Premium
Mycophenolate mofetil for treatment of steroid‐refractory acute graft‐versus‐host disease after pediatric hematopoietic stem cell transplantation
Author(s) -
Inagaki Jiro,
Kodama Yuichi,
Fukano Reiji,
Noguchi Maiko,
Okamura Jun
Publication year - 2015
Publication title -
pediatric transplantation
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 0.457
H-Index - 69
eISSN - 1399-3046
pISSN - 1397-3142
DOI - 10.1111/petr.12545
Subject(s) - medicine , mycophenolate , hematopoietic stem cell transplantation , refractory (planetary science) , graft versus host disease , transplantation , stem cell , hematopoietic stem cell , disease , haematopoiesis , surgery , physics , astrobiology , biology , genetics
A standard treatment is yet to be established for steroid‐refractory acute a GVHD following HSCT . The effects of MMF have not been well studied in children with a GVHD . We evaluated the effectiveness of oral MMF in 14 children with steroid‐refractory a GVHD (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.