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: Mihailo Dragana Janić, Cedomir Dimitrije Brasanac, Jovan Srda Janković, Bogdan Lidija Dokmanović, Radmio Nada Krstovski, Janko Nada Kraguljac Kurtović
• Journal: Pediatric blood & cancer (Pediatr Blood Cancer) Vol. 53 Issue 6 Pg. 1117-9 (Dec 2009) ISSN: 1545-5017 [Electronic] United States
• PMID: 19588524 (Publication Type: Case Reports, Journal Article)

Chemical References

• Immunosuppressive Agents
• Sirolimus

Topics

• Autoimmune Diseases (drug therapy)
• Female
• Humans
• Immunosuppressive Agents (therapeutic use)
• Infant
• Lymph Nodes (pathology)
• Lymphatic Diseases (drug therapy, pathology)
• Lymphoproliferative Disorders (drug therapy, immunology, pathology)
• Remission Induction (methods)
• Sirolimus (therapeutic use)