ReviewReliable assessment of the effects of treatment on mortality and major morbidity, II: observational studies
Section snippets
Assessment of adverse effects of treatment
Observational studies can have an important role in the identification of large adverse effects of treatments, particularly on infrequent outcomes that are not likely to be related to the indications for, or contraindications to, the treatment of interest (Panel 1). Perhaps one of the best illustrations of this is the detection of increased risks of abnormal fetal limb development after maternal use of thalidomide.2 A decade later, observational studies also detected the many-fold increased
CONCLUSIONS: Improving health care by the appropriate use of epidemiological evidence
Both randomised trials and observational studies can contribute important evidence about the effects of treatment on mortality and major non-fatal outcomes. The appropriate role for each type of study is determined primarily by the extent to which random error and bias can be guaranteed to be sufficiently small for the question posed to be answered reliably. Observational studies can often reduce random error substantially by involving very large numbers of individuals with a specific disease
References (72)
- et al.
Reliable assessment of the effects of treatmenton mortality and major morbidity, I: clinical trials
Lancet
(2001) - et al.
Risk of Stevens-Johnson syndrome and toxic epidermal necrolysis during first weeks of antiepileptic therapy: a case-control study
Lancet
(1999) - et al.
General and local administration of penicillin
Lancet
(1943) - et al.
Do inhibitors of angiotensin-Iconverting enzyme protect against risk of cancer?
Lancet
(1998) - et al.
Statins and the risk of dementia
Lancet
(2000) - et al.
Beta blockade during and after myocardial infarction: an overview of the randomized trials
Prog Cardiovasc Dis
(1985) - et al.
Low-dose and high-dose acetylsalicylic acid for patients undergoing carotid endar terectomy: a randomised controlled trial
Lancet
(1999) - et al.
Reporting bias in retrospective ascertainment of drug-induced embryopathy
Lancet
(1999) - et al.
Principles of epidemiological research on drug effects
Lancet
(1999) - et al.
Principles of epidemiological research on drug effects
Lancet
(1999)
Association of road-traffic accidents with benzodiazepine use
Lancet
Ups and downs for HRT and heart disease
Lancet
For the UK Heart Investigation. Digoxin and mortality in chronic heart failure
Lancet
Safety concerns about digoxin after acute myocardial infarction
Lancet
Reserpine use in relation to breast cancer
Lancet
Calcium-channel blockade and incidence of cancer in aged populations
Lancet
Blood pressure, stroke, and coronary heart disease. Part 1, prolonged differences in blood pressure: prospective observational studies corrected for the regression dilution bias
Lancet
The saga of thalidomide
N Engl J Med
Adenocarcinoma of the vagina: association of maternal stilbestrol therapy with tumor appearance in young women
N Engl J Med
The prevalence of cardiac valvular insufficiency assessed by transthoracic echocardiography in obese patients treated with appetite-suppressant drugs
N Engl J Med
Hormone therapy to prevent disease and prolong life in postmenopausal women
Ann Intern Med
Use of HRT and the subsequent risk of cancer
J Epidemiol Biostats
Results of treatment in malignant hypertension: a seven-year experience in 94 cases
BMJ
Oral fluid therapy of cholera among Bangladesh refugees
Johns Hopkins Med J
Observational evidence
Ann N Y Acad Sci
On behalf of the SAVE investigators. Effect of captopril on mortality and morbidity in patients with left ventricular dysfunction after myocardial infarction: results of the survival and ventricular enlargement trial
N Engl J Med
Effect of enalapril on mortality and the development of heart failure in asymptomatic patients with reduced left ventricular ejection fractions
N Engl J Med
Effects of an angiotensin-converting-enzyme inhibitor, ramipril, on cardiovascular events in high-risk patients
N Engl J Med
Hormone replacement therapy, heart disease, and other considerations
Annu Rev Public Health
Hormone replacement therapy
BMJ
Adverse outcomes of underuse of β-blockers in elderly survivors of acute myocardial infarction
JAMA
Effects of betablocker therapy on mortality in patients with heart failure: a systematic overview of randomized controlled trials
Eur Heart J
The Cardiac Insufficiency Bisoprolol Study II (CIBIS II): a randomised trial
Lancet
Effects of controlled-release metoprolol on total mortality, hospitalizations, and well-being in patients with heart failure: the Metroprolol CR/XL Randomized Intervention Trial in Congestive Heart Failure (MERIT-HF)
JAMA
Underestimation of risk associations due to regression dilution in long-term follow-up of prospective studies
Am J Epidemiol
Is blood pressure treatment as effective in a population setting as in controlled trials? Results from a prospective study
J Hypertens
Cited by (213)
Empagliflozin in patients admitted to hospital with COVID-19 (RECOVERY): a randomised, controlled, open-label, platform trial
2023, The Lancet Diabetes and EndocrinologyResearch methodology in acupuncture and moxibustion for managing primary dysmenorrhea: A scoping review
2022, Complementary Therapies in MedicineClinical Trials in Perioperative Medicine
2022, Perioperative Quality ImprovementHydroxychloroquine for COVID-19: Balancing contrasting claims
2020, European Journal of Internal MedicineAdherence and acceptability of light therapies to improve sleep in intrinsic circadian rhythm sleep disorders and neuropsychiatric illness: a systematic review
2020, Sleep HealthCitation Excerpt :For this reason, although 51/78 studies reported no adverse effects (or reported ‘no serious adverse effects’, 2 studies), it was often not possible to confidently distinguish between studies where no adverse effects occurred, where no adverse effects were identified or where adverse effects might have been identified but not reported in the manuscript. It should also be noted that most studies were not powered to detect rare adverse effects, which requires large trials or observational studies.104 Only 6 of 47 studies reported separate numbers for adverse effects in the active intervention versus control condition, limiting conclusions.
Retrospective Real-World Studies of Paclitaxel and Mortality: Defining the Many Faces of Bias
2020, JACC: Cardiovascular Interventions