title: 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.
abstract: 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.
authors.affiliation: Haematology Unit, I.R.C.C.S. Istituto Giannina Gaslini, Genoa, Italy. mauriziomiano@gaslini.org.
authors.forename: Maurizio
authors.initials: M
authors.lastname: Miano
authors.affiliation: Haematology Unit, Paediatric Department, University of Turin, Turin, Italy.
authors.forename: Ugo
authors.initials: U
authors.lastname: Ramenghi
authors.affiliation: Department of Paediatrics, Unit of Paediatric Haematology and Oncology, University of Catania, Catania, Italy.
authors.forename: Giovanna
authors.initials: G
authors.lastname: Russo
authors.affiliation: Haematology-Oncology Unit, I.R.C.C.S. Policlinico Fondazione San Matteo Pavia, Pavia, Italy.
authors.forename: Laura
authors.initials: L
authors.lastname: Rubert
authors.affiliation: Paediatric and Onco-Haematology Unit, University Hospital of Parma, Parma, Italy.
authors.forename: Angelica
authors.initials: A
authors.lastname: Barone
authors.affiliation: Paediatric Haematology-Oncology Unit, Ospedale Pediatrico Meyer, Florence, Italy.
authors.forename: Fabio
authors.initials: F
authors.lastname: Tucci
authors.affiliation: Paediatric Haematology and Oncology Unit, "G. Di Cristina" Children's Hospital, Palermo, Italy.
authors.forename: Piero
authors.initials: P
authors.lastname: Farruggia
authors.affiliation: Paediatric Unit, Rovereto Hospital, APSS Trento, Rovereto, Italy.
authors.forename: Angelamaria
authors.initials: A
authors.lastname: Petrone
authors.affiliation: Haematology Unit, Paediatric Department, University of Turin, Turin, Italy.
authors.forename: Anna
authors.initials: A
authors.lastname: Mondino
authors.affiliation: Department of Paediatrics, Unit of Paediatric Haematology and Oncology, University of Catania, Catania, Italy.
authors.forename: Laura
authors.initials: L
authors.lastname: Lo Valvo
authors.affiliation: Haematology Unit, Paediatric Department, University of Turin, Turin, Italy.
authors.forename: Nicoletta
authors.initials: N
authors.lastname: Crescenzio
authors.affiliation: Department of Paediatrics, Unit of Paediatric Haematology and Oncology, University of Catania, Catania, Italy.
authors.forename: Francesco
authors.initials: F
authors.lastname: Bellia
authors.affiliation: Haematology Unit, I.R.C.C.S. Istituto Giannina Gaslini, Genoa, Italy.
authors.forename: Irene
authors.initials: I
authors.lastname: Olivieri
authors.affiliation: Haematology Unit, I.R.C.C.S. Istituto Giannina Gaslini, Genoa, Italy.
authors.forename: Elena
authors.initials: E
authors.lastname: Palmisani
authors.affiliation: Infectious Diseases Unit, I.R.C.C.S. Istituto Giannina Gaslini, Genoa, Italy.
authors.forename: Ilaria
authors.initials: I
authors.lastname: Caviglia
authors.affiliation: Haematology Unit, I.R.C.C.S. Istituto Giannina Gaslini, Genoa, Italy.
authors.forename: Carlo
authors.initials: C
authors.lastname: Dufour
authors.affiliation: Haematology Unit, I.R.C.C.S. Istituto Giannina Gaslini, Genoa, Italy.
authors.forename: Francesca
authors.initials: F
authors.lastname: Fioredda
mesh_terms: D000293:Adolescent; D000744:Anemia, Hemolytic, Autoimmune / Q000175:diagnosis / Q000188:drug therapy*; D000903:Antibiotics, Antineoplastic / Q000009:adverse effects / Q000627:therapeutic use*; D002648:Child; D002675:Child, Preschool; D018450:Disease Progression; D005260:Female; D006801:Humans; D007223:Infant; D007558:Italy; D008297:Male; D009173:Mycophenolic Acid / Q000009:adverse effects / Q000627:therapeutic use*; D016017:Odds Ratio; D016553:Purpura, Thrombocytopenic, Idiopathic / Q000175:diagnosis / Q000188:drug therapy*; D012008:Recurrence; D019233:Retreatment; D012189:Retrospective Studies; D013921:Thrombocytopenia / Q000175:diagnosis / Q000188:drug therapy*; D016896:Treatment Outcome
chemical_list: D000903:Antibiotics, Antineoplastic; D009173:Mycophenolic Acid
keywords: Evans syndrome; autoimmune disease; childhood ITP; immunodeficiency; mycophenolate mofetil