Observational study designs for comparative effectiveness research: An alternative approach to close evidence gaps in head-and-neck cancer

International Journal of Radiation Oncology Biology Physics

Volumn 88, Issue 1, 2014, Pages 106-114

  Goulart, Bernardo H L ^a,c   Ramsey, Scott D ^a,c   Parvathaneni, Upendra ^b,c  

^a USA   (United States)

^b UNIVERSITY OF WASHINGTON SCHOOL OF MEDICINE   (United States)

^c UNIVERSITY OF WASHINGTON   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

DESIGN; DESIGN OF EXPERIMENTS; HEALTH CARE; RANDOM PROCESSES;

ANALYTIC TECHNIQUE; CLINICAL PRACTICES; COMPARATIVE EFFECTIVENESS RESEARCHES; HEAD-AND-NECK CANCER; HEALTH-CARE DECISIONS; OBSERVATIONAL STUDY; OSTEORADIONECROSIS; RANDOMIZED CONTROLLED TRIAL;

DISEASES;

CETUXIMAB; CISPLATIN;

ARTICLE; CANCER CONTROL; CANCER PATIENT; CANCER PROGNOSIS; CANCER RADIOTHERAPY; CANCER RECURRENCE; CANCER REGISTRY; CANCER SURVIVAL; CANCER THERAPY; CHEMORADIOTHERAPY; CLINICAL DECISION MAKING; CLINICAL PRACTICE; COMPARATIVE EFFECTIVENESS; COMPUTER ASSISTED EMISSION TOMOGRAPHY; CONFOUNDING VARIABLE; CONSERVATIVE TREATMENT; COST EFFECTIVENESS ANALYSIS; ELECTRONIC MEDICAL RECORD; EVIDENCE BASED MEDICINE; FOLLOW UP; GOLD STANDARD; HEAD AND NECK CANCER; HEALTH CARE ACCESS; HEALTH CARE COST; HEALTH CARE DELIVERY; HEALTH CARE QUALITY; HEALTH CARE UTILIZATION; HUMAN; HYPOPHARYNX CANCER; INSTRUMENTAL VARIABLE ANALYSIS; INTENSITY MODULATED RADIATION THERAPY; LARYNX CANCER; MEDICARE; OBSERVATIONAL STUDY; OROPHARYNX CARCINOMA; OUTCOME ASSESSMENT; OVERALL SURVIVAL; PATIENT COMPLIANCE; PATIENT SELECTION; PRIORITY JOURNAL; PROPENSITY SCORE; QUALITY OF LIFE; RANDOMIZATION; RANDOMIZED CONTROLLED TRIAL (TOPIC); RISK ASSESSMENT; SELF REPORT; SQUAMOUS CELL CARCINOMA; STUDY DESIGN; SYSTEMATIC ERROR; TONSIL CANCER; TREATMENT RESPONSE; WART VIRUS; XEROSTOMIA;

EID: 84890543058     PISSN: 03603016     EISSN: 1879355X     Source Type: Journal    
DOI: 10.1016/j.ijrobp.2013.05.050     Document Type: Review

References (72)

1. Institute of Medicine. Initial national priorities for comparative effectiveness research. Available at: http://www.iom.edu/Reports/2009/ ComparativeEffectivenessResearchPriorities.aspx. Accessed May, 22, 2013.
2. Organization for Economic Cooperation and Development. Why is health spending in the United States so high? Available at: http://www.oecd.org/ unitedstates/. Accessed May, 22, 2013.
3. A.I. Mushlin, and H. Ghomrawi Health care reform and the need for comparative-effectiveness research N Engl J Med 362 2010 e6
4. D.F. Martin, M.G. Maguire, and S.L. Fine Identifying and eliminating the roadblocks to comparative-effectiveness research N Engl J Med 363 2010 105 107
5. Federal Council for Comparative Effectiviness Research Report to the President and the Congress 2009 US Department of Health and Human Services Washington, DC 1 77
6. G.H. Lyman, and M. Levine Comparative effectiveness research in oncology: An overview J Clin Oncol 30 2012 4181 4184
7. B.R. Luce, L.C. Paramore, and B. Parasuraman Can managed care organizations partner with manufacturers for comparative effectiveness research? Am J Manag Care 14 2008 149 156
8. A.M. Garber, and S.R. Tunis Does comparative-effectiveness research threaten personalized medicine? N Engl J Med 360 2009 1925 1927
9. J.M. Lee, M. Turini, and M.F. Botteman Economic burden of head and neck cancer. A literature review Eur J Health Econ 5 2004 70 80
10. K.R. Yabroff, J. Lund, and D. Kepka Economic burden of cancer in the United States: Estimates, projections, and future research Cancer Epidemiol Biomarkers Prev 20 2011 2006 2014
11. S.J. Wong, P.M. Harari, and A.S. Garden Longitudinal Oncology Registry of Head and Neck Carcinoma (LORHAN): Analysis of chemoradiation treatment approaches in the United States Cancer 117 2011 1679 1686
12. J.A. Bonner, P.M. Harari, and J. Giralt Radiotherapy plus cetuximab for squamous-cell carcinoma of the head and neck N Engl J Med 354 2006 567 578
13. P. Blanchard, B. Baujat, and V. Holostenco Meta-analysis of chemotherapy in head and neck cancer (MACH-NC): A comprehensive analysis by tumour site Radiother Oncol 100 2011 33 40
14. N.C. Sheets, S.B. Wheeler, and R.E. Kohler Costs of care in a matched pair comparison of intensity-modulated radiation therapy (IMRT) versus conventional radiation therapy (CRT) for the treatment of head and neck cancer Am J Clin Oncol 2013 March 4 Epub ahead of print
15. Oxford Centre for Evidence Based Medicine. Levels of evidence (March 2009). Available at: http://www.cebm.net/index.aspx?o=1025. Oxford, UK: Centre for Evidence Based Medicine, University of Oxford; 2009. Accessed February, 26, 2013.
16. N.F. Marko, and R.J. Weil The role of observational investigations in comparative effectiveness research Value Health 13 2010 989 997
17. H. Walach, T. Falkenberg, and V. Fonnebo Circular instead of hierarchical: Methodological principles for the evaluation of complex interventions BMC Med Res Methodol 6 2006 29
18. J. Concato, N. Shah, and R.I. Horwitz Randomized, controlled trials, observational studies, and the hierarchy of research designs N Engl J Med 342 2000 1887 1892
19. T.D. Kopsell Epidemiologic Methods - Studying the Occurrence of Illness 2003 Oxford University Press New York
20. O.M. Hahn, and R.L. Schilsky Randomized controlled trials and comparative effectiveness research J Clin Oncol 30 2012 4194 4201
21. D.L. Hershman, and J.D. Wright Comparative effectiveness research in oncology methodology: Observational data J Clin Oncol 30 2012 4215 4222
22. J.L. Warren, C.N. Klabunde, and D. Schrag Overview of the SEER-Medicare data: Content, research applications, and generalizability to the United States elderly population Med Care 40 2002 IV-3-18
23. K.K. Ang, J. Harris, and R. Wheeler Human papillomavirus and survival of patients with oropharyngeal cancer N Engl J Med 363 2010 24 35
24. B. O'Sullivan, S.H. Huang, and L.L. Siu Deintensification candidate subgroups in human papillomavirus-related oropharyngeal cancer according to minimal risk of distant metastasis J Clin Oncol 31 2013 543 550
25. M.T. Andreoli, S.M. Andreoli, and M.G. Shrime Radiotherapy in parotid acinic cell carcinoma: Does it have an impact on survival? Arch Otolaryngol Head Neck Surg 138 2012 463 466
26. J.H. Turner, and D.D. Reh Incidence and survival in patients with sinonasal cancer: A historical analysis of population-based data Head Neck 34 2012 877 885
27. B.M. Beadle, K.P. Liao, and M.S. Chambers Evaluating the impact of patient, tumor, and treatment characteristics on the development of jaw complications in patients treated for oral cancers: A SEER-Medicare analysis Head Neck 2012 Nov 14 Epub ahead of print
28. A.Y. Chen, S. Fedewa, and A. Pavluck Improved survival is associated with treatment at high-volume teaching facilities for patients with advanced stage laryngeal cancer Cancer 116 2010 4744 4752
29. National Cancer Institute. Surveillance, Epidemiology, and End Results (SEER). Available at http://seer.cancer.gov/registries/list.html. Accessed February, 28, 2013.
30. J.L. Warren, L.C. Harlan, and A. Fahey Utility of the SEER-Medicare data to identify chemotherapy use Med Care 40 2002 IV-55-61
31. S.D. Ramsey, N. Howlader, and R.D. Etzioni Chemotherapy use, outcomes, and costs for older persons with advanced non-small-cell lung cancer: Evidence from Surveillance, Epidemiology and End Results-Medicare J Clin Oncol 22 2004 4971 4978
32. Agency for Healthcare Research and Quality. Overview of the Nationwide Inpatient Sample (NIS). Available at: http://www.hcup-us.ahrq.gov/nisoverview. jsp. Accessed May, 22, 2013.
33. C.G. Gourin, and K.D. Frick National trends in oropharyngeal cancer surgery and the effect of surgeon and hospital volume on short-term outcomes and cost of care Laryngoscope 122 2012 543 551
34. B.J. Kennedy, H.R. Menck, and G.D. Steele Jr. National Cancer Data Base. A clinical assessment of patients with cancer Cancer 69 1992 276
35. Truven Health Analytics. Marketscan Research. Available at: http://marketscan.truvenhealth.com/marketscanportal/. Accessed May, 22, 2013.
36. ClinicalTrials.gov. Head and Neck Cancer Registry (LORHAN). Available at: http://clinicaltrials.gov/ct2/show/NCT01080313. Accessed May, 22, 2013.
37. J.R. Palta, J.A. Efstathiou, and J.E. Bekelman Developing a national radiation oncology registry: From acorns to oaks Prac Rad Onc 2 2012 10 17
38. American Society of Clinical Oncology. CancerLinQ: Building a transformation in cancer care. Available at: http://www.asco.org/institute- quality/cancerlinq. Accessed May, 23, 2013.
39. E. Glatstein Restrictions of a statistical mind: Clinical relevance versus p values or when less is more Int J Radiat Oncol Biol Phys 68 2007 322 323
40. E. von Elm, D.G. Altman, and M. Egger The Strengthening the Reporting of Observational Studies in Epidemiology (STROBE) statement: Guidelines for reporting observational studies Ann Intern Med 147 2007 573 577
41. R.B. D'Agostino Jr. Propensity score methods for bias reduction in the comparison of a treatment to a non-randomized control group Stat Med 17 1998 2265 2281
42. J.W. Hogan, and T. Lancaster Instrumental variables and inverse probability weighting for causal inference from longitudinal observational studies Stat Methods Med Res 13 2004 17 48
43. A. Basu, J.J. Heckman, and S. Navarro-Lozano Use of instrumental variables in the presence of heterogeneity and self-selection: An application to treatments of breast cancer patients Health Econ 16 2007 1133 1157
44. J.P. Vandenbroucke When are observational studies as credible as randomised trials? Lancet 363 2004 1728 1731
45. J.J. Jacobson, J.B. Epstein, and F.C. Eichmiller The cost burden of oral, oral pharyngeal, and salivary gland cancers in three groups: Commercial insurance, Medicare, and Medicaid Head Neck Oncol 4 2012 15
46. B. O'Sullivan, P. Warde, and B. Grice The benefits and pitfalls of ipsilateral radiotherapy in carcinoma of the tonsillar region Int J Radiat Oncol Biol Phys 51 2001 332 343
47. K.E. Rusthoven, D. Raben, and C. Schneider Freedom from local and regional failure of contralateral neck with ipsilateral neck radiotherapy for node-positive tonsil cancer: Results of a prospective management approach Int J Radiat Oncol Biol Phys 74 2009 1365 1370
48. A. Eisbruch, H.M. Kim, and J.E. Terrell Xerostomia and its predictors following parotid-sparing irradiation of head-and-neck cancer Int J Radiat Oncol Biol Phys 50 2001 695 704
49. K.S. Chao, N. Majhail, and C.J. Huang Intensity-modulated radiation therapy reduces late salivary toxicity without compromising tumor control in patients with oropharyngeal carcinoma: A comparison with conventional techniques Radiother Oncol 61 2001 275 280
50. T. Gupta, J. Agarwal, and S. Jain Three-dimensional conformal radiotherapy (3D-CRT) versus intensity modulated radiation therapy (IMRT) in squamous cell carcinoma of the head and neck: A randomized controlled trial Radiother Oncol 104 2012 343 348
51. E.H. Pow, D.L. Kwong, and A.S. McMillan Xerostomia and quality of life after intensity-modulated radiotherapy vs. conventional radiotherapy for early-stage nasopharyngeal carcinoma: Initial report on a randomized controlled clinical trial Int J Radiat Oncol Biol Phys 66 2006 981 991
52. M.K. Kam, S.F. Leung, and B. Zee Prospective randomized study of intensity-modulated radiotherapy on salivary gland function in early-stage nasopharyngeal carcinoma patients J Clin Oncol 25 2007 4873 4879
53. C.M. Nutting, J.P. Morden, and K.J. Harrington Parotid-sparing intensity modulated versus conventional radiotherapy in head and neck cancer (PARSPORT): A phase 3 multicentre randomised controlled trial Lancet Oncol 12 2011 127 136
54. D.M. Cannon, and N.Y. Lee Recurrence in region of spared parotid gland after definitive intensity-modulated radiotherapy for head and neck cancer Int J Radiat Oncol Biol Phys 70 2008 660 665
55. A.M. Chen, D.G. Farwell, and Q. Luu Marginal misses after postoperative intensity-modulated radiotherapy for head and neck cancer Int J Radiat Oncol Biol Phys 80 2011 1423 1429
56. Induction chemotherapy plus radiation compared with surgery plus radiation in patients with advanced laryngeal cancer. The Department of Veterans Affairs Laryngeal Cancer Study Group N Engl J Med 324 1991 1685 1690
57. J.L. Lefebvre, D. Chevalier, and B. Luboinski Larynx preservation in pyriform sinus cancer: Preliminary results of a European Organization for Research and Treatment of Cancer phase III trial. EORTC Head and Neck Cancer Cooperative Group J Natl Cancer Inst 88 1996 890 899
58. A.A. Forastiere, H. Goepfert, and M. Maor Concurrent chemotherapy and radiotherapy for organ preservation in advanced laryngeal cancer N Engl J Med 349 2003 2091 2098
59. H.T. Hoffman, K. Porter, and L.H. Karnell Laryngeal cancer in the United States: Changes in demographics, patterns of care, and survival Laryngoscope 116 2006 1 13
60. A.K. Chaturvedi, E.A. Engels, and R.M. Pfeiffer Human papillomavirus and rising oropharyngeal cancer incidence in the United States J Clin Oncol 29 2011 4294 4301
61. ClinicalTrials.gov. Radiation therapy with cisplatin or cetuximab in treating patients with oropharyngeal cancer. Available at: http:// clinicaltrials.gov/ct2/show/NCT01302834. Accessed March, 12, 2013.
62. J.M. Albert, and P. Das Quality assessment in oncology Int J Radiat Oncol Biol Phys 83 2012 773 781
63. J.M. Albert, and P. Das Quality indicators in radiation oncology Int J Radiat Oncol Biol Phys 85 2013 904 911
64. M.D. Fesinmeyer, V. Mehta, and D. Blough Effect of radiotherapy interruptions on survival in Medicare enrollees with local and regional head-and-neck cancer Int J Radiat Oncol Biol Phys 78 2010 675 681
65. S.V. Porceddu, G. Adams, and R. Gundelach Does fluorodeoxyglucose PET add to the management of the neck following curative radiotherapy in head and neck cancer compared with computed tomography? Exp Rev Anticancer Ther 13 2013 279 284
66. A. Meirovitz, C.A. Murdoch-Kinch, and M. Schipper Grading xerostomia by physicians or by patients after intensity-modulated radiotherapy of head-and-neck cancer Int J Radiat Oncol Biol Phys 66 2006 445 453
67. A.D. Jensen, J. Krauss, and K. Potthoff Phase II study of induction chemotherapy with TPF followed by radioimmunotherapy with cetuximab and intensity-modulated radiotherapy (IMRT) in combination with a carbon ion boost for locally advanced tumours of the oro-, hypopharynx and larynx - TPF-C-HIT BMC Cancer 11 2011 182
68. A.A. Donatelli-Lassig, S.A. Duffy, and K.E. Fowler The effect of neck dissection on quality of life after chemoradiation Otolaryngol Head Neck Surg 139 2008 511 518
69. M.C. Weinstein, J.E. Siegel, and M.R. Gold Recommendations of the Panel on Cost-effectiveness in Health and Medicine JAMA 276 1996 1253 1258
70. D.J. Sher, R.B. Tishler, and D. Annino Cost-effectiveness of CT and PET-CT for determining the need for adjuvant neck dissection in locally advanced head and neck cancer Ann Oncol 21 2010 1072 1077
71. A. Rabalais, R.R. Walvekar, and J.T. Johnson A cost-effectiveness analysis of positron emission tomography-computed tomography surveillance versus up-front neck dissection for management of the neck for N2 disease after chemoradiotherapy Laryngoscope 122 2012 311 314
72. J.H. Yong, J. Beca, and B. O'Sullivan Cost-effectiveness of intensity-modulated radiotherapy in oropharyngeal cancer Clin Oncol (R Coll Radiol) 24 2012 532 538