Mycophenolate mofetil for the treatment of children with immune thrombocytopenia and Evans syndrome. A retrospective data review from the Italian association of paediatric haematology/oncology.

Mycophenolate mofetil ( MMF) has been shown to be effective in children with immune thrombocytopenia ( ITP) and Evans syndrome ( ES), but data from larger series and details on the timing of the response are lacking. We evaluated 56 children treated with MMF for ITP ( n = 40) or ES ( n = 16), which was primary or secondary to autoimmune lymphoproliferative syndrome -related syndrome ( ARS). Thirty-five of the 54 evaluable patients (65%) achieved a partial (18%) or complete (46%) response after a median (range) of 20 (7-137) and 37 (7-192) d, respectively. ITP and ES patients responded in 58% and 81% of cases ( P = not significant, ns), with complete response in 32% and 81% ( P = 0·01), respectively. 60% and 73% of children with primary disease and ARS responded ( P = ns) with complete response in 34% and 68% of cases ( P = 0·01), respectively. Six of 35 (17%) children relapsed after a median of 283 d (range 189-1036). Limited toxicity was observed in four patients. The median durations of treatment and follow-up were seven and 12·7 months, respectively. This is the largest reported cohort of patients treated with MMF for ITP/ ES. The results show that MMF is effective and safe and provides a relatively quick response, suggesting that it has a potential role as an alternative to more aggressive and expensive second/further-line treatments. [ABSTRACT FROM AUTHOR]