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Use of mycophenolate mofetil for chronic, refractory immune cytopenias in children with autoimmune lymphoproliferative syndrome
Author(s) -
Koneti Rao V.,
Dugan Faith,
Dale Janet K.,
Davis Joie,
Tretler Jean,
Hurley John K.,
Fleisher Thomas,
Puck Jennifer,
Straus Stephen E.
Publication year - 2005
Publication title -
british journal of haematology
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.907
H-Index - 186
eISSN - 1365-2141
pISSN - 0007-1048
DOI - 10.1111/j.1365-2141.2005.05496.x
Subject(s) - mycophenolate , medicine , autoimmune lymphoproliferative syndrome , refractory (planetary science) , immunology , mycophenolic acid , lymphoproliferative disorders , immune system , lymphoproliferative disease , lymphoma , transplantation , biology , apoptosis , programmed cell death , astrobiology , biochemistry , fas receptor
Summary Autoimmune lymphoproliferative syndrome (ALPS) is a disorder of apoptosis associated most often with heritable FAS mutations leading to lymphadenopathy, hypersplenism and chronic refractory autoimmune cytopenias. Mycophenolate mofetil (MMF) was used to treat cytopenias in 13 ALPS patients aged 9 months to 17 years from a cohort of 118 children (aged < 18 years) and 82 adults. Twelve responded for a median follow‐up of 49 weeks (range 38–240 weeks), defined by maintenance of adequate blood counts and reduction in dosage or cessation of other immunosuppressive agents. This preliminary experience suggests that MMF may spare steroid usage in patients with ALPS‐associated cytopenias.