Progression-free survival as a surrogate for overall survival in oncology trials: a methodological systematic review.

Abstract:

BACKGROUND:Progression-free survival (PFS) is a surrogate endpoint widely used for overall survival (OS) in oncology. Validation of PFS as a surrogate must be done for each indication and each intervention. We aimed to identify all studies evaluating the validity of PFS as a surrogate for OS in oncology, and to describe their methodological characteristics. METHODS:We conducted a systematic review by searching MEDLINE via PubMed and the Cochrane Library with no limitation on time, selected relevant studies and extracted data in duplicate on how surrogacy was evaluated (meta-analytic approach, assessment of correlation and level of evaluation). RESULTS:We identified 91 studies evaluating the validity of PFS as a surrogate for OS in 24 cancer localisations. Although a meta-analytic approach was used in 83 (91%) studies, the methods used to validate PFS as a surrogate of OS were heterogeneous across studies. Of the 47 studies concluding that PFS is a good surrogate for OS, for 15 (32%), there was no quantitative argument for surrogacy. CONCLUSIONS:Although most studies used a meta-analytic approach as recommended, our methodological review highlights heterogeneity in methods and reporting, which stresses the importance of developing and applying clear recommendations in this area.

journal_name

Br J Cancer

authors

Belin L,Tan A,De Rycke Y,Dechartres A

doi

10.1038/s41416-020-0805-y

subject

Has Abstract

pub_date

2020-05-01 00:00:00

pages

1707-1714

issue

11

eissn

0007-0920

issn

1532-1827

pii

10.1038/s41416-020-0805-y

journal_volume

122

pub_type

杂志文章
  • The impact of antigen density and antibody affinity on antibody-dependent cellular cytotoxicity: relevance for immunotherapy of carcinomas.

    abstract::Antibody-dependent cellular cytotoxicity (ADCC) is considered to be the major mechanism through which tumour cells, upon treatment with anti-tumour MAbs, are eliminated in vivo. However, the relative importance of various parameters that influence the efficacy of ADCC is unclear. Here we present in vitro data on the i...

    journal_title:British journal of cancer

    pub_type: 杂志文章

    doi:10.1038/bjc.1998.518

    authors: Velders MP,van Rhijn CM,Oskam E,Fleuren GJ,Warnaar SO,Litvinov SV

    更新日期:1998-08-01 00:00:00

  • Results from a seven-year programme of breast self-examination in 89,010 women.

    abstract::This report presents the results of a study into the effect of breast self-examination (BSE) in a large defined population within the City of Nottingham since 1979. We have examined the effect of breast self-examination in a group of patients invited to attend for education in BSE compared with a group of historical c...

    journal_title:British journal of cancer

    pub_type: 杂志文章

    doi:10.1038/bjc.1989.294

    authors: Locker AP,Caseldine J,Mitchell AK,Blamey RW,Roebuck EJ,Elston CW

    更新日期:1989-09-01 00:00:00

  • The validity of the labelling index in tumour studies.

    abstract::The distribution of labelled cells through 5 different mouse tumours was measured after a single injection of [3H]-thymidine [( 3H]-TdR) or [3H]-deoxyuridine [( 3H]-UdR). All the tumours had areas where the percentage of labelled cells (the labelling index, LI) was high and areas where the LI was very low. The total a...

    journal_title:British journal of cancer

    pub_type: 杂志文章

    doi:10.1038/bjc.1985.3

    authors: Hamilton E,Dobbin J

    更新日期:1985-01-01 00:00:00

  • Prognostic value of prostate circulating cells detection in prostate cancer patients: a prospective study.

    abstract::In clinically organ-confined prostate cancer patients, bloodstream tumour cell dissemination generally occurs, and may be enhanced by surgical prostate manipulation. To evaluate cancer-cell seeding impact upon patient recurrence-free survival, 155 patients were prospectively enrolled then followed. Here, 57 patients p...

    journal_title:British journal of cancer

    pub_type: 杂志文章

    doi:10.1038/sj.bjc.6604912

    authors: Eschwège P,Moutereau S,Droupy S,Douard R,Gala JL,Benoit G,Conti M,Manivet P,Loric S

    更新日期:2009-02-24 00:00:00

  • Phenotypic effects of overexpression of the MMAC1 gene in prostate epithelial cells.

    abstract::The prostate cancer cell lines PC3 and LNCaP have been shown to lack expression of the tumour suppressor gene MMAC1/PTEN, in contrast to the immortalized non-tumorigenic epithelial lines PNT1a and PNT2. We have measured the effects of reintroduction of wild type (wt) and mutant MMAC1 genes on to these genetic backgrou...

    journal_title:British journal of cancer

    pub_type: 杂志文章

    doi:10.1054/bjoc.2000.1400

    authors: Sharrard RM,Maitland NJ

    更新日期:2000-10-01 00:00:00

  • DNA methylation status is more reliable than gene expression at detecting cancer in prostate biopsy.

    abstract:BACKGROUND:We analysed critically the potential usefulness of RNA- and DNA-based biomarkers in supporting conventional histological diagnostic tests for prostate carcinoma (PCa) detection. METHODS:Microarray profiling of gene expression and DNA methylation was performed on 16 benign prostatic hyperplasia (BPH) and 32 ...

    journal_title:British journal of cancer

    pub_type: 杂志文章

    doi:10.1038/bjc.2014.337

    authors: Paziewska A,Dabrowska M,Goryca K,Antoniewicz A,Dobruch J,Mikula M,Jarosz D,Zapala L,Borowka A,Ostrowski J

    更新日期:2014-08-12 00:00:00

  • Chemotherapy of advanced breast cancer: a controlled randomized trial of cyclophosphamide versus a four-drug combination.

    abstract::Ninety-nine patients with advanced breast cancer were randomized to receive either cyclophosphamide continuously or a combination of cyclophosphamide, methotrexate, 5-fluorouracil and vinblastine given intermittently. The number and duration of objective responses were greater in patients receiving the combination but...

    journal_title:British journal of cancer

    pub_type: 临床试验,杂志文章,随机对照试验

    doi:10.1038/bjc.1975.284

    authors: Rubens RD,Knight RK,Hayward JL

    更新日期:1975-12-01 00:00:00

  • EpCAM is overexpressed in local and metastatic prostate cancer, suppressed by chemotherapy and modulated by MET-associated miRNA-200c/205.

    abstract:BACKGROUND:Expression of epithelial cell adhesion molecule (EpCAM) is deregulated in epithelial malignancies. Beside its role in cell adhesion, EpCAM acts as signalling molecule with tumour-promoting functions. Thus, EpCAM is part of the molecular network of oncogenic receptors and considered an interesting therapeutic...

    journal_title:British journal of cancer

    pub_type: 杂志文章

    doi:10.1038/bjc.2014.366

    authors: Massoner P,Thomm T,Mack B,Untergasser G,Martowicz A,Bobowski K,Klocker H,Gires O,Puhr M

    更新日期:2014-08-26 00:00:00

  • Normal tissues toxicities triggered by combined anti-angiogenic and radiation therapies: hurdles might be ahead.

    abstract:BACKGROUND:Combined-modality therapy is a promising approach to improve the therapeutic index of radiotherapy. However, these improvements could come at the cost of increased toxicities. Clinical trials evaluating anti-tumour efficacy of bevacizumab combined with radiotherapy have encountered unexpected side effects. T...

    journal_title:British journal of cancer

    pub_type: 杂志文章

    doi:10.1038/bjc.2012.236

    authors: Mangoni M,Vozenin MC,Biti G,Deutsch E

    更新日期:2012-07-10 00:00:00

  • E-cadherin relates to EGFR expression and lymph node metastasis in primary breast carcinoma.

    abstract::Expression of the calcium-dependent cell-cell adhesion molecule E-cadherin has been examined in 187 primary breast carcinomas using an immunohistochemical technique. The pattern and extent of reactivity has been correlated with clinicopathological data including tumour type, grade and lymph node status and with other ...

    journal_title:British journal of cancer

    pub_type: 杂志文章

    doi:10.1038/bjc.1996.522

    authors: Jones JL,Royall JE,Walker RA

    更新日期:1996-10-01 00:00:00

  • Combinatorial experimental protocols for Erbicin-derived immunoagents and Herceptin.

    abstract::Erbicin is a human anti-ErbB2 single-chain antibody fragment with high affinity and selectivity for ErbB2-positive cancer cells. Two anti-ErbB2 immunoconjugates, called Erb-hRNase and Erb-hcAb, have been prepared and found to be selectively cytotoxic on ErbB2-positive cancer cells in vitro and vivo. In Erb-hRNase, Erb...

    journal_title:British journal of cancer

    pub_type: 杂志文章

    doi:10.1038/sj.bjc.6604022

    authors: De Lorenzo C,Troise F,Cafaro V,D'Alessio G

    更新日期:2007-11-19 00:00:00

  • Prevalence and determinants of human papillomavirus genital infection in men.

    abstract::Four-hundred-forty-five husbands of women with invasive cervical carcinoma, 165 of women with in situ cervical cancer, and 717 of control women (age range 19-82 years) were interviewed and a sample of exfoliated cells from the penis obtained in seven case-control studies conducted by the International Agency for Resea...

    journal_title:British journal of cancer

    pub_type: 杂志文章

    doi:10.1038/sj.bjc.6600194

    authors: Franceschi S,Castellsagué X,Dal Maso L,Smith JS,Plummer M,Ngelangel C,Chichareon S,Eluf-Neto J,Shah KV,Snijders PJ,Meijer CJ,Bosch FX,Muñoz N

    更新日期:2002-03-04 00:00:00

  • Telomerase activity in benign and malignant human thyroid tissues.

    abstract::Telomerase is a specialized ribonucleoprotein polymerase that directs the synthesis of telomerase repeats at chromosome ends. Accumulating evidence has indicated that telomerase is stringently repressed in normal human somatic tissues but reactivated in cancers and immortal cells, suggesting that activation of telomer...

    journal_title:British journal of cancer

    pub_type: 杂志文章

    doi:10.1038/bjc.1998.363

    authors: Cheng AJ,Lin JD,Chang T,Wang TC

    更新日期:1998-06-01 00:00:00

  • KRAS mutational status affects oxaliplatin-based chemotherapy independently from basal mRNA ERCC-1 expression in metastatic colorectal cancer patients.

    abstract:BACKGROUND:In this study, we evaluated the possibility that KRAS mutational status might be predictive of oxaliplatin (OXA) efficacy. We also explored the role of excision repair cross complementing group-1 (ERCC-1). METHODS:Ninety anti-epidermal growth factor receptor-naive advanced colorectal cancer patients were re...

    journal_title:British journal of cancer

    pub_type: 杂志文章

    doi:10.1038/bjc.2012.526

    authors: Basso M,Strippoli A,Orlandi A,Martini M,Calegari MA,Schinzari G,Di Salvatore M,Cenci T,Cassano A,Larocca LM,Barone C

    更新日期:2013-01-15 00:00:00

  • Effects of varying the interval between courses of methotrexate on its myelotoxic and anti-leukaemic activities.

    abstract::The toxicity produced by two courses of methotrexate separated by different intervals has been studied in matched groups of rats. The maximum degree of neutropenia reached when courses were separated by 8 days or more was no greater than that seen after a single course of methotrexate. However, when courses of neutrop...

    journal_title:British journal of cancer

    pub_type: 杂志文章

    doi:10.1038/bjc.1977.2

    authors: Harding B,Culvenor J,MacLennan IC

    更新日期:1977-01-01 00:00:00

  • Restored T-cell activation mechanisms in human tumour-infiltrating lymphocytes from melanomas and colorectal carcinomas after exposure to interleukin-2.

    abstract::We investigated the effects of interleukin-2 (IL-2) exposure on T-cell signal transduction molecules and apoptosis markers in tumour-infiltrating lymphocytes (TIL) isolated from 20 melanoma and 16 colorectal carcinoma metastases and expanded in vitro for therapeutic reinfusion. Before IL-2 culture, TIL showed undetect...

    journal_title:British journal of cancer

    pub_type: 杂志文章

    doi:10.1038/sj.bjc.6600679

    authors: De Paola F,Ridolfi R,Riccobon A,Flamini E,Barzanti F,Granato AM,Mordenti GL,Medri L,Vitali P,Amadori D

    更新日期:2003-01-27 00:00:00

  • Molecular targeting of retinoic acid metabolism in neuroblastoma: the role of the CYP26 inhibitor R116010 in vitro and in vivo.

    abstract::Isomerisation to all-trans-retinoic acid (ATRA) is widely accepted as the key mechanism underlying the favourable clinical properties of 13-cis-retinoic acid (13cisRA). As intracellular metabolism of ATRA by CYP26 may result in clinical resistance to 13cisRA, an increase in efficacy may be achieved through modulation ...

    journal_title:British journal of cancer

    pub_type: 杂志文章

    doi:10.1038/sj.bjc.6603779

    authors: Armstrong JL,Taylor GA,Thomas HD,Boddy AV,Redfern CP,Veal GJ

    更新日期:2007-06-04 00:00:00

  • Lack of Bcl10 mutations in testicular germ cell tumours and derived cell lines.

    abstract::Aberrations within Bcl10, a gene involved in execution of apoptosis, has most recently been found in a variety of cancers, including cell lines of testicular germ cell tumours of adolescents and adults (TGCTs). To study this in more detail, we screened exons 2 and 3 of this gene for mutations in a larger series of cel...

    journal_title:British journal of cancer

    pub_type: 杂志文章

    doi:10.1038/sj.bjc.6690563

    authors: van Schothorst EM,Mohkamsing S,van Gurp RJ,Oosterhuis JW,van der Saag PT,Looijenga LH

    更新日期:1999-07-01 00:00:00

  • External multicentre validation of a nomogram predicting the risk of relapse in patients with borderline ovarian tumours.

    abstract:BACKGROUND:The Obermair nomogram was recently developed to predict the risk of relapse in patients with borderline ovarian tumours (BOTs) based on five readily available clinical, biological, and pathological characteristics. We set out to externally validate and assess its robustness using a multi-institutional BOT da...

    journal_title:British journal of cancer

    pub_type: 杂志文章,多中心研究

    doi:10.1038/bjc.2013.678

    authors: Bendifallah S,Uzan C,Fauvet R,Morice P,Darai E

    更新日期:2013-11-26 00:00:00

  • Post-neoadjuvant cellular dissociation grading based on tumour budding and cell nest size is associated with therapy response and survival in oesophageal squamous cell carcinoma.

    abstract:BACKGROUND:Cellular Dissociation Grade (CDG) composed of tumour budding and cell nest size has been shown to independently predict prognosis in pre-therapeutic biopsies and primary resections of oesophageal squamous cell carcinoma (ESCC). Here, we aimed to evaluate the prognostic impact of CDG in ESCC after neoadjuvant...

    journal_title:British journal of cancer

    pub_type: 杂志文章

    doi:10.1038/s41416-019-0623-2

    authors: Jesinghaus M,Boxberg M,Wilhelm D,Münch S,Dapper H,Quante M,Schlag C,Lange S,Budczies J,Konukiewitz B,Mollenhauer M,Schlitter AM,Becker KF,Feith M,Friess H,Steiger K,Combs SE,Weichert W

    更新日期:2019-12-01 00:00:00

  • Targeting SDF-1/CXCR4 to inhibit tumour vasculature for treatment of glioblastomas.

    abstract::Local recurrence of glioblastomas is a major cause of patient mortality after definitive treatment. This review discusses the roles of the chemokine stromal cell-derived factor-1 and its receptor CXC chemokine receptor 4 (CXCR4) in affecting the sensitivity of glioblastomas to irradiation. Blocking these molecules pre...

    journal_title:British journal of cancer

    pub_type: 杂志文章,评审

    doi:10.1038/bjc.2011.169

    authors: Tseng D,Vasquez-Medrano DA,Brown JM

    更新日期:2011-06-07 00:00:00

  • Anti-Her-2/neu antibody induces apoptosis in Her-2/neu overexpressing breast cancer cells independently from p53 status.

    abstract::Anti-Her-2/neu antibody is known to induce apoptosis in HER-2/neu overexpressing breast cancer cells. However, exact regulatory mechanisms mediating and controlling this phenomenon are still unknown. In the present study, we have investigated the effect of anti-Her-2/neu antibody on apoptosis of HER-2/neu overexpressi...

    journal_title:British journal of cancer

    pub_type: 杂志文章

    doi:10.1054/bjoc.2001.2197

    authors: Brodowicz T,Kandioler D,Tomek S,Ludwig C,Rudas M,Kunstfeld R,Koestler W,Hejna M,Budinsky A,Wiltschke C,Zielinski CC

    更新日期:2001-11-30 00:00:00

  • Cytokeratin 7 and 20 staining for the diagnosis of lung and colorectal adenocarcinoma.

    abstract::The origin of metastatic adenocarcinoma lesions can sometimes be difficult to diagnose. The objectives of our study were to establish the cytokeratin staining pattern of primary and metastatic lung and colorectal adenocarcinomas, and to determine if this helps to identify the site of origin of metastatic lesions. We r...

    journal_title:British journal of cancer

    pub_type: 杂志文章

    doi:10.1038/sj.bjc.6600326

    authors: Kummar S,Fogarasi M,Canova A,Mota A,Ciesielski T

    更新日期:2002-06-17 00:00:00

  • Risk of cancer in patients with epistaxis and haemoptysis.

    abstract::This corrects the article DOI: 10.1038/bjc.2017.85. ...

    journal_title:British journal of cancer

    pub_type: 杂志文章

    doi:10.1038/bjc.2017.494

    authors: Ording AG,Veres K,Farkas DK,Adelborg K,Sørensen HT

    更新日期:2018-03-20 00:00:00

  • Neonatal growth and breast cancer risk in adulthood.

    abstract::Birth size of a woman has been positively associated with her breast cancer risk, particularly before menopause, but no study has investigated neonatal growth in relation to this risk. We conducted a case-control study nested within a population-based cohort of women, born in Sweden between 1901 and 1961, covering all...

    journal_title:British journal of cancer

    pub_type: 杂志文章

    doi:10.1038/sj.bjc.6604702

    authors: Lagiou P,Hsieh CC,Trichopoulos D,Adami HO,Hall P,Chie L,Ekbom A

    更新日期:2008-11-04 00:00:00

  • Chemical modification and immunogenicity of membrane fractions from mouse tumour cells.

    abstract::A crude membrane fraction isolated from mouse tumour cells was treated with various chemicals. The effects on the immunogenicity of the membrane sample were tested in syngeneic mice for tumour protection, using a challenge dose of 10(5) viable tumour cells. Best protection was obtained after immunization of mice with ...

    journal_title:British journal of cancer

    pub_type: 杂志文章

    doi:10.1038/bjc.1978.236

    authors: Staab HJ,Anderer FA

    更新日期:1978-10-01 00:00:00

  • Accelerated BEP: a phase I trial of dose-dense BEP for intermediate and poor prognosis metastatic germ cell tumour.

    abstract:BACKGROUND:We used bleomycin, etoposide, cisplatin (BEP), the most effective regimen in the treatment of germ cell tumours (GCTs) and increased dose-density by using pegfilgrastim to shorten cycle length. Our aim was to assess safety and tolerability. METHODS:Sixteen male patients with intermediate or poor prognosis m...

    journal_title:British journal of cancer

    pub_type: 杂志文章

    doi:10.1038/bjc.2011.309

    authors: Rimmer Y,Chester J,Joffe J,Stark D,Shamash J,Powles T,White J,Wason J,Parashar D,Armstrong G,Mazhar D,Williams MV

    更新日期:2011-09-06 00:00:00

  • Number of apoptotic cells as a prognostic marker in invasive breast cancer.

    abstract::Apoptosis plays an important role in tumorigenesis. Tumour growth is determined by the rate of cell proliferation and cell death. We counted the number of apoptotic cells in haematoxylin and eosin (H&E)-stained tumour sections in series of 172 grade I and II invasive breast cancers with long-term follow-up. The number...

    journal_title:British journal of cancer

    pub_type: 杂志文章

    doi:10.1054/bjoc.1999.0928

    authors: de Jong JS,van Diest PJ,Baak JP

    更新日期:2000-01-01 00:00:00

  • Avoidable deaths and random variation in patients' survival.

    abstract:BACKGROUND:Random error in the numbers of avoidable deaths among cancer patients has not been considered in earlier studies. METHODS:Methods to obtain valid confidence intervals (CIs) for numbers of avoidable deaths were developed. The excess mortality rates were estimated for patients diagnosed with colon cancer in f...

    journal_title:British journal of cancer

    pub_type: 杂志文章

    doi:10.1038/bjc.2012.169

    authors: Seppä K,Hakulinen T,Läärä E

    更新日期:2012-05-22 00:00:00

  • Influence of lodgement site on the proliferation of metastases of Walker 256 carcinoma in the rat.

    abstract::The growth of s.c. Walker 256 carcinoma was found to be independent of secondary growths induced by i.v. injection. Tumour cells injected i.v. lodged mainly in the lungs, with small clusters of cells in the lymph nodes. The rate of cellular proliferation of these secondary growths of Walker carcinoma was significantly...

    journal_title:British journal of cancer

    pub_type: 杂志文章

    doi:10.1038/bjc.1978.12

    authors: Bellamy D,Hinsull SM

    更新日期:1978-01-01 00:00:00