Future directions in the treatment of neuroendocrine tumors: Consensus report of the National Cancer Institute Neuroendocrine Tumor Clinical Trials Planning Meeting

Matthew H. Kulke, Lillian L. Siu, Joel E. Tepper, George Fisher, Deborah Jaffe, Daniel G. Haller, Lee M. Ellis, Jacqueline K. Benedetti, Emily K. Bergsland, Timothy James Hobday, Eric Van Cutsem, James Pingpank, Kjell Oberg, Steven J. Cohen, Mitchell C. Posner, James C. Yao

Research output: Contribution to journalArticle

195 Citations (Scopus)

Abstract

Neuroendocrine tumors (NETs) arise from a variety of anatomic sites and share the capacity for production of hormones and vasoactive peptides. Because of their perceived rarity, NETs have not historically been a focus of rigorous clinical research. However, the diagnosed incidence of NETs has been increasing, and the estimated prevalence in the United States exceeds 100,000 individuals. The recent completion of several phase III studies, including those evaluating octreotide, sunitinib, and everolimus, has demonstrated that rigorous evaluation of novel agents in this disease is both feasible and can lead to practice-changing outcomes. The NET Task Force of the National Cancer Institute GI Steering Committee convened a clinical trials planning meeting to identify key unmet needs, develop appropriate study end points, standardize clinical trial inclusion criteria, and formulate priorities for future NET studies for the US cooperative group program. Emphasis was placed on the development of well-designed clinical trials with clearly defined efficacy criteria. Key recommendations include the evaluation of pancreatic NET separately from NETs of other sites and the exclusion of patients with poorly differentiated histologies from trials focused on low-grade histologies. Studies evaluating novel agents for the control of hormonal syndromes should avoid somatostatin analog washout periods when possible and should include quality-of-life end points. Because of the observed long survival after progression of many patients, progression-free survival is recommended as a feasible and relevant primary end point for both phase III studies and phase II studies where a delay in progression is expected in the absence of radiologic responses.

Original languageEnglish (US)
Pages (from-to)934-943
Number of pages10
JournalJournal of Clinical Oncology
Volume29
Issue number7
DOIs
StatePublished - Mar 1 2011

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Neuroendocrine Tumors
National Cancer Institute (U.S.)
Clinical Trials
Therapeutics
Histology
Octreotide
Peptide Hormones
Advisory Committees
Direction compound
Somatostatin
Disease-Free Survival
Quality of Life
Survival
Incidence
Research

ASJC Scopus subject areas

  • Cancer Research
  • Oncology

Cite this

Future directions in the treatment of neuroendocrine tumors : Consensus report of the National Cancer Institute Neuroendocrine Tumor Clinical Trials Planning Meeting. / Kulke, Matthew H.; Siu, Lillian L.; Tepper, Joel E.; Fisher, George; Jaffe, Deborah; Haller, Daniel G.; Ellis, Lee M.; Benedetti, Jacqueline K.; Bergsland, Emily K.; Hobday, Timothy James; Van Cutsem, Eric; Pingpank, James; Oberg, Kjell; Cohen, Steven J.; Posner, Mitchell C.; Yao, James C.

In: Journal of Clinical Oncology, Vol. 29, No. 7, 01.03.2011, p. 934-943.

Research output: Contribution to journalArticle

Kulke, MH, Siu, LL, Tepper, JE, Fisher, G, Jaffe, D, Haller, DG, Ellis, LM, Benedetti, JK, Bergsland, EK, Hobday, TJ, Van Cutsem, E, Pingpank, J, Oberg, K, Cohen, SJ, Posner, MC & Yao, JC 2011, 'Future directions in the treatment of neuroendocrine tumors: Consensus report of the National Cancer Institute Neuroendocrine Tumor Clinical Trials Planning Meeting', Journal of Clinical Oncology, vol. 29, no. 7, pp. 934-943. https://doi.org/10.1200/JCO.2010.33.2056
Kulke, Matthew H. ; Siu, Lillian L. ; Tepper, Joel E. ; Fisher, George ; Jaffe, Deborah ; Haller, Daniel G. ; Ellis, Lee M. ; Benedetti, Jacqueline K. ; Bergsland, Emily K. ; Hobday, Timothy James ; Van Cutsem, Eric ; Pingpank, James ; Oberg, Kjell ; Cohen, Steven J. ; Posner, Mitchell C. ; Yao, James C. / Future directions in the treatment of neuroendocrine tumors : Consensus report of the National Cancer Institute Neuroendocrine Tumor Clinical Trials Planning Meeting. In: Journal of Clinical Oncology. 2011 ; Vol. 29, No. 7. pp. 934-943.
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