Analyzing phase III studies in hospice/palliative care. A solution that sits between intention-to-treat and per protocol analyses: The palliative-modified ITT analysis

Journal of Pain and Symptom Management

Volumn 44, Issue 4, 2012, Pages 595-603

  Currow, David C ^a   Plummer, John L ^b   Kutner, Jean S ^c   Samsa, Greg P ^d   Abernethy, Amy P ^a,d  

^a FLINDERS UNIVERSITY   (Australia)

^b FLINDERS MEDICAL CENTRE   (Australia)

^c UNIVERSITY OF COLORADO SCHOOL OF MEDICINE   (United States)

^d DUKE UNIVERSITY MEDICAL CENTER   (United States)

Author keywords

Clinical trials; Intention to treat analyses; Palliative care research; Per protocol on treatment analyses; Phase III studies; Randomized controlled trials

Indexed keywords

ARTICLE; CLINICAL PROTOCOL; DEATH; DISEASE COURSE; HEALTH CARE QUALITY; HOSPICE CARE; HUMAN; INTENTION TO TREAT ANALYSIS; OUTCOME ASSESSMENT; PALLIATIVE THERAPY; PER PROTOCOL ANALYSIS; PHASE 3 CLINICAL TRIAL (TOPIC); RANDOMIZATION; RANDOMIZED CONTROLLED TRIAL (TOPIC); STATISTICAL ANALYSIS; STUDY DESIGN;

EID: 84869238412     PISSN: 08853924     EISSN: 18736513     Source Type: Journal    
DOI: 10.1016/j.jpainsymman.2011.10.028     Document Type: Article

References (17)

1. Lachin JM. Statistical considerations in the intention-to-treat analysis. Control Clin Trials 2000; 21:167-189.
2. Food and Drug Administration. International Conference on Harmonization: guidance on general considerations for clinical trials. Fed Regist 1997;62:66113-66119.
3. ICH Harmonised Tripartite Guideline. Statistical principles for clinical trials. Stat Med 1999;18: 1905-1942.
4. Gillings D, Koch G. The application of the principle of intention-to-treat to the analysis of clinical trials. Drug Inf J 1999;25:411-424.
5. Hollis S, Campbell F. What is meant by intention to treat analysis? Survey of published randomized controlled trials. Br Med J 1999;319: 670-674. (Pubitemid 29423954)
6. Kruse RL, Alper BS, Reust C, et al. Randomized controlled trials; research design; random allocation; intention to treat. J Fam Pract 2002;51: 969-971.
7. Wiens BL, Zhao W. The role of intention to treat analysis of noninferiority studies. Clin Trials 2007;4:286-291. (Pubitemid 47400540)
8. Mularski RA, Rosenfeld K, Coons SJ, et al. Measuring outcomes in randomized prospective trials in palliative care. J Pain Symptom Manage 2007; 34(Suppl 1):S7eS19. (Pubitemid 46970813)
9. Whalen GF, Kutner J, Byock I, et al. Implementing palliative care studies. J Pain Symptom Manage 2007;34(Suppl 1):S40eS48. (Pubitemid 46970816)
10. Abernethy AP, Currow DC, Hunt R, et al. A pragmatic 2 x 2 x 2 factorial cluster randomized controlled trial of educational outreach visiting and case conferencing in palliative care-methodology of the Palliative Care Trial [ISRCTN 81117481]. Contemp Clin Trials 2006;27:83-100. (Pubitemid 41758441)
11. Shih WJ, Quan H. Testing for treatment differences with dropouts present in clinical trialsda composite approach. Stat Med 1997;16: 1225-1239. (Pubitemid 27252594)
12. Yusuf S, Wittes J, Probstfield J, Tyroler HA. Analysis and interpretation of treatment effects in subgroups of patients in randomized clinical trials. JAMA 1991;266:93-98.
13. Shelby-James TM, Hardy J, Agar M, et al. Designing and conducting randomized controlled trials in palliative care: a summary of discussions from the 2010 clinical research forum of the Australian Palliative Care Clinical Studies Collaborative (PaCCSC). Palliat Med 2011. [Epub ahead of print].
14. Eagar K, Gordon R, Green J, Smith M. An Australian casemix classification for palliative care: lessons and policy implications of a national study. Palliat Med 2004;18:227-233. (Pubitemid 38771662)
15. Abernethy AP, Shelby-James TM, Fazekas BS, Woods D, Currow DC. The Australian-modified Karnofsky Performance Status (AKPS) scale: a revised scale for contemporary palliative care clinical practice. BMC Pall Care 2005;4:7.
16. Zubrod CG, Schneiderman M, Frei E, et al. Appraisal of methods for the study of chemotherapy of cancer in man: comparative therapeutic trial of nitrogen mustard and triethylene thiophosphoramide. J Chron Dis 1960;11:7-33.
17. Rubin DB. Causal inference through potential outcomes and principal stratification: application to studies with "censoring" due to death. Stat Sci 2006;21:299-309.