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Table 2 Key results and main author conclusions from Method A studies

From: A literature review on the representativeness of randomized controlled trial samples and implications for the external validity of trial results

Study Real-world data source Key differences (real-world versus RCT patients) Main author conclusionsb
Cardiology
Badano et al., 2003 [15] MC-PR Older, more female, higher rates of concomitant diabetes, greater LVF clinical impairment Different
Björklund et al., 2004 [17] MC-PR Older, more female and more CV risk factors Different
Costantino et al., 2009a [21] SC-PR Older, more female, lower NYHA class Different
Dhruva et al., 2008 [22] ID Older, more female Different
Ezekowitz et al., 2012 [24] MC-PR Older, more female, more co-morbidities/prior cancer Different
Golomb et al., 2012 [27] MC-PR Increased self-rated physical activity with increasing age Different
Hutchinson-Jaffe et al., 2010 [29] MC-PR Older, more female, more co-morbidities, less guideline-recommended treatment/procedures Different
Melloni et al., 2010 [37] MC-PR More female Different
Steinberg et al., 2007 [62] MC-PR Older, more co-morbidities/CVD history NE
Uijen et al., 2007a [44] MC-PR Older, more female, higher CVD risk Different
Wagner et al., 2011 [65] ID Older, more chronic diseases NE
Mental health
Kushner et al., 2009 [57] MC-PR Greater depression severity (some scales), lower preference for novel experiences NE
Rabinowitz et al., 2003a [59] MC-PR No major differences Similar
Riedel et al., 2005 [60] SC-PR Older, longer duration of illness, more internistic co-morbidities/hospitalizations Similar
Surman et al., 2010a [42] SC-PR More co-morbidities, anxiety/depression, alcohol/substance dependence Different
Zarin et al., 2005a [49] MC-PR Older, more female/Caucasian Different
Oncology
Baquet et al., 2009 [52] MC-PR Fewer females (non-sex-specific tumor RCTs), fewer males (sex-specific tumor RCTs) NE
Elting et al., 2006 [23] SC-PR Older, more females/chronic co-morbidities, worse health/performance status Different
Fraser et al., 2011a [25] MC-PR Worse disease prognosis, more drug-related toxicity, lower drug dose intensity Different
Jennens et al., 2006 [30] MC-PR Older Different
Kalata et al., 2009 [31] MC-PR Older, more females, worse prognosis Different
Mengis et al., 2003a [38] SC-PR Older, worse performance status, more infections/AML-MDS subtypes Different
van der Linden et al., 2014 [45] MC-PR Older, more females, poor prognostic factors Different
Yennurajalingam et al., 2013 [48] SC-PR Older, more males, higher symptom intensity scores Different
Yessaian et al., 2005 [66] MC-PR No major differences Similar
  1. Please see Additional files 2 and 3 for more detailed results
  2. aStudies that employed Methods A and B; in these studies RCT and real-world populations were compared, the authors then used the eligibility criteria from the RCT of interest to determine how many patients would hypothetically have been eligible or ineligible for that trial. Results presented in this table are for Method A only (see Table 3 for Method B results). bDifferent: authors explicitly comment, in their opinion, that there were meaningful differences between populations that suggested they were not representative, that the data could not be extrapolated or were not applicable to real-world settings, and/or that external validity is impacted; NE: authors do not explicitly comment on external validity or do not comment on external validity despite demonstration of differences in baseline characteristics; Similar: authors comment that populations are similar and/or that RCT results are generalizable to the overall disease population
  3. AML acute myeloid leukemia, CV cardiovascular, CVD cardiovascular disease, ID insurance data; LVF left ventricular function, MC-PR patient records - multicenter (including multicenter registries), MDS myelodysplastic syndrome, NYHA New York Heart Association, RCT randomized controlled trial, SC-PR patient records - single center