Treatment and Treatment-Response Criteria

Soter NA, Austen KF, Wasserman SI. Oral disodium cromoglycate in the treatment of systemic mastocytosis. N Engl J Med. 1979;301:465-469.

Metcalfe DD. The treatment of mastocytosis: an overview. J Invest Dermatol. 1991;96:55S-56S; discussion 56S-59S.

Austen KF. Systemic mastocytosis. N Engl J Med. 1992;326:639-640.

Kluin-Nelemans HC, Jansen JH, Breukelman H, Wolthers BG, Kluin PM, Kroon HM, Willemze R. Response to interferon alfa-2b in a patient with systemic mastocytosis. N Engl J Med. 1992;326:619-623.

Weide R, Ehlenz K, Lorenz W, Walthers E, Klausmann M, Pfluger KH. Successful treatment of osteoporosis in systemic mastocytosis with interferon alpha-2b. Ann Hematol. 1996;72:41-43.

Lehmann T, Beyeler C, Lammle B, Hunziker T, Vock P, Olah AJ, Dahinden C, Gerber NJ. Severe osteoporosis due to systemic mast cell disease: successful treatment with interferon alpha-2B. Br J Rheumatol. 1996;35:898-900.

Worobec AS. Treatment of systemic mast cell disorders. Hematol Oncol Clin North Am. 2000;14:659-687.

Tefferi A, Li CY, Butterfield JH, Hoagland HC. Treatment of systemic mast-cell disease with cladribine. N Engl J Med. 2001;344:307-309.

Worobec AS, Metcalfe DD. Mastocytosis: current treatment concepts. Int Arch Allergy Immunol. 2002;127:153-155.

Kluin-Nelemans HC, Oldhoff JM, Van Doormaal JJ, Van 't Wout JW, Verhoef G, Gerrits WB, van Dobbenburgh OA, Pasmans SG, Fijnheer R. Cladribine therapy for systemic mastocytosis. Blood. 2003;102:4270-4276.

Valent P, Akin C, Sperr WR, Escribano L, Arock M, Horny HP, Bennett JM, Metcalfe DD. Aggressive systemic mastocytosis and related mast cell disorders: current treatment options and proposed response criteria. Leuk Res. 2003;27:635-641.

Pardanani A, Elliott M, Reeder T, Li CY, Baxter EJ, Cross NC, Tefferi A. Imatinib for systemic mast-cell disease. Lancet. 2003;362:535-536.

Valent P, Akin C, Sperr WR, Horny HP, Arock M, Lechner K, Bennett JM, Metcalfe DD. Diagnosis and treatment of systemic mastocytosis: state of the art. Br J Haematol. 2003;122:695-717.

Akin C, Brockow K, D'Ambrosio C, et al. Effects of tyrosine kinase inhibitor STI571 on human mast cells bearing wild-type or mutated c-kit. Exp Hematol. 2003;31:686-692.

Tefferi A, Pardanani A. Clinical, genetic, and therapeutic insights into systemic mast cell disease. Curr Opin Hematol. 2004;11:58-64.

Valent P, Ghannadan M, Akin C, et al. On the way to targeted therapy of mast cell  neoplasms: identification of molecular targets in neoplastic mast cells and evaluation of arising treatment concepts. Eur J Clin Invest. 2004;34:41-52.

Hauswirth AW, Simonitsch-Klupp I, Uffmann M, Koller E, Sperr WR, Lechner K, Valent P. Response to therapy with interferon alpha-2b and prednisolone in aggressive systemic mastocytosis: report of five cases and review of the literature. Leuk Res. 2004;28:249-257.

Penack O, Sotlar K, Noack F, Horny HP, Thiel E, Notter M. Cladribine therapy in a patient with an aleukemic subvariant of mast cell leukemia. Ann Hematol. 2005;84:692-693.

Gotlib J, Berube C, Growney JD, Chen CC, George TI, Williams C, Kajiguchi T, Ruan J, Lilleberg SL, Durocher JA, Lichy JH, Wang Y, Cohen PS, Arber DA, Heinrich MC, Neckers L, Galli SJ, Gilliland DG, Coutré SE. Activity of the tyrosine kinase inhibitor PKC412 in a patient with mast cell leukemia with the D816V KIT mutation. Blood. 2005;106:2865-2870.

Pardanani A, Akin C, Valent P. Pathogenesis, clinical features, and treatment advances in mastocytosis. Best Pract Res Clin Haematol. 2006;19:595-615.

Florian S, Esterbauer H, Binder T, Müllauer L, Haas OA, Sperr WR, Sillaber C, Valent P. Systemic mastocytosis (SM) associated with chronic eosinophilic leukemia (SM-CEL): detection of FIP1L1/PDGFRalpha, classification by WHO criteria, and response to therapy with imatinib. Leuk Res. 2006;30:1201-1205.

Gotlib J, George TI, Linder A, et al. Phase II Trial of the Tyrosine Kinase Inhibitor PKC412 in Advanced Systemic Mastocytosis: Preliminary Results. Blood. 2006;108:3609.

Purtill D, Cooney J, Sinniah R, et al. Dasatinib therapy for systemic mastocytosis: four cases. Eur J Haematol. 2008;80:456-458.

Verstovsek S, Tefferi A, Cortes J, O'Brien S, Garcia-Manero G, Pardanani A, Akin C, Faderl S, Manshouri T, Thomas D, Kantarjian H.  Phase II study of dasatinib in Philadelphia chromosome-negative acute and chronic myeloid diseases, including systemic mastocytosis. Clin Cancer Res. 2008;14:3906-3915.

Aichberger KJ, Sperr WR, Gleixner KV, Kretschmer A, Valent P. Treatment responses to cladribine and dasatinib in rapidly progressing aggressive mastocytosis. Eur J Clin Invest. 2008;38:869-873.

Kluin-Nelemans HC, Ferenc V, van Doormaal JJ, van Iperen C, Peters WG, Akin C, Valent P. Lenalidomide therapy in systemic mastocytosis. Leuk Res. 2009;33:e19-22.

Ustun C, Corless CL, Savage N, Fiskus W, Manaloor E, Heinrich MC, Lewis G, Ramalingam P, Kepten I, Jillella A, Bhalla K. Chemotherapy and dasatinib induce long-term hematologic and molecular remission in systemic mastocytosis with acute myeloid leukemia with KIT(D816V). Leuk Res. in press.

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