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1.
Am J Hematol ; 99(6): 1095-1102, 2024 Jun.
Artigo em Inglês | MEDLINE | ID: mdl-38581211

RESUMO

Systemic mastocytosis (SM) corresponds to a rare and heterogeneous spectrum of diseases characterized by the accumulation of atypical mast cells (MCs). Advanced mastocytosis (Adv-SM) is associated with poor survival; in contrast, patients with non-advanced SM (non-Adv-SM) usually have a normal life expectancy but may experience poor quality of life. Despite recent therapeutic progress including tyrosine kinase inhibitors, new treatment options are needed for refractory and/or intolerant patients with both severely symptomatic and Adv-SM. In vitro, the mTOR pathway is activated in MCs from patients bearing the KIT D816V mutation. Furthermore, rapamycin induces the apoptosis of KIT D816V MCs selectively. In this nationwide study, we report the outcomes of patients diagnosed with SM and treated with a mammalian target of rapamycin inhibitor (imTOR) within the French National Reference Center for mastocytosis (CEREMAST). All patients registered were relapsing, treatment-refractory, or ineligible for other cytoreductive therapy. Non-Adv-SM patients received imTOR as a monotherapy (rapamycin/everolimus), and Adv-SM patients received imTOR as a monotherapy or in combination with cytarabine. The objective response rate (ORR) in non-Adv-SM was 60% (partial response in 40% and major response in 20%), including reductions in skin involvement, mediator release symptoms, and serum tryptase. In the Adv-SM group, the ORR was 20% (including one major response and one partial response, both in patients with a KIT D816V mutation), which enabled a successful bridge to allogeneic stem cell transplantation in one patient. Our results suggest that imTOR treatment has potential benefits in patients with SM harboring a KIT D816V mutation.


Assuntos
Inibidores de MTOR , Mastocitose Sistêmica , Sirolimo , Humanos , Mastocitose Sistêmica/tratamento farmacológico , Projetos Piloto , Feminino , Masculino , Pessoa de Meia-Idade , Adulto , França , Idoso , Sirolimo/uso terapêutico , Sirolimo/efeitos adversos , Inibidores de MTOR/uso terapêutico , Proteínas Proto-Oncogênicas c-kit/genética , Proteínas Proto-Oncogênicas c-kit/antagonistas & inibidores , Everolimo/uso terapêutico , Everolimo/efeitos adversos , Resultado do Tratamento , Serina-Treonina Quinases TOR/antagonistas & inibidores , Idoso de 80 Anos ou mais
3.
Presse Med ; 44(6 Pt 1): 639-46, 2015 Jun.
Artigo em Francês | MEDLINE | ID: mdl-26033556

RESUMO

Influenza is a respiratory disease caused by influenza viruses. The virus is responsible for pandemics by emergence of new viral strains, then for seasonal flu by antigenic drift. Seasonal flu is more frequent and severe in pregnant women, with increased risk of pneumonia and increased risk of hospitalization (but no increased death reported). Pandemic flu is more severe in pregnant women, with increased risk of pneumonia and increased mortality. Influenza vaccination is recommended for all women who are or will be pregnant (in any trimester) during influenza season. After closed contact with a flu case or in case of symptoms compatible with flu, early oseltamivir-based treatment (prophylactic in the first situation, curative in the latter one) is recommended, at any term of pregnancy. The occurrence of flu symptoms in a pregnant woman requires medical evaluation to confirm the diagnosis or identify any alternative infection requiring appropriate therapy like listeriosis, chorioamniotitis, pyelonephritis or viral primo-infection.


Assuntos
Influenza Humana , Complicações Infecciosas na Gravidez/virologia , Feminino , Humanos , Vacinas contra Influenza , Influenza Humana/diagnóstico , Influenza Humana/prevenção & controle , Gravidez , Complicações Infecciosas na Gravidez/diagnóstico , Complicações Infecciosas na Gravidez/prevenção & controle
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