title: Rapid regression of lymphadenopathy upon rapamycin treatment in a child with autoimmune lymphoproliferative syndrome.
abstract: Autoimmune lymphoproliferative syndrome (ALPS) is a genetic disorder of the immune system caused by inadequate induction of apoptosis via the Fas pathway, mainly characterized by generalized lymphadenopathy, splenomegaly, and autoimmune cytopenias, as well as increased risk of lymphoma. Although the clinical course of ALPS is highly variable, without treatment long-term prognosis is unsatisfactory for most patients. ALPS has been treated with most of the existing immunosuppressive agents, with variable success. We hereby present a case of a child with ALPS whose greatly enlarged lymph nodes rapidly regressed upon initiation of rapamycin, a novel potential therapeutic agent in the treatment of ALPS.
authors: University Children's Hospital, Beograd (Belgrade), Republic of Serbia. dragana.janic@udk.bg.ac.yu
authors: Mihailo Dragana
authors: MD
authors: Janić
authors: Cedomir Dimitrije
authors: CD
authors: Brasanac
authors: Jovan Srda
authors: JS
authors: Janković
authors: Bogdan Lidija
authors: BL
authors: Dokmanović
authors: Radmio Nada
authors: RN
authors: Krstovski
authors: Janko Nada
authors: JN
authors: Kraguljac Kurtović
mesh_terms: D001327:Autoimmune Diseases / Q000188:drug therapy*; D005260:Female; D006801:Humans; D007166:Immunosuppressive Agents / Q000627:therapeutic use; D007223:Infant; D008198:Lymph Nodes / Q000473:pathology; D008206:Lymphatic Diseases / Q000188:drug therapy / Q000473:pathology; D008232:Lymphoproliferative Disorders / Q000188:drug therapy* / Q000276:immunology / Q000473:pathology; D012074:Remission Induction / Q000379:methods; D020123:Sirolimus / Q000627:therapeutic use*
chemical_list: D007166:Immunosuppressive Agents; D020123:Sirolimus