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Table 3 Studies comparing cancer outcomes within and outside clinical trials

From: Are cancer patients better off if they participate in clinical trials? A mixed methods study

ReferencePopulationTypeDatesEn-rolledaEligible controlsTreatment similaritybPotential confounders and methods of control in adjusted analysis:cTrial effect observed:1
Accounted for:Unaccounted for:Unadjusted:Adjusted:d
1. Arrieta et al. [28]Advanced non-small lung cancerRC2007–2014143/889NoNoNBD: Age, wood smoke exposure, exposure to asbestos, tobacco pack-years, brain metastases, ECOG PS, tobacco smoking group
MVA: Disease stage, histology, sex, KRAS mutational status, EGFR mutational status, comorbiditye
RoC: Measurable disease, age
> 18 years, ECOG PS ≤2
SgPA: Received chemotherapye, positive EGFR mutatione
Treatment centre
2. Bertelli et al. [29]Epithelial ovarian, fallopian tube, primary peritoneal cancer stage IV or sub-optimally
debulked stage III
RC2006–2010 (trial)
2012–2015 (real-world)
60/248NoYesRoC: Patients managed by the South Wales Gynaecological Cancer Multidisciplinary Team, received at least one dose of bevacizumab as part of first-line treatmentNA: Sex
NE: Stage,
comorbidity, age, WHO PS, chemotherapy regimen
NoNot done
3. De Placido et al. [30]HER2- positive metastatic breast cancerRC2008–2011 (trial)
2012–2015 (real-world)
155/402NoYesNBD: Age, prior neoadjuvant therapy type
RoC: Inclusion criteria: Received tratuzumab and pertu-zumab with taxane as first-line treatment for metastatic disease, no earlier RCT participation in a neo/adjuvant or metastatic setting
SgPA: HR status, previously treated vs untreated with adjuvant and neoadjuvant tratuzumab, visceral vs non-visceral disease,  > 2 metastatic sites vs 2–5 metastatic sites vs > 5 metastatic sites
NE: Comorbidity, PS, SES, treatment
centre, sex
BD: Brain metastasis, FISH / SISH / CISH statu
4. Field et al. [31]Gliobla-stomaRC1998–201161/481NoNoMVA: Age, IRSAD/SES, ECOG PS, operation type/biopsy
RoC: Two co-located hospitals
NE: First-line clinical trial, comorbidity, tumour location, sex, multifocal disease, operation type, extent of resection, no. of operations, country of birth interpreter, residence, treatment centre, diabetes, smoking, tumour diameter
NA: Stage
5. Goldman et al. [32]Advanced stage melanoma unresecta-ble stage III or IVPC2006–2014115/203NoYesMVA: Sex, primary thickness, primary ulceration, ECOG PS, LDH (stage IV diagnosis), organs with metastases (treatment initiation), metastatic involvement
RoC: Exclusion criteria: Treatments not administered by NYU oncologists, if records omitted pertinent data, patient lost to follow up, therapy administered adjuvantly
StrA: Immunotherapy, targeted therapy
NA: Stage, treatment
SE: SES, comorbidity
BD: Age, prior systemic treatment
6. Khera et al. [33]Acute leukaemia, myelo-dysplasia (MDS), chronic myeloid or myelo-monocytic leukaemia or myelo-fibrosisRC2004–2010494/1353YesYesMVA: Treatment, patient age at transplantation, diagnosis, disease risk, graft source, interval between diagnosis and HCT, KPS, HLA match, donor-recipient sex match, GVHD prophylaxis and use of ATG/alemtuzumab, regimen intensity, ethnicity, treatment centree
RoC: Inclusion criteria: Age < 66 years, HCT. Exclusion criteria: Presence of donor-specific anti-HLA antibodies prior allogeneic or autologous transplantation < 12 months prior, HIV infection, pregnancy or breast-feeding, cardiac insufficiency or coronary artery disease requiring treatment, active infection, concomitant enrolment in a phase 1 study, a serum level of creatinine, bilirubin, ALT or AST that was greater than two times the upper limit of the normal range, forced vital capacity, forced expiratory volume in 1 s and/or diffusing capacity of the lung for carbon monoxide less than 50% of the predicted value
StrA: Race, secondary leukaemia/MDS with prior auto, comorbidity, peripheral blood, bone marrow, sex (NBD)
BD: CMV match, donor age, year of transplantation
7. Le Du et al. [34]Metastatic breast cancer
RC2000–2010285/367YesNoNBD: PR status, radiation, age
previous chemotherapy regimen
MVA: Race, HER2 status, hormone receptor status, number of metastatic organs, previous neoadjuvant vs adjuvant chemotherapy, nuclear grade
RoC: Sex, brain metastases, specific comorbidity, SES/treatment centre; patients living in Harris Country (MD Anderson location), eligible for a clinical trial according to usual inclusion and exclusion criteria for MBC trials and could benefit from the MD Anderson financial assistance programme support
SgPA: ER positive, HER2 positive, triple negative breast cancer
BD: Meta-static sites
8. Svensson et al. [35]Metastatic castration-resistant prostate cancer (mCRPC)RC2008–2010 (trial)
119/1195NoYesNBD: PSA, time since diagnosis at treatment start, Gleason score
RoC: Inclusion criteria: male diagnosed with mCRPC, aged ≥18 years at time of treatment, treated for mCRPC with abiraterone
NE: PS, SES comorbidity, treatment centre
BD: Age
NA: Sex
NoNot done
9. Temple-ton et al. [36]Metastatic castration-resistant prostate cancerRC2001–201243/314NoYesNBD: Comorbidities, Gleason score, extent of disease: bones and visceral metastases, BMI, PSA-dt
MVA: ECOG PS, year of first administration, haemoglobin, log LDH, log time from initial diagnosis, log PSA μg/l, baseline albumin, log ALP
RoC: Exclusion criteria: Patients receiving weekly docetaxel, docetaxel in the context of neoadjuvant or adjuvant trials or as second-line chemotherapy
NA: Sex, treatment centre
BD: Age, extent of disease (lymph nodes)
  1. ALP Alkaline phosphatases, ALT Alanine aminotransferases, AST Aspartate aminotransferases, ATG Antithymocyte globulin, BD Baseline Difference recorded, BMI Body mass index, CISH Chromogenic in situ hybridization, CMV Cytomegalovirus, ECOG Eastern Cooperative Oncology Group, EGFR Epidermal growth factor receptor, ER Oestrogen receptor, FISH Fluorescence in situ hybridization, GVDH Chronic graft-versus-host disease, HCT Hematopoietic cell transplantation, HER2 Human epidermal growth factor receptor, HIV Human immunodeficiency virus, HLA Human leukocyte antigens, HR Hormone receptor, IRSAD Index of Relative Socioeconomic Advantage and Disadvantage, KPS Karnofsky performance score, LHD Lactate dehydrogenases, MVA MultiVariate/MultiVariable/MulticoVariate Analysis, NA Not Applicable, NBD No Baseline Difference, NE Not Evaluated, NYU New York University, PC Prospective Cohort, PS Performance status, PR Progesterone receptor, PSA Prostate specific antigen, PSA-dt Prostate specific antigen - doubling time, RC Retrospective Cohort, RCT Randomised controlled trial, RoC Restriction of cohort, SES Socioeconomic Status, SISH Silver in situ hybridization, SgpA Subgroup Analysis, StrA Stratified Analysis, WHO World Health Organisation
  2. a Values are number of participants in trial/non-trial group
  3. b Similarity between the treatment received by the trial participants and the treatment offered in the control group (for randomised controlled trials) and that was received by non-trial participants
  4. c We assessed whether each study attempted to account for possible confounding by age, sex (where applicable). PS, comorbidity, SES, stage (where applicable) and treatment centre
  5. d Adjusting for confounders by using multivariate or multivariable models, stratification, subgroup analysis, restriction of cohort
  6. e The methods section describes that this was adjusted in multivariate analysis but the results are not presented in the result section
  7. 1p < 0·05, unless otherwise noted
  8. 2 The Kaplan-Meier analyse showed a trial effect (p = 0.007), but the univariate Cox proportional hazard ratio did not (p = 0.089)