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- Title
Use of mycophenolate mofetil for chronic, refractory immune cytopenias in children with autoimmune lymphoproliferative syndrome.
- Authors
Rao, V Koneti; Dugan, Faith; Dale, Janet K; Davis, Joie; Tretler, Jean; Hurley, John K; Fleisher, Thomas; Puck, Jennifer; Straus, Stephen E
- Abstract
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.
- Publication
British journal of haematology, 2005, Vol 129, Issue 4, p534
- ISSN
0007-1048
- Publication type
Journal Article
- DOI
10.1111/j.1365-2141.2005.05496.x