Patient-reported outcomes from two global multicenter clinical trials of children and adults with tropomyosin receptor kinase (TRK) fusion cancer receiving larotrectinib.

Abstract

6602 Background: TRK fusions involving NTRK1, NTRK2, and NTRK3 occur in diverse tumor types in children and adults. Larotrectinib, a selective TRK inhibitor, was recently approved by the FDA based on high objective response rates, durable disease control, and a favorable toxicity profile in in patients with tumors harboring TRK fusions (Drilon et al, NEJM 2018; Laetsch et al, Lancet Oncol 2018). To explore the impact of larotrectinib on quality of life (QoL), patient-reported outcomes (PROs) are reported here for pediatric patients (SCOUT; NCT02637687) and adolescents and adults (NAVIGATE; NCT02576431). Methods: Patients completed questionnaires assessing QoL (i.e., PedsQL, FACES, EORTC QLQ-C30, EQ-5D-5L) at baseline and planned cycle visits. Data were analyzed using descriptive statistics, longitudinal data models, and graphical displays. Results: As of July 2017, 37 patients (13 pediatric and 24 adolescent or adult) completed questionnaires at baseline and had at least one post-baseline measurement. Improvements compared to baseline in PROs were seen in both groups of patients in all functional domains of the PedsQL (physical, emotional, social, and school) and of the EORTC QLQ-C30 (physical, role, cognitive, emotional, and social). Number of patients with MID-improvement for PedsQL (Total Score), EORTC QLQ-C30 (Global Health Score), and EQ-5D-5L (VAS) are presented in the table below. Improvements were rapid (by cycle 3 or 5), seen across most tumor types and sustained a minimum of 2 cycles. Conclusions: Although necessarily viewed with caution due to relatively small sample sizes, these results nonetheless show a meaningful early improvement in QoL in pediatric and adult patients with TRK fusion cancer treated with larotrectinib. Clinical trial information: NCT02637687 and NCT02576431. [Table: see text]