title: Use of mycophenolate mofetil for chronic, refractory immune cytopenias in children with autoimmune lymphoproliferative syndrome.
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.
authors.affiliation: Laboratory of Clinical Infectious Diseases, National Institute of Allergy and Infectious Diseases/NIH, 10 Center Drive, Bethesda, MD 20892, USA.
authors.forename: V Koneti
authors.initials: VK
authors.lastname: Rao
authors.forename: Faith
authors.initials: F
authors.lastname: Dugan
authors.forename: Janet K
authors.initials: JK
authors.lastname: Dale
authors.forename: Joie
authors.initials: J
authors.lastname: Davis
authors.forename: Jean
authors.initials: J
authors.lastname: Tretler
authors.forename: John K
authors.initials: JK
authors.lastname: Hurley
authors.forename: Thomas
authors.initials: T
authors.lastname: Fleisher
authors.forename: Jennifer
authors.initials: J
authors.lastname: Puck
authors.forename: Stephen E
authors.initials: SE
authors.lastname: Straus
mesh_terms: D000293:Adolescent; D000744:Anemia, Hemolytic, Autoimmune / Q000188:drug therapy / Q000276:immunology; D001327:Autoimmune Diseases / Q000188:drug therapy* / Q000276:immunology; D002648:Child; D002675:Child, Preschool; D005500:Follow-Up Studies; D006801:Humans; D007166:Immunosuppressive Agents / Q000009:adverse effects / Q000627:therapeutic use*; D007223:Infant; D007958:Leukocyte Count; D008232:Lymphoproliferative Disorders / Q000188:drug therapy* / Q000276:immunology; D008297:Male; D009173:Mycophenolic Acid / Q000009:adverse effects / Q000031:analogs & derivatives* / Q000627:therapeutic use*; D009503:Neutropenia / Q000188:drug therapy / Q000276:immunology; D010146:Pain / Q000139:chemically induced; D011355:Prodrugs; D013921:Thrombocytopenia / Q000188:drug therapy / Q000276:immunology
chemical_list: D007166:Immunosuppressive Agents; D011355:Prodrugs; D009173:Mycophenolic Acid