The effect of itraconazole on the clinical outcomes of patients with advanced non-small cell lung cancer receiving platinum-based chemotherapy: a randomized controlled study.

Itraconazole is an oral antifungal that has a been reported to have anticancer effect in non-small cell lung cancer (NSCLC) through inhibition of angiogenesis. The aim is to evaluate the effect of using itraconazole on the clinical outcome of metastatic NSCLC. This was a prospective randomized controlled open-label study conducted on 60 chemotherapy-naive metastatic NSCLC. Patients were simply randomized to either Control group who received platinum-based chemotherapy for a maximum of six cycles or Itraconazole group who received the same chemotherapy regimen in addition to itraconazole 200 mg daily for 21 days starting from day 1 in each cycle. Primary outcome was 1-year progression-free survival (PFS) while secondary outcomes included overall response rate (ORR), 1-year overall survival (OS) and tolerability. The two groups were comparable at baseline with no significant difference between groups regarding demographics and clinical characteristics. The ORR in Control group was 66.7% versus 90% in Itraconazole group (p value 0.028). There was a significant difference between groups regarding PFS where the mean 1-year PFS was 5.415 months in Control group versus 6.556 months in Itraconazole group (p value = 0.002). However, there was no significant difference between groups with respect to 1-year OS. All adverse effects reported were tolerable except for one patient who developed grade 2 cardiotoxicity in Itraconazole group requiring itraconazole discontinuation. Itraconazole use was beneficial in NSCLC in terms of 1-year PFS and ORR which was not reflected by improvement in 1-year OS.Clinical trial.gov registration number: NCT03664115, date of registration: September 10, 2018.