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Expert Perspectives on Incorporating Targeted Therapies Into Multidisciplinary Management of Advanced Systemic Mastocytosis
  • CME
  • CE

Prithviraj Bose, MD
person default
Ilene A. Galinsky, NP
Caitlin R. Rausch, PharmD
Released: April 12, 2022
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  1. Ustun C, Arock M, Kluin-Nelemans HC, et al. Advanced systemic mastocytosis: from molecular and genetic progress to clinical practice. Haematologica. 2016;101:1133-1143.
  2. Sperr WR, Kundi M, Alvarez-Twose I, et al. International prognostic scoring system for mastocytosis (IPSM): a retrospective cohort study. Lancet Haematol. 2019;6:e638-e649.
  3. Schwaab J, Hartmann NCO, Naumann N, et al. Importance of adequate diagnostic workup for correct diagnosis of advanced systemic mastocytosis. J Allergy Clin Immunol Pract. 2020;8:3121-3127.
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  11. Jennings SV, Slee VM, Finnerty CC, et al. Symptoms of mast cell activation: the patient perspective. Ann Allergy Asthma Immunol. 2021;127:407-409.
  12. Valent P, Sperr WR, Sotlar K, et al. The serum tryptase test: an emerging robust biomarker in clinical hematology. Expert Rev Hematol. 2014;7:683-690.
  13. Gilreath JA, Tchertanov L, Deininger MW. Novel approaches to treating advanced systemic mastocytosis. Clin Pharmacol. 2019;11:77-92.
  14. Midostaurin [prescribing information]. East Hanover, NJ: Novartis Pharmaceuticals Corporation; 2021.
  15. Gotlib J, Kluin-Nelemans HC, George TI, et al. Efficacy and safety of midostaurin in advanced systemic mastocytosis. N Engl J Med. 2016;374:2530-2541.
  16. Gotlib J, Reiter A, Radia DH, et al. Efficacy and safety of avapritinib in advanced systemic mastocytosis: interim analysis of the phase 2 PATHFINDER trial. Nat Med. 2021;27:2192-2199.
  17. DeAngelo DJ, Radia DH, George TI, et al. Safety and efficacy of avapritinib in advanced systemic mastocytosis: the phase 1 EXPLORER trial. Nat Med. 2021;27:2183-2191.
  18. Shomali W, Gotlib J. Response criteria in advanced systemic mastocytosis: evolution in the era of KIT inhibitors. Int J Mol Sci. 2021;22:2983.
  19. Avapritinib [prescribing information]. Cambridge, MA: Blueprint Medicines Corporation; 2021.
  20. Deininger MW, DeAngelo DJ, Radia DH, et al. Effective control of advance systemic mastocytosis with avapritinib: mutational analysis from the Explorer clinical study. Presented at: 63rd American Society of Hematology Annual Meeting & Exposition; December 11-14, 2021. Abstract 318.
  21. George TI, Karner KH, Moser KA, et al. Efficacy of avapritinib in patients with advanced systemic mastocytosis: hematologic and bone marrow responses from the phase 2 open-label, single-arm, PATHFINDER study. Presented at: 63rd American Society of Hematology Annual Meeting & Exposition; December 11-14, 2021. Abstract 2565.
  22. Imatinib [prescribing information]. East Hanover, NJ: Novartis Pharmaceuticals Corporation; 2020.
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  24. Akin C, Oude Elberink H, Gotlib J, et al. Results from Pioneer: a randomized, double-blind, placebo-controlled, phase 2 study of avapritinib in patients with indolent systemic mastocytosis. Presented at: 62nd American Society of Hematology Annual Meeting & Exposition; December 5-8, 2020. Abstract 1248.
  25. Guarnieri A, Chicarelli M, Cable L, et al. Preclinical data identifies bezuclastinib as a differentiated KIT inhibitor with unique selectivity to KIT D816V and minimal evidence of brain penetration. Presented at: AACR-NCI-EORTC Virtual International Conference on Molecular Targets and Cancer Therapeutics; October 7-10, 2021. Abstract P257.
  26. Dave N, Devlin M, Rodstrom J, et al. Safety and pharmacokinetics of BLU-263, a next-generation KIT inhibitor, in normal healthy volunteers. Presented at: American Association for Cancer Research Annual Meeting; April 9-14, 2021. Abstract CT122.
  27. Pardanani A. How I treat patients with indolent and smoldering mastocytosis (rare conditions but difficult to manage). Blood. 2013;121:3085-3094.
  28. Galinsky I, Coleman M, Fechter L. Midostaurin: nursing perspectives on managing treatment and adverse events in patients with flt3 mutation-positive acute myeloid leukemia and advanced systemic mastocytosis. Clin J Oncol Nurs. 2019;23:599-608.
  29. Tefferi A, Kittur J, Farrukh F, et al. Cladribine therapy for advanced and indolent systemic mastocytosis: Mayo Clinic experience in 42 consecutive cases. Br J Haematol. 2022;196:975-983.
  30. Castells M, Butterfield J. Mast cell activation syndrome and mastocytosis: initial treatment options and long-term management. J Allergy Clin Immunol Pract. 2019;7:1097-1106.
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