Clinical, radiological and molecular responses to combination chemotherapy with MAPK pathway inhibition in relapsed and refractory Langerhans cell histiocytosis.
Br J Haematol
; 204(5): 1882-1887, 2024 May.
Article
em En
| MEDLINE
| ID: mdl-38501390
ABSTRACT
Optimal therapeutic approaches for advanced Langerhans cell histiocytosis (LCH) are not known. We assessed the safety and efficacy of combined chemotherapy with MAPK pathway inhibition in 10 patients with refractory systemic disease and/or LCH-associated neurodegeneration. Overall response rate was 9/10 (90%) for the entire cohort 5/5 (100%) for patients with systemic disease and 6/7 (86%) for patients with central nervous system disease. BRAFV600E+ peripheral blood fraction decreased in 5/6 (83%). Toxicities included fever, skin rash, myalgias, neuropathy, cytopenias and hypocalcaemia. Prospective trials are required to optimize combination strategies, determine potential to achieve cure and compare outcomes to chemotherapy or MAPK inhibitor monotherapy.
Palavras-chave
Texto completo:
1
Coleções:
01-internacional
Temas:
Geral
Base de dados:
MEDLINE
Assunto principal:
Histiocitose de Células de Langerhans
/
Sistema de Sinalização das MAP Quinases
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Inibidores de Proteínas Quinases
Limite:
Female
/
Humans
/
Male
Idioma:
En
Revista:
Br J Haematol
Ano de publicação:
2024
Tipo de documento:
Article
País de afiliação:
Estados Unidos