–74) and was increased by 1.5–2 times if manifestations of GVHD did not improve. Within four wk of treatment, seven patients (50%) achieved CR, and four (29%) had a PR. Within eight wk, 11 patients (79%) achieved CR without using additional agents. Overall, 12 patients are alive and in remission with a median follow‐up of 35 months (range, 14–86). The median maximum dose of MMF was 60 mg/kg/day (range, 34–107). No fatal toxicity was observed, including MMF‐related infections. MMF appears to be highly effective for steroid‐refractory aGVHD when used at a higher dose than has been described previously. Larger studies and pharmacokinetic analysis are required to evaluate its efficacy and toxicity and find the optimal dose of MMF in children.</p> </abstract>