Treatment with sirolimus results in complete responses in patients with autoimmune lymphoproliferative syndrome

British Journal of Haematology

Volumn 145, Issue 1, 2009, Pages 101-106

  Teachey, David T ^a,b   Greiner, Robert ^c   Seif, Alix ^b   Attiyeh, Edward ^b   Bleesing, Jack ^d   Choi, John ^b   Manno, Catherine ^b   Rappaport, Eric ^b   Schwabe, Dirk ^e   Sheen, Cecilia ^b   Sullivan, Kathleen E ^f   Zhuang, Hongming ^b   Wechsler, Daniel S ^c   Grupp, Stephan A ^b  

^a CHILDREN S HOSPITAL OF PHILADELPHIA   (United States)

^b UNIVERSITY OF PENNSYLVANIA   (United States)

^c DUKE UNIVERSITY MEDICAL CENTER   (United States)

^d CINCINNATI CHILDREN'S HOSPITAL MEDICAL CENTER   (United States)

^e UNIVERSITY HOSPITAL FRANKFURT   (Germany)

^f UNIVERSITY OF PENNSYLVANIA   (United States)

Author keywords

Autoimmunity; MTOR; Rapamycin; Signal transduction

Indexed keywords

CYCLOPHOSPHAMIDE; CYCLOSPORIN A; ETANERCEPT; HYDROXYCHLOROQUINE; IMMUNOGLOBULIN; MAMMALIAN TARGET OF RAPAMYCIN INHIBITOR; MERCAPTOPURINE; METHOTREXATE; MYCOPHENOLIC ACID 2 MORPHOLINOETHYL ESTER; RAPAMYCIN; RECOMBINANT INTERLEUKIN 1 RECEPTOR BLOCKING AGENT; RITUXIMAB; STEROID;

ARTHRITIS; ARTICLE; AUTOIMMUNE LYMPHOPROLIFERATIVE SYNDROME; CERVICAL LYMPH NODE; CLINICAL ARTICLE; COLITIS; COMPUTER ASSISTED TOMOGRAPHY; CYTOPENIA; DISEASE CLASSIFICATION; DRUG INDICATION; DRUG MEGADOSE; DRUG RESPONSE; HUMAN; HYPERTENSION; IMMUNOSUPPRESSIVE TREATMENT; LUNG EMBOLISM; LYMPHADENOPATHY; LYMPHOCYTE COUNT; LYMPHOPROLIFERATIVE DISEASE; MALE; MUCOSA INFLAMMATION; POSITRON EMISSION TOMOGRAPHY; PRIORITY JOURNAL; SPLENOMEGALY; T LYMPHOCYTE; THROMBOCYTOPENIA; THRUSH; TREATMENT DURATION; TREATMENT OUTCOME; TREATMENT RESPONSE;

ANEMIA, HEMOLYTIC, AUTOIMMUNE; CHILD; CHILD, PRESCHOOL; DRUG ADMINISTRATION SCHEDULE; FOLLOW-UP STUDIES; HUMANS; IMMUNOSUPPRESSIVE AGENTS; INFANT; MALE; POSITRON-EMISSION TOMOGRAPHY; SIROLIMUS;

EID: 61949157161     PISSN: 00071048     EISSN: 13652141     Source Type: Journal    
DOI: 10.1111/j.1365-2141.2009.07595.x     Document Type: Article

References (15)

1. Abdel-Karim, I.A. Giles, F.J. (2008) Mammalian target of rapamycin as a target in hematological malignancies. Current Problems in Cancer, 32, 161 177.
2. + regulatory T cells of both healthy subjects and type 1 diabetic patients. Journal of Immunology, 177, 8338 8347.
3. Bleesing, J.J., Straus, S.E. Fleisher, T.A. (2000) Autoimmune lymphoproliferative syndrome. A human disorder of abnormal lymphocyte survival. Pediatric Clinics of North America, 47, 1291 1310.
4. Bleesing, J.J., Brown, M.R., Straus, S.E., Dale, J.K., Siegel, R.M., Johnson, M., Lenardo, M.J., Puck, J.M. Fleisher, T.A. (2001) Immunophenotypic profiles in families with autoimmune lymphoproliferative syndrome. Blood, 98, 2466 2473.
5. Brusko, T.M., Putnam, A.L. Bluestone, J.A. (2008) Human regulatory T cells: role in autoimmune disease and therapeutic opportunities. Immunological Reviews, 223, 371 390.
6. Dale, J., Price, S., J, T., Aldridge, P., Montague, S., David, J., Gill, F., Hartmann, K., Stork, L., Gnarra, D., Kirshnamurti, L., Puck, J.M., Fleisher, T. Rao, V.K. (2007) Use of rituximab for refractory autoimmune cytopenias associated with autoimmune lymphoproliferative syndrome (ALPS). Blood (ASH Annual Meeting Abstracts), 110, 1319.
7. Ferrer, E., Moral, M.A. Bozzo, J. (2007) Spotlight on autoimmune lymphoproliferative syndrome. Drug News Perspect, 20, 260 262.
8. Fischer, K., Voelkl, S., Heymann, J., Przybylski, G.K., Mondal, K., Laumer, M., Kunz-Schughart, L., Schmidt, C.A., Andreesen, R. Mackensen, A. (2005) Isolation and characterization of human antigen-specific TCR alpha beta+ CD4(-)CD8- double-negative regulatory T cells. Blood, 105, 2828 2835.
9. Koneti Rao, V., Dugan, F., Dale, J.K., Davis, J., Tretler, J., Hurley, J.K., Fleisher, T., Puck, J. Straus, S.E. (2005) Use of mycophenolate mofetil for chronic, refractory immune cytopenias in children with autoimmune lymphoproliferative syndrome. British Journal of Haematology, 129, 534 538.
10. Nagata, S. Golstein, P. (1995) The Fas death factor. Science, 267, 1449 1456.
11. Noris, M., Casiraghi, F., Todeschini, M., Cravedi, P., Cugini, D., Monteferrante, G., Aiello, S., Cassis, L., Gotti, E., Gaspari, F., Cattaneo, D., Perico, N. Remuzzi, G. (2007) Regulatory T cells and T cell depletion: role of immunosuppressive drugs. Journal of the American Society of Nephrology, 18, 1007 1018.
12. Rieux-Laucat, F., Le Deist, F. Fischer, A. (2003) Autoimmune lymphoproliferative syndromes: genetic defects of apoptosis pathways. Cell Death and Differentiation, 10, 124 133.
13. Sneller, M.C., Wang, J., Dale, J.K., Strober, W., Middelton, L.A., Choi, Y., Fleisher, T.A., Lim, M.S., Jaffe, E.S., Puck, J.M., Lenardo, M.J. Straus, S.E. (1997) Clincial, immunologic, and genetic features of an autoimmune lymphoproliferative syndrome associated with abnormal lymphocyte apoptosis. Blood, 89, 1341 1348.
14. Teachey, D.T., Obzut, D.A., Axsom, K., Choi, J.K., Goldsmith, K.C., Hall, J., Hulitt, J., Manno, C.S., Maris, J.M., Rhodin, N., Sullivan, K.E., Brown, V.I. Grupp, S.A. (2006) Rapamycin improves lymphoproliferative disease in murine autoimmune lymphoproliferative syndrome (ALPS). Blood, 108, 1965 1971.
15. Teachey, D.T., Seif, A.E., Brown, V.I., Bruno, M., Bunte, R.M., Chang, Y.J., Choi, J.K., Fish, J.D., Hall, J., Reid, G.S., Ryan, T., Sheen, C., Zweidler-McKay, P. Grupp, S.A. (2008) Targeting Notch signaling in autoimmune and lymphoproliferative disease. Blood, 111, 705 714.