Vemurafenib combined with chemotherapy achieved sustained remission in pediatric LCH: a multi-center observational study.
J Cancer Res Clin Oncol
; 150(1): 12, 2024 Jan 17.
Article
em En
| MEDLINE
| ID: mdl-38231288
ABSTRACT
BACKGROUND:
Langerhans cell histiocytosis (LCH) is a myeloid neoplasia with potentially fatal consequences, and about 2/3 of cases involve the BRAFV600E kinase-activated mutation. Vemurafenib, a BRAF inhibitor, has demonstrated significant clinical improvements in LCH. However, the high relapse rate of LCH following cessation of vemurafenib therapy remains a major challenge, and alternative treatment strategies require further investigation.METHODS:
In this retrospective multi-center study, we evaluated the efficacy and safety of vemurafenib combined with conventional chemotherapy in patients with severe or refractory LCH.RESULTS:
Seventeen patients were enrolled in the study, with eleven classified as risk organ involvement (RO +). Six received the combination therapy as the primary treatment, and eleven after being refractory to prior chemotherapy. The overall response rate was 94.1%. Progression-free survival among all 17 patients was 70.6% (12/17) at a median follow-up of 32 months, and relapse-free survival among the 15 patients with discontinuation after a response was 73.3%(11/15) at a median follow-up of 34 months. Five of six patients (83.3%) with myeloid BRAFV600E mutations demonstrated molecular remission. The overall survival rate was 100%. Adverse events were mostly classified as grades 1 or 2.CONCLUSION:
Our data suggest that the combination of vemurafenib and chemotherapy can achieve sustained clinical and molecular level relief in children with LCH, and side effects are tolerable.Palavras-chave
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Base de dados:
MEDLINE
Assunto principal:
Proteínas Proto-Oncogênicas B-raf
/
Inibidores de Proteínas Quinases
Idioma:
En
Ano de publicação:
2024
Tipo de documento:
Article