Clinical trial designs for rare diseases: Studies developed and discussed by the International Rare Cancers Initiative

被引：104

作者：

Bogaerts, Jan ^{[1
]}

Sydes, Matthew R. ^{[2
]}

Keat, Nicola ^{[3
]}

McConnell, Andrea ^{[3
]}

Benson, Al ^{[27
]}

Ho, Alan ^{[4
]}

Rotha, Arnaud ^{[28
]}

Fortpied, Catherine ^{[1
]}

Eng, Cathy ^{[5
]}

Peckitt, Clare ^{[6
]}

Coens, Corneel ^{[1
]}

Pettaway, Curtis ^{[5
]}

Arnold, Dirk ^{[7
]}

Hall, Emma ^{[8
]}

Marshall, Ernie ^{[9
]}

Sclafani, Francesco ^{[6
]}

Hatcher, Helen ^{[10
,11
]}

Earl, Helena ^{[10
,11
]}

Ray-Coquard, Isabelle ^{[12
]}

Paul, James ^{[13
]}

Blay, Jean-Yves ^{[12
]}

Whelan, Jeremy ^{[14
]}

Panageas, Kathy ^{[4
]}

Wheatley, Keith ^{[15
]}

Harrington, Kevin ^{[8
]}

Licitra, Lisa ^{[16
]}

Billingham, Lucinda ^{[17
,18
,19
]}

Hensley, Martee ^{[4
]}

McCabe, Martin

Patel, Poulam M. ^{[29
]}

Carvajal, Richard ^{[4
]}

Wilson, Richard ^{[20
]}

Glynne-Jones, Rob ^{[30
]}

McWilliams, Rob ^{[21
]}

Leyvraz, Serge ^{[22
]}

Rao, Sheela ^{[6
]}

Nicholson, Steve ^{[23
]}

Filiaci, Virginia ^{[24
]}

Negrouk, Anastassia ^{[1
]}

Lacombe, Denis ^{[1
]}

Dupont, Elisabeth ^{[25
]}

Pauporte, Iris ^{[26
]}

Welch, John J. ^{[25
]}

Law, Kate ^{[3
]}

Trimble, Ted ^{[25
]}

Seymour, Matthew

机构：

[1] EORTC, Brussels, Belgium

[2] UCL, Med Res Council Clin Trial Unit, London WC1E 6BT, England

[3] Canc Res UK, London, England

[4] Mem Sloan Kettering Canc Ctr, New York, NY USA

[5] Univ Texas MD Anderson Canc Ctr, Houston, TX USA

[6] Royal Marsden, London, England

[7] Univ Canc Ctr Hamburg, Hubertus Wald Tumorzentrum, Hamburg, Germany

[8] Inst Canc Res, London SW3 6JB, England

[9] Univ Liverpool, Liverpool L69 3BX, Merseyside, England

[10] Univ Cambridge, Dept Oncol, Cambridge CB2 1TN, England

[11] NIHR Cambridge Biomed Res Ctr, Cambridge, England

[12] Leon Berard Canc Ctr, Lyon, France

[13] Univ Glasgow, Inst Canc Sci, Canc Res UK Clin Trials Unit, Glasgow G12 8QQ, Lanark, Scotland

[14] Univ Coll London Hosp, Biomed Res Ctr, NIHR, London, England

[15] Univ Birmingham, Birmingham B15 2TT, W Midlands, England

[16] Ist Nazl Tumori, I-20133 Milan, Italy

[17] Univ Birmingham, Canc Res UK Clin Trials Unit, Birmingham B15 2TT, W Midlands, England

[18] Univ Birmingham, MRC Midland Hub Trials Methodol Res, Birmingham B15 2TT, W Midlands, England

[19] Univ Manchester, Manchester M13 9PL, Lancs, England

[20] Queens Univ Belfast, Belfast BT7 1NN, Antrim, North Ireland

[21] Mayo Clin, Rochester, MN USA

[22] CHU Vaudois, Lausanne, Switzerland

[23] Locum Consultant Med Oncol, London, England

[24] NRG Oncol, Gynecol Oncol Grp, Orange, CA USA

[25] US Natl Canc Inst, Ctr Global Hlth, Bethesda, MD USA

[26] Natl Canc Inst, Boulogne, France

[27] Northwestern Univ, Robert H Lurie Comprehens Canc Ctr, Evanston, IL 60208 USA

[28] Univ Hosp Geneva, Med Oncol, Geneva, Switzerland

[29] Univ Nottingham, Nottingham NG7 2RD, England

[30] Mt Vernon Canc Ctr, Northwood, Middx, England

来源：

EUROPEAN JOURNAL OF CANCER | 2015年 / 51卷 / 03期

基金：

英国医学研究理事会;

关键词：

Rare cancers; Clinical trials; Randomised controlled trials; Methodology; Frequentist; Bayesian; Multi-arm; UTERINE LEIOMYOSARCOMA; ADJUVANT THERAPY; SURVIVAL; EPIDEMIOLOGY; STATISTICS; CARCINOMA; STAMPEDE; MELANOMA; OUTCOMES; SARCOMA;

D O I：

10.1016/j.ejca.2014.10.027

中图分类号：

R73 [肿瘤学];

学科分类号：

100214 [肿瘤学];

摘要：

Background: The past three decades have seen rapid improvements in the diagnosis and treatment of most cancers and the most important contributor has been research. Progress in rare cancers has been slower, not least because of the challenges of undertaking research. Settings: The International Rare Cancers Initiative (IRCI) is a partnership which aims to stimulate and facilitate the development of international clinical trials for patients with rare cancers. It is focused on interventional usually randomised clinical trials with the clear goal of improving outcomes for patients. The key challenges are organisational and methodological. A multi-disciplinary workshop to review the methods used in ICRI portfolio trials was held in Amsterdam in September 2013. Other as-yet unrealised methods were also discussed. Results: The IRCI trials are each presented to exemplify possible approaches to designing credible trials in rare cancers. Researchers may consider :these for use in future trials and understand the choices made for each design. Interpretation: Trials can be designed using a wide array of possibilities. There is no 'one size fits all' solution. In order to make progress in the rare diseases, decisions to change practice will have to be based on less direct evidence from clinical trials than in more common diseases. (C) 2014 The Authors. Published by Elsevier Ltd.

引用

页码：271 / 281

页数：11

共 43 条

[1]

American Cancer Society, CANC FACTS FIG

[2]

[Anonymous], 2013, J Clin Oncol, DOI DOI 10.1200/JCO.2013.31.15_SUPPL.9031

[3]

[Anonymous], AN CANC INC STAT

[4]

Phase II evaluation of temozolomide in metastatic choroidal melanoma [J].

Bedikian, AY ;

Papadopoulos, N ;

Plager, C ;

Eton, O ;

Ring, S .

MELANOMA RESEARCH, 2003, 13 (03) :303-306

[5]

Introduction to Bayesian methods III: use and interpretation of Bayesian tools in design and analysis [J].

Berry, DA .

CLINICAL TRIALS, 2005, 2 (04) :295-300

[6]

Berry DA, 2005, CLIN TRIALS, V2, P64

[7]

Berry DA, 2005, CLIN TRIALS, V2, P1

[8]

Small Bowel Cancer in the United States Changes in Epidemiology, Treatment, and Survival Over the Lost 20 Years [J].

Bilimoria, Karl Y. ;

Bentrem, David J. ;

Wayne, Jeffrey D. ;

Ko, Clifford Y. ;

Bennett, Chales L. ;

Talamonti, Mark S. .

ANNALS OF SURGERY, 2009, 249 (01) :63-71

[9]

Cancer Research UK, 2014, CANCERSTATS

[10]

Effect of Selumetinib vs Chemotherapy on Progression-Free Survival in Uveal Melanoma A Randomized Clinical Trial [J].

Carvajal, Richard D. ;

Sosman, Jeffrey A. ;

Quevedo, Jorge Fernando ;

Milhem, Mohammed M. ;

Joshua, Anthony M. ;

Kudchadkar, Ragini R. ;

Linette, Gerald P. ;

Gajewski, Thomas F. ;

Lutzky, Jose ;

Lawson, David H. ;

Lao, Christopher D. ;

Flynn, Patrick J. ;

Albertini, Mark R. ;

Sato, Takami ;

Lewis, Karl ;

Doyle, Austin ;

Ancell, Kristin ;

Panageas, Katherine S. ;

Bluth, Mark ;

Hedvat, Cyrus ;

Erinjeri, Joseph ;

Ambrosini, Grazia ;

Marr, Brian ;

Abramson, David H. ;

Dickson, Mark Andrew ;

Wolchok, Jedd D. ;

Chapman, Paul B. ;

Schwartz, Gary K. .

JAMA-JOURNAL OF THE AMERICAN MEDICAL ASSOCIATION, 2014, 311 (23) :2397-2405

← 1 2 3 4 5 →