Mycophenolate mofetil for treatment of steroid-refractory acute graft-versus-host disease after pediatric hematopoietic stem cell transplantation.

Abstract:

: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.

journal_name

Pediatr Transplant

authors

Inagaki J,Kodama Y,Fukano R,Noguchi M,Okamura J

doi

10.1111/petr.12545

subject

Has Abstract

pub_date

2015-09-01 00:00:00

pages

652-8

issue

6

eissn

1397-3142

issn

1399-3046

journal_volume

19

pub_type

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