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Original Article

Phase 2 study of everolimus monotherapy in patients with nonfunctioning neuroendocrine tumors or pheochromocytomas/paragangliomas†

  1. Do-Youn Oh MD, PhD1,
  2. Tae-Won Kim MD, PhD2,
  3. Young Suk Park MD, PhD3,
  4. Sang Joon Shin MD4,
  5. Seong Hoon Shin MD5,
  6. Eun-Kee Song MD6,
  7. Hyo Jin Lee MD7,
  8. Kewn-wook Lee MD, PhD8,
  9. Yung-Jue Bang MD, PhD1,‡,*

Article first published online: 26 JUN 2012

DOI: 10.1002/cncr.27675

Issue

Cancer

Cancer

Volume 118, Issue 24, pages 6162–6170, 15 December 2012

Additional Information

How to Cite

Oh, D.-Y., Kim, T.-W., Park, Y. S., Shin, S. J., Shin, S. H., Song, E.-K., Lee, H. J., Lee, K.-w. and Bang, Y.-J. (2012), Phase 2 study of everolimus monotherapy in patients with nonfunctioning neuroendocrine tumors or pheochromocytomas/paragangliomas. Cancer, 118: 6162–6170. doi: 10.1002/cncr.27675

Author Information

  1. 1

    Department of Internal Medicine, Seoul National University Hospital, Cancer Research Institute, Seoul National University College of Medicine, Seoul, Korea

  2. 2

    Department of Oncology, Asan Medical Center, Seoul, Korea

  3. 3

    Division of Hematology-Oncology, Department of Medicine, Samsung Medical Center, Sungkyunkwan University, School of Medicine, Seoul, South Korea

  4. 4

    Division of Medical Oncology, Department of Oncology, Yonsei Cancer Center, Yonsei University College of Medicine, Seoul, Korea

  5. 5

    Department of Internal Medicine, Kosin University Gospel Hospital, Kosin University College of Medicine, Busan, Korea

  6. 6

    Division of Hematology/Oncology, Chonbuk National University Hospital, Chonbuk National University Medical School, Jeonju, Korea

  7. 7

    Department of Internal Medicine, Chungnam National University Hospital, Daejeon, Korea

  8. 8

    Department of Internal Medicine, Seoul National University Bundang Hospital, Seoul, Korea

  1. ‡

    Fax: (011) 82-2-762-9662

*Department of Internal Medicine, Seoul National University Hospital, Cancer Research Institute, Seoul National University, 101 Daehak-ro, Jongno-gu, Seoul 110-744

  1. †

    Presented in part at the European Multidisciplinary Cancer Congress; September 23-27, 2011; Stockholm, Sweden.

  2. ‡

    Fax: (011) 82-2-762-9662

Publication History

  1. Issue published online: 3 DEC 2012
  2. Article first published online: 26 JUN 2012
  3. Manuscript Accepted: 30 APR 2012
  4. Manuscript Revised: 10 APR 2012
  5. Manuscript Received: 19 FEB 2012

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Keywords:

  • everolimus;
  • neuroendocrine tumor;
  • carcinoid;
  • pheochromocytoma;
  • paraganglioma;
  • positron emission tomography

Abstract

BACKGROUND:

The current study was conducted to evaluate the efficacy and safety of everolimus in the treatment of patients with nonfunctioning neuroendocrine tumors (NETs) or pheochromocytomas/paragangliomas.

METHODS:

Patients with histologically confirmed nonfunctioning NETs or pheochromocytomas/paragangliomas and with documented disease progression before study enrollment were eligible for the current study. Everolimus was administered daily at a dose of 10 mg for 4 weeks. Response was assessed by Response Evaluation Criteria In Solid Tumors (RECIST; version 1.0) every 8 weeks. The primary endpoint was the 4-month progression-free survival rate (PFSR). The hypothesis of the current study was that the 4-month PFSR would increase from 50% to 65%. Safety was evaluated using the National Cancer Institute's Common Terminology Criteria for Adverse Events (version 3.0).

RESULTS:

A total of 34 patients were enrolled. Of these, 27 patients had nonfunctioning NETs, 5 had pheochromocytomas, and 2 had paragangliomas. The 4-month PFSR was 78%. Partial response (PR) was observed in 3 patients. Twenty-eight patients had stable disease (SD) and 2 patients developed progressive disease (PD). The response rate (RR) and overall disease control rate (DCR) were 9.0% (95% confidence interval [95% CI], 0%-18.6%) and 93.9% (95% CI, 85.8%-100%), respectively. The PFS was 15.3 months (95% CI, 4.6 months-26.0 months). Of the patients with nonfunctioning NETs, 3 achieved a PR and 23 had SD (RR, 11.1%; DCR, 100%); the PFS was 17.1 months (95% CI, 11.1 months-23.0 months) and the 4-month PFSR was 90.0%. Twenty-one patients (80.8%) demonstrated tumor shrinkage. In 7 patients with pheochromocytomas/paragangliomas, 5 achieved SD, and 2 developed PD. The PFS was 3.8 months (95% CI, 0.5 months-7.0 months) and the 4-month PFSR was 42.9%. Four patients demonstrated tumor shrinkage. The major grade 3/4 adverse events were thrombocytopenia (14.7%), hyperglycemia (5.9%), stomatitis (5.9%), and anemia (5.9%).

CONCLUSIONS:

Everolimus was associated with high therapeutic efficacy and tolerability in patients with nonfunctioning NETs, and demonstrated modest efficacy in patients with pheochromocytomas/paragangliomas. Cancer 2012. © 2012 American Cancer Society.

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