Diagnosis and treatment of systemic mastocytosis in Brazil: Recommendations of a multidisciplinary expert panel

Velloso, Elvira D. Rodrigues Pereira; Padulla, Geórgia A.; Cerqueira, Ana Maria Mósca de; Sousa, Adriana Martins de; Sandes, Alex Freire; Traina, Fabiola; Seguro, Fernanda Salles; Nogueira, Frederico Lisboa; Pereira, Grazielly de Fátima; Boechat, José Laerte; Pagnano, Katia Borgia Barbosa; Marchi, Luan Lima; Ensina, Luis Felipe; Giavina-Bianchi, Mara; Aun, Marcelo Vivolo; Agondi, Rosana Câmara; Santos, Fabio Pires de Souza; Giavina-Bianchi, Pedro

doi:10.1016/j.htct.2022.04.006

Acessibilidade / Reportar erro

Brasil

Hematology, Transfusion and Cell Therapy

Español English

Brasil

Español English

sumário « anterior atual seguinte »

Sumário

Special article • Hematol., Transfus. Cell Ther. 44 (4) • 2022 • https://doi.org/10.1016/j.htct.2022.04.006 copy

Diagnosis and treatment of systemic mastocytosis in Brazil: Recommendations of a multidisciplinary expert panel

Authorship SCIMAGO INSTITUTIONS RANKINGS

ABSTRACT

Introduction:

Systemic Mastocytosis comprises a group of neoplastic diseases characterized by clonal expansion and infiltration of mast cells into several organs. The diagnosis and treatment of this disease may be challenging for non-specialists. Objective: Make suggestions or recommendations in Systemic Mastocytosis based in a panel of Brazilian specialists.

Method and results:

An online expert panel with 18 multidisciplinary specialists was convened to propose recommendations on the diagnosis and treatment of Systemic Mastocytosis in Brazil. Recommendations were based on discussions of topics and multiple-choice questions and were graded using the Oxford Centre for Evidence-Based Medicine 2011 Levels of Evidence Chart.

Conclusion:

Twenty-two recommendations or suggestions were proposed based on a literature review and graded according to the findings.

Keywords:
Mastocytosis; Expert panel; Adults; Children; Diagnosis; Treatment

Drug	Mechanism of action	Dose, route of administration	Overall response rate	Year, ref.
Interferon	Probably inhibits the degranulation of mast cells.	1.2 to 5 million units subcutaneous or intramuscular (3 times weekly) combination with corticosteroids	49 to 53%	2009⁸⁷87 Alvarez-Twose I, Matito A, Morgado JM, Munoz LS-,Jara-Acevedo M, García-Montero A, et al. Imatinib in systemic mastocytosis: a phase IV clinical trial in patients lacking exon 17 KIT mutations and review of the literature. Oncotarget. 2016;8(40):68950–63. 2011⁹⁰90 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(8):1009–16. 2017⁸⁸88 Weis Bjerrum O. Interferon-a treatment in systemic mastocytosis. Curr Drug Targets. 2011;12(3):433–6.
Cladribine	Synthetic purine analogue cytoreductive	0.13 to 0.17 mg/kg in infusion or subcutaneously, days 1-5; repeated at 4 - 12 weeks until 9 courses	50 to 55%	2009⁹¹91 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-chlorodeoxyadenosine. Am J Hematol. 2009;84(12):790–4. 2015⁹⁰90 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(8):1009–16. 2018⁸⁹89 Lei D, Akin C, Kovalszki A. Management of mastocytosis in pregnancy: a review. J Allergy Clin Immunol Pract. 2017;5 (5):1217–23.
Imatinib	Inhibitor type II of protein tyrosine kinases, such as ABL1, PDGFR, ARG and KIT (resistant to D816V mutation)	100 to 400 mg orally twice daily	35to 50%	2006⁸³83 Mannelli F. Catching the clinical and biological diversity for an appropriate therapeutic approach in systemic mastocytosis. Ann Hematol. 2021;100(2):337-44. 2009⁸⁴84 Droogendijk HJ, Kluin-Nelemans HJC, van Doormaal JJ, Oranje AP, van de Loosdrecht AA, van Daele PLA. Imatinib mesylate in the treatment of systemic mastocytosis. Cancer. 2006;107(2):345-51. 2019⁸⁶86 Ustun C, DeRemer DL, Akin C. Tyrosine kinase inhibitors in the treatment of systemic mastocytosis. Leuk Res. 2011;35 (9):1143-52.
Midostaurin	Type I multikinase inhibitor that inhibits the protein product of KIT	100 mg orally twice daily	60to 69%	2016⁹³93 Gotlib J, Kluin-Nelemans 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(26):2530–41. 2018⁹⁴94 DeAngelo DJ, George TI, Linder A, Langford C, Perkins C, Ma J, et al. Efficacy and safety of midostaurin in patients with advanced systemic mastocytosis: 10-year median follow-up of a phase II trial. Leukemia. 2018;32(2):470–8.
Avapritinib	Type I multikinase inhibitor with highly selective and potent activity against mutated KIT (including D816V)	200 mg orally once daily	75%	2021⁹⁷97 Chandesris M-O, Damaj G, Canioni D, Brouzes C, Lhermitte L, Hanssens K, et al. Midostaurin in advanced systemic mastocytosis. N Engl J Med. 2016;374(26):2605–6.

Associação Brasileira de Hematologia, Hemoterapia e Terapia Celular (ABHH) R. Dr. Diogo de Faria, 775 cj 133, 04037-002, São Paulo / SP - Brasil - São Paulo - SP - Brazil
E-mail: htct@abhh.org.br

Acompanhe os números deste periódico no seu leitor de RSS

[1] *Corresponding author at: Service of Hematology, Transfusion and Cell Therapy and Laboratory of Medical Investigation in Pathogenesis and Directed Therapy in Onco-Immuno-Hematology, Hospital das Clínicas da Faculdade de Medicina da Universidade de São Paulo (LIM-31 HCFMUSP), São Paulo, SP CEP 05403-000, Brazil. E-mail address: elvira.velloso@fm.usp.br (E.D.R.P. Velloso).