dc.contributor.author | Doz, F | |
dc.contributor.author | van Tilburg, CM | |
dc.contributor.author | Geoerger, B | |
dc.contributor.author | Højgaard, M | |
dc.contributor.author | Øra, I | |
dc.contributor.author | Boni, V | |
dc.contributor.author | Capra, M | |
dc.contributor.author | Chisholm, J | |
dc.contributor.author | Chung, HC | |
dc.contributor.author | DuBois, SG | |
dc.contributor.author | Gallego-Melcon, S | |
dc.contributor.author | Gerber, NU | |
dc.contributor.author | Goto, H | |
dc.contributor.author | Grilley-Olson, JE | |
dc.contributor.author | Hansford, JR | |
dc.contributor.author | Hong, DS | |
dc.contributor.author | Italiano, A | |
dc.contributor.author | Kang, HJ | |
dc.contributor.author | Nysom, K | |
dc.contributor.author | Thorwarth, A | |
dc.contributor.author | Stefanowicz, J | |
dc.contributor.author | Tahara, M | |
dc.contributor.author | Ziegler, DS | |
dc.contributor.author | Gavrilovic, IT | |
dc.contributor.author | Norenberg, R | |
dc.contributor.author | Dima, L | |
dc.contributor.author | De La Cuesta, E | |
dc.contributor.author | Laetsch, TW | |
dc.contributor.author | Drilon, A | |
dc.contributor.author | Perreault, S | |
dc.date.accessioned | 2021-12-07T09:27:15Z | |
dc.date.available | 2021-12-07T09:27:15Z | |
dc.identifier.citation | Neuro-Oncology | |
dc.identifier.issn | 1522-8517 | |
dc.identifier.uri | https://repository.icr.ac.uk/handle/internal/4912 | |
dc.identifier.eissn | 1523-5866 | |
dc.identifier.doi | 10.1093/neuonc/noab274 | |
dc.description.abstract | 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. In 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. | |
dc.language | eng | |
dc.language.iso | eng | |
dc.publisher | Oxford University Press (OUP) | |
dc.rights.uri | https://creativecommons.org/licenses/by/4.0 | |
dc.title | Efficacy and safety of larotrectinib in TRK fusion-positive primary central nervous system tumors | |
dc.type | Journal Article | |
dcterms.dateAccepted | 2021-11-27 | |
rioxxterms.version | AM | |
rioxxterms.versionofrecord | 10.1093/neuonc/noab274 | |
rioxxterms.licenseref.uri | https://creativecommons.org/licenses/by/4.0 | |
rioxxterms.type | Journal Article/Review | |
dc.relation.isPartOf | Neuro-Oncology | |
pubs.notes | No embargo | |
pubs.organisational-group | /ICR | |
pubs.organisational-group | /ICR/Primary Group | |
pubs.organisational-group | /ICR/Primary Group/ICR Divisions | |
pubs.organisational-group | /ICR/Primary Group/ICR Divisions/Clinical Studies | |
pubs.organisational-group | /ICR/Primary Group/ICR Divisions/Clinical Studies/Sarcoma Clinical Trials in Children and Young People | |
pubs.organisational-group | /ICR/Primary Group/ICR Divisions/Clinical Studies/Sarcoma Clinical Trials in Children and Young People/Sarcoma Clinical Trials in Children and Young People (hon.) | |
pubs.organisational-group | /ICR/Primary Group/ICR Divisions/Molecular Pathology | |
pubs.organisational-group | /ICR/Primary Group/ICR Divisions/Molecular Pathology/Sarcoma Clinical Trials in children and young people | |
pubs.organisational-group | /ICR/Primary Group/ICR Divisions/Molecular Pathology/Sarcoma Clinical Trials in children and young people/Sarcoma Clinical Trials in Children and Young People (hon.) | |
pubs.organisational-group | /ICR/Primary Group/Royal Marsden Clinical Units | |
pubs.publication-status | Published online | |
pubs.embargo.terms | No embargo | |
icr.researchteam | Sarcoma Clinical Trials in children and young people | |
dc.contributor.icrauthor | Chisholm, Julia | |