title: A multicenter phase II study of everolimus in patients with progressive unresectable adenoid cystic carcinoma.
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.
  
  
  BACKGROUND
  ClinicalTrials.gov identifier, NCT01152840.
authors.forename: Dong-Wan
authors.initials: DW
authors.lastname: Kim
authors.forename: Do-Youn
authors.initials: DY
authors.lastname: Oh
authors.forename: Seong Hoon
authors.initials: SH
authors.lastname: Shin
authors.forename: Jin Hyoung
authors.initials: JH
authors.lastname: Kang
authors.forename: Byoung Chul
authors.initials: BC
authors.lastname: Cho
authors.forename: Joo-Seop
authors.initials: JS
authors.lastname: Chung
authors.forename: HyeJin
authors.initials: H
authors.lastname: Kim
authors.forename: Keon Uk
authors.initials: KU
authors.lastname: Park
authors.forename: Jung Hye
authors.initials: JH
authors.lastname: Kwon
authors.forename: Ji-Youn
authors.initials: JY
authors.lastname: Han
authors.forename: Mi-Jung
authors.initials: MJ
authors.lastname: Kim
authors.affiliation: Department of Internal Medicine, Seoul National University Hospital, 101 Daehak-ro, Jongno-gu, Seoul 110-744, Republic of Korea. bangyj@snu.ac.kr.
authors.forename: Yung-Jue
authors.initials: YJ
authors.lastname: Bang
mesh_terms: D000328:Adult; D000368:Aged; D000970:Antineoplastic Agents / Q000008:administration & dosage / Q000009:adverse effects / Q000627:therapeutic use*; D003528:Carcinoma, Adenoid Cystic / Q000175:diagnosis / Q000188:drug therapy* / Q000401:mortality / Q000473:pathology*; D000068338:Everolimus; D005260:Female; D019788:Fluorodeoxyglucose F18; D006801:Humans; D008297:Male; D008875:Middle Aged; D009362:Neoplasm Metastasis; D009367:Neoplasm Staging; D049268:Positron-Emission Tomography; D020123:Sirolimus / Q000008:administration & dosage / Q000009:adverse effects / Q000031:analogs & derivatives* / Q000627:therapeutic use; D016896:Treatment Outcome; D047368:Tumor Burden
chemical_list: D000970:Antineoplastic Agents; D019788:Fluorodeoxyglucose F18; D000068338:Everolimus; D020123:Sirolimus