Use of alternative methodologies for evaluation of composite end points in trials of therapies for critical limb ischemia

American Heart Journal

Volumn 164, Issue 3, 2012, Pages 277-284

  Subherwal, Sumeet ^a   Anstrom, Kevin J ^a   Jones, William S ^a   Felker, Michael G ^a   Misra, Sanjay ^b   Conte, Michael S ^c   Hiatt, William R ^d   Patel, Manesh R ^a  

^a DUKE CLINICAL RESEARCH INSTITUTE   (United States)

^b MAYO CLINIC   (United States)

^c UNIVERSITY OF CALIFORNIA   (United States)

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

Author keywords

[No Author keywords available]

Indexed keywords

PROSTANOID;

BELOW KNEE AMPUTATION; CRITICAL LIMB ISCHEMIA; ENDARTERECTOMY; HEART ASSIST DEVICE; HEART FAILURE; HUMAN; LIMB AMPUTATION; MORBIDITY; MORTALITY; PRIORITY JOURNAL; QUALITY OF LIFE; REVASCULARIZATION; REVIEW; SMOKING CESSATION; STATISTICAL ANALYSIS; TREATMENT OUTCOME; TREATMENT PLANNING; ULCER; WOUND CARE; WOUND HEALING;

EID: 84866321753     PISSN: 00028703     EISSN: 10976744     Source Type: Journal    
DOI: 10.1016/j.ahj.2012.07.002     Document Type: Editorial

References (43)

1. S. Nikol Therapeutic angiogenesis with intramuscular NV1FGF improves amputation-free survival in patients with critical limb ischemia Mol Ther 16 2008 972 978 (Pubitemid 351587109)
2. J. Belch Effect of fibroblast growth factor NV1FGF on amputation and death: a randomised placebo-controlled trial of gene therapy in critical limb ischaemia Lancet 377 2011 1929 1937
3. E.P. Brass Parenteral therapy with lipo-ecraprost, a lipid-based formulation of a PGE1 analog, does not alter six-month outcomes in patients with critical leg ischemia J Vasc Surg 43 2006 752 759
4. R.J. Powell Results of a double-blind, placebo-controlled study to assess the safety of intramuscular injection of hepatocyte growth factor plasmid to improve limb perfusion in patients with critical limb ischemia Circulation 118 2008 58
5. S. Rajagopalan Regional angiogenesis with vascular endothelial growth factor in peripheral arterial disease: a phase II randomized, double-blind, controlled study of adenoviral delivery of vascular endothelial growth factor 121 in patients with disabling intermittent claudication Circulation 108 2003 1933 1938 (Pubitemid 37296537)
6. R. Morishita Safety evaluation of clinical gene therapy using hepatocyte growth factor to treat peripheral arterial disease Hypertension 44 2004 203 209 (Pubitemid 39014240)
7. E. Tateishi-Yuyama Therapeutic angiogenesis for patients with limb ischaemia by autologous transplantation of bone-marrow cells: a pilot study and a randomised controlled trial Lancet 360 2002 427 435
8. W.S. Jones, and B.H. Annex Growth factors for therapeutic angiogenesis in peripheral arterial disease Curr Opin Cardiol 22 2007 458
9. R.J. Powell Safety and efficacy of patient specific intramuscular injection of HGF plasmid gene therapy on limb perfusion and wound healing in patients with ischemic lower extremity ulceration: Results of the HGF-0205 trial J Vasc Surg 52 2010 1525 1530
10. M. Werk Inhibition of restenosis in femoropopliteal arteries. Paclitaxel-coated versus uncoated balloon: femoral paclitaxel randomized pilot trial Circulation 118 2008 1358 1365
11. G. Tepe Local delivery of paclitaxel to inhibit restenosis during angioplasty of the leg N Engl J Med 358 2008 689 699 (Pubitemid 351240748)
12. M.C. Stoner Mid-term results with laser atherectomy in the treatment of infrainguinal occlusive disease J Vasc Surg 46 2007 289.e1 295.e1
13. M.S. Conte Suggested objective performance goals and clinical trial design for evaluating catheter-based treatment of critical limb ischemia J Vasc Surg 50 2009 1462.e3 1473.e3
14. A. Kumar FDA perspective on objective performance goals and clinical trial design for evaluating catheter-based treatment of critical limb ischemia J Vasc Surg 50 2009 1474 1476
15. T.G. Brott Stenting versus endarterectomy for treatment of carotid-artery stenosis N Engl J Med 363 2010 11 23
16. E.P. Brass Risk assessment in drug development for symptomatic indications: a framework for the prospective exclusion of unacceptable cardiovascular risk Clin Pharmacol Ther 79 2006 165 172
17. G.L. Grunkemeier, R. Jin, and A. Starr Prosthetic heart valves: objective performance criteria versus randomized clinical trial Ann Thorac Surg 82 2006 776 780
18. E. Chen FDA perspective on clinical trial design for cardiovascular devices Ann Thorac Surg 82 2006 773
19. M.K. Parides Progress versus precision: challenges in clinical trial design for left ventricular assist devices Ann Thorac Surg 82 2006 1140 1146
20. S.J. Pocock The win ratio: a new approach to the analysis of composite endpoints in clinical trials based on clinical priorities Eur Heart J 33 2012 176 182
21. G.M. Felker, K.J. Anstrom, and J.G. Rogers A global ranking approach to end points in trials of mechanical circulatory support devices J Card Fail 14 2008 368 372 (Pubitemid 351749834)
22. G.M. Felker, and A.S. Maisel A global rank end point for clinical trials in acute heart failure Circ Heart Fail 3 2010 643 646
23. P.W. Armstrong Refining clinical trial composite outcomes: An application to the Assessment of the Safety and Efficacy of a New Thrombolytic-3 (ASSENT-3) trial Am Heart J 161 2011 848 854
24. P.C. O'Brien Procedures for comparing samples with multiple endpoints Biometrics 40 1984 1079 1087
25. R. Califf, L. Harrelson-Woodlief, and E. Topol Left ventricular ejection fraction may not be useful as an end point of thrombolytic therapy comparative trials Circulation 82 1990 1847 1853 (Pubitemid 20367987)
26. N. Diehm A call for uniform reporting standards in studies assessing endovascular treatment for chronic ischaemia of lower limb arteries Eur Heart J 28 2007 798 805 (Pubitemid 47343592)
27. L.A. MoyÈ, B.R. Davis, and C.M. Hawkins Analysis of a clinical trial involving a combined mortality and adherence dependent interval censored endpoint Stat Med 11 1992 1705 1717
28. D.M. Finkelstein, and D.A. Schoenfeld Combining mortality and longitudinal measures in clinical trials Stat Med 18 1999 1341 1354 (Pubitemid 29261493)
29. R.P. McMahon, and F.E. Harrell Jr. Power calculation for clinical trials when the outcome is a composite ranking of survival and a nonfatal outcome Control Clin Trials 21 2000 305 312 (Pubitemid 30456291)
30. S. Matoba Long-term clinical outcome after intramuscular implantation of bone marrow mononuclear cells (Therapeutic Angiogenesis by Cell Transplantation [TACT] trial) in patients with chronic limb ischemia Am Heart J 156 2008 1010 1018
31. D.J. Adam Bypass versus angioplasty in severe ischaemia of the leg (BASIL): multicentre, randomised controlled trial Lancet 366 2005 1925 1934 (Pubitemid 41713982)
32. C. Randon Angioplasty or primary stenting for infrapopliteal lesions: results of a prospective randomized trial Cardiovasc Intervent Radiol 33 2010 260 269
33. D.H. Walter Intraarterial administration of bone marrow mononuclear cells in patients with critical limb ischemia/clinical perspective Circ Cardiovasc Interv 4 2011 26 37
34. Y. Tang Size and power estimation for the Wilcoxon-Mann-Whitney test for ordered categorical data Stat Med 30 2011 3461 3470
35. E.J.G. Peters Functional status of persons with diabetes-related lower-extremity amputations Diabetes Care 24 2001 1799 1804 (Pubitemid 33716485)
36. J.M. Lachin Worst-rank score analysis with informatively missing observations in clinical trials Control Clin Trials 20 1999 408 422 (Pubitemid 29460918)
37. E. Lim Composite outcomes in cardiovascular research: a survey of randomized trials Ann Intern Med 149 2008 612 617
38. G. Cordoba Definition, reporting, and interpretation of composite outcomes in clinical trials: systematic review BMJ 2010 341
39. N. Freemantle Composite outcomes in randomized trials JAMA 289 2003 2554
40. E.M. Antman The TIMI risk score for unstable angina/non-ST elevation MI: a method for prognostication and therapeutic decision making JAMA 284 2000 835 842 (Pubitemid 30643610)
41. C.B. Granger Predictors of hospital mortality in the Global Registry of Acute Coronary Events Arch Intern Med 163 2003 2345 2353 (Pubitemid 37310196)
42. L. Norgren Inter-society consensus for the management of peripheral arterial disease (TASC II) J Vasc Surg 45 2007 5
43. J.A. Bakal, C.M. Westerhout, and P.W. Armstrong Impact of weighted composite compared to traditional composite endpoints for the design of randomized controlled trials Stat Methods Med Res 2012 [Epub ahead of print]