Use of mycophenolate mofetil for chronic, refractory immune cytopenias in children with autoimmune lymphoproliferative syndrome.
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.
Rao
V Koneti
VK
Laboratory of Clinical Infectious Diseases, National Institute of Allergy and Infectious Diseases/NIH, 10 Center Drive, Bethesda, MD 20892, USA.
Dugan
Faith
F
Dale
Janet K
JK
Davis
Joie
J
Tretler
Jean
J
Hurley
John K
JK
Fleisher
Thomas
T
Puck
Jennifer
J
Straus
Stephen E
SE
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
D007166:Immunosuppressive Agents; D011355:Prodrugs; D009173:Mycophenolic Acid