Mastocytosis: Update on pharmacotherapy and future directions

Expert Opinion on Pharmacotherapy

Volumn 14, Issue 15, 2013, Pages 2033-2045

  Cardet, Juan Carlos ^a   Akin, Cem ^a   Lee, Min Jung ^a  

^a BRIGHAM AND WOMEN S HOSPITAL   (United States)

Author keywords

Antihistamines; Mastocytosis; Tyrosine kinase inhibitors

Indexed keywords

ACETYLSALICYLIC ACID; ADRENALIN; ALENDRONIC ACID; AZELASTINE; BISPHOSPHONIC ACID DERIVATIVE; CETIRIZINE; CHLORPHENIRAMINE; CIMETIDINE; CROMOGLYCATE DISODIUM; CYPROHEPTADINE; DESLORATADINE; DIPHENHYDRAMINE; DOXEPIN; ETIDRONIC ACID; FAMOTIDINE; FEXOFENADINE; GLUCOCORTICOID; HYDROXYZINE; KETOTIFEN; LEVOCETIRIZINE; LORATADINE; METHOXSALEN; MONTELUKAST; PAMIDRONIC ACID; POLIDOCANOL; RANITIDINE; RISEDRONIC ACID; TIXOCORTOL; UNINDEXED DRUG; ZINC SULFATE;

ABDOMINAL CRAMP; ABDOMINAL PAIN; ANAPHYLACTIC SHOCK; ANAPHYLAXIS; ASCITES; BLOATING; BONE MARROW TRANSPLANTATION; BONE PAIN; COMORBIDITY; CONSTIPATION; CORTICOSTEROID THERAPY; CYTOREDUCTIVE SURGERY; DIARRHEA; DIZZINESS; DOSAGE SCHEDULE COMPARISON; DRUG ABSORPTION; DRUG DOSE INCREASE; DRUG MEGADOSE; DYSPEPSIA; FATIGUE; FLUSHING; GASTROESOPHAGEAL REFLUX; GASTROINTESTINAL SYMPTOM; HEADACHE; HEART VENTRICLE ARRHYTHMIA; HUMAN; HYPOTENSION; INSOMNIA; IRRITABILITY; LEUKEMIA; LOW DRUG DOSE; MASTOCYTOSIS; MEDLINE; MUSCULOSKELETAL PAIN; NAUSEA; OSTEOPOROSIS; OSTEOSCLEROSIS; PHOTOTHERAPY; PROGNOSIS; PRURITUS; QT PROLONGATION; RESPIRATORY DISTRESS; REVIEW; RHINORRHEA; RISK REDUCTION; SARCOMA; SIDE EFFECT; SKIN DISEASE; STEATORRHEA; SYSTEMIC MASTOCYTOSIS; THROAT DISEASE; TORSADE DES POINTES; TREATMENT RESPONSE; ULTRAVIOLET A RADIATION; URTICARIA; URTICARIA PIGMENTOSA; VOMITING;

HUMANS; IMMUNOLOGIC FACTORS; MASTOCYTOSIS; PROTEIN KINASE INHIBITORS;

EID: 84884541237     PISSN: 14656566     EISSN: 17447666     Source Type: Journal    
DOI: 10.1517/14656566.2013.824424     Document Type: Review

References (111)

1. Valent P, Horny HP, Escribano L, et al. Diagnostic criteria and classification of mastocytosis: a consensus proposal. Leuk Res 2001;25(7):603-25
2. Valent P, Akin C, Escribano L, et al. Standards and standardization in mastocytosis: consensus statements on diagnostics, treatment recommendations and response criteria. Eur J Clin Invest 2007;37(6):435-53
3. Fuller SJ. New insights into the pathogenesis, diagnosis, and management of mastocytosis. Hematol Oncol Clin North Am 2012;26(6):1143-68
4. Escribano L, Akin C, Castells M, et al. Current options in the treatment of mast cell mediator-related symptoms in mastocytosis. Inflamm Allergy Drug Targets 2006;5(1):61-77
5. Escribano L, Akin C, Castells M, et al. Mastocytosis: current concepts in diagnosis and treatment. Ann Hematol 2002;81(12):677-90
6. Wilson TM, Metcalfe DD, Robyn J. Treatment of systemic mastocytosis. Immunol Allergy Clin North Am 2006;26(3):549-73
7. Worobec AS. Treatment of systemic mast cell disorders. Hematol Oncol Clin North Am 2000;14(3):659-87, vii
8. Metcalfe DD. The treatment of mastocytosis: an overview. J Invest Dermatol 1991;96(3 Suppl):55S-6S; discussion 56S-59S, 60S-65S
9. Akin C. Molecular diagnosis of mast cell disorders: a paper from the 2005 William Beaumont Hospital Symposium on Molecular Pathology. J Mol Diagn 2006;8(4):412-19
10. Castells M, Austen KF. Mastocytosis: mediator-related signs and symptoms. Int Arch Allergy Immunol 2002;127(2):147-52
11. Valent P, Akin C, Arock M, et al. Definitions, criteria and global classification of mast cell disorders with special reference to mast cell activation syndromes: a consensus proposal. Int Arch Allergy Immunol 2012;157(3):215-25
12. Worobec AS, Metcalfe DD. Mastocytosis: current treatment concepts. Int Arch Allergy Immunol 2002;127(2):153-5
13. Simons FERA, Cezmi A. Histamine and H1 antihistamines. In: Adkinson NF, editor. Middleton's allergy: principles and practice. 7th edition. Mosby; China: 2009
14. Friedman BS, Santiago ML, Berkebile C, et al. Comparison of azelastine and chlorpheniramine in the treatment of mastocytosis. J Allergy Clin Immunol 1993;92(4):520-6
15. Frieri M, Alling DW, Metcalfe DD. Comparison of the therapeutic efficacy of cromolyn sodium with that of combined chlorpheniramine and cimetidine in systemic mastocytosis. Results of a double-blind clinical trial. Am J Med 1985;78(1):9-14
16. Gasior-Chrzan B, Falk ES. Systemic mastocytosis treated with histamine H1 and H2 receptor antagonists. Dermatology 1992;184(2):149-52
17. Marone G, Spadaro G, Granata F, et al. Treatment of mastocytosis: pharmacologic basis and current concepts. Leuk Res 2001;25(7):583-94
18. Pratt CM, Hertz RP, Ellis BE, et al. Risk of developing life-threatening ventricular arrhythmia associated with tefenadine in comparison with over-the-counter antihistamines, ibuprofen and clemastine. Am J Cardiol 1994;73(5):346-52
19. Berg MJ, Bernhard H, Schentag JJ. Cimetidine in systemic mastocytosis. Drug Intell Clin Pharm 1981;15(3):180-3
20. Bredfeldt JE, O'Laughlin JC, Durham JB, et al. Malabsorption and gastric hyperacidity in systemic mastocytosis. Results of cimetidine therapy. Am J Gastroenterol 1980;74(2):133-7
21. Hirschowitz BI, Groarke JF. Effect of cimetidine on gastric hypersecretion and diarrhea in systemic mastocytosis. Ann Intern Med 1979;90(5):769-71
22. Achord JL, Langford H. The effect of cimetidine and propantheline on the symptoms of a patient with systemic mastocytosis. Am J Med 1980;69(4):610-14
23. Johnson GJ, Silvis SE, Roitman B, et al. Long-term treatment of systemic mastocytosis with histamine H2 receptor antagonists. Am J Gastroenterol 1980;74(6):485-9
24. Edwards AMH, Stephen T. The Chromones: sodium cromolyn and nedocromil sodium. In: Adkinson NF, editor. Middleton's allergy: principles and practice. 7th edition. Mosby; China: 2009
25. Alexander RR. Disodium cromoglycate in the treatment of systemic mastocytosis involving only bone. Acta Haematol 1985;74(2):108-10
26. Soter NA, Austen KF, Wasserman SI. Oral disodium cromoglycate in the treatment of systemic mastocytosis. N Engl J Med 1979;301(9):465-9
27. Horan RF, Sheffer AL, Austen KF. Cromolyn sodium in the management of systemic mastocytosis. J Allergy Clin Immunol 1990;85(5):852-5
28. Mallet AI, Norris P, Rendell NB, et al. The effect of disodium cromoglycate and ketotifen on the excretion of histamine and N tau-methylimidazole acetic acid in urine of patients with mastocytosis. Br J Clin Pharmacol 1989;27(1):88-91
29. Dolovich J, Punthakee ND, MacMillan AB, et al. Systemic mastocytosis: control of lifelong diarrhea by ingested disodium cromoglycate. Can Med Assoc J 1974;111(7):684-5
30. Zachariae H, Herlin T, Larsen PO. Oral disodium cromoglycate in mastocytosis. Acta Derm Venereolo 1981;61(3):272-3
31. Businco L, Cantani A, Businco E, et al. Systemic mastocytosis in a 5-year-old child: successful treatment with disodium cromoglycate. Clin Allergy 1984;14(2):147-52
32. Czarnetzki BM, Behrendt H. Urticaria pigmentosa: clinical picture and response to oral disodium cromoglycate. Br J Dermatol 1981;105(5):563-7
33. Lindskov R, Lange Wantzin G, Knudsen L, et al. Urticaria pigmentosa treated with oral disodium cromoglycate. Dermatologica 1984;169(1):49-52
34. Welch EA, Alper JC, Bogaars H, et al. Treatment of bullous mastocytosis with disodium cromoglycate. J Am Acad Dermatol 1983;9(3):349-53
35. Cardet JC, Castells MC, Hamilton MJ. Immunology and clinical manifestations of non-clonal mast cell activation syndrome. Curr Allergy Asthma Rep 2013;13(1):10-18
36. Patterson R, Fitzsimons EJ, Choy AC, et al. Malignant and corticosteroid-dependent idiopathic anaphylaxis: successful responses to ketotifen. Ann Allergy Asthma Immunol 1997;79(2):138-44
37. Dykewicz MS, Wong SS, Patterson R, et al. Evaluation of ketotifen in corticosteroid-dependent idiopathic anaphylaxis. Ann Allergy 1990;65(5):406-10
38. Kettelhut BV, Berkebile C, Bradley D, et al. A double-blind, placebo-controlled, crossover trial of ketotifen versus hydroxyzine in the treatment of pediatric mastocytosis. J Allergy Clin Immunol 1989;83(5):866-70
39. Ting S. Ketotifen and systemic mastocytosis. J Allergy Clin Immunol 1990;85(4):818
40. Graves L III, Stechschulte DJ, Morris DC, et al. Inhibition of mediator release in systemic mastocytosis is associated with reversal of bone changes. J Bone Min Res 1990;5(11):1113-19
41. Povoa P, Ducla-Soares J, Fernandes A, et al. A case of systemic mastocytosis; therapeutic efficacy of ketotifen. J Intern Med 1991;229(5):475-7
42. Castells M, Metcalfe DD, Escribano L. Diagnosis and treatment of cutaneous mastocytosis in children: practical recommendations. Am J Clin Dermatol 2011;12(4):259-70
43. Schleimer RP. Pharmacology of glucocorticoids in allergic disease. In: Adkinson NF, editor. Middleton's allergy: principles and practice. 7th edition. Mosby
44. Friedman BS, Metcalfe DD. Effects of tixocortol pivalate on gastrointestinal disease in systemic mastocytosis: a preliminary study. Clin Exp Allergy 1991;21(2):183-8
45. Barton J, Lavker RM, Schechter NM, et al. Treatment of urticaria pigmentosa with corticosteroids. Arch Dermatol 1985;121(12):1516-23
46. Boyce JA. Eicosanoids in asthma, allergic inflammation, and host defense. Curr Mol Med 2008;8(5):335-49
47. Wenzel SE. Antileukotriene therapy in asthma. In: Adkinson NF, editor. Middleton's allergy: principles and practice. 7th edition. Mosby
48. Castells M. Mast cell mediators in allergic inflammation and mastocytosis. Immunol Allergy Clin North Am 2006;26(3):465-85
49. Butterfield JH. Increased leukotriene E4 excretion in systemic mastocytosis. Prostaglandins Other Lipid Mediat 2010;92(1-4):73-6
50. Tolar J, Tope WD, Neglia JP. Leukotriene-receptor inhibition for the treatment of systemic mastocytosis. N Engl J Med 2004;350(7):735-6
51. Turner PJ, Kemp AS, Rogers M, et al. Refractory symptoms successfully treated with leukotriene inhibition in a child with systemic mastocytosis. Pediatr Dermatol 2012;29(2):222-3
52. Roberts LJ II, Sweetman BJ, Lewis RA, et al. Increased production of prostaglandin D2 in patients with systemic mastocytosis. N Engl J Med 1980;303(24):1400-4
53. Roberts LJ II, Turk JW, Oates JA. Shock syndrome associated with mastocytosis: pharmacologic reversal of the acute episode and therapeutic prevention of recurrent attacks. Adv Shock Res 1982;8:145-52
54. Meade EA, Smith WL, DeWitt DL. Differential inhibition of prostaglandin endoperoxide synthase (cyclooxygenase) isozymes by aspirin and other non-steroidal anti-inflammatory drugs. J Biol Chem 1993;268(9):6610-14
55. Butterfield JH. Survey of aspirin administration in systemic mastocytosis. Prostaglandins Other Lipid Mediat 2009;88(3-4):122-4
56. Butterfield JH, Weiler CR. Prevention of mast cell activation disorder-associated clinical sequelae of excessive prostaglandin D(2) production. Int Arch Allergy Immunol 2008;147(4):338-43
57. Lorcerie B, Arveux I, Chauffert B, et al. Aspirin and systemic mastocytosis. Lancet 1989;2(8672):1155
58. Crawhall JC, Wilkinson RD. Systemic mastocytosis: management of an unusual case with histamine (H1 and H2) antagonists and cyclooxygenase inhibition. Clin Invest Med 1987;10(1):1-4
59. Greenhawt M, Akin C. Mastocytosis and allergy. Curr Opin Allergy Clin Immunol 2007;7(5):387-92
60. Simons FE, Gu X, Silver NA, et al. EpiPen Jr versus EpiPen in young children weighing 15 to 30 kg at risk for anaphylaxis. J Allergy Clin Immunol 2002;109(1):171-5
61. Turk J, Oates JA, Roberts LJ II. Intervention with epinephrine in hypotension associated with mastocytosis. J Allergy Clin Immunol 1983;71(2):189-92
62. Lieberman P, Nicklas RA, Oppenheimer J, et al. The diagnosis and management of anaphylaxis practice parameter: 2010 update. J Allergy Clin Immunol 2010;126(3):477-80; e1-42
63. Simons FE, Ardusso LR, Bilo MB, et al. World allergy organization guidelines for the assessment and management of anaphylaxis. World Allergy Organ J 2011;4(2):13-37
64. Muraro A, Roberts G, Clark A, et al. The management of anaphylaxis in childhood: position paper of the European academy of allergology and clinical immunology. Allergy 2007;62(8):857-71
65. Barete S, Assous N, de Gennes C, et al. Systemic mastocytosis and bone involvement in a cohort of 75 patients. Ann Rheum Dis 2010;69(10):1838-41
66. Bellido T, Plotkin LI. Novel actions of bisphosphonates in bone: preservation of osteoblast and osteocyte viability. Bone 2011;49(1):50-5
67. Brumsen C, Hamdy NA, Papapoulos SE. Osteoporosis and bone marrow mastocytosis: dissociation of skeletal responses and mast cell activity during long-term bisphosphonate therapy. J Bone Min Res 2002;17(4):567-9
68. Cundy T, Beneton MN, Darby AJ, et al. Osteopenia in systemic mastocytosis: natural history and responses to treatment with inhibitors of bone resorption. Bone 1987;8(3):149-55
69. Marshall A, Kavanagh RT, Crisp AJ. The effect of pamidronate on lumbar spine bone density and pain in osteoporosis secondary to systemic mastocytosis. Br J Rheumatol 1997;36(3):393-6
70. Godt O, Proksch E, Streit V, et al. Short-and long-term effectiveness of oral and bath PUVA therapy in urticaria pigmentosa and systemic mastocytosis. Dermatology 1997;195(1):35-9
71. Czarnetzki BM, Rosenbach T, Kolde G, et al. Phototherapy of urticaria pigmentosa: clinical response and changes of cutaneous reactivity, histamine and chemotactic leukotrienes. Arch Dermatol Res 1985;277(2):105-13
72. Kinsler VA, Hawk JL, Atherton DJ. Diffuse cutaneous mastocytosis treated with psoralen photochemotherapy: case report and review of the literature. Br J Dermatol 2005;152(1):179-80
73. Bowling J, Cork MJ. Severe pruritus in a patient with urticaria pigmentosa treated with topical 5% urea and 3% polidocanol cream. J Dermatolog Treat 2003;14(3):190-1
74. Correia O, Duarte AF, Quirino P, et al. Cutaneous mastocytosis: two pediatric cases treated with topical pimecrolimus. Dermatol Online J 2010;16(5):8
75. Carter MC, Robyn JA, Bressler PB, et al. Omalizumab for the treatment of unprovoked anaphylaxis in patients with systemic mastocytosis. J Allergy Clin Immunol 2007;119(6):1550-1
76. Douglass JA, Carroll K, Voskamp A, et al. Omalizumab is effective in treating systemic mastocytosis in a nonatopic patient. Allergy 2010;65(7):926-7
77. Siebenhaar F, Kuhn W, Zuberbier T, et al. Successful treatment of cutaneous mastocytosis and Meniere disease with anti-IgE therapy. J Allergy Clin Immunol 2007;120(1):213-15
78. Kontou-Fili K. High omalizumab dose controls recurrent reactions to venom immunotherapy in indolent systemic mastocytosis. Allergy 2008;63(3):376-8
79. Molderings GJ, Raithel M, Kratz F, et al. Omalizumab treatment of systemic mast cell activation disease: experiences from four cases. Intern Med 2011;50(6):611-15
80. Bonadonna P, Zanotti R, Muller U. Mastocytosis and insect venom allergy. Curr Opin Allergy Clin Immunol 2010;10(4):347-53
81. Oude Elberink JN, de Monchy JG, Kors JW, et al. Fatal anaphylaxis after a yellow jacket sting, despite venom immunotherapy, in two patients with mastocytosis. J Allergy Clin Immunol 1997;99(1 Pt 1):153-4
82. Haeberli G, Bronnimann M, Hunziker T, et al. Elevated basal serum tryptase and hymenoptera venom allergy: relation to severity of sting reactions and to safety and efficacy of venom immunotherapy. Clin Exp Allergy 2003;33(9):1216-20
83. Bonadonna P, Perbellini O, Passalacqua G, et al. Clonal mast cell disorders in patients with systemic reactions to Hymenoptera stings and increased serum tryptase levels. J Allergy Clin Immunol 2009;123(3):680-6
84. Gonzalez de Olano D, Alvarez-Twose I, Esteban-Lopez MI, et al. Safety and effectiveness of immunotherapy in patients with indolent systemic mastocytosis presenting with Hymenoptera venom anaphylaxis. J Allergy Clin Immunol 2008;121(2):519-26
85. Boissan M, Feger F, Guillosson JJ, et al. c-Kit and c-kit mutations in mastocytosis and other hematological diseases. J Leukoc Biol 2000;67(2):135-48
86. Ustun C, DeRemer DL, Akin C. Tyrosine kinase inhibitors in the treatment of systemic mastocytosis. Leuk Res 2011;35(9):1143-52
87. Akin C, Fumo G, Yavuz AS, et al. A novel form of mastocytosis associated with a transmembrane c-kit mutation and response to imatinib. Blood 2004;103(8):3222-5
88. 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(8):686-92
89. Frost MJ, Ferrao PT, Hughes TP, et al. Juxtamembrane mutant V560GKit is more sensitive to imatinib (STI571) compared with wild-type c-kit whereas the kinase domain mutant D816VKit is resistant. Mol Cancer Ther 2002;1(12):1115-24
90. Vega-Ruiz A, Cortes JE, Sever M, et al. Phase II study of imatinib mesylate as therapy for patients with systemic mastocytosis. Leuk Res 2009;33(11):1481-4
91. Lim KH, Pardanani A, Butterfield JH, et al. Cytoreductive therapy in 108 adults with systemic mastocytosis: outcome analysis and response prediction during treatment with interferon-alpha, hydroxyurea, imatinib mesylate or 2-chlorodeoxyadenosine. Am J Hematol 2009;84(12):790-4
92. Pagano L, Valentini CG, Caira M, et al. Advanced mast cell disease: an Italian Hematological Multicenter experience. Int J Hematol 2008;88(5):483-8
93. von Bubnoff N, Gorantla SH, Kancha RK, et al. The systemic mastocytosis-specific activating cKit mutation D816V can be inhibited by the tyrosine kinase inhibitor AMN107. Leukemia 2005;19(9):1670-1
94. Verstovsek S, Akin C, Manshouri T, et al. Effects of AMN107, a novel aminopyrimidine tyrosine kinase inhibitor, on human mast cells bearing wild-type or mutated codon 816 c-kit. Leuk Res 2006;30(11):1365-70
95. Hochhaus A, Ottmann OG, Lauber S, et al. A phase II study of nilotinib, a novel inhibitor of c-Kit, PDGFR, and Bcr-Abl, administered to patients with systemic mastocytosis. Blood 2006;108
96. Shah NP, Lee FY, Luo R, et al. Dasatinib (BMS-354825) inhibits KITD816V, an imatinib-resistant activating mutation that triggers neoplastic growth in most patients with systemic mastocytosis. Blood 2006;108(1):286-91
97. Schittenhelm MM, Shiraga S, Schroeder A, et al. Dasatinib (BMS-354825), a dual SRC/ABL kinase inhibitor, inhibits the kinase activity of wild-type, juxtamembrane, and activation loop mutant KIT isoforms associated with human malignancies. Cancer Res 2006;66(1):473-81
98. Verstovsek S, Tefferi A, Cortes J, et al. Phase II study of dasatinib in Philadelphia chromosome-negative acute and chronic myeloid diseases, including systemic mastocytosis. Clin Cancer Res 2008;14(12):3906-15
99. Purtill D, Cooney J, Sinniah R, et al. Dasatinib therapy for systemic mastocytosis: four cases. Eur J Haematol 2008;80(5):456-8
100. Meyer T, Regenass U, Fabbro D, et al. A derivative of staurosporine (CGP 41 251) shows selectivity for protein kinase C inhibition and in vitro anti-proliferative as well as in vivo anti-tumor activity. Int J Cancer 1989;43(5):851-6
101. Gotlib J, Kluin-Nelemans HC, George T, et al. KIT Inhibitor midostaurin in patients with advanced systemic mastocytosis: results of a planned interim analysis of the Global CPKC412D2201 Trial. 54th ASH Annual meeting and Exposition; Atlanta, GA; 2012
102. Dubreuil P, Letard S, Ciufolini M, et al. Masitinib (AB1010), a potent and selective tyrosine kinase inhibitor targeting KIT. PLoS One 2009;4(9):e7258
103. Hahn KA, Legendre AM, Shaw NG, et al. Evaluation of 12-and 24-month survival rates after treatment with masitinib in dogs with nonresectable mast cell tumors. Am J Vet Res 2010;71(11):1354-61
104. Paul C, Sans B, Suarez F, et al. Masitinib for the treatment of systemic and cutaneous mastocytosis with handicap: a phase 2a study. Am J Hematol 2010;85(12):921-5
105. Lehmann T, Lammle B. IFNalpha treatment in systemic mastocytosis. Ann Hematol 1999;78(10):483-4
106. Hauswirth AW, Simonitsch-Klupp I, Uffmann M, 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(3):249-57
107. Pardanani A. Systemic mastocytosis in adults: 2011 update on diagnosis, risk stratification, and management. Am J Hematol 2011;86(4):362-71
108. Simon J, Lortholary O, Caillat-Vigneron N, et al. Interest of interferon alpha in systemic mastocytosis. The French experience and review of the literature. Pathol Biol 2004;52(5):294-9
109. Hermine O, Hirsh I, Damaj G, et al. Long term efficacy and safety of cladribine in adult systemic mastocytosis: a French multicenter study of 44 patients. Blood 2010;116:1982
110. Horny HP MD, Bennett JM, et al. WHO classification of mastocytosis. WHO classification of tumours of haematopoietic and lymphoid tissues. IARC Press; Lyon, France: 2008. p. 54-63
111. Valent P, Akin C, Sperr WR, et al. Aggressive systemic mastocytosis and related mast cell disorders: current treatment options and proposed response criteria. Leuk Res 2003;27(7):635-41