Catching the clinical and biological diversity for an appropriate therapeutic approach in systemic mastocytosis.
Ann Hematol
; 100(2): 337-344, 2021 Feb.
Article
em En
| MEDLINE
| ID: mdl-33156374
ABSTRACT
Systemic mastocytosis (SM) is a rare disease calling for integrated approaches involving onco-hematologic competences for appropriate clinical management and treatment. The wide variability of manifestations and disease course claims for an accurate risk stratification, currently relying on the appraisal of the benefit/risk ratio of treatment modalities within indolent and advanced variants according to WHO classification. More objective parameters are progressively incorporated and integrated into comprehensive models, on which to support the adoption of therapeutic strategies, since the mere clinical distinction between mediator-related signs/symptoms and "true" organ damage can sometimes be complicated. The development of novel targeted drugs is progressively extending the therapeutic alternatives available, which ranges from conventional agents such as interferon and cladribine, to the more modern approach based on KIT inhibition. Ultimately, the choice of the most appropriate therapy should be rationalized on the basis of the clinical picture and molecular data. The focus of the present review is on the areas still open in the current evaluation of SM patients, particularly when considering the need of a treatment.
Palavras-chave
Texto completo:
1
Base de dados:
MEDLINE
Assunto principal:
Interferons
/
Cladribina
/
Proteínas Proto-Oncogênicas c-kit
/
Mastocitose Sistêmica
Tipo de estudo:
Prognostic_studies
Limite:
Humans
Idioma:
En
Ano de publicação:
2021
Tipo de documento:
Article