- 1.
- 2.
Buyse
M.
Towards validation of statistically reliable biomarkers.
Eur J Cancer
2007;5:89–95. 10.1016/S1359-6349(07)70028-9. [
CrossRef]
- 3.
Joffe
MM, Greene
T.
Related causal frameworks for surrogate outcomes.
Biometrics
2009;65:530–8. 10.1111/j.1541-0420.2008.01106.x. [
PubMed: 18759836] [
CrossRef]
- 4.
Beck
A, Ward
C, Mendelson
M, Mock
J, Erbaugh
J.
An inventory for measuring depression.
Arch Gen Psychiatry
1961;4:561–71. 10.1001/archpsyc.1961.01710120031004. [
PubMed: 13688369] [
CrossRef]
- 5.
Pearl
J.
Causality. 2nd edn.
New York, NY;
Cambridge University Press; 2009. 10.1017/CBO9780511803161. [
CrossRef]
- 6.
Rubin
DB.
Estimating causal effects of treatment in randomized and non-randomized studies.
J Educ Psychol
1974;66:688–701. 10.1037/h0037350. [
CrossRef]
- 7.
Angrist
JD, Imbens
GW, Rubin
DB.
Identification of causal effects using instrumental variables.
J Am Stat Assoc
1996;91:444–55. 10.1080/01621459.1996.10476902. [
CrossRef]
- 8.
Barnard
J, Frangakis
CE, Hill
JL, Rubin
DB.
Principal stratification approach to broken randomized experiments: a case study of school choice vouchers in New York City.
J Am Stat Assoc
2003;98:299–311. 10.1198/016214503000071. [
CrossRef]
- 9.
Manski
CF. Nonparametric bounds on treatment effects. Am Econ Rev
1990;80:319–23.
- 10.
Balke
A, Pearl
J.
Bounds on treatment effects from studies with imperfect compliance.
J Am Stat Assoc
1997;92:1172–6. 10.1080/01621459.1997.10474074. [
CrossRef]
- 11.
- 12.
Freeman
D, Dunn
G, Startup
H, Kingdon
D.
The effects of reducing worry in patients with persecutory delusions: study protocol for a randomized controlled trial.
Trials
2012;13:223. 10.1186/1745-6215-13-223. [
PMC free article: PMC3551833] [
PubMed: 23171601] [
CrossRef]
- 13.
Dunn
G, Fowler
D, Rollinson
R, Freeman
D, Kuipers
E, Smith
B, et al.
Effective elements of cognitive behaviour therapy for psychosis: results of a novel type of subgroup analysis based on principal stratification.
Psychol Med
2012;42:1057–68. 10.1017/S0033291711001954. [
PMC free article: PMC3315767] [
PubMed: 21939591] [
CrossRef]
- 14.
Dunn
G, Bentall
R.
Modelling treatment-effect heterogeneity in randomized controlled trials of complex interventions (psychological treatments).
Stat Med
2007;26:4719–45. 10.1002/sim.2891. [
PubMed: 17476649] [
CrossRef]
- 15.
- 16.
Baron
RM, Kenny
DA.
The moderator–mediator variable distinction in social psychological research: conceptual, strategic, and statistical considerations.
J Pers Soc Psychol
1986;51:1173–82. 10.1037/0022-3514.51.6.1173. [
PubMed: 3806354] [
CrossRef]
- 17.
Kraemer
HC, Fairburn
CG, Agras
WS.
Mediators and moderators of treatment effects in randomized clinical trials.
Arch Gen Psychiatry
2002;59:877–83. 10.1001/archpsyc.59.10.877. [
PubMed: 12365874] [
CrossRef]
- 18.
Judd
CM, Kenny
DA.
Process analysis – estimating mediation in treatment evaluations.
Eval Rev
1981;5:602–19. 10.1177/0193841X8100500502. [
CrossRef]
- 19.
MacKinnon
DP. Introduction to Statistical Mediation Analysis.
New York, NY:
Taylor & Francis Group; 2008.
- 20.
Birchwood
M, Peters
E, Tarrier
N, Dunn
G, Lewis
S, Wykes
T, et al.
A multi-centre, randomised controlled trial of cognitive therapy to prevent harmful compliance with command hallucinations.
BMC Psychiatry
2011;11:155. 10.1186/1471-244X-11-155. [
PMC free article: PMC3191332] [
PubMed: 21961763] [
CrossRef]
- 21.
Barrowclough
C, Haddock
G, Wykes
T, Beardmore
R, Conrod
P, Craig
T, et al.
Integrated motivational interviewing and cognitive behavioural therapy for people with psychosis and comorbid substance misuse: randomised controlled trial.
BMJ
2010;341:c6325. 10.1136/bmj.c6325. [
PMC free article: PMC2991241] [
PubMed: 21106618] [
CrossRef]
- 22.
- 23.
Green
J, Charman
T, McConachie
H, Aldred
C, Slonims
V, Howlin
H, et al.
Parent-mediated communication-focused treatment in children with autism (PACT): a randomised controlled trial.
Lancet
2010;375:2152–60. 10.1016/S0140-6736(10)60587-9. [
PMC free article: PMC2890859] [
PubMed: 20494434] [
CrossRef]
- 24.
Bruce
ML, Ten Have
TR, Reynolds
CF, Katz
II, Schulberg
HC, Mulsant
BH, et al.
Reducing suicidal ideation and depressive symptoms in depressed older primary care patients – a randomized controlled trial.
JAMA
2004;291:1081–91. 10.1001/jama.291.9.1081. [
PubMed: 14996777] [
CrossRef]
- 25.
Ten Have
TR, Joffe
MM, Lynch
KG, Brown
GK, Maisto
SA, Beck
AT.
Causal mediation analysis with rank preserving models.
Biometrics
2007;63:926–34. 10.1111/j.1541-0420.2007.00766.x. [
PubMed: 17825022] [
CrossRef]
- 26.
Bellamy
SL, Lin
JY, Ten Have
TR.
An introduction to causal modelling in clinical trials.
Clin Trials
2007;4:58–73. 10.1177/1740774506075549. [
PubMed: 17327246] [
CrossRef]
- 27.
- 28.
Lord
C, Risi
S, Lambrecht
L, Cook
EH
Jr, Leventhal
BL, DiLavore
PC, et al.
The autism diagnostic observation schedule-generic: a standard measure of social and communication deficits associated with the spectrum of autism.
J Autism Dev Disord
2000;30:205–23. 10.1023/A:1005592401947. [
PubMed: 11055457] [
CrossRef]
- 29.
MacKinnon
DP, Dwyer
JH.
Estimating mediated effects in prevention studies.
Eval Rev
1993;17:144–58. 10.1177/0193841X9301700202. [
CrossRef]
- 30.
Emsley
R, Dunn
G, White
IR.
Mediation and moderation of treatment effects in randomised controlled trials of complex interventions.
Stat Methods Med Res
2010;19:237–70. 10.1177/0962280209105014. [
PubMed: 19608601] [
CrossRef]
- 31.
Emsley
R, Dunn
G.
Evaluation of Potential Mediators in Randomized Trials of Complex Interventions (Psychotherapies). In Berzuini
C, Dawid
P, Bernardinelli
L, editors.
Causality: Statistical Perspectives and Applications.
Chichester:
Wiley; 2012. pp. 290–309. 10.1002/9781119945710.ch20. [
CrossRef]
- 32.
Robins
JM, Greenland
S.
Identifiability and exchangeability for direct and indirect effects.
Epidemiology
1992;3:143–55. 10.1097/00001648-199203000-00013. [
PubMed: 1576220] [
CrossRef]
- 33.
Pearl
J. Direct and Indirect Effects. In Breese
J, Koller
D, editors. Proceedings of the Seventeenth Conference on Uncertainty in Artificial Intelligence.
San Francisco, CA:
Morgan Kaufmann; 2011. pp. 411–20.
- 34.
Cai
ZH, Kuroki
M, Pearl
J, Tian
J.
Bounds on direct effects in the presence of confounded intermediate variables.
Biometrics
2008;64:695–701. 10.1111/j.1541-0420.2007.00949.x. [
PubMed: 18162106] [
CrossRef]
- 35.
Wooldridge
JM. Econometric Analysis of Cross Section and Panel Data.
Cambridge, MA:
MIT Press; 2002.
- 36.
VanderWeele
TJ, Vansteelandt
S.
Conceptual issues concerning mediation, interventions and composition.
Stat Interface
2009;2:457–68. 10.4310/SII.2009.v2.n4.a7. [
CrossRef]
- 37.
- 38.
Emsley
R, Liu
H, Dunn
G, Valeri
L, VanderWeele
TJ. Paramed: a command to perform causal mediation analysis using parametric models. 2015; in preparation.
- 39.
Herting
JR.
Evaluating and rejecting true mediation models: a cautionary note.
Prevent Sci
2002;3:285–9. 10.1023/A:1020828709115. [
PubMed: 12458766] [
CrossRef]
- 40.
Holland
PW.
Causal inference, path analysis and recursive structural equation models (with discussion).
Sociol Methodol
1988;18:449–84. 10.2307/271055. [
CrossRef]
- 41.
Kaufman
JS, MacLehose
R, Kaufman
S.
A further critique of the analytic strategy of adjusting for covariates to identify biologic mediation.
Epidemiol Perspect Innovations
2004;1. [
PMC free article: PMC526390] [
PubMed: 15507130]
- 42.
Kaufman
S, Kaufman
JS, MacLehose
RF, Greenland
S, Poole
C.
Improved estimation of controlled direct effects in the presence of unmeasured confounding of intermediate variables.
Stat Med
2005;24:1683–702. 10.1002/sim.2057. [
PubMed: 15742358] [
CrossRef]
- 43.
Tritchler
D.
Explanatory analyses of randomised studies.
Biometrics
1996;52:1450–6. 10.2307/2532858. [
PubMed: 8962463] [
CrossRef]
- 44.
Tritchler
D.
Reasoning about data with directed graphs.
Stat Med
1999;18:2067–76. 10.1002/(SICI)1097-0258(19990830)18:16<2067::AID-SIM182>3.0.CO;2-2. [
PubMed: 10441763] [
CrossRef]
- 45.
McDonald
RP.
Haldane’s lungs: a case study in path analysis.
Mul Behav Res
1997;32:1–38. 10.1207/s15327906mbr3201_1. [
PubMed: 26751104] [
CrossRef]
- 46.
Wooldridge
JM. Introductory Econometrics: A Modern Approach. 2nd edn.
Ohio, OH:
Thompson Learning; 2003.
- 47.
Gennetian
LA, Morris
PA, Bos
JM, Bloom
HS. Constructing Instrumental Variables From Experimental Data to Explore how Treatments Produce Effects. In Bloom
HS, editor. Learning More From Social Experiments: Evolving Analytic Approaches. 1st edn.
New York, NY:
Russell Sage Foundation; 2005. pp. 75–114.
- 48.
Gennetian
LA, Magnuson
K, Morris
PA.
From statistical associations to causation: what developmentalists can learn from instrumental variables techniques coupled with experimental data.
Develop Psychol
2008;44:381–94. 10.1037/0012-1649.44.2.381. [
PMC free article: PMC3208329] [
PubMed: 18331130] [
CrossRef]
- 49.
Sobel
ME.
Identification of causal parameters in randomised studies with mediating variables.
J Educ Behav Stat
2008;33:230–51. 10.3102/1076998607307239. [
CrossRef]
- 50.
Fischer-Lapp
K, Goetghebeur
E.
Practical properties of some structural mean analyses of the effect of compliance in randomized trials.
Control Clin Trials
1999;20:531–46. 10.1016/S0197-2456(99)00027-6. [
PubMed: 10588294] [
CrossRef]
- 51.
Albert
JM.
Mediation analysis via potential outcomes models.
Stat Med
2008;27:1282–304. 10.1002/sim.3016. [
PubMed: 17691077] [
CrossRef]
- 52.
Ten Have
TR, Joffe
M.
A review of causal estimation of effects in mediation analyses.
Stat Methods Med Res
2012;21:77–107. 10.1177/0962280210391076. [
PubMed: 21163849] [
CrossRef]
- 53.
Fuller
WA.
Measurement Error Models.
New York, NY:
Wiley; 1987. 10.1002/9780470316665. [
CrossRef]
- 54.
Carroll
RJ, Ruppert
D, Stefanski
LA, Crainiceanu
CM.
Measurement Error in Non-Linear Models. 2nd edn.
London:
Chapman & Hall; 2006. 10.1201/9781420010138. [
CrossRef]
- 55.
Bollen
K.
Structural Equations with Latent Variables. 2nd edn.
New York, NY:
John Wiley & Sons, Inc; 1989. 10.1002/9781118619179. [
CrossRef]
- 56.
Dunn
G. Statistical Evaluation of Measurement Errors. 2nd edn.
London:
Arnold; 2004.
- 57.
Dunn
G.
Regression models for method comparison data.
J Biopharm Stat
2007;17:739–56. 10.1080/10543400701329513. [
PubMed: 17613651] [
CrossRef]
- 58.
Dunn
G.
The problem of measurement error in modelling the effect of compliance in a randomised trial.
Stat Med
1999;18:2863–77. 10.1002/(SICI)1097-0258(19991115)18:21<2863::AID-SIM238>3.0.CO;2-I. [
PubMed: 10523747] [
CrossRef]
- 59.
Goetghebeur
E, Vansteelandt
S.
Structural mean models for compliance analysis in randomised clinical trials and the impact of errors in exposure.
Stat Methods Med Res
2005;14:397–415. 10.1191/0962280205sm407oa. [
PubMed: 16178139] [
CrossRef]
- 60.
Dunn
G, Everitt
BS, Pickles
A. Modelling Covariances and Latent Variables in EQS.
London:
Chapman & Hall; 1993.
- 61.
Weir
CJ, Walley
RJ.
Statistical evaluation of biomarkers as surrogate endpoints: literature review.
Stat Med
2006;25:183–203. 10.1002/sim.2319. [
PubMed: 16252272] [
CrossRef]
- 62.
Daniels
MJ, Hughes
MD.
Meta-analysis for the evaluation of potential surrogate markers.
Stat Med
1997;16:1965–82. 10.1002/(SICI)1097-0258(19970915)16:17<1965::AID-SIM630>3.0.CO;2-M. [
PubMed: 9304767] [
CrossRef]
- 63.
Burzykowski
T, Molenberghs
G, Buyse
M. The Evaluation of Surrogate Endpoints.
New York, NY:
Springer; 2006.
- 64.
Florens
JP, Heckman
JJ, Meghir
C, Vytlacil
E.
Identification of treatment effects using control functions in models with continuous, endogenous treatment and heterogeneous effects.
Econometrica
2008;76:1191–206. 10.3982/ECTA5317. [
CrossRef]
- 65.
Emsley
RA, Dunn
G, Liu
H, Clarke
P, White
IR, Windmeijer
F. Estimating rank preserving models using instrumental variables for causal mediation analysis. 2015; in preparation.
- 66.
- 67.
Beck
AT, Brown
GK, Steer
RA.
Psychometric characteristics of the scale for suicide ideation with psychiatric outpatients.
Behav Res Ther
1997;35:1039–46. 10.1016/S0005-7967(97)00073-9. [
PubMed: 9431735] [
CrossRef]
- 68.
- 69.
Gunderson
JG, Frank
AF, Katz
HM, Vannicelli
ML, Frosch
JP, Knapp
PH.
Effects of psychotherapy in schizophrenia: II. Comparative outcome of two forms of treatment.
Schizophr Bull
1984;10:564–98. 10.1093/schbul/10.4.564. [
PubMed: 6151246] [
CrossRef]
- 70.
Jo
B.
Estimation of intervention effects with noncompliance: alternative model specifications.
J Educ Behav Stat
2002;27:385–409. 10.3102/10769986027004385. [
CrossRef]
- 71.
Dunn
G, Maracy
M, Tomenson
B.
Estimating treatment effects from randomized clinical trials with noncompliance and loss to follow-up: the role of instrumental variable methods.
Stat Method Med Res
2005;14:369–95. 10.1191/0962280205sm403oa. [
PubMed: 16178138] [
CrossRef]
- 72.
Dunn
G, Maracy
M, Dowrick
C, Ayuso-Mateos
JL, Dalgard
OS, Page
H, et al.
Estimating psychological treatment effects from an RCT with both non-compliance and loss to follow-up.
Br J Psychiatry
2013;183:323–31. 10.1192/bjp.183.4.323. [
PubMed: 14519610] [
CrossRef]
- 73.
Little
RJA, Rubin
DB.
Statistical Analysis with Missing Data. 2nd edn.
Hoboken, NJ:
Wiley; 2002. 10.1002/9781119013563. [
CrossRef]
- 74.
Frangakis
CE, Rubin
DB.
Addressing complications of intention-to-treat analysis in the combined presence of all-or-none treatment-noncompliance and subsequent missing outcomes.
Biometrika
1999;86:365–79. 10.1093/biomet/86.2.365. [
CrossRef]
- 75.
Lewis
S, Tarrier
N, Haddock
G, Bentall
R, Kinderman
P, Kingdon
D, et al.
Randomised controlled trial of cognitive–behavioural therapy in early schizophrenia: acute-phase outcomes.
Br J Psychiatry
2002;181:S91–7. 10.1192/bjp.181.43.s91. [
PubMed: 12271807] [
CrossRef]
- 76.
Tarrier
N, Lewis
S, Haddock
G, Bentall
R, Drake
R, Kinderman
P, et al.
Cognitive–behavioural therapy in first-episode and early schizophrenia – 18-month follow-up of a randomised controlled trial.
Br J Psychiatry
2004;184:231–9. 10.1192/bjp.184.3.231. [
PubMed: 14990521] [
CrossRef]
- 77.
Kay
SR, Fiszbein
A, Opler
LA.
The positive and negative syndrome scale (PANSS) for schizophrenia.
Schiz Bull
1987;13:261–76. 10.1093/schbul/13.2.261. [
PubMed: 3616518] [
CrossRef]
- 78.
Efron
B, Tibshirani
RJ. An Introduction to the Bootstrap.
London:
Chapman & Hall; 1993.
- 79.
Muthén
LK, Muthén
BO. Mplus User’s Guide.
Los Angeles, CA:
Muthén & Muthén; 1998–2012.
- 80.
Pickles
A, Green
J and the
PACT consortium.
Therapeutic mechanism in the MRC Pre-school Autism Communication Trial: implications for study design and parent focussed therapy for children.
J Child Psychol Psychiatry
2015;56:162–70. 10.1111/jcpp.12291. [
PubMed: 25039961] [
CrossRef]
- 81.
Garety
P, Fowler
D, Freeman
D, Bebbington
P, Dunn
G, Kuipers
E.
Cognitive–behavioural therapy and family intervention for relapse prevention and symptom reduction in psychosis: randomised controlled trial.
Br J Psychiatry
2008;192:412–23. 10.1192/bjp.bp.107.043570. [
PubMed: 18515890] [
CrossRef]
- 82.
- 83.
Muthén
B, Khoo
ST.
Longitudinal studies of achievement growth using latent variable modeling.
Learn Individ Differences
1998;10:73–101. 10.1016/S1041-6080(99)80135-6. [
CrossRef]
- 84.
McArdle
JJ.
Latent variable modeling of differences and changes with longitudinal data.
Ann Rev Psychol
2009;60:577–605. 10.1146/annurev.psych.60.110707.163612. [
PubMed: 18817479] [
CrossRef]
- 85.
- 86.
Asparouhov
T, Muthén
B. Auxiliary variables in mixture modeling: 3-Step approaches using Mplus. Mplus Web Notes: No. 15 Version 8, 5
August
2014.
- 87.
Bullock
JG, Green
DP, Ha
SE.
Yes, but what’s the mechanism? (Don’t expect an easy answer).
J Personality Soc Psychol
2010;98:550–8. 10.1037/a0018933. [
PubMed: 20307128] [
CrossRef]
- 88.
Burgess
S, Thompson
SG.
Avoiding bias from weak instruments in Mendelian randomisation studies.
Int J Epidemiol
2011;40:755–64. 10.1093/ije/dyr036. [
PubMed: 21414999] [
CrossRef]
- 89.
Burgess
S, Thompson
SG.
Bias in causal estimates from Mendelian randomisation studies with weak instruments.
Stat Med
2011;30:1312–23. 10.1002/sim.4197. [
PubMed: 21432888] [
CrossRef]
- 90.
Davey Smith
G, Ebrahim
S.
‘Mendelian randomisation’: can genetic epidemiology contribute to understanding environmental determinants of disease?
Int J Epidemiology
2003;32:1–22. 10.1093/ije/dyg070. [
PubMed: 12689998] [
CrossRef]
- 91.
Lawlor
DA, Harbord
RM, Sterne
JAC, Timpson
N, Davey Smith
G.
Mendelian randomisation: using genes as instruments for making causal inferences in epidemiology.
Stat Med
2008;27:1133–63. 10.1002/sim.3034. [
PubMed: 17886233] [
CrossRef]
- 92.
Didelez
V, Sheehan
NA.
Mendeleian randomisation as an instrumental variable approach to causal inference.
Stat Methods Med Res
2007;16:309–30. 10.1177/0962280206077743. [
PubMed: 17715159] [
CrossRef]
- 93.
Garety
PA, Kuipers
E, Fowler
D, Freeman
D, Bebbington
PE.
A cognitive model of the positive symptoms of psychosis.
Psychol Med
2001;31:189–95. 10.1017/S0033291701003312. [
PubMed: 11232907] [
CrossRef]
- 94.
Garety
P, Freeman
D.
Cognitive approaches to delusions: a critical review of theories and evidence.
Br J Clin Psychol
1999;38:113–54. 10.1348/014466599162700. [
PubMed: 10389596] [
CrossRef]
- 95.
Freeman
D.
Suspicious minds: the psychology of persecutory delusions.
Clin Psychol Rev
2007;27:425–57. 10.1016/j.cpr.2006.10.004. [
PubMed: 17258852] [
CrossRef]
- 96.
Emsley
RA, VanderWeele
TJ. Mediation and sensitivity analysis using two or more trials. 2015; in preparation.
- 97.
VanderWeele
TJ. Explanation in Causal Analysis: Methods for Mediation and Interaction.
New York, NY:
Oxford University Press; 2015.
- 98.
Spencer
SJ, Zanna
MP, Fong
GT.
Establishing a causal chain: why experiments are often more effective than mediational analyses in examining psychological processes.
J Pers Soc Psychol
2005;89:845–51. 10.1037/0022-3514.89.6.845. [
PubMed: 16393019] [
CrossRef]
- 99.
Stone-Romero
EF, Rosopa
PJ.
The relative validity of inferences about mediation as a function of research design characteristics.
Org Res Methods
2008;11:326–52. 10.1177/1094428107300342. [
CrossRef]
- 100.
Young
KY, Laird
A, Zhou
ZX.
The efficiency of clinical trial designs for predictive biomarker validation.
Clin Trials
2010;7:557–66. 10.1177/1740774510370497. [
PubMed: 20571132] [
CrossRef]
- 101.
- 102.
Dunn
G, Emsley
R, Liu
H, Landau
S.
Integrating biomarker information within trials to evaluate treatment mechanisms and efficacy for personalised medicine.
Clin Trials
2013;10:712–22. 10.1177/1740774513499651. [
PubMed: 24000376] [
CrossRef]
- 103.
Imai
K, Tingley
D, Yamamoto
T.
Experimental designs for identifying causal mechanisms.
J R Stat Soc A
2013;76:5–51. 10.1111/j.1467-985X.2012.01032.x. [
CrossRef]
- 104.
Imai
K, Keele
L, Tingley,
D, Yamamoto
T.
Unpacking the black box of causality: learning about causal mechanisms from experimental and observational studies.
Am Political Sci Rev
2011;105:765–89. 10.1017/S0003055411000414. [
CrossRef]
- 105.
- 106.
- 107.
- 108.
- 109.
