Efficacy and safety of midostaurin in patients with advanced systemic mastocytosis: 10-year median follow-up of a phase II trial (original) (raw)

References

  1. Horny HP, Metcalfe DD, Bennett JM, Bain BJ, Akin C, Escribano L _et al_Mastocytosis. In: Swerdlow SH, Campo E, Harris NL, Jaffe ES, Pileri SA, Stein H et al(eds) WHO Classification of Tumors of Hematopoietic and Lymphoid Tissues. International Agency for Research and Cancer (IARC): Lyon, 2008; pp 54–63.
    Google Scholar
  2. Valent P, Horny HP, Escribano L, Longley BJ, Li CY, Schwartz LB et al. Diagnostic criteria and classification of mastocytosis: a consensus proposal. Leuk Res 2001; 25: 603–625.
    Article CAS PubMed Google Scholar
  3. Lim KH, Tefferi A, Lasho TL, Finke C, Patnaik M, Butterfield JH et al. Systemic mastocytosis in 342 consecutive adults: survival studies and prognostic factors. Blood 2009; 113: 5727–5736.
    Article CAS PubMed Google Scholar
  4. Pardanani A . Systemic mastocytosis in adults: 2017 update on diagnosis, risk, stratification, and management. Am J Hematol 2016; 91: 1146–1159.
    Article CAS PubMed Google Scholar
  5. Valent P, Akin C, Sperr WR, Escribano L, Arock M, Horny HP et al. Aggressive systemic mastocytosis and related mast cell disorders: current treatment options and proposed response criteria. Leuk Res 2003; 27: 635–641.
    Article CAS PubMed Google Scholar
  6. Sperr WR, Horny HP, Valent P . Spectrum of associated clonal hematologic non-mast cell lineage disorders occurring in patients with systemic mastocytosis. Int Arch Allergy Immunol 2002; 127: 140–142.
    Article CAS PubMed Google Scholar
  7. Georgin-Lavialle S, Lhermitte L, Dubreuil P, Chandesris MO, Hermine O, Damaj G . Mast cell leukemia. Blood 2013; 121: 1285–1295.
    Article CAS PubMed Google Scholar
  8. Valent P, Sotlar K, Sperr WR, Escribano L, Yavuz S, Reiter A et al. Refined diagnostic criteria and classification of mast cell leukemia (MCL) and myelomastocytic leukemia (MML): a consensus report. Ann Oncol 2014; 25: 691–700.
    Article Google Scholar
  9. Delaporte E, Piérard E, Wolthers BG, Desreumaux P, Janin A, Cortot A et al. Interferon-alpha in combination with corticosteroids improves systemic mast cell disease. Br J Dermatol 1995; 132: 479–482.
    Article CAS PubMed Google Scholar
  10. Hauswirth AW, Simonitsch-Klupp I, Uffmann M, Koller E, Sperr WR, Lechner K et al. 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.
    Article CAS PubMed Google Scholar
  11. Lim KH, Pardanani A, Butterfield JH, Li CY, Tefferi A . Cytoreductive therapy in 108 adults with systemic mastocytosis: outcome analysis and response prediction during treatment with interferon-alpha, hydroxyurea, imatinib mesylate or 2-chlorodeoxy-adenosine. Am J Hematol 2009; 84: 790–794.
    Article CAS PubMed Google Scholar
  12. Kluin-Nelemans HC, Oldhoff JM, Van Doormaal JJ, Van’t Wout JW, Verhoef G, Gerrits WB et al. Cladribine therapy for systemic mastocytosis. Blood 2003; 102: 4270–4276.
    Article CAS PubMed Google Scholar
  13. Tefferi A, Li CY, Butterfield JH, Hoagland HC . Treatment of systemic mast-cell disease with cladribine. N Engl J Med 2001; 344: 307–309.
    Article CAS PubMed Google Scholar
  14. Pardanani A, Hoffbrand AV, Butterfield JH, Tefferi A . Treatment of systemic mast cell disease with 2-chlorodeoxyadenosine. Leuk Res 2004; 28: 127–131.
    Article CAS PubMed Google Scholar
  15. Barete S, Lortholary O, Damaj G, Hirsch I, Chandesris MO, Elie C et al. Long-term efficacy and safety of cladribine (2-CdA) in adult patients with mastocytosis. Blood 2015; 126: 1009–1016.
    Article CAS PubMed Google Scholar
  16. Garcia-Montero AC, Jara-Acevedo M, Teodosio C, Sanchez ML, Nunez R, Prados A et al. KIT mutation in mast cells and other bone marrow hematopoietic cell lineages in systemic mast cell disorders: a prospective study of the Spanish Network on Mastocytosis (REMA) in a series of 113 patients. Blood 2006; 108: 2366–2372.
    Article CAS PubMed Google Scholar
  17. Kristensen T, Vestergaard H, Møller MB . Improved detection of the KIT D816V mutation in patients with systemic mastocytosis using a quantitative and highly sensitive real-time qPCR assay. J Mol Diagn 2011; 13: 180–188.
    Article CAS PubMed PubMed Central Google Scholar
  18. Arock M, Sotlar K, Akin C, Broesby-Olsen S, Hoermann G, Escribano L et al. KIT mutation analysis in mast cell neoplasms: recommendations of the European Competence Network on Mastocytosis. Leukemia 2015; 29: 1223–1232.
    Article CAS PubMed PubMed Central Google Scholar
  19. Fabbro D, Ruetz S, Bodis S, Pruschy M, Csermak K, Man A et al. PKC412–a protein kinase inhibitor with a broad therapeutic potential. Anticancer Drug Des 2000; 15: 17–28.
    CAS PubMed Google Scholar
  20. Growney JD, Clark JJ, Adelsperger J, Stone R, Fabbro D, Griffin JD et al. Activation mutations of human c-KIT resistant to imatinib mesylate are sensitive to the tyrosine kinase inhibitor PKC412. Blood 2005; 106: 721–724.
    Article CAS PubMed PubMed Central Google Scholar
  21. Gotlib J, Berube C, Growney JD, Chen CC, George TI, Williams C et al. Activity of the tyrosine kinase inhibitor PKC412 in a patient with mast cell leukemia with the D816V KIT mutation. Blood 2005; 106: 2865–2870.
    Article CAS PubMed PubMed Central Google Scholar
  22. Cheson BD, Bennett JM, Kantarjian H, Pinto A, Schiffer CA, Nimer SD et al. Report of an international working group to standardize response criteria for myelodysplastic syndromes. Blood 2000; 96: 3671–3674.
    CAS PubMed Google Scholar
  23. Cheson BD, Greenberg PL, Bennett JM, Lowenberg B, Wijermans PW, Nimer SD et al. Clinical application and proposal for modification of the International Working Group (IWG) response criteria in myelodysplasia. Blood 2006; 108: 419–425.
    Article CAS PubMed Google Scholar
  24. Simon R . Optimal two-stage designs for phase II clinical trials. Control Clin Trials 1989; 10: 1–10.
    Article CAS PubMed Google Scholar
  25. Verstovsek S, Kantarjian H, Mesa RA, Pardanani AD, Cortes-Franco J, Thomas AD et al. Safety and efficacy of INCB018424, a JAK1 and JAK2 inhibitor, in myelofibrosis. N Engl J Med 2010; 363: 1117–1127.
    Article CAS PubMed PubMed Central Google Scholar
  26. Gotlib J, Kluin-Nelemens HC, George TI, Akin C, Sotlar K, Hermine O et al. Efficacy and safety of midostaurin in advanced systemic mastocytosis. N Engl J Med 2016; 374: 2530–2541.
    Article CAS PubMed Google Scholar
  27. van Anrooij B, Kluin-Nelemans JC, Safy M, Flokstra-de Blok BM, Oude Elberink JN . Patient-reported disease-specific quality-of-life and symptom severity in systemic mastocytosis. Allergy 2016; 71: 1585–1593.
    Article CAS PubMed Google Scholar
  28. Ware JE, Kosinski M, Keller SD . A 12-Item Short-Form Health Survey: construction of scales and preliminary tests of reliability and validity. Med Care 1996; 34: 220–233.
    Article PubMed Google Scholar
  29. Portenoy RK, Thaler HT, Kornblith AB, Lepore JM, Friedlander-Klar H, Kiyasu E et al. The Memorial Symptom Assessment Scale: an instrument for the evaluation of symptom prevalence, characteristics and distress. Eur J Cancer 1994; 30A: 1326–1336.
    Article CAS PubMed Google Scholar
  30. Krauth MT, Mirkina I, Herrmann H, Baumgartner C, Kneidinger M, Valent P . Midostaurin (PKC412) inhibits immunoglobulin E-dependent activation and mediator release in human blood basophils and mast cells. Clin Exp Allergy 2009; 39: 1711–1720.
    Article CAS PubMed Google Scholar
  31. Saglio G, Kim DW, Issaragrisil S, le Coutre P, Etienne G, Lobo C et al. Nilotinib versus imatinib for newly diagnosed chronic myeloid leukemia. N Engl J Med 2010; 362: 2251–2259.
    Article CAS PubMed Google Scholar
  32. Ma Y, Zeng S, Metcalfe DD, Akin C, Dimitrijevic S, Butterfield JH et al. The c KIT mutation causing human mastocytosis is resistant to STI571 and other KIT kinase inhibitors; kinases with enzymatic site mutations show different inhibitor sensitivity profiles than wild-type kinases and those with regulatory-type mutations. Blood 2002; 100: 1068–1071.
    Article Google Scholar
  33. Vega-Ruiz A, Cortes JE, Sever M, Manshouri T, Quintás-Cardama A, Luthra R et al. Phase II study of imatinib mesylate as therapy for patients with systemic mastocytosis. Leuk Res 2009; 33: 1481–1484.
    Article CAS PubMed PubMed Central Google Scholar
  34. Pagano L, Valentini CG, Caira M, Rondoni M, Van Lint MT, Candoni A et al. Advanced mast cell disease: an Italian hematological multicenter experience. Int J Hematol 2008; 88: 483–488.
    Article PubMed Google Scholar
  35. Verstovsek S, Tefferi A, Cortes J, O’Brien S, Garcia-Manero G, Pardanani A et al. Phase II study of dasatinib in Philadelphia chromosome-negative acute and chronic myeloid diseases, including systemic mastocytosis. Clin Cancer Res 2008; 14: 3906–3915.
    Article CAS PubMed PubMed Central Google Scholar
  36. Hochhaus A, Baccarani M, Giles FJ, le Coutre PD, Müller MC, Reiter A et al. Nilotinib in patients with systemic mastocytosis: analysis of the phase 2, open-label, single-arm nilotinib registration study. J Cancer Res Clin Oncol 2015; 141: 2047–2060.
    Article CAS PubMed PubMed Central Google Scholar
  37. Lortholary O, Chandesris MO, Livideanu CB, Paul C, Guillet G, Jassem E et al. Masitinib for treatment of severely symptomatic indolent systemic mastocytosis: a randomized, placebo-controlled, phase 3 study. Lancet 2017; 389: 612–620.
    Article CAS PubMed PubMed Central Google Scholar
  38. Valent P, Sotlar K, Sperr WR, Reiter A, Arock M, Horny HP . Chronic mast cell leukemia: a novel leukemia-variant with distinct morphological and clinical features. Leuk Res 2015; 39: 1–5.
    Article PubMed Google Scholar
  39. Valent P, Berger J, Cerny-Reiterer S, Peter B, Eisenwort G, Hoermann G et al. Chronic mast cell leukemia (MCL) with KIT S476I: a rare entity defined by leukemic expansion of mature mast cells and absence of organ damage. Ann Hematol 2015; 94: 223–231.
    Article PubMed Google Scholar
  40. Ustun C, Reiter A, Scott BL, Nakamura R, Damaj G, Kreil S et al. Hematopoietic stem-cell transplantation for advanced systemic mastocytosis. J Clin Oncol 2014; 32: 3264–3274.
    Article PubMed PubMed Central Google Scholar
  41. Schwaab J, Schnittger S, Sotlar K, Walz C, Fabarius A, Pfirrmann M et al. Comprehensive mutational profiling in advanced systemic mastocytosis. Blood 2013; 122: 2460–2466.
    Article CAS PubMed Google Scholar
  42. Jawhar M, Schwaab J, Schnittger S, Meggendorfer M, Pfirrmann M, Sotlar K et al. Additional mutations in SRSF2, ASXL1 and/or RUNX1 identify a high-risk group of patients with KIT D816V(+) advanced systemic mastocytosis. Leukemia 2016; 30: 136–143.
    Article CAS PubMed Google Scholar
  43. Jawhar M, Schwaab J, Meggendorfer M, Naumann N, Horny HP, Sotlar K et al. The clinical and molecular diversity of mast cell leukemia with or without associated hematologic neoplasm. Haematologica 2017; 102: 1035–1043.
    Article CAS PubMed PubMed Central Google Scholar
  44. Jawhar M, Schwaab J, Naumann N, Horny HP, Sotlar K, Haferlach T et al. Response and progression on midostaurin in advanced systemic mastocytosis: KIT D816V and other molecular markers. Blood 2017; 130: 137–145.
    Article CAS PubMed Google Scholar

Download references