From: Are cancer patients better off if they participate in clinical trials? A mixed methods study
Reference | Population | Type | Dates | En-rolleda | Eligible controls | Treatment similarityb | Potential confounders and methods of control in adjusted analysis:c | Trial effect observed:1 | ||
---|---|---|---|---|---|---|---|---|---|---|
Accounted for: | Unaccounted for: | Unadjusted: | Adjusted:d | |||||||
1. Arrieta et al. [28] | Advanced non-small lung cancer | RC | 2007–2014 | 143/889 | No | No | NBD: 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 | NE: SES NA: Treatment centre | Yes | Yes |
2. Bertelli et al. [29] | Epithelial ovarian, fallopian tube, primary peritoneal cancer stage IV or sub-optimally debulked stage III | RC | 2006–2010 (trial) 2012–2015 (real-world) | 60/248 | No | Yes | RoC: Patients managed by the South Wales Gynaecological Cancer Multidisciplinary Team, received at least one dose of bevacizumab as part of first-line treatment | NA: Sex NE: Stage, comorbidity, age, WHO PS, chemotherapy regimen | No | Not done |
3. De Placido et al. [30] | HER2- positive metastatic breast cancer | RC | 2008–2011 (trial) 2012–2015 (real-world) | 155/402 | No | Yes | NBD: 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 | No | No |
4. Field et al. [31] | Gliobla-stoma | RC | 1998–2011 | 61/481 | No | No | MVA: 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 | Yes | Yes |
5. Goldman et al. [32] | Advanced stage melanoma unresecta-ble stage III or IV | PC | 2006–2014 | 115/203 | No | Yes | MVA: 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 centre SE: SES, comorbidity BD: Age, prior systemic treatment | Yes | Yes |
6. Khera et al. [33] | Acute leukaemia, myelo-dysplasia (MDS), chronic myeloid or myelo-monocytic leukaemia or myelo-fibrosis | RC | 2004–2010 | 494/1353 | Yes | Yes | MVA: 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 NE: SES | No | No |
7. Le Du et al. [34] | Metastatic breast cancer (MBC) | RC | 2000–2010 | 285/367 | Yes | No | NBD: 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 | NE: PS BD: Meta-static sites | No | No |
8. Svensson et al. [35] | Metastatic castration-resistant prostate cancer (mCRPC) | RC | 2008–2010 (trial) 2011–2016 (real-world) | 119/1195 | No | Yes | NBD: 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 | No | Not done |
9. Temple-ton et al. [36] | Metastatic castration-resistant prostate cancer | RC | 2001–2012 | 43/314 | No | Yes | NBD: 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 | NE: SES NA: Sex, treatment centre BD: Age, extent of disease (lymph nodes) | Mixed2 | No |