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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