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Pediatr Blood Cancer. 2011 Dec 15;57(7):1239-43. doi: 10.1002/pbc.23226. Epub 2011 Jun 16.

Rapamycin does not control hemophagocytic lymphohistiocytosis in LCMV-infected perforin-deficient mice.

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  • 1Division of Hematology/Oncology, Duke University Medical Center, Durham, North Carolina, USA.


Hemophagocytic lymphohistiocytosis (HLH) is an immunodysregulatory disorder for which more effective treatments are needed. The macrolide rapamycin has immunosuppressive properties, making it an attractive candidate for controlling the aberrant T cell activation that occurs in HLH. To investigate its therapeutic potential, we used rapamycin to treat Lymphocytic Choriomeningitis Virus (LCMV)-infected perforin-deficient (Prf1(-/-)) mice according to a well-established model of HLH. At the regimens tested, rapamycin did not improve weight loss, splenomegaly, hemophagocytosis, cytopenias, or proinflammatory cytokine production in LCMV-infected Prf1(-/-) animals. Thus, single agent rapamycin appears ineffective in treating the clinical and laboratory manifestations of LCMV-induced HLH.

Copyright © 2011 Wiley Periodicals, Inc.

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