A multicenter phase II study of everolimus in patients with progressive unresectable adenoid cystic carcinoma

Dong-Wan Kim, Do-Youn Oh, Seong Hoon Shin, Jin Hyoung Kang, Byoung Chul Cho, Joo-Seop Chung, HyeJin Kim, Keon Uk Park, Jung Hye Kwon, Ji-Youn Han, Mi-Jung Kim, Yung-Jue Bang, Dong-Wan Kim, Do-Youn Oh, Seong Hoon Shin, Jin Hyoung Kang, Byoung Chul Cho, Joo-Seop Chung, HyeJin Kim, Keon Uk Park, Jung Hye Kwon, Ji-Youn Han, Mi-Jung Kim, Yung-Jue Bang

Abstract

Background: The aim of this study was to examine the efficacy and safety of everolimus in patients with progressive unresectable adenoid cystic carcinoma (ACC).

Methods: Histologically confirmed ACC patients with documented disease progression within 12 months prior to the study entry were eligible. Everolimus was given at a dose of 10 mg daily until progression or occurrence of unacceptable toxicities. The primary endpoint was a 4-month progression-free survival (PFS).

Results: A total of 34 patients were enrolled. The 4-month PFS probability was 65.5% (95% one-sided confidence interval [CI], 47.7 to infinity). Median PFS duration was 11.2 months (95% CI, 3.6 to 15.8). Complete or partial response was not achieved. Twenty-seven (79.4%, 95% CI, 63.2 to 89.6) patients showed stable disease (SD). Tumor shrinkage within SD criteria was observed in 15 patients (44.1%) and SD lasting 6 months was observed in 13 patients (38.2%). Four patients had disease progression. Among the 18 patients with both pre- and post-treatment (at 8 weeks) FDG-PET scans available, 8 patients (44.4%) showed a partial metabolic response, defined as a ≥25% reduction in maximum standardized uptake values (SUVmax). The most common adverse events were stomatitis, anemia, asthenia, and leukopenia. No unexpected everolimus related toxicities were reported.

Conclusions: Everolimus showed promising efficacy and good tolerability in progressive unresectable ACC.

Trial registration: ClinicalTrials.gov identifier, NCT01152840.

Figures

Figure 1
Figure 1
Progression free survival(intention-to-treat population,N = 34).
Figure 2
Figure 2
Best percent changes in tumor size by patients(response evaluable patients,N = 31).
Figure 3
Figure 3
Percent changes in maximum standardized uptake values(SUVmax)after 2 cycles of everolimus treatment(FDG-PET evaluable patients,N = 18).

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