Efficacy and safety of larotrectinib in TRK fusion-positive primary central nervous system tumors.
Neuro Oncol
; 24(6): 997-1007, 2022 06 01.
Article
en En
| MEDLINE
| ID: mdl-34850167
ABSTRACT
BACKGROUND:
Larotrectinib is a first-in-class, highly selective tropomyosin receptor kinase (TRK) inhibitor approved to treat adult and pediatric patients with TRK fusion-positive cancer. The aim of this study was to evaluate the efficacy and safety of larotrectinib in patients with TRK fusion-positive primary central nervous system (CNS) tumors.METHODS:
Patients with TRK fusion-positive primary CNS tumors from two clinical trials (NCT02637687, NCT02576431) were identified. The primary endpoint was investigator-assessed objective response rate (ORR).RESULTS:
As of July 2020, 33 patients with TRK fusion-positive CNS tumors were identified (median age 8.9 years; range 1.3-79.0). The most common histologies were high-grade glioma (HGG; n = 19) and low-grade glioma (LGG; n = 8). ORR was 30% (95% confidence interval [CI] 16-49) for all patients. The 24-week disease control rate was 73% (95% CI 54-87). Twenty-three of 28 patients (82%) with measurable disease had tumor shrinkage. The 12-month rates for duration of response, progression-free survival, and overall survival were 75% (95% CI 45-100), 56% (95% CI 38-74), and 85% (95% CI 71-99), respectively. Median time to response was 1.9 months (range 1.0-3.8 months). Duration of treatment ranged from 1.2-31.3+ months. Treatment-related adverse events were reported for 20 patients, with grade 3-4 in 3 patients. No new safety signals were identified.CONCLUSIONS:
In patients with TRK fusion-positive CNS tumors, larotrectinib demonstrated rapid and durable responses, high disease control rate, and a favorable safety profile.Palabras clave
Texto completo:
1
Base de datos:
MEDLINE
Asunto principal:
Glioma
/
Neoplasias
/
Antineoplásicos
Límite:
Adult
/
Child
/
Humans
Idioma:
En
Revista:
Neuro Oncol
Asunto de la revista:
NEOPLASIAS
/
NEUROLOGIA
Año:
2022
Tipo del documento:
Article
País de afiliación:
Francia