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2015 ; 10
(9
): e0136640
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gab.com Text
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English Wikipedia
The Role of End-of-Life Issues in the Design and Reporting of Cancer Clinical
Trials: A Structured Literature Review
#MMPMID26327232
Gaertner J
; Weingärtner V
; Lange S
; Hausner E
; Gerhardus A
; Simon ST
; Voltz R
; Becker G
; Schmacke N
PLoS One
2015[]; 10
(9
): e0136640
PMID26327232
show ga
BACKGROUND: Randomized controlled trials (RCTs) are important sources of
information on the benefits and harms patients may expect from treatment options.
The aim of this structured literature review by the German Institute for Quality
and Efficiency in Health Care was to explore whether and how the end-of-life
(EoL) situation of patients with advanced cancer is considered in RCTs
investigating anti-cancer treatments. METHODS: Our journal pool comprised 19
medical journals, namely five preselected key general medical journals as well as
14 specialist journals (mainly cancer) identified via a scoping search. We
systematically searched these journals in MEDLINE to identify RCTs investigating
anti-cancer treatments for the following four cancer types: glioblastoma, lung
cancer (stage IIIb-IV), malignant melanoma (stage IV), and pancreatic cancer
(search via OVID; November 2012). We selected a representative sample of 100
publications, that is, the 25 most recent publications for each cancer type. EoL
was defined as a life expectancy of ? two years. We assessed the information
provided on (1) the descriptions of the terminal stage of the disease, (2) the
therapeutic goal (i.e. the intended therapeutic benefit of the intervention
studied), (3) the study endpoints assessed, (4) the authors' concluding appraisal
of the intervention's effects, and (5) the terminology referring to the patients'
EoL situation. RESULTS: Median survival was ? one year for each of the four
cancer types. Descriptions of the terminal stage of the disease were ambiguous or
lacking in 29/100 publications. One or more therapeutic goals were mentioned in
51/100 publications; these goals were patient-relevant in 38 publications
(survival alone: 30/38; health-related quality of life (HRQoL) or HRQoL and
survival: 6/38; symptom control or symptom control and survival: 2/38). Primary
endpoints included survival (50%), surrogates (44%), and safety (3%).
Patient-reported outcomes (PROs) were assessed in 36/100 RCTs. The implications
of treatment-related harms for the patients were discussed in 22/100 appraisals.
Terminology referring to the patients' EoL situation (e.g. "terminal") was
scarce, whereas terms suggesting control of the disease (e.g. "cancer control")
were common. CONCLUSIONS: The EoL situation of patients with advanced cancer
should be more carefully considered in clinical trials. Although the
investigation and robust reporting of PROs is a prerequisite for informed
decision-making in healthcare, they are rarely defined as endpoints and HRQoL is
rarely mentioned as a therapeutic goal. Suggestions for improving standards for
study design and reporting are presented.