ǂa ǂretrospective analysis of patients treated through an access program

Povzetek

Introduction: Rearranged during transfection (RET) gene fusions are rare genetic drivers in non-small cell lung cancer (NSCLC). Selective RET-inhibitors such as selpercatinib have shown therapeutic activity in early clinical trials; however, their efficacy in the real-world setting is unknown. Methods: A retrospective efficacy and safety analysis was performed on data from RET fusion-positive NSCLC patients who participated in a selpercatinib access program (named patient protocol) between August 2019 and January 2021. Results: Data from 50 patients with RET fusion-positive advanced NSCLC treated with selpercatinib at 27 centers in 12 countries was analyzed. Most patients were Non-Asian (90%), female (60%), never-smokers (74%), with a median age of 65 years (range, 38-89). 32% of the patients had known brain metastasis at the time of selpercatinib treatment. Overall, 13 patients were treatment-naïve, while 37 were pretreated with a median of three lines of therapy (range, 1-8). The objective response rate (ORR) was 68% [95% confidence interval (CI), 53-81] in the overall population. The disease control rate was 92%. The median progression-free survival was 15.6 months (95% CI, 8.8-22.4) after a median follow-up of 9 months. In patients with measurable brain metastases (n=8) intracranial ORR reached 100%. In total, 88% of patients experienced treatment-related adverse events (TRAEs), a large majority of them being grade 1 or 2. The most common grade >/=3 TRAEs were increased liver enzyme levels (in 10% of patients), prolonged QTc time (4%), abdominal pain (4%), hypertension (4%), and fatigue/asthenia (4%). None of patients discontinued selpercatinib treatment for safety reasons. No new safety concerns were observed, nor where there any treatment-related death. Conclusions: In this real-world setting, the selective RET-inhibitor selpercatinib demonstrated durable systemic and intracranial antitumor activity in RET fusion-positive NSCLC and was well tolerated.

Ključne besede

podatki iz resničnega življenja;selperactinib;tarčna terapija;zaviralci tirozinskih kinaz;real-world data;selpercatinib;targeted therapy;tyrosine kinase inhibitor;

Podatki

Jezik: Angleški jezik
Leto izida:
Tipologija: 1.01 - Izvirni znanstveni članek
Založnik: Sage Journals
UDK: 616-006
COBISS: 66935555 Povezava se bo odprla v novem oknu
ISSN: 1758-8359
Št. ogledov: 736
Št. prenosov: 337
Ocena: 0 (0 glasov)
Metapodatki: JSON JSON-RDF JSON-LD TURTLE N-TRIPLES XML RDFA MICRODATA DC-XML DC-RDF RDF

Ostali podatki

Sekundarne ključne besede: Carcinoma, non-small cell lung;Drug therapy;Genetics;Molecular targeted therapy;Nedrobnocelični karcinom pljuč;Terapija z zdravili;Genetika;Molekularna tarčna terapija;
Komentar vira: Nasl. z nasl. zaslona; Soavtorica iz Slovenije: Katja Mohorčič; Opis vira z dne 14. 6. 2021;
Strani: str. 1-17
Zvezek: ǂVol. ǂ13
Čas izdaje: 2021
DOI: 10.1177/17588359211019675
ID: 13032370