Rapid regression of lymphadenopathy upon rapamycin treatment in a child with autoimmune lymphoproliferative syndrome.
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.
Janić
Mihailo Dragana
MD
University Children's Hospital, Beograd (Belgrade), Republic of Serbia. dragana.janic@udk.bg.ac.yu
Brasanac
Cedomir Dimitrije
CD
Janković
Jovan Srda
JS
Dokmanović
Bogdan Lidija
BL
Krstovski
Radmio Nada
RN
Kraguljac Kurtović
Janko Nada
JN
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*
D007166:Immunosuppressive Agents; D020123:Sirolimus