Clofarabine monotherapy in aggressive, relapsed and refractory Langerhans cell histiocytosis.
Br J Haematol
; 204(5): 1888-1893, 2024 May.
Article
em En
| MEDLINE
| ID: mdl-38501389
ABSTRACT
Over 50% of patients with systemic LCH are not cured with front-line therapies, and data to guide salvage options are limited. We describe 58 patients with LCH who were treated with clofarabine. Clofarabine monotherapy was active against LCH in this cohort, including heavily pretreated patients with a systemic objective response rate of 92.6%, higher in children (93.8%) than adults (83.3%). BRAFV600E+ variant allele frequency in peripheral blood is correlated with clinical responses. Prospective multicentre trials are warranted to determine optimal dosing, long-term efficacy, late toxicities, relative cost and patient-reported outcomes of clofarabine compared to alternative LCH salvage therapy strategies.
Palavras-chave
Texto completo:
1
Coleções:
01-internacional
Base de dados:
MEDLINE
Assunto principal:
Histiocitose de Células de Langerhans
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Clofarabina
Limite:
Adolescent
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Adult
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Aged
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Child
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Child, preschool
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Female
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Humans
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Infant
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Male
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Middle aged
Idioma:
En
Revista:
Br J Haematol
Ano de publicação:
2024
Tipo de documento:
Article
País de afiliação:
Estados Unidos