Title |
Complete Cytogenetic Response and Major Molecular Response as Surrogate Outcomes for Overall Survival in First-Line Treatment of Chronic Myelogenous Leukemia: A Case Study for Technology Appraisal on the Basis of Surrogate Outcomes Evidence
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Published in |
Value in Health (Elsevier Science), September 2013
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DOI | 10.1016/j.jval.2013.07.004 |
Pubmed ID | |
Authors |
Ciani Oriana, Hoyle Martin, Pavey Toby, Cooper Chris, Garside Ruth, Rudin Claudius, Taylor Rod |
Abstract |
In 2012, the National Institute for Health and Care Excellence assessed dasatinib, nilotinib, and standard-dose imatinib as first-line treatment of chronic phase chronic myelogenous leukemia (CML). Licensing of these alternative treatments was based on randomized controlled trials assessing complete cytogenetic response (CCyR) and major molecular response (MMR) at 12 months as primary end points. We use this case study to illustrate the validation of CCyR and MMR as surrogate outcomes for overall survival in CML and how this evidence was used to inform National Institute for Health and Care Excellence's recommendation on the public funding of these first-line treatments for CML. |
X Demographics
Geographical breakdown
Country | Count | As % |
---|---|---|
United Kingdom | 1 | 33% |
Italy | 1 | 33% |
Unknown | 1 | 33% |
Demographic breakdown
Type | Count | As % |
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Members of the public | 3 | 100% |
Mendeley readers
Geographical breakdown
Country | Count | As % |
---|---|---|
United States | 1 | 2% |
Switzerland | 1 | 2% |
Brazil | 1 | 2% |
Unknown | 56 | 95% |
Demographic breakdown
Readers by professional status | Count | As % |
---|---|---|
Student > Master | 10 | 17% |
Researcher | 8 | 14% |
Student > Ph. D. Student | 7 | 12% |
Student > Bachelor | 5 | 8% |
Other | 4 | 7% |
Other | 12 | 20% |
Unknown | 13 | 22% |
Readers by discipline | Count | As % |
---|---|---|
Medicine and Dentistry | 16 | 27% |
Mathematics | 4 | 7% |
Nursing and Health Professions | 3 | 5% |
Business, Management and Accounting | 3 | 5% |
Unspecified | 3 | 5% |
Other | 14 | 24% |
Unknown | 16 | 27% |