Table 2 Study objectives and variables

Primary: safety of larotrectinib in adult and pediatric patients with locally advanced or metastatic TRK fusion cancer

TEAEs in real-world practice conditions

  • Incidence

  • Severity

  • Seriousness

  • Outcome

  • Causality assessment (ARs)

Secondary:

  • Effectiveness of larotrectinib as per investigator assessment in the total study population as well as in patient subgroups (including, but not limited to, age, NTRK gene, NTRK gene fusion partner, testing methodology, country/region, and prior therapy [type and/or number of lines of therapy])

  • Patient demographic and baseline characteristics

  • Disease history (including pathology report at baseline and subsequently as applicable)

  • Comorbidities

  • Method for detecting NTRK gene fusion (including testing report at baseline and subsequently as applicable)

  • ECOG, Lansky, or Karnofsky performance status

  • Tumor assessments (ORR, DCR, DOR, TTR, PFS, OS) per investigator assessment

  • Date of radiologic or clinical progression on larotrectinib

  • Larotrectinib treatment patterns

  • Larotrectinib use (initiation and termination dates, dosage, and dose modification)

  • Long-term effects of larotrectinib on growth, developmental milestones, and sexual development in the pediatric cohort

  • Long-term effects of larotrectinib on neurologic outcomes in all patients

  • Developmental milestones; age at adrenarche or menarche (if applicable); Tanner scale

  • Neurological examination

  • Laboratory examination data; and date of death/last follow-up

Exploratory:

  • Effectiveness of larotrectinib

  • Tumor assessments (ORR, DCR, DOR, TTR, PFS, OS) based on radiologic assessments of tumor response per IRC,

as applicable

  • Procedures avoided because of the use of larotrectinib (e.g., amputation or other disfiguring procedures) in patients with infantile fibrosarcoma

  • Whether an amputation was considered

for the patient prior to treatment with larotrectinib (e.g., in patients with

infantile fibrosarcoma)

  • Number of patients who underwent surgery with a curative intent (excluding amputation) because of the use of larotrectinib

  • Surgery (with curative intent) while on larotrectinib

  • Description of systemic treatments prior to larotrectinib therapy

  • Previous systemic therapy (initiation and termination dates, doses, duration of treatment, best tumor response to therapy, date of radiological progression on therapy, reasons for discontinuation)