Design and conduct of phase II studies of targeted anticancer therapy: Recommendations from the task force on methodology for the development of innovative cancer therapies (MDICT)

被引：51

作者：

Booth, Christopher M. ^{[1
]}

Calvert, A. Hilary ^{[2
]}

Giaccone, Giuseppe ^{[3
]}

Lobbezoo, Marinus W. ^{[4
]}

Eisenhauer, Elizabeth A. ^{[1
]}

Seymour, Lesley K. ^{[1
]}

机构：

[1] Queens Univ, Canada Clin Trials Grp, Natl Canc Inst Canada, Kingston, ON K7L 3N6, Canada

[2] Med Sch Newcastle Upon Tyne, No Inst Canc Res, Newcastle Upon Tyne NE2 4HH, Tyne & Wear, England

[3] NCI, Med Oncol Branch, Bethesda, MD 20892 USA

[4] NDDO Res Fdn, Amsterdam, Netherlands

来源：

EUROPEAN JOURNAL OF CANCER | 2008年 / 44卷 / 01期

关键词：

clinical trials/phase II; drug therapy; neoplasms; receptors; growth factors/antagonists; inhibitors;

D O I：

10.1016/j.ejca.2007.07.031

中图分类号：

R73 [肿瘤学];

学科分类号：

100214 ;

摘要：

The Methodology for the Development of Innovative Cancer Therapies (MDICT) task force considered aspects of the design and conduct of phase II studies for molecular targeted agents during their 2007 meeting. The task force recommended that multinomial end-points and designs should be considered for phase II studies of targeted agents, that both single arm as well as randomised designs remain appropriate in certain settings, and that further assessment of novel endpoints (tumour growth kinetic assessment, biomarker or functional imaging) and designs (randomised discontinuation or Bayesian adaptive design) be encouraged. The MDICT cautioned on the use of small randomised trials which have a number of statistical pitfalls and dangers and strongly encouraged the complete reporting, including negative trials, in the scientific literature. (c) 2007 Elsevier Ltd. All rights reserved.

引用

页码：25 / 29

页数：5

共 13 条

[1] We should desist using RECIST, at least in GIST [J].

Benjamin, Robert S. ;

Choi, Haesun ;

Macapinlac, Homer A. ;

Burgess, Michael A. ;

Patel, Shreyaskumar R. ;

Chen, Lei L. ;

Podoloff, Donald A. ;

Charnsangavej, Chuslip .

JOURNAL OF CLINICAL ONCOLOGY, 2007, 25 (13) :1760-1764

[2] Bayesian clinical trials [J].

Berry, DA .

NATURE REVIEWS DRUG DISCOVERY, 2006, 5 (01) :27-36

[3]

BOOTH CM, 2007, IN PRESS EUR J CANC

[4] Specific killing of BRCA2-deficient tumours with inhibitors of poly(ADP-ribose) polymerase [J].

Bryant, HE ;

Schultz, N ;

Thomas, HD ;

Parker, KM ;

Flower, D ;

Lopez, E ;

Kyle, S ;

Meuth, M ;

Curtin, NJ ;

Helleday, T .

NATURE, 2005, 434 (7035) :913-917

[5] Modified RECIST criteria for assessment of response in malignant pleural mesothelioma [J].

Byrne, MJ ;

Nowak, AK .

ANNALS OF ONCOLOGY, 2004, 15 (02) :257-260

[6] Efficacy and safety of imatinib mesylate in advanced gastrointestinal stromal tumors [J].

Demetri, GD ;

von Mehren, M ;

Blanke, CD ;

Van den Abbeele, AD ;

Eisenberg, B ;

Roberts, PJ ;

Heinrich, MC ;

Tuveson, DA ;

Singer, S ;

Janicek, M ;

Fletcher, JA ;

Silverman, SG ;

Silberman, SL ;

Capdeville, R ;

Kiese, B ;

Peng, B ;

Dimitrijevic, S ;

Druker, BJ ;

Corless, C ;

Fletcher, CDM ;

Joensuu, H .

NEW ENGLAND JOURNAL OF MEDICINE, 2002, 347 (07) :472-480

[7] Application of a new multinomial phase II stopping rule using response and early progression [J].

Dent, S ;

Zee, B ;

Dancey, J ;

Hanauske, A ;

Wanders, J ;

Eisenhauer, E .

JOURNAL OF CLINICAL ONCOLOGY, 2001, 19 (03) :785-791

[8] Evaluation of randomized discontinuation design [J].

Freidlin, B ;

Simon, R .

JOURNAL OF CLINICAL ONCOLOGY, 2005, 23 (22) :5094-5098

[9] Adaptive randomized study of idarubicin and cytarabine versus troxacitabine and cytarabine versus troxacitabine and idarubicin in untreated patients 50 years or older with adverse karyotype acute myeloid leukemia [J].

Giles, FJ ;

Kantarjian, HM ;

Cortes, JE ;

Garcia-Manero, G ;

Verstovsek, S ;

Faderl, S ;

Thomas, DA ;

Ferrajoli, A ;

O'Brien, S ;

Wathen, JK ;

Xiao, LC ;

Berry, DA ;

Estey, EH .

JOURNAL OF CLINICAL ONCOLOGY, 2003, 21 (09) :1722-1727

[10] Phase II placebo-controlled randomized discontinuation trial of sorafnib in patients with metastatic renal cell carcinoma [J].

Ratain, Mark J. ;

Eisen, Tim ;

Stadler, Walter M. ;

Flaherty, Keith T. ;

Kaye, Stan B. ;

Rosner, Gary L. ;

Gore, Martin ;

Desai, Apurva A. ;

Patnaik, Amita ;

Xiong, Henry Q. ;

Rowinsky, Lric ;

Abbruzzese, James L. ;

Xia, Chenghua ;

Simantov, Ronit ;

Schwartz, Brian ;

O'Dwyer, Peter J. .

JOURNAL OF CLINICAL ONCOLOGY, 2006, 24 (16) :2505-2512

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