Statistical challenges in the evaluation of treatments for small patient populations (Q38093778)

28 August 2017

Statistical challenges in the evaluation of treatments for small patient populations (English)

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28 August 2017

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3

Boris Freidlin

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28 August 2017

Edward L Korn

1

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PubMed ID

23536014

28 August 2017

Lisa M McShane

series ordinal

2

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PubMed ID

23536014

28 August 2017

language of work or name

English

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publication date

1 March 2013

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Science Translational Medicine

28 August 2017

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28 August 2017

5

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28 August 2017

178sr3

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28 August 2017

178

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Effect of VX-770 in persons with cystic fibrosis and the G551D-CFTR mutation

28 August 2017

1 reference

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A CFTR potentiator in patients with cystic fibrosis and the G551D mutation

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCITRANSLMED.3004018

Multi-arm clinical trials of new agents: some design considerations

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCITRANSLMED.3004018

Data monitoring committees and interim monitoring guidelines

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCITRANSLMED.3004018

Discrete sequential boundaries for clinical trials

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCITRANSLMED.3004018

A Multiple Testing Procedure for Clinical Trials

21 January 2018

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https://api.crossref.org/works/10.1126%2FSCITRANSLMED.3004018

Monitoring for lack of benefit: a critical component of a randomized clinical trial

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCITRANSLMED.3004018

A general inefficacy interim monitoring rule for randomized clinical trials

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCITRANSLMED.3004018

Role of independent data-monitoring committees in randomized clinical trials sponsored by the National Cancer Institute

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCITRANSLMED.3004018

A randomized trial of fetal endoscopic tracheal occlusion for severe fetal congenital diaphragmatic hernia

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCITRANSLMED.3004018

Outcome--adaptive randomization: is it useful?

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCITRANSLMED.3004018

Adaptive randomization versus interim monitoring

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCITRANSLMED.3004018

Efficacy and tolerability of a new formulation of pancrelipase delayed-release capsules in children aged 7 to 11 years with exocrine pancreatic insufficiency and cystic fibrosis: a multicenter, randomized, double-blind, placebo-controlled, two-perio

21 January 2018

1 reference

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Treatment of hereditary angioedema with danazol. Reversal of clinical and biochemical abnormalities

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCITRANSLMED.3004018

Controlled trials in single subjects. 2. Limitations of use.

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCITRANSLMED.3004018

Testing and interval estimation for two-sample survival comparisons with small sample sizes and unequal censoring

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCITRANSLMED.3004018

Surrogate end points in clinical trials: are we being misled?

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCITRANSLMED.3004018

Mortality and morbidity in patients receiving encainide, flecainide, or placebo. The Cardiac Arrhythmia Suppression Trial

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCITRANSLMED.3004018

The Cardiac Arrhythmia Suppression Trial: first CAST ... then CAST-II.

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCITRANSLMED.3004018

Design issues of randomized phase II trials and a proposal for phase II screening trials

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCITRANSLMED.3004018

Design issues in randomized phase II/III trials

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCITRANSLMED.3004018

Disease-free survival versus overall survival as a primary end point for adjuvant colon cancer studies: individual patient data from 20,898 patients on 18 randomized trials

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCITRANSLMED.3004018

Overall survival as the outcome for randomized clinical trials with effective subsequent therapies

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCITRANSLMED.3004018

Efficacy and safety of a specific inhibitor of the BCR-ABL tyrosine kinase in chronic myeloid leukemia

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCITRANSLMED.3004018

Hematologic and cytogenetic responses to imatinib mesylate in chronic myelogenous leukemia

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCITRANSLMED.3004018

Effects of a selective inhibitor of the Abl tyrosine kinase on the growth of Bcr-Abl positive cells

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCITRANSLMED.3004018

Imatinib compared with interferon and low-dose cytarabine for newly diagnosed chronic-phase chronic myeloid leukemia

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCITRANSLMED.3004018

De novo CIAS1 mutations, cytokine activation, and evidence for genetic heterogeneity in patients with neonatal-onset multisystem inflammatory disease (NOMID): a new member of the expanding family of pyrin-associated autoinflammatory diseases

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCITRANSLMED.3004018

Chronic infantile neurological cutaneous and articular syndrome is caused by mutations in CIAS1, a gene highly expressed in polymorphonuclear cells and chondrocytes

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCITRANSLMED.3004018

Neonatal-onset multisystem inflammatory disease responsive to interleukin-1beta inhibition

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCITRANSLMED.3004018

Sustained response and prevention of damage progression in patients with neonatal-onset multisystem inflammatory disease treated with anakinra: a cohort study to determine three- and five-year outcomes

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCITRANSLMED.3004018

Induction and maintenance infliximab therapy for the treatment of moderate-to-severe Crohn's disease in children.

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCITRANSLMED.3004018

New approaches to drug development in pediatric oncology

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCITRANSLMED.3004018

Much ado about not...enough data: high-dose chemotherapy with autologous stem cell rescue for breast cancer

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCITRANSLMED.3004018

Improved early event-free survival with imatinib in Philadelphia chromosome-positive acute lymphoblastic leukemia: a children's oncology group study

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCITRANSLMED.3004018

The Will Rogers phenomenon. Stage migration and new diagnostic techniques as a source of misleading statistics for survival in cancer

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCITRANSLMED.3004018

Prospective trial of a pediatric ventricular assist device.

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCITRANSLMED.3004018

Berlin Heart EXCOR Pediatric ventricular assist device Investigational Device Exemption study: study design and rationale.

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCITRANSLMED.3004018

Rare diseases and the assessment of intervention: what sorts of clinical trials can we use?

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCITRANSLMED.3004018

Conditional power calculations for clinical trials with historical controls.

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCITRANSLMED.3004018

Progression-free survival in gastrointestinal stromal tumours with high-dose imatinib: randomised trial

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCITRANSLMED.3004018

Outcome of patients with advanced gastro-intestinal stromal tumours crossing over to a daily imatinib dose of 800 mg after progression on 400 mg.

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCITRANSLMED.3004018

Clinical trial of a farnesyltransferase inhibitor in children with Hutchinson-Gilford progeria syndrome

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCITRANSLMED.3004018

Disease progression in Hutchinson-Gilford progeria syndrome: impact on growth and development.

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCITRANSLMED.3004018

The Path to Personalized Medicine

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCITRANSLMED.3004018

Anaplastic lymphoma kinase inhibition in non-small-cell lung cancer

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCITRANSLMED.3004018

Effective incorporation of biomarkers into phase II trials

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCITRANSLMED.3004018

Trastuzumab in combination with chemotherapy versus chemotherapy alone for treatment of HER2-positive advanced gastric or gastro-oesophageal junction cancer (ToGA): a phase 3, open-label, randomised controlled trial

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCITRANSLMED.3004018

Raised conditional level of significance for the 2 × 2-table when testing the equality of two probabilities

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCITRANSLMED.3004018

Tumour necrosis factor α and interleukin 1β in relapse of Crohn's disease

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCITRANSLMED.3004018

10.1126/SCITRANSLMED.3004018

21 January 2018

Identifiers

DOI

1 reference

28 August 2017

1 reference

PubMed ID

23536014